ACTIvATeS: ACTive Treatment for Idiopathic AdolescenT Scoliosis - A feasibility study

Current treatments

In the UK, surveys of current surgical practice9 and advice information provided at initial diagnosis have previously been conducted;10 however, a survey of current conservative treatments provided in the NHS has not been undertaken. Currently, it is thought that the main components of conservative management of scoliosis in the UK are monitoring and a limited use of bracing.

Rationale for treatments

Braces are an intrusive and uncomfortable intervention. Two publications reported bracing protocols that lasted up to 4 years and required the brace to be worn for 23 hours per day. 11,12 The effectiveness of bracing protocols is unclear,13 although a recent trial reported by Weinstein et al. 14 suggests that there is promise of efficacy.

Exercise is a promising intervention for which there is an emerging, although low-quality, evidence base. 15,16 The rationale is to use exercises that promote spinal realignment and thus either improve or halt progression of the curvature. Exercise is, potentially, a low-cost intervention and, even if not effective in all patients, it may be of substantial benefit if the relative risk reduction in progression to curvature of > 45° or requirement for surgery was reduced in a modest proportion of those participating. It is thought that exercise should be applied as early in the disease process as possible.

Exercise therapy is not commonly provided in the UK and the USA, although it is routinely provided in parts of Europe. 17 Within continental Europe, there are various schools of exercise therapy for AIS. The Schroth method (originating in Germany), Scientific Exercises Approach to Scoliosis (SEAS; Italy), the Lyon method (France) and the DoboMed method (Poland) are among the most well-known and reported in the literature. However, there is little evidence to support the choice of one form of therapy over another or evidence of their clinical effectiveness and cost-effectiveness.

The rationale for exercise to manage AIS is that a number of underlying impairments in spinal muscular function and postural ability contribute to or accompany the development of curvature and are potentially reversible. Electromyography (EMG) of trunk muscles in AIS patients indicates disrupted patterns of muscle recruitment under static and dynamic conditions, in a broad range of postures. These asymmetries extend to the paraspinal lumbar and abdominal oblique muscles and are associated with a disparity in trunk isometric rotation strength between sides. 18–21 In keeping with differential muscle activity, there are differences in muscle fibre type distribution on the convex and the concave sides of the curve. AIS patients exhibit greater balance control problems and proprioceptive impairments. 22,23 AIS may be associated with distorted body schema, resulting in a mismatch between actual body alignment and patients’ internal bodily representation of the body. 24

Hence, to improve function and reduce or stabilise curvature, exercise programmes must include strengthening of all affected muscle groups; exercises to encourage the appropriate magnitude and timing of muscle activation; proprioceptive elements; and postural/body awareness components. These programmes will require careful tailoring to each individual. There is evidence that such approaches can remediate underlying impairments in EMG activation and strength, although effects may well be limited to curves with a Cobb angle of < 50°. 19–21,25,26

Although there is a theoretical basis for exercise in AIS, there is little robust evidence that it has a significant effect on curve progression. There are relatively few studies investigating this important outcome,15 and only one randomised controlled trial (RCT)27 had been published at the time of the application (March 2011). A small number of prospective cohort studies have attempted to evaluate the European schools of exercise including SEAS and Schroth. 15 Although these studies show a favourable outcome in support of exercises, the research is problematic for a variety of reasons. These include a lack of control group, failure to randomise, the reporting of very small statistically significant but not necessarily clinically important differences in Cobb angle, poor statistical analysis, limited descriptions of baseline characteristics of participants, and no reporting or poor adherence to treatment and the exercise programmes evaluated. 28 Clear evidence that these approaches are beneficial is lacking.

Economic considerations

There is no information on the cost-effectiveness of the various exercise approaches and whether or not they offer a viable alternative to surgery and bracing. Many patients with similar spine deformities report lower scores in physical functioning than the general population. 29 The impact of treatment of illness on health-related quality of life (HRQoL) can be measured with ‘utility’ scores that represent people’s preferences towards a particular health state. The current study used two preference-based HRQoL instruments, namely the Health Utilities Index (HUI) and the European Quality of Life-5 Dimensions (EQ-5D). These systems employ multidomain health status questionnaires completed by individuals to obtain information on self-assessed health status. Preference-based scores for those health states are then calculated using published scoring functions for the two systems. The published scoring functions are based on preferences obtained from random samples of the general population; the Canadian and UK adult populations for the Health Utilities Index Mark 2 (HUI2), the Canadian adult population for the Health Utilities Index Mark 3 (HUI3) and the UK adult population for the EQ-5D. These multiattribute preference-based instruments are significantly less resource- and time-consuming than direct utility or preference elicitation approaches, such as the standard gamble and time-trade-off approaches, which can directly measure the utility that patients attach to their health states. HUI and EQ-5D measures have been shown to be valid, reliable and responsive to changes in health status over time. 30–32

There is no evidence in the literature with respect to comparisons of the EQ-5D and the HUI in patients with AIS. One study by Adobor et al. 33 evaluated the repeatability, reliability, internal consistency and concurrent validity between the Scoliosis Research Society-22 patient questionnaire (SRS-22) and EQ-5D in AIS. Their results showed a moderate correlation between these two instruments. The authors claimed that one of the reasons for this was that the EQ-5D has been validated for use in adult populations with back pain, but not in adolescents with spine deformity (as in the ACTIvATeS study). The advantage of preference-based measures such as the HUI and EQ-5D is that they can generate health utility scores for comparative economic evaluation purposes. In addition, the HUI has been widely used in the literature to describe the self-rated health of youths and young adults. 29,34–36

Relevant evidence with regards to head-to-head comparison of the HUI and EQ-5D measures in adolescents is provided by a recent study by Oluboyede et al. ,37 which attempted to understand the practicality, validity and reliability of using these two measures for adolescents who have experienced a self-harm episode. Their results showed that the HUI had a higher rate of missing data, and adolescents had difficulties interpreting some of its questions. No missing data were observed for any questions in the EQ-5D in the population of 49 adolescents. In a study of children with meningitis38 there was evidence that, although the EQ-5D may be preferable because it has been used in many different patient populations and is the preferred measure of HRQoL in the National Institute for Health and Care Excellence (NICE) health technology assessments, the HUI classifications are more precise and discriminative. This study also indicated that both instruments would produce the same HRQoL weights or utility scores in the meningitis context.

Following structured literature searches we have found no evidence to indicate the cost of AIS in the UK. A study conducted in the USA suggested the overall national cost of AIS to be around US$1.13B (based on 2006–7 prices). 39 Earlier evidence from the World Health Organization suggests that the cost from musculoskeletal diseases in general stems mainly from hospital admissions, physician visits, nursing home services, medications and non-health-related sources. The contribution of these different health-care services to economic costs may vary by musculoskeletal disease, age and sex. Treatment of AIS is often a lengthy process and, therefore, requires considerable contributions from different providers within the health-care system.

Qualitative study

Understanding why patients and their families make the decisions they do about their health care can help us to provide the appropriate treatments. Although parents are often used in research to elicit young people’s experiences, we believed that we needed to seek information directly from the young people in order to determine the feasibility of the proposed intervention. 40 An adult may not give such a useful account of the young person’s experience. 41 This is the first qualitative study to explore the experiences of young people with AIS undergoing an exercise programme. Previous research has focused on young people’s experiences with scoliosis surgery and bracing. 42–45

Identification of new eligible studies

We have rerun the published search strategies to identify new eligible trials and searched databases up to 12 February 2014. A further study was also identified from an e-mail alert of new research from a journal publisher (see Table 1). We attempted to source grey literature using the OpenGrey database (OpenGrey V1.0, Institut de l’Information Scientifique et Technique, Vandoeuvre-lès-Nancy, France).

