Efficacy and Mechanism Evaluation

Volume 11 (2024)

19 issues

Extended Research Article

Clinical effectiveness of subsensory sacral neuromodulation in adults with faecal incontinence: the SUBSoNIC crossover RCT and mechanistic study

Paul F Vollebregt
,
Yan Li Goh
,
Claire L Chan
,
Thomas Dudding
,
Paul Furlong
,
Shaheen Hamdy
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 19
  • Published:
    21 November 2024

Extended Research Article

The efficacy of sildenafil therapy in dismal prognosis early-onset intrauterine growth restriction: the STRIDER RCT

Andrew Sharp
,
Christine Cornforth
,
Richard Jackson
,
Jane Harrold
,
Mark A Turner
,
Louise Kenny
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 18
  • Published:
    31 October 2024

Extended Research Article

Infliximab versus alpha interferon in the treatment of Behçet's disease: the BIO BEHÇET'S RCT

Robert J Moots
,
Farida Fortune
,
Richard Jackson
,
Tony Thornburn
,
Ann W Morgan
,
Dan Carr
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 17
  • Published:
    31 October 2024

Extended Research Article

Efficacy and safety of ketogenic diet in infants with epilepsy: KIWE RCT

Natasha E Schoeler
,
Louise Marston
,
Laura Lyons
,
Sally Halsall
,
Ruchika Jain
,
Siobhan Titre-Johnson
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 16
  • Published:
    9 October 2024

Extended Research Article

Development and evaluation of machine learning methods in whole-body magnetic resonance imaging with diffusion weighted imaging for staging of patients with cancer: the MALIBO diagnostic test accuracy study

Andrea Rockall
,
Xingfeng Li
,
Nicholas Johnson
,
Ioannis Lavdas
,
Shalini Santhakumaran
,
A Toby Prevost
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 15
  • Published:
    9 October 2024

Extended Research Article

Determining the optimal route of faecal microbiota transplant in patients with ulcerative colitis: the STOP-Colitis pilot RCT

Mohammed N Quraishi
,
Catherine A Moakes
,
Mehmet Yalchin
,
Jonathan Segal
,
Natalie J Ives
,
Laura Magill
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 14
  • Published:
    29 August 2024

Extended Research Article

Bezafibrate as treatment in males for Barth syndrome: CARDIOMAN, a double-blind, placebo-controlled crossover RCT

Guido Pieles
,
Colin Steward
,
Lucy Dabner
,
Laura Collet
,
Lucy Culliford
,
Karen Sheehan
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 13
  • Published:
    22 August 2024

Extended Research Article

High-sensitivity cardiac troponin I-guided combination angiotensin receptor blockade and beta blocker therapy to prevent anthracycline cardiotoxicity: the Cardiac CARE RCT

Peter Henriksen
,
Morag MacLean
,
Marek Atter
,
Steff Lewis
,
Aryelly Rodriguez
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 12
  • Published:
    15 August 2024

Extended Research Article

Efficacy and safety of eculizumab in children with Shiga-toxin-producing Escherichia coli haemolytic uraemic syndrome: the ECUSTEC RCT

Natalie Ives
,
Rebecca Woolley
,
Moin A Saleem
,
Catherine A Moakes
,
Aoife Waters
,
Rodney D Gilbert
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 11
  • Published:
    25 July 2024

Extended Research Article

Prophylactic zoledronic acid therapy to prevent or modify Paget's disease of bone progression in adults with SQSTM1 mutations: the ZiPP RCT

Jonathan Phillips
,
Deepak Subedi
,
Steff C Lewis
,
Catriona Keerie
,
Stuart H Ralston
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 10
  • Published:
    27 June 2024

Extended Research Article

Gene therapy for choroideremia using an adeno-associated viral vector encoding Rab escort protein 1: the REGENERATE open-label trial

Jasmina Cehajic-Kapetanovic
,
Marco P Bellini
,
Laura J Taylor
,
Imran H Yusuf
,
Taha Soomro
,
Lyndon da Cruz
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 9
  • Published:
    21 May 2024

Extended Research Article

The effect of closed-loop glucose control on C-peptide secretion in youth with newly diagnosed type 1 diabetes: the CLOuD RCT

Charlotte K Boughton
,
Janet M Allen
,
Julia Ware
,
Malgorzata E Wilinska
,
Sara Hartnell
,
Ajay Thankamony
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 8
  • Published:
    21 May 2024

Extended Research Article

Automated closed-loop insulin delivery for the management of type 1 diabetes during pregnancy: the AiDAPT RCT

Tara TM Lee
,
Corinne Collett
,
Simon Bergford
,
Sara Hartnell
,
Eleanor M Scott
,
Robert S Lindsay
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 7
  • Published:
    30 April 2024

Extended Research Article

Intravenous immunoglobulin treatment for encephalitis in children aged 6 months to 16 years: the IgNiTE RCT

Mildred A Iro
,
Manish Sadarangani
,
Michael Absoud
,
Liberty Cantrell
,
Wui K Chong
,
Christopher Clark
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 6
  • Published:
    9 April 2024

Extended Research Article

Withdrawal of renin-angiotensin system inhibitors' effect on estimated glomerular filtration rate in adults with advanced kidney disease: the STOP-ACEi RCT

Sunil Bhandari
,
Samir Mehta
,
Arif Khwaja
,
John Cleland
,
Natalie Ives
,
Elizabeth Brettell
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 5
  • Published:
    27 March 2024

Extended Research Article

Rituximab compared to intravenous cyclophosphamide in adults with connective tissue disease-associated interstitial lung disease: the RECITAL RCT

Toby M Maher
,
Veronica A Tudor
,
Peter Saunders
,
Fernando Zanghelini
,
Carlota Grossi Sampedro
,
Georgios Xydopoulos
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 4
  • Published:
    27 February 2024

Extended Research Article

Autologous stem cell transplantation with low-dose cyclophosphamide to improve mucosal healing in adults with refractory Crohn's disease: the ASTIClite RCT

James O Lindsay
,
Daniel Hind
,
Lizzie Swaby
,
Hannah Berntsson
,
Mike Bradburn
,
Uday Bannur C
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 3
  • Published:
    20 February 2024

Extended Research Article

Resuscitation with pre-hospital blood products in adults with trauma-related haemorrhagic shock: the RePHILL RCT

Nicholas Crombie
,
Heidi A Doughty
,
Jonathan RB Bishop
,
Amisha Desai
,
Emily F Dixon
,
James M Hancox
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 2
  • Published:
    9 January 2024

Extended Research Article

Evaluation of efficacy, outcomes and safety of infant haemodialysis and ultrafiltration in clinical use: I-KID a stepped wedge cluster RCT

Heather Lambert
,
Shaun Hiu
,
Malcolm Coulthard
,
John N S Matthews
,
Ruth Wood
,
Jean Crosier
et al.
  • Journal:
    Efficacy and Mechanism Evaluation Volume: 11, Issue: 1
  • Published:
    9 January 2024