Literature DB >> 29492470

Systemic Sclerosis Trial Design Moving Forward.

Sindhu R Johnson1, Dinesh Khanna2, Yannick Allanore3, Marco Matucci-Cerinic4, Daniel E Furst5.   

Abstract

The 2013 ACR-EULAR classification criteria for systemic sclerosis (SSc) have shifted the construct of SSc. The new reality is that patients recruited for trials may not be so severe and not so advanced. We can now look for therapeutics that might stop disease evolution and/or prevent organ involvement. This article highlights recent advances in research methodology, and broadens the potential range of design and analytic considerations when planning a SSc trial.

Entities:  

Keywords:  Bayesian; Systemic sclerosis; cross-over trial; enriched enrolment randomized withdrawal design; randomized controlled trials; randomized placebo phase design; scleroderma

Year:  2018        PMID： 29492470      PMCID： PMC5826575          DOI： 10.5301/jsrd.5000198

Source DB:  PubMed          Journal:  J Scleroderma Relat Disord        ISSN： 2397-1983


  24 in total

Review 1.  Systemic sclerosis - continuing progress in developing clinical measures of response.

Authors:  Daniel Furst; Dinesh Khanna; Marco Matucci-Cerinic; Philip Clements; Virginia Steen; Janet Pope; Peter Merkel; Ivan Foeldvari; James Seibold; David Pittrow; Richard Polisson; Vibeke Strand
Journal:  J Rheumatol       Date:  2007-05       Impact factor: 4.666

2.  2013 classification criteria for systemic sclerosis: an American College of Rheumatology/European League against Rheumatism collaborative initiative.

Authors:  Frank van den Hoogen; Dinesh Khanna; Jaap Fransen; Sindhu R Johnson; Murray Baron; Alan Tyndall; Marco Matucci-Cerinic; Raymond P Naden; Thomas A Medsger; Patricia E Carreira; Gabriela Riemekasten; Philip J Clements; Christopher P Denton; Oliver Distler; Yannick Allanore; Daniel E Furst; Armando Gabrielli; Maureen D Mayes; Jacob M van Laar; James R Seibold; Laszlo Czirjak; Virginia D Steen; Murat Inanc; Otylia Kowal-Bielecka; Ulf Müller-Ladner; Gabriele Valentini; Douglas J Veale; Madelon C Vonk; Ulrich A Walker; Lorinda Chung; David H Collier; Mary Ellen Csuka; Barri J Fessler; Serena Guiducci; Ariane Herrick; Vivien M Hsu; Sergio Jimenez; Bashar Kahaleh; Peter A Merkel; Stanislav Sierakowski; Richard M Silver; Robert W Simms; John Varga; Janet E Pope
Journal:  Arthritis Rheum       Date:  2013-10-03

Review 3.  Enriched enrollment: definition and effects of enrichment and dose in trials of pregabalin and gabapentin in neuropathic pain. A systematic review.

Authors:  Sebastian Straube; Sheena Derry; Henry J McQuay; R Andrew Moore
Journal:  Br J Clin Pharmacol       Date:  2008-04-11       Impact factor: 4.335

4.  Etanercept in children with polyarticular juvenile rheumatoid arthritis. Pediatric Rheumatology Collaborative Study Group.

Authors:  D J Lovell; E H Giannini; A Reiff; G D Cawkwell; E D Silverman; J J Nocton; L D Stein; A Gedalia; N T Ilowite; C A Wallace; J Whitmore; B K Finck
Journal:  N Engl J Med       Date:  2000-03-16       Impact factor: 91.245

5.  N of 1 randomized trials for investigating new drugs.

Authors:  G H Guyatt; A Heyting; R Jaeschke; J Keller; J D Adachi; R S Roberts
Journal:  Control Clin Trials       Date:  1990-04

6.  The plasma cell signature in autoimmune disease.

Authors:  Katie Streicher; Christopher A Morehouse; Christopher J Groves; Bhargavi Rajan; Fernanda Pilataxi; Kim P Lehmann; Philip Z Brohawn; Brandon W Higgs; Kathleen McKeever; Steven A Greenberg; David Fiorentino; Laura K Richman; Bahija Jallal; Ronald Herbst; Yihong Yao; Koustubh Ranade
Journal:  Arthritis Rheumatol       Date:  2014-01       Impact factor: 10.995

7.  Physicians' preferences for active-controlled versus placebo-controlled trials of new antihypertensive drugs.

Authors:  Scott D Halpern; Peter A Ubel; Jesse A Berlin; Raymond R Townsend; David A Asch
Journal:  J Gen Intern Med       Date:  2002-09       Impact factor: 5.128

8.  Rituximab in the treatment of refractory adult and juvenile dermatomyositis and adult polymyositis: a randomized, placebo-phase trial.