Full articles of studies that seemed eligible from the abstract were examined independently by two assessors and checked for eligibility based on the original criteria for inclusion.

According to the original Cochrane review criteria, studies were eligible for the review if they were RCTs, quasi-RCTs or observational studies that included participants diagnosed with AIS (defined as at least a 10° Cobb angle) who were older than 10 years but had not reached the end of bone growth.

Studies were excluded if participants had any type of secondary scoliosis (congenital, neurological, metabolic, post-traumatic, etc.). The experimental interventions in this review included all types of SSE that are considered to be ‘specific movements performed with a therapeutic aim of reducing the deformity’. Sports, active recreational activities and generalised physiotherapy were not considered to be specific exercises for the treatment of scoliosis, and studies including these types of activities were excluded.

Control interventions included no treatment, different types of SSE, usual therapy, different doses or schedules of exercises or other non-surgical treatments. Comparison could include exercises versus no treatment; another treatment versus that treatment plus exercises; exercises versus another conservative treatment; exercises versus usual physiotherapy; and comparisons between different types of exercises or different doses/schedules of exercises.

Risk of bias assessment

After identifying eligible studies, a risk of bias assessment was carried out using the criteria published in the original review (see Table 6). This was done independently by two reviewers who then compared findings. Any differences were resolved through discussion.

Each study was then classified as at high or low risk of bias using the same criteria as the previous review. 17 A study was considered to be at low risk of bias if it fulfilled the three key criteria related to randomisation, allocation concealment and outcome assessor blinding, as well as any three of the other criteria.

Data synthesis

Data extraction was carried out independently by two assessors using a standardised form. Data were compared and any discrepancies were resolved through discussion between the two assessors.

Meta-analysis was not performed owing to lack of homogeneity between studies. The variability between exercise approaches assessed, outcome measures used and timings of follow-up was too great to allow us to combine study findings. We assessed the overall quality of evidence for each outcome using an adapted Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach as used in the original Cochrane review of exercises for AIS17 and as recommended by the Cochrane Back Review Group. 46

In accordance with the GRADE approach:

• High-quality evidence was defined as consistent findings among at least two RCTs with low risk of bias that are generalisable to the population in question (consistency is defined as 75% or more of the studies with similar results); sufficient data, with narrow confidence intervals; no known or suspected reporting biases; further research is very unlikely to change confidence in the estimate of effect.

• Moderate-quality evidence was defined as failure to meet one of the factors described in the high-quality definition. Further research is likely to have an important impact on confidence in the estimate of effect and may change the estimate.

• Low-quality evidence was defined as failure to meet two of the factors described in the high-quality definition. Further research is very likely to have an important impact on confidence in the estimate of effect and is likely to change the estimate.

• Very low-quality evidence was defined as failure to meet three of the factors described in the high-quality definition. Any estimate of effect is very uncertain.

• No evidence was defined as complete lack of evidence from RCTs.

Characteristics of included randomised studies

Characteristics of the five randomised studies and the outcomes they measured are described in Tables 2 and 3. 27,49–52 Characteristics of the two non-randomised studies are presented in Tables 4 and 5. 15,48

For randomised studies, samples sizes ranged from 20 participants to 110 participants, and all studies included males and females. The mean age of participants at baseline ranged from 1051 to 15 years. 27 Three studies included participants diagnosed with AIS irrespective of curve type, with the remaining two studies only including participants with specific curves: S-shaped or double curves27 and structural thoracic curves. 51

Most studies used criteria based on the Cobb angle to select patients and there was some variability here. The inclusion criteria in three studies included those patients with curves between 10° and 30°,49 those with curves from 10° to 25°52 and those with curves between 10° and 45°, respectively. 50 Two studies did not define inclusion criteria using the Cobb angle. 27,51

The mean Cobb angle of participants recruited to studies varied between approximately 15°,51 19°,52 25°27 and 30°. 50 One study did not report Cobb angles at baseline. 49

The most common lengths of follow-up were 3 months49,50 and 6 months post inclusion. 27,51 One study followed up participants to 12 months after skeletal maturity was reached (mean time to follow-up was 52 months). 52

Outcome measures varied across the studies (see Table 3). The Cobb angle was the only outcome measured in more than one study,27,51 whereas two other studies also collected additional physical measurements. 15,48 Only one study reported the Cobb angle alongside a patient-reported measure. 52 Two studies did not include the Cobb angle as an outcome. 49,50 One used postural parameters, including craniovertebral angle and spinal balance, along with a functional measure as outcomes,49 whereas the other evaluated back endurance, quality of life and self-efficacy. 50

The studies reported a variety of different approaches to exercises for scoliosis (see Table 2). No one study evaluated the same approach, although there are some similarities between the approaches used. The concept of active self-correction (ASC) underpinned the majority of the approaches,50–52 facilitated by varying degrees of assistive manual therapeutic techniques. Of the remaining studies, one included stretches of tightened structures on the concave side of the curve and strengthening of trunk muscles with and without the inclusion of exercises to correct adapted forward head posture,49 whereas the other evaluated a gymnastic-based exercise programme where participants exercised in asymmetrical postures to correct the S-shaped curve. 27

The amount of time that participants were expected to carry out exercises was variable. Although descriptions of interventions were not always clear, it appears that three studies required participants to carry out daily exercise programmes, although the amount of time spent exercising varied between approaches. 27,49,50 For example, an exercise programme based on the Schroth programme required participants to carry out 30–45 minutes of training per day. 50

Those undergoing global postural re-education attended treatment sessions twice per week. 51 Participants taking part in an ASC programme were required to attend a weekly 1-hour workout session at the institution supplemented by two 30-minute exercise sessions at home per week. 52

The duration of exercise interventions also varied between studies. These varied from 10 weeks49 and 12 weeks,51 to 6 months. 27,50 One study required participants to exercise until they reached skeletal maturity, which resulted in a mean duration of exercise intervention of 42 months. 52

Risk of bias for randomised studies

One randomised study was considered to be at low risk of bias52 and the remaining studies were considered to be at high risk of bias (Tables 6 and 7). 27,49–51

Risk of bias for non-randomised studies

Both observational studies were considered to be at high risk of bias. Both studies were rated as being at high or unclear risk of bias for at least three of the six criteria (see Table 7). Both studies were considered to be at low risk of bias for ascertainment of exposure and completeness of follow-up. The self-selection process used in Negrini et al. 15 is a source of bias because it was based on participant and physician choice; however, it is reported that there were no statistically significant differences between the groups at baseline for any scoliosis parameters, which suggests that risk of bias had been minimised.

Levels of evidence

Respondents

Thirty-three trusts were approached, which involved 106 individual clinicians (three of the 36 trusts were covered by clinicians working at other trusts). A response was received from 19 trusts (78%) and 39 (37%) individual clinicians. Eleven questionnaires had incomplete data, leaving 28 complete questionnaires. All respondents were consultant spinal surgeons who stated that patients with AIS were managed in their department.

Consultants who responded were generally experienced, with the majority having been managing AIS patients for at least 6 years (22/28 respondents). A similar proportion had been performing surgery for at least 6 years (21/28). Approximately half of the respondents maintained a private list of patients (17/28). The median number of consultants providing treatment in departments was 3 [interquartile range (IQR) 3–4]. Results are shown in Table 11.

Patient population

Referrals were received equally from primary and secondary care. Consultants reported rarely taking self-referrals. There was a wide variation in the numbers of patients seen; the mean [standard deviation (SD)] number of new NHS patients with AIS per month was 10 [SD 6.2 (range 2–28) new NHS patients with AIS per month]. Similarly, there was a wide variation in the number of follow-up patients per month [mean 23 (SD 16, range 4–80) follow-up patients per month].

Patient management

A total of 92% (22/24) of respondents stated that most or all pre- and post-pubescent patients were monitored by Cobb angle. A total of 67% and 74% of consultants stated that respiratory function was monitored in no or rare cases in pre- and post-pubescent patients, respectively.

A majority of clinicians reported following-up pre-pubescent patients at 6-monthly intervals (74%), and post-pubescent patients at 12-monthly intervals (63%). Use of radiography mirrored this frequency of follow-up, with a majority of consultants radiographing pre-pubescent patients every 6 months (74%) and post-pubescent patients every 12 months (82%).

Most consultants stated that only selected pre- and post-pubescent patients would go on to have surgery (89% and 96% of respondents, respectively). A total of 60% of respondents said selected pre-pubescent patients were braced, whereas 93% said they either never or rarely braced post-pubescent patients. This was corroborated by reporting that 60% of consultants believed that bracing could help in selected patients, whereas 85% believed that bracing is never or rarely helpful in post-pubescent patients.

Use of physiotherapy for conservative management

A total of 78% of respondents rarely or never referred patients to NHS physiotherapy. The remainder of consultants only referred selected patients. When consultants do refer patients to physiotherapy, they report doing this mostly to NHS outpatient departments.

The majority of consultants believed that physiotherapy could not limit curve progression (65% and 73% of clinicians for pre- and post-pubescent patients, respectively). An even greater proportion of consultants believed that physiotherapy could not reverse curve progression. The remaining consultants believed it can only rarely have an effect.

Involvement in research

Approximately half of the respondents were involved in research for AIS (54%), and a majority were happy for their patients to be involved in a RCT that randomised to exercise or watchful waiting (78%).

Respondents

Thirty-six trusts were approached, which involved 57 individual physiotherapists. A response was received from 28 of the 36 trusts (78%) and from 42 individual physiotherapists (74%).

Five (14%) physiotherapists reported that their trusts did not provide any physiotherapy treatment for AIS patients, despite being listed as a NHS trust that has a consultant providing care for patients with scoliosis.

Of the 37 physiotherapists who responded, eight provided pre- and post-operative care only, seven provided conservative physiotherapy management and 22 provided both.

Respondents were experienced, with the majority having at least 6 years’ experience managing AIS patients (0–5 years, n = 8; 6–10 years, n = 8; 10–15 years, n = 7; 16 + years, n = 5). The median number of physiotherapists providing treatment in departments was 3 (IQR 1–5). Results are shown in Table 12.

Patient population

Physiotherapists reported that the most common referral source was secondary care (n = 20 for most or all patients). Only in rare cases were referrals received from primary care or self-referrals. On average, NHS departments managed three new AIS patients per month [mean 2.8 (SD 2.2) new AIS patients per month] and five follow-up patients per month [mean 5.4 (SD 5.0) follow-up patients per month]. For those departments that did provide outpatient care, a median of five (IQR 4–6) sessions were given, with respondents often specifying that this depended on individual patients. There was a wide variation in the length of time patients were kept on the caseload, ranging from a few weeks up to 4 years (or until skeletal maturity).

The reason for referral to physiotherapy was for exercise prescription. Referrals were unlikely to be received for a curve having a particular presentation (n = 12/19 said never or rarely) or for management/monitoring of bracing (n = 11/19 said no patients), and there seemed to be no discrimination of referrals owing to the age (n = 14/18 said no patients) or sex (n = 16/18 said no patients) of the patients. More than 50% of physiotherapists (16/37) reported that no or few patients were using a brace.

Patient management

In most or all cases, treatments consisted of core stability exercises, strengthening, stretching, postural correction and sensorimotor retraining. Manipulation, acupuncture and electrotherapy were never or rarely used. In most cases, conservative treatments of AIS (i.e. not pre- or post-operative physiotherapy) were provided at outpatient one-to-one appointments. Inpatient sessions or outpatient group sessions were never or rarely used.

Training

Eight (22%) physiotherapists had received training and used specialist scoliosis approaches.

Beliefs about the effectiveness of physiotherapy

Beliefs that physiotherapy and exercise can limit curve progression were mixed. Responses varied between ‘never’, ‘rarely’ and ‘in selected cases’, with no real difference for pre- and post-pubescent adolescents. Few physiotherapists believed that physiotherapy and exercise could reverse curve progression, and then only in selected patients. The most common response to the question of whether or not physiotherapists believed bracing was helpful was ‘only in selected patients’.

Involvement in research

Two physiotherapists (5%) were currently involved in research for AIS. A total of 16 (43%) respondents said they would be happy for patients to be involved in a RCT that randomised them to exercises or watchful waiting, with a number of comments about requiring consultant permission. Ten (27%) respondents were interested in their patients participating in a full trial.

Current practice

Current practice regarding conservative treatment for AIS was assessed as part of a nation-wide survey of orthopaedic consultants and specialist physiotherapists (see Chapter 3 for more details). The results indicate that the main components of conservative management of scoliosis in the UK are ‘watchful waiting’ and, in a minority of cases, bracing. Although some NHS trusts offer other services, including referral to physiotherapy for advice and/or exercises, these are not routinely offered within the NHS in the UK, and survey responses suggest that only a small percentage of patients are referred to physiotherapy. The majority of consultants at these centres rarely or never refer patients to physiotherapy for conservative management of AIS.

Once diagnosed with AIS, patients generally will come under the care of a specialist spinal consultant. Depending on the size of the curve and prognosis regarding potential progression, a monitoring plan will be instituted to maintain regular evaluation of the spinal deformity. Typically, this will occur every 6 or 12 months and will involve some form of imaging, although the timing will alter depending on prognosis and the speed of any changes. For 90% of patients with AIS, monitoring is the only ‘treatment’ they will receive.

Clinical guidelines

We searched for but did not find UK guidelines concerning conservative management of AIS. The Society on Scoliosis Orthopaedic and Rehabilitation Treatment, an international group dedicated to scoliosis treatment and research, has produced a series of papers detailing the results of consensus exercises using Delphi techniques. They state the aims of conservative management to be stopping curve progression; preventing pulmonary dysfunction; treating any pain; and improving appearance via postural correction. 59 A series of recommendations regarding conservative treatment were also made. These include:

• SSE programmes should be used and should include education, postural self-correction and integration of postural correction into activities of daily living (ADLs).

• Exercise programmes should be individualised according to patients’ needs and curve type.

• SSEs should be performed regularly.

No specific recommendations were provided regarding the type of exercises or their exact frequency or delivery method.

Evidence base

The majority of the studies included in the systematic review update described in Chapter 2 were not published when the ACTIvATeS trial intervention was developed. At that time, the evidence base for the effectiveness of exercise in AIS consisted of the Cochrane review published in August 2012,17 which included publication of studies up to 30 March 2011. This review included two studies (154 participants): the RCT by Wan et al. 27 and the prospective controlled cohort study by Negrini et al. 15

In order to ensure that we had the most up-to-date information, we reran the search strategies used in the original March 2011 review. This yielded one new study,51 a RCT of Global Posture Re-education evaluated with 20 patients. All of these were considered to be at high risk of bias in the latest update. The basic exercise approach was broadly similar in two of the studies. However, the exercise intervention used by Wan et al. 27 differs greatly from the consensus within Europe where exercises are used routinely as treatment for AIS (see Table 2 for further intervention details).