Authors:  Chester V Oddis; Ann M Reed; Rohit Aggarwal; Lisa G Rider; Dana P Ascherman; Marc C Levesque; Richard J Barohn; Brian M Feldman; Michael O Harris-Love; Diane C Koontz; Noreen Fertig; Stephanie S Kelley; Sherrie L Pryber; Frederick W Miller; Howard E Rockette
Journal:  Arthritis Rheum       Date:  2013-02

9.  Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.

Authors:  Marilyn J Manco-Johnson; Thomas C Abshire; Amy D Shapiro; Brenda Riske; Michele R Hacker; Ray Kilcoyne; J David Ingram; Michael L Manco-Johnson; Sharon Funk; Linda Jacobson; Leonard A Valentino; W Keith Hoots; George R Buchanan; Donna DiMichele; Michael Recht; Deborah Brown; Cindy Leissinger; Shirley Bleak; Alan Cohen; Prasad Mathew; Alison Matsunaga; Desiree Medeiros; Diane Nugent; Gregory A Thomas; Alexis A Thompson; Kevin McRedmond; J Michael Soucie; Harlan Austin; Bruce L Evatt
Journal:  N Engl J Med       Date:  2007-08-09       Impact factor: 91.245

10.  Anticoagulation and survival in pulmonary arterial hypertension: results from the Comparative, Prospective Registry of Newly Initiated Therapies for Pulmonary Hypertension (COMPERA).

Authors:  Karen M Olsson; Marion Delcroix; H Ardeschir Ghofrani; Henning Tiede; Doerte Huscher; Rudolf Speich; Ekkehard Grünig; Gerd Staehler; Stephan Rosenkranz; Michael Halank; Matthias Held; Tobias J Lange; Juergen Behr; Hans Klose; Martin Claussen; Ralf Ewert; Christian F Opitz; C Dario Vizza; Laura Scelsi; Anton Vonk-Noordegraaf; Harald Kaemmerer; J Simon R Gibbs; Gerry Coghlan; Joanna Pepke-Zaba; Uwe Schulz; Matthias Gorenflo; David Pittrow; Marius M Hoeper
Journal:  Circulation       Date:  2013-09-30       Impact factor: 29.690
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  4 in total

1.  Exhaled Nitric Oxide in Systemic Sclerosis Lung Disease.

Authors:  Natalie K Kozij; John T Granton; Philip E Silkoff; John Thenganatt; Shobha Chakravorty; Sindhu R Johnson
Journal:  Can Respir J       Date:  2017-02-14       Impact factor: 2.409

2.  Systemic sclerosis: state of the art on clinical practice guidelines.

Authors:  Vanessa Smith; Carlo Alberto Scirè; Rosaria Talarico; Paolo Airo; Tobias Alexander; Yannick Allanore; Cosimo Bruni; Veronica Codullo; Virgil Dalm; Jeska De Vries-Bouwstra; Alessandra Della Rossa; Oliver Distler; Ilaria Galetti; David Launay; Gemma Lepri; Alexis Mathian; Luc Mouthon; Barbara Ruaro; Alberto Sulli; Angela Tincani; Els Vandecasteele; Amber Vanhaecke; Marie Vanthuyne; Frank Van den Hoogen; Ronald Van Vollenhoven; Alexandre E Voskuyl; Elisabetta Zanatta; Stefano Bombardieri; Gerd Burmester; Fonseca João Eurico; Charissa Frank; Eric Hachulla; Frederic Houssiau; Ulf Mueller-Ladner; Matthias Schneider; Jacob M van Laar; Ana Vieira; Maurizio Cutolo; Marta Mosca; Marco Matucci-Cerinic
Journal:  RMD Open       Date:  2018-10-18

3.  Evaluation of Scleroderma Clinical Trials Consortium training recommendations on modified Rodnan skin score assessment in scleroderma.

Authors:  Andrea H L Low; Sue-Ann Ng; Veronica Berrocal; Benjamin Brennan; Grace Chan; Swee-Cheng Ng; Dinesh Khanna
Journal:  Int J Rheum Dis       Date:  2019-03-06       Impact factor: 2.454

4.  Predictors of disease worsening defined by progression of organ damage in diffuse systemic sclerosis: a European Scleroderma Trials and Research (EUSTAR) analysis.

Authors:  Mike Becker; Nicole Graf; Rafael Sauter; Yannick Allanore; John Curram; Christopher P Denton; Dinesh Khanna; Marco Matucci-Cerinic; Janethe de Oliveira Pena; Janet E Pope; Oliver Distler
Journal:  Ann Rheum Dis       Date:  2019-06-21       Impact factor: 19.103
  4 in total

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