The Controlled Trials Register (International Standard Randomised Controlled Trial Number Register) was also searched for any current trials investigating exercises as treatment for AIS, but no further trials were identified at that time.

Expert opinion

A crucial part of the development of the intervention was the advice received by clinicians. Most notably, a consensus meeting was held with 17 specialist physiotherapists from across England to gain further understanding of normal practice within typical NHS clinics and to assist in the design of the exercise intervention. Invitations were made through professional forums (interactive Chartered Society of Physiotherapy), links with large NHS centres and private providers (Scoliosis SOS Clinic, London, UK).

It needed to be feasible to perform the SSE intervention within NHS outpatient clinics, taking into account normal appointment duration and commonly available rehabilitation materials. A list of exercises described in the literature, along with others proposed by various therapists, were examined in detail and decisions made as to which of these were the most important. A consensus was reached through voting on inclusion of individual exercises. The results of this were used to guide the design of the programme used in the feasibility study. Discussions as to what the control intervention should consist of were facilitated by the study team. Feedback regarding the design of the online tools and paperwork associated with the trial were also provided by patients and clinicians.

Content of the exercise intervention

Each participant was assessed by their physiotherapist and given an individualised exercise programme based on the presenting spinal deformity. The first step of the exercise programme was to teach the participant to correct their posture (ASC to reduce the spinal deformity). Participants were also given additional exercises that progressively challenged their ability to maintain the corrected posture by altering position, adding load or resistance, adding movement or distractions and incorporating it into activities of daily living. Exercises to address any secondary impairments (e.g. balance, ROM, strength) were also included to be used where required.

Alongside the exercise programme and adherence strategies, the physiotherapist also provided education, advice and support for the participant and family, similar to that provided in the control intervention. All treatments provided during each session were recorded in a detailed log.

Adherence

Adherence is defined as how closely a patient’s behaviour is aligned with a prescriber’s recommendations. 64 Adherence with any exercise programme is vital to ensure that there is sufficient dosage received in order to effect a change. Unfortunately, patient adherence with home treatment programmes is typically low. Reasons for this include a lack of understanding of how home treatment may directly benefit patients personally; patients’ lack of confidence in their own ability; intrusion into normal life; and various other barriers (both real and imagined). This may be compounded by the age of patients with AIS (i.e. adolescents).

Many of the approaches in Europe provide extensive supervised sessions. However, it is not feasible within the UK NHS to provide supervision for the duration and frequency reported in the literature (e.g. 5 days/week for 4 weeks65). We aimed to maximise adherence to the prescribed exercise regimen by integrating evidence-based adherence strategies66,67 into the exercise programme to counter these factors, utilising behavioural methods and techniques including exercise contracts, an online exercise diary and an online chat forum (see Appendices 4 and 5). 68 The behavioural strategies have been successful in other areas of health intervention for adolescents. Exercise diaries are effective in improving adherence to home exercise programmes, particularly if the participant is aware that the diary is being monitored by a health professional. 69

The online forum was designed as a means by which the participants in the exercise intervention arm could communicate with each other and discuss issues both related and unrelated to AIS as well as their involvement in the ACTIvATeS study. Participants could also use the forum to ask questions of the physiotherapists. The forum was moderated by the central study team.

The online exercise diary was part of the same website as the forum. It consisted of a calendar and a series of drop-down menus from which physiotherapists could select exercises for the home exercise programme. Each exercise was represented by a photograph or a video and came with pre-loaded instructions, which could be modified. Participants were then able to access the online diary at home and check-off exercises when they had performed them.

Summary of scoliosis-specific exercise programme intervention

The programme involved between six and nine appointments spread over 6 months and was designed following a review of the available literature and a professional consensus meeting of specialist UK physiotherapists. We added a number of additional elements designed to increase long-term effectiveness, including standardised progression and specific adherence strategies. The programme included components to address all the aims described earlier in this section. An individualised programme could be created by selecting and progressing each exercise according to each participant’s capabilities.

The programme was performed at home as well as in the clinic in order to stimulate psychomotor or muscular adaptation. 70 The experimental intervention can be summarised as follows:

• SSE programme involving between six and nine contact sessions with the physiotherapist spread over 6 months

• information and advice to patients about their condition and self-management

• an emphasis on teaching the participant to be able to achieve ASC of spinal deformity

• a menu of exercises aiming to:

  • achieve maintenance of the self-corrected position during movement including integration into ADLs

  • address secondary factors including restricted range of movement, strength, sensorimotor and balance deficits that may inhibit ASC

• a home exercise plan with exercises being performed daily

• a standardised protocol for progression and regression of exercise difficulty

• strategies to improve programme adherence

• participants continued to attend for orthopaedic review as per standard practice at their centre.

Attendance and treatment logs

Physiotherapists were asked to complete treatment for all participants within 6 months of randomisation in order to gauge short-term effects. Fifty-eight participants took part in the randomised pilot feasibility study. Treatment for participants in the exercise arm was completed, on average, within 6 months of randomisation at two sites and within 9 months of randomisation at a further site (Table 13). Final figures for this will not be known until those participants still receiving treatment have completed the course.

Table 14 presents attendance rates for the different treatment arms by centre. Table 15 details how many sessions were attended by the participants in each arm.

All 29 control arm participants had completed their treatment by report date, with only one having more than one session (two sessions in total). None of the control participants declined further treatment or failed to attend.

Of the 29 exercise arm participants, 16 had either partially or fully completed treatment by report date (including three who declined further treatment), 10 were still receiving treatment, two had not responded to attempts to book an initial session and one remained unclassified.

Three out of the four NHS trusts were able to commit to up to nine sessions over a 6-month period within their current NHS resources. One trust was unable to manage between six and nine sessions for exercise programme participants, owing to limited resources. This was reflected in the median number of treatment sessions for the exercise arm participants, which was seven or higher at the two trusts with participants who had completed treatment sessions but only 3.5 at the trust with resource issues.

It is expected that the overall median number of sessions (currently five) will increase to above the pre-defined minimum threshold of six sessions once the fourth site completes treatment of their exercise arm participants.

All sites were asked to hold the initial treatment session within 4 weeks of randomisation, and, ideally, within 2 weeks. Three of the four sites were successful in complying with this request (see Table 13). At two of the four sites, patients attended the initial treatment session at a median time of approximately 2 weeks from randomisation date. At one trust, the initial treatment session occurred at a median time of between 3 and 4 weeks. Participants were seen initially at a median time of 6 weeks at the remaining site. There was no difference between arms for time to initial attendance.

Table 16 details the content of each treatment session as recorded by the physiotherapists on the treatment logs for each participant. Very high figures for provision of the different information categories were recorded for both the control and exercise arms. The low figures for brace information (provided to just over 50% of participants) reflect the low number of participants being braced. The core component of the exercise programme (ASC) was provided to 94% (15/16) of exercise arm participants, with high figures for stabilising, balance and abdominal (core) strengthening exercises. In terms of treatment contamination, one control participant had a previous exercise programme reviewed and progressed when she attended her trial assessment and advice session.

Use of the online exercise diary

At baseline, the vast majority (98%) of participants had daily access to the internet in their home, suggesting that there would not be an issue with accessing the online exercise diary.

Of the 18 participants who had completed the treatment process, 14 had used the online exercise diary (Table 17). Three participants reported problems with access to the online facilities owing to incompatibility issues with some internet browsers. Solutions to these problems were provided, although these participants in general either did not use the diary or reported low usage rates.

There was wide variation in the number of exercises prescribed, and, therefore, the number of exercise days counted for the 16 participants who were partial or full treatment completers (range 51–1164 exercise days counted). The six participants who completed treatment sessions (attended between six and nine sessions) completed, on average, 80% of the prescribed exercises.

The 10 participants who partially completed treatment sessions (attended < 6 sessions) completed, on average, 24% of the prescribed exercises.

Use of the online forum

Only four of the exercise programme participants used the online forum, providing seven posts. Attempts were made to increase activity with various challenges and information requests but this had little impact. Feedback from the qualitative study indicated that participants were reluctant to use the forum owing to the presence of other adults and also because of the availability of other social media sites.

Therapists’ ratings of adherence and ability to correct curve

After the last treatment session physiotherapists were asked the following:

Overall, how well do you think the participant completed the programme according to what was agreed? (On a scale of 1 to 7, where 1 = not at all and 7 = completely.) Physiotherapists’ mean (SD) rating of adherence was 5.6 (SD 1.4).

Feedback on control intervention

We asked all of the participants of the control intervention to provide written feedback on their experience of the trial intervention and to return this to the study team in a pre-paid envelope. We asked the participants and their parents, ‘How do you feel about the treatment that you received as part of the trial?’ and ‘How do you feel about the treatment that your child received as part of the trial?’, respectively.

We received five forms from the 29 participants who had completed their treatment. All recorded favourable comments about the control intervention, although some indicated that they would have preferred to have had the exercise intervention if they could have chosen.

Design

A multicentre randomised feasibility study.

Settings

Four centres were selected from 35 NHS trust hospitals specialising in scoliosis management and surgery. The 35 trusts were those listed by SAUK/British Scoliosis Research Foundation as having clinicians actively managing and performing surgery for patients with scoliosis. We originally planned to run the randomised study in three of these NHS trusts. The sites were chosen to represent a range of the types of hospital likely to be eligible to recruit for a main study to ensure we got a representative estimate of recruitment rate and baseline variability. Some of the three sites we had worked with before, others we had not worked with previously. Some sites had not participated in research previously and had no specialist physiotherapy service. Sites were of a median size for scoliosis centres listed by SAUK/British Scoliosis Research Foundation. As a result of delays in obtaining governance approvals, we added an additional site to ensure that we reached our sample size target. The fourth NHS trust was chosen based on collaborations with researchers and clinicians that had evolved in the early stages of developing the study as well as on the availability of an ISIS system at the centre.

Participants

Inclusion criteria:

• 10- to 16-year-olds with AIS

• mild to moderate AIS, defined by a Cobb angle of between 10° and 50° (measured radiographically).

Exclusion criteria:

• individuals who had had previous surgery or were on a waiting list for spinal surgery within the next 6 months

• individuals with non-idiopathic scoliosis, for example congenital malformations, syringomyelia, neurofibromatosis, spina bifida, polio or cerebral palsy.

Other treatments including bracing and previous physiotherapy were not exclusion criteria.

Interventions

The control intervention consisted of one or two sessions of education and advice delivered by a physiotherapist. This was compared with an experimental intervention consisting of between six and nine sessions of education, advice and a programme of SSEs delivered by a physiotherapist, supported by a daily home exercise programme. Full details of the interventions and their development are provided in Chapter 4.

Procedures

Randomised feasibility study procedures are summarised in a flow chart in Appendix 1.

Baseline assessment

Baseline assessments were conducted by research clinicians who had received training in the measurement protocol. The assessment consisted of self-report questionnaires and physical measurements to capture characteristics, as shown in Table 18.

TABLE 18 - Outcome measurements

N, no; PODCI, Pediatric Outcomes Data Collection Instrument; SAE, serious adverse event; SAQ, Spinal Appearance Questionnaire; Y, yes.

Type and method of measure	Measurement (units/scale)	Time points taken
Primary outcomes
Spine curvature	Primary Cobb angle from radiograph (°)	Baseline and 6 months
Characteristics
Age, sex, ethnic group	Self-report questionnaire	Baseline
Height, weight, body mass index	Standing and seated height (m) Weight (kg)	Baseline and 6 months
Pubescent status	Onset of puberty and age of occurrence	Baseline
Skeletal maturity	Risser classification (0–5) from radiograph Pre or post menarche	Baseline and 6 months
Treatments received	Current bracing status Previous exercise treatments received	Baseline and 6 months
Secondary outcomes
Disease-specific function	SRS-2275	Baseline and 6 months
Participant perceived spinal deformity	SAQ76	Baseline and 6 months
Generic function	PODCI77	Baseline and 6 months
HRQoL	EQ-5D HUI	Baseline and 6 months
Coronal and sagittal curve balance	Coronal and sagittal balance calculated from radiographs	Baseline and 6 months
Topographical spinal deformity	ISIS-2 system measurements	Baseline and 6 months
Treatment preference	Control or experimental intervention (with the awareness these were randomly allocated)	Baseline
Global perceived change in scoliosis	Seven categories ranging from vastly improved to vastly worsened	6 months
Perceived benefit or harm from study treatments	Five categories ranging from substantial benefit to substantial harm	6 months
Treatment satisfaction	Seven categories ranging from extremely satisfied to extremely dissatisfied	6 months
Exercise performance	Participant-reported frequency Online exercise diary output	6 months
Brace wearing status	Currently wearing brace (Y/N)	Baseline and 6 months
Progression to surgery	Listed for or received surgery	6 months
Resource use for economic analysis		6 months
AEs	AEs or SAEs	During treatment and 6 months

We anticipated that the primary outcome measure for a main study would be progression/stabilisation of curvature quantified by Cobb angle. This is measured using standing posteroanterior radiographs of the full spine and assessing the lateral curvature of the spine. To do this, the most tilted vertebra above and below the apex of the curve is identified. The angle between intersecting lines drawn perpendicular to the top of the top vertebra and the bottom of the bottom vertebra is the Cobb angle (Figure 2).

FIGURE 2.

Measurement of Cobb angle.

Other measures that we collected from radiograph were the Risser classification and coronal and sagittal spinal balance. Risser classification divides the steps of ossification and fusion of the iliac apophysis into six stages (0–5), where stage 0 describes no apophyseal ossification and stage 5 represents complete ossification and fusion. This provides an indication of skeletal maturity, which in turn helps to predict growth potential and velocity and, therefore, likelihood of curve progression. 78 Risser classification is probably the most widely used indicator of skeletal maturity, although correct classification relies on a sufficient radiograph of visualisation of the entire pelvis.

Coronal balance was evaluated by measuring the horizontal distances between two lines in millimetres (mm); one vertical plumb line is drawn downwards through the centre of the C7 vertebral body and another vertical line is drawn upwards from the centre of the S1 vertebral body. The spine was considered to be balanced when the difference between these two lines was < 40 mm. 79 Sagittal balance was evaluated by measuring the distance between a vertical line drawn up from the posterosuperior aspect of the S1 vertebral body and the plumb line down from the centre of the T1 vertebral body. Sagittal balance can be divided as negative or neutral/positive. 79

Details and timings of most recent radiographic images were obtained from patient records. Cobb angles, spinal balance distances and Risser grades were calculated/recorded by spinal consultants at individual centres.

Some NHS trusts have invested in topographical measurement equipment that allows for assessment of a representation of the Cobb angle and rib hump by means of a digital photographic image rather than exposure to X-rays. The ISIS-2 system was developed as a low-cost method to reduce the dependence on serial radiography and to reduce radiation exposure when monitoring spinal deformity. 80 The system produces a version of the Cobb angle (although this is known to not correlate completely with radiographic versions) and volumetric asymmetry. One of the study objectives was to evaluate the feasibility of using the ISIS-2 topographical measurement system in all centres in the main study.

Disease-specific quality of life was measured using the SRS-22, the most widely used patient-reported outcome measure, which contains 22 questions covering five domains (function/activity, pain, self-perceived image, mental health and satisfaction). It has been demonstrated to be valid, reliable and responsive to change, with a minimally clinically important difference of 13 points out of 100 for the sum score. 81

Patient-perceived spinal deformity was measured using the Spinal Appearance Questionnaire (SAQ). In its refined format, it has two domains – appearance (10 items) and satisfaction (4 items) – and has been shown to be valid and reliable,82 although a minimally clinically important difference has yet to be derived.

A generic orthopaedic function measure was also collected in the form of the Pediatric Outcomes Data Collection Instrument (PODCI). This covers eight domains (upper extremity function, transfers and mobility, physical function and sports, comfort, happiness, satisfaction with symptoms, expectations of treatment and global functioning) on an overall scale of 0–100, with 100 being the highest level of function. It has been shown to have good psychometric properties. 77,83

Clinical reporting/analysis

Pilot RCT data are summarised and reported in accordance with Consolidated Standards of Reporting Trials (CONSORT) guidelines for RCTs where possible. 85 The purpose of the statistical analysis was not to make comparisons between groups but to compare against the progression criteria we set. We report the number of participants approached, the numbers meeting the eligibility criteria, the numbers agreeing to randomisation, the numbers attending the trial treatment sessions, the overall proportion of people who attended 60% of the sessions and use of the online exercise diary. Completion of measures is recorded alongside descriptive data. Only an observed data set has been used, as imputation was not appropriate.

Withdrawals from treatment, withdrawals from the trial, AEs and serious adverse events (SAEs) are reported.

Health economics reporting/analysis

The study collected data on the use of services within the health-care system and other sectors of the economy, as well as on broader costs to society, in an attempt to capture relevant components that comprise the overall cost of treating AIS. Key items included data completeness and the costs of the control and experimental interventions.

Data were collected from information gathered via the study participant questionnaires completed by participants, their parents and research physiotherapists at baseline and 6 months post randomisation. Questionnaires captured the frequency of use of community-based health and social care services, number and duration of admissions to inpatient wards, number of diagnostic tests, use of outpatient services (classified as orthopaedic/spinal, paediatric, physiotherapy, orthotics/bracing), medication use and equipment provided, indirect costs borne by parents and carers as a result of attending hospital visits, as well as direct non-medical costs (including travel expenses), attributable to the child’s health state. The parent-completed questionnaires recorded employment status, weekly working hours, self-reported annual income and work-days lost as a result of the child’s health state.

To measure effectiveness, two multiattribute utility measures were piloted in the study, the EuroQol EQ-5D-3L questionnaire and the HUI, at baseline and 6 months.

The practicalities and difficulties associated with an assessment of the cost to providers, individuals and, more broadly, to society entailed by the introduction of the exercise intervention, along with the identification of appropriate sources of unit cost data, were addressed. A NHS and Personal Social Services perspective was adopted for the costing component of the feasibility study. 86

Data on consumable use were taken from the study records. Similarly, physiotherapist training time, length of treatment sessions and programming input were obtained using primary research methods.

Valuation of resource-use cost data

The unit costs for resources used for the implementation of the exercise programme were mainly obtained from the ACTIvATeS feasibility study records, apart from the unit costs of physiotherapists’ time, which were obtained from the Personal Social Services Research Unit’s Unit Costs of Health and Social Care 2013 cost compendium87 (Table 19; see also Tables 39–41). Unit costs for the broader resources used by adolescents during the study were obtained from a range of primary and secondary sources (see Table 1). Estimation of unit costs used followed recent guidelines on costing health and social care services as part of health economic evaluations. Unit costs for hospital- and community-based health and social care services were derived from the NHS Reference Costs (2012–13) (Department of Health 2013)88 and the Personal Social Services Research Unit’s Unit Costs of Health and Social Care 2013 cost compendium. 87 A list of all medications used was collated, and costs for individual preparations related to idiopathic scoliosis were identified for inclusion in a prospective trial-based economic evaluation. This list included musculoskeletal drugs, central nervous system drugs, endocrine system drugs, skin drugs, ear drugs, nose and oropharynx drugs, obstetrics drugs, gynaecology and urinary tract disorder drugs, and nutrition and blood agents. The drug prescription costs were obtained from the Health and Social Care Information Centre Prescription Cost Analysis database. 87 All unit costs were expressed in GBP (£) and valued at 2012–13 prices.

Calculation of health utilities

The EQ-5D is the generic, multiattribute, preference-based measure preferred by NICE for broader cost-effectiveness comparative purposes. The EQ-5D consists of two principal measurement components. The first is a descriptive system, which defines HRQoL in terms of five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Responses to each of these dimensions are divided into three ordinal levels – first, no problems; second, some or moderate problems; and third, severe or extreme problems – generating a total of 243 possible health states. Utility scores can be produced from the responses to the descriptive system using a UK-specific tariff,89 calculated from a time-trade-off study of 3336 people from the UK general population, taking values between –0.594 and 1.0. The second measurement component of the EQ-5D, the vertical visual analogue scale (VAS), which ranges from 100 (best imaginable health state) to 0 (worst imaginable health state), was also included in ACTIvATeS.

The HUI is a family of generic health profiles and preference-based systems for the purposes of measuring health status, reporting HRQoL and producing utility scores. The HUI 15-item questionnaire for usual health status assessment completed by adolescents in the study covers both the HUI2 and the HUI3 health status classification systems. The HUI2 and HUI3 health status classification systems are complementary. Together they provide descriptive measures of ability or disability for health-state attributes and descriptions of comprehensive health status. Utility scores have interval-scale measurement properties. The multiattribute scales of overall HRQoL are defined such that the score for dead = 0.00 and the score for perfect health = 1.00. Both HUI2 and HUI3 allow for negative scores of HRQoL that represent health states considered worse than dead. The lowest possible HRQoL scores are –0.03 for HUI2 and –0.36 for HUI3. 30,90,91

Ethical approval and monitoring

Sites and therapists

Four centres recruited patients for the study:

1. Frenchay Hospital, Bristol, UK

2. Royal Orthopaedic Hospital, Birmingham, UK

3. Nuffield Orthopaedic Centre, Oxford, UK

4. James Cook Hospital, Middlesbrough, UK.

Seven physiotherapists delivered interventions. These senior physiotherapists had a range of experience in treating scoliosis (9–40 years qualified, Agenda for Change bands 6–8a), with a minority having taken specialist training in prescribing SSEs.

Screening of patients

Screening and recruitment took place between December 2012 and October 2013. Clinic screening data are summarised in Table 20, and subsequent telephone screening is summarised in Table 21. There are some missing data at case or item level (age).

Screening logs recorded 1632 patients being screened in the spinal clinics of the four centres. Of these, 177 (11%) patients were eligible. The range of proportions of eligible patients between the centres varied from 7% to 83%, although the upper range is somewhat artificial as a result of the strategic screening that was conducted at the James Cook Hospital, Middlesbrough. Of those who were eligible to participate, 25% were 10- to 13-year-olds and 75% were 14- to 16-year-olds.

The main reason for ineligibility was that the patient did not have a diagnosis of AIS (≈60% of patients). Overall, 76% (n = 135, between-centre range 65–81%) of eligible patients were willing to be contacted by the research team. Of the 18 patients not willing to consider participation, the main reason given was lack of time (n = 11, 61% of patients). No patients gave the reason of not being willing to travel for study.

Of the 148 patients logged to be contacted by telephone (note that missing data mean that this figure is different from the 135 recorded in the clinic screening data), just over half (n = 82, 55%) were successfully contacted and the vast majority of these were still eligible (n = 77, 93%). The 25%/75% split between the age categories persisted. A total of 54 patients are recorded as having a clinic appointment arranged, highlighting the issue of some missing data (as we have records of 60 attending clinics and 58 patients being randomised; see Figure 5).

Seventeen patients were not willing to arrange a research clinic appointment, with the reasons stated varying from not having time to commit to the study (n = 5), not being willing to travel (bearing in mind that some of the patients travel a long way to the specialist centres; n = 6) and other miscellaneous reasons (n = 4). One participant gave the reason of not being willing to be randomised. There were no major differences between older and younger age categories in terms of willingness to be contacted and willingness to arrange a research clinic if successfully contacted. A slightly higher proportion of 10- to 13-year-olds were unable to be contacted [n = 12 (44%), compared with n = 26 (30%) for 14- to 16-year-olds].

Recruitment of participants

Sixty patients were booked for a research clinic appointment between January and October 2013. Of these, one patient was unwilling to be involved in the study and one had been placed on a surgical list between agreeing to attend and the research clinic appointment. Therefore, 58 patients consented and were recruited to the study.

The original timetable detailed the start of recruitment as September 2012 but delays were experienced with contracting and governance approvals, so that the first screening began in December 2012. (Governance approvals took between 5 months and 6 months to obtain.) Figure 3 illustrates the expected and observed overall recruitment of participants by month. Figure 4 illustrates recruitment by individual sites by month.

FIGURE 3.

Cumulative monthly overall recruitment.

FIGURE 4.

Cumulative monthly recruitment by individual sites. FRE, Frenchay Hospital, Bristol, UK; JCH, James Cook Hospital, Middlesbrough, UK; NOC, Nuffield Orthopaedic Centre, Oxford, UK; ROH, Royal Orthopaedic Hospital, Birmingham, UK.

Table 22 displays projected and actual recruitment by centre. The mean projected recruitment rate per centre per month was 1.4 (50/36), whereas the actual recruitment rate per centre per month was 1.8 (58/33).

Randomisation

Participants were randomised in equal proportion to the advice and education and exercise programme arms (n = 29 in each). Table 23 demonstrates that randomisation was well balanced at each centre.

Figure 5 presents the CONSORT flow diagram for the pilot RCT.

FIGURE 5.

The CONSORT flow diagram.

Baseline data

Baseline health economic measures

A total of 57 adolescents completed the HUI as part of the baseline questionnaire, 28 in the usual-care arm of the feasibility study and 29 in the exercise programme. For this cohort HUI, EQ-5D and EQ-5D VAS scores are all approximately 0.8, or 80%, of the maximum score (Table 29) for both treatment arms.

Follow-up data

Allocation concealment

Research clinicians were asked to record at the 6-month follow-up whether or not they had been unblinded and the reasons for this. For 8 out of the 24 participants (33%) followed up at 6 months, the research clinicians reported being unblinded. Reasons for this included being told by parent (n = 3), told by participant (n = 2) and implied by participant (n = 2).

Health economics outcomes

Quality assurance

We conducted a quality assurance visit for each research clinician and physiotherapist at each of the four sites to ensure research and treatment protocols were being adhered to. All visits were graded as satisfactory.

Recruitment

Our first aim was to determine the rate of recruitment and if pre-specified recruitment rate could be achieved. Once delays with governance approvals had been addressed, the recruitment rate exceeded the original target at three of the four sites and, overall, equated to almost two patients per centre per month on average. Recruiting to a trial that randomises patients to either a control group of advice and active monitoring or a programme of SSEs is feasible.

The 58 participants recruited to this feasibility study will undergo a 12-month follow-up as part of a Doctor of Philosophy (PhD) project carried out by one of the ACTIvATeS team. Should the modifications to the trial methods and interventions be minor, these participants could be retained for analysis as part of a main trial (we have ethical approval to follow them up annually should this be the case). The first participant was due to have their 12-month follow-up in February 2014 and, therefore, their data would expire in February 2015.

Randomisation and interventions

The second objective was to determine the acceptability of randomisation and the compliance to allocated treatment arm (60%). Randomisation was equally distributed between the arms, well balanced at each of the centres, and characteristics of participants in each arm were similar, indicating that the procedure worked well. All participants of the control intervention attended at least one session, indicating that the process of blinding the participants to allocation until the initial physiotherapy appointment worked well. Maintaining blinding for outcome assessors is always challenging when participants are unblinded. Implementing a formalised reminder system to participants prior to follow-up would reduce the incidence of this for a main study.

Adherence to attending treatment sessions and performing home exercises in the exercise programme arm of the study was more variable, although where physiotherapists had the resources to provide at least six individualised sessions a majority of patients attended. Some considerations should be given to whether or not adherence could be increased through small alterations in the format of the intervention; for example, participants may benefit from telephone follow-up contact. Data from the treatment logs and quality assurance visits suggest good adherence by the physiotherapists with the treatment protocols of each arm.

This study has been conducted at a time when there is potential for conservative clinical practice to change more rapidly than normal following the robust bracing study reported by Weinstein et al. in 2013. 14 This may result in an increase in the number of patients offered bracing as a conservative treatment, which may impact on the effect or the ability to estimate the effect of SSE interventions. There were no changes in bracing status for any of the participants who had completed follow-up at the time of freezing the database for purposes of reporting. Currently, we maintain the recommendation given prior to conducting the study that sensitivity analyses should be undertaken to investigate the effect of bracing on results of effectiveness of SSE.

Data collection

We aimed to determine data quality and completeness from the feasibility study. Data quality and completeness were generally very good, most likely as a result of the face-to-face data collection in research clinics. From the quality assurance visits, evidence suggested that the training in obtaining consent and data collection for research clinicians was sufficient to conduct the baseline and follow-up evaluation with accuracy and precision.

The one area where there was substantial incomplete data was for some of the radiography measures, particularly the Risser grading. Generating and implementing a clear imaging protocol would reduce this occurrence for a main study.

Estimating variability of sample for main trial sample size calculation

We aimed to estimate the SD of a sample recruited from a generalisable selection of secondary care settings. For this cohort of 58 patients, the baseline SD for the Cobb angle was 10°. This was lower than the SD we anticipated from the previous literature and stated in our original application of 15°, providing the possibility that the estimated sample size could be reduced. However, to measure our secondary outcomes and conduct an economic analysis it is likely that our original estimate of 400–500 participants is still accurate.

Feasibility of using the ISIS-2 system as outcome measure

We stated that we would explore the feasibility of using the ISIS-2 measurement system in the main trial owing to its advantage of reducing exposure to radiation. Discussing the validity of the measurements with clinical experts and the ISIS-2 creators highlighted that the measurements generated by the system are not directly analogous to the radiographic Cobb angle and hence clinicians and researchers do not place as much confidence or emphasis in this measure.

There were also difficulties with capturing these data; at times the system was not working or staff were unavailable to run the system. Furthermore, the four sites involved in the feasibility study constitute the majority of centres that have the ISIS-2 system currently in place. If this were to be used as an outcome measure for a main trial it would be necessary to purchase approximately 20 units, which would incur at least an additional £150,000.

We suggest that the ISIS-2 system is not worth investing in further for the purposes of a main study, but we would see it as a beneficial secondary outcome at sites where an ISIS-2 system is already in place and being used as part of routine clinical monitoring.

Health economics and the cost of the interventions

The current feasibility study has shown that is it possible to collect the data necessary for a full cost-effectiveness analysis of the ACTIvATeS exercise intervention compared with usual care. There were no practical issues in accessing information to collect and evaluate the elements of the exercise programme per se. Similarly, participant inputs on use of health and social care services and broader service use, and measurement of preference-based HRQoL outcomes, were accessible through the participant questionnaires. It is a requirement for both clinical effectiveness and cost-effectiveness analyses that follow-up be of sufficient length to capture skeletal maturity and potential avoidance of future surgery to evaluate potential offset costs of the exercise intervention.

Study limitations

Imprecision is inherent in data from small samples and, therefore, it is inappropriate for the data generated from this feasibility study to be used to carry out inferential statistical comparisons. This feasibility study was not conducted to evaluate efficacy or safety definitively, although we have proposed that it could be an indicator for safety. To date, there is no evidence of harm occurring as a result of the trial interventions.

We have delivered a report of a study evaluating the feasibility of conducting a definitive RCT of SSEs for idiopathic scoliosis. Some uncertainty remains in some of the conclusions, as only half of the cohort have completed the short-term follow-up. Data collection is ongoing. This is particularly important for assessing progression rates to surgery as set out in the original protocol.

Completing a feasibility study successfully is not a guarantee of main trial success. Despite having worked with some of the sites in previous research projects, considerable time and effort were required for the governance approval, set-up and maintenance of the four pilot sites and this may be more difficult with the research-naive and new sites that would be required for a large multicentre main trial.

Participants

Sixteen people involved in the randomised feasibility trial, from the three original pilot sites (Oxford, Birmingham and Bristol), were interviewed by FT:

• Six girls (age range 10–16 years), three of whom had significant pain before the trial (one had no improvement in pain). Table 45 presents baseline characteristics for qualitative and overall cohorts. The qualitative study cohort was of similar age. All were female, all were white, and all were similar in terms of sexual maturity but at lower risk of curve progression (from Risser grading, although the large number of missing data introduce considerable uncertainty) and with smaller curves. They had similar scores on the SRS-22 questionnaire (disability) and SAQ (self-image).

• Six parents; two interviews were with mothers on their own and two interviews were with mother and father together.

• Four physiotherapists (two experienced in providing the exercise intervention for patients with AIS and two with more limited experience).

Interview duration ranged from 35 minutes to 2 hours.

This section reports findings from the nested qualitative study relevant to a future trial. Findings are organised into:

1. outcomes

2. being in the ACTIvATeS trial

3. trial components

4. additional themes impacting on a future trial (experience of health care, specific worries, unmet information needs).

Outcomes

This section relates to trial outcomes:

1. Motivations for being in the trial (desired outcomes).

2. What are the important outcomes from AIS interventions (relevant outcomes)?

3. Positive outcomes from the trial (actual outcomes).

Being in the trial

This section focuses on relevant experiences of being in the trial for physiotherapist, parent and child.

Trial components

This section describes physiotherapists’, children’s and parents’ perceptions of the outcome measurement (questionnaire and physical testing) and trial components (diary, study information pack, goal-setting, patient forum, exercises, contracts).

Additional themes impacting on success of interventions for adolescent idiopathic scoliosis

The findings highlighted several areas with a potential impact on the success of interventions for AIS and on recruitment into a larger trial:

1. experience of health-care services

2. specific concerns related to AIS

3. information needs and accessing information about AIS.

The exercise intervention

The overall feedback from all participants about the exercise intervention was that it was acceptable. Some physiotherapists expressed concerns over the complexity of the exercises but this was not reflected in the experiences of the patients who appeared to cope well with the programme. No indication was given by the young people or parents that it was too onerous or difficult to perform but, instead, feedback suggested that they enjoyed doing the exercises and benefited from them. Elements of the intervention designed to increase adherence with the programme, including the online diary, goal-setting and contract, were generally viewed in a positive light. One suggestion was made to include text messages to remind participants to do their exercises. The one element of the online resources that was not utilised was the forum, and participants gave examples of other places they had sought peer support, which suggests it was not needed.

Need for an active approach

The findings support the usefulness of a more active approach to the management of chronic conditions in adolescents. ‘Feeling in control’ was an important outcome described by all participants. Physiotherapists described the difficulties of negotiating the disappointment of families who were allocated to the non-intervention arm, which they regarded as not ‘giving you a chance to improve’. This could potentially threaten the validity of treatment outcomes if those in the comparison groups perform worse on the outcome measures because they do not consider that they are ‘doing something’ (resentful demoralisation). Participants describe how they would try other interventions rather than ‘do nothing’ in the comparator arm. We did not interview patients in the non-exercise group and should consider what people in this group are doing beyond ‘watchful waiting’.

Modifiable outcomes: ‘bony curve’ or ‘posture’

The findings also raise queries about the participants’ (both physiotherapists’ and families’) expected modifiable outcomes of the trial. Participants make a distinction between:

1. ‘bony curve’, which they perceive as not modifiable (with the exception of one family)

2. aesthetic or ‘postural’ changes, which are modifiable and important.

This is further complicated by the difficulty of actually measuring:

• changes in the ‘bony curve’

• habitual changes in postural change

• consciously controlled changes in posture (e.g. when having radiography, a person can adapt their posture).

Trial procedures

The participants and their parents were generally happy with taking part in physical measurements and completing questionnaires. There were some suggestions that the questionnaires were long and repetitive, and one participant who wore a brace was unsure if the questionnaires were related to her curve or her brace.

Fitting service around families

All participants discuss the fundamental difficulties of fitting treatments around the commitments of school, often at a crucial time at school.

Unmet information need

The findings suggest that hearing other people’s stories and experiences about their scoliosis would be a useful adjunct to improve the experience of families with AIS.

Training to support positive outcomes

The findings suggest possible areas for further training to support a future trial:

1. ‘The family is the patient’: physiotherapists recognised the importance of a holistic approach to the family, for example the ability to empower families to face worries about the future and the impact of scoliosis on school and life. They recognised that working with adolescents required skills in this area that they had developed during the trial.

2. Accurate information about scoliosis. Physiotherapists and families focused on the importance of accurate information about scoliosis. Families in particular wanted to know more about the potential paths so that they could prepare for them (e.g. what are the odds of needing surgery?).

3. Physiotherapists discussed the importance of mentoring and learning ‘on the job’ as part of training. Those inexperienced in providing exercises for AIS developed skills ‘on the job’.

4. Physiotherapists could find it challenging to set goals for patients who had no pain or functional limitation.

5. Issues related to research: equipoise and standardisation of treatment protocols.

Limitations

From the comparisons of characteristics of the qualitative participants to the whole cohort, it is apparent that there are important differences. We cannot know what the experience was for boys and for patients from ethnic minority groups. There was limited exploration of the experiences of the participants of the control group through written feedback. These should be explored in a further qualitative study.