期刊
ALIMENTARY PHARMACOLOGY & THERAPEUTICS
卷 42, 期 10, 页码 1200-1210出版社
WILEY
DOI: 10.1111/apt.13408
关键词
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资金
- UK National Institute for Health Research
- AbbVie
- Abbott/Abbvie
- ActoGeniX NV
- Amgen
- AM-Pharma BV
- Boehringer-Ingelheim
- ChemoCentryx
- Centocor/Jansen Biologics
- Cosmo Technologies
- Elan/Biogen
- EnGene Inc
- Ferring Pharmaceuticals
- Gilead Sciences
- Given Imaging
- GSK
- Merck Research Laboratories
- Merck Serono
- Millenium Pharmaceuticals
- Novo Nordisk
- NPS Pharmaceuticals
- PDL Biopharma
- Pfizer
- Receptos
- Salix Pharmaceuticals
- Schering Plough
- Shire Pharmaceuticals
- Sigmoid Pharma Ltd
- Teva Pharmaceuticals
- Tillotts Pharma AG
- UCB Pharma
- Falk
- Janssen
- Merck
- Jansen
- Takeda
- UCB
- Shire
- Abbott
- AGI Therapeutics Inc
- Alba Therapeutics Corp
- Albireo
- Alfa Wasserman
- Anaphore
- Astellas
- Athersys Inc
- Atlantic Healthcare Ltd
- Aptalis
- BioBalance Corp
- Bristol-Myers Squibb
- Celgene
- Celek Pharmaceuticals
- Cellerix SL
- Cerimon Pharmaceuticals
- CoMentis
- Coronado Biosciences
- Cytokine Pharmasciences
- Eagle Pharmaceuticals
- Eli Lilly
- Enteromedics
- Exagen Diagnostics Inc
- Flexio Therapeutics Inc
- Funxional Therapeutics Ltd
- Genzyme Corp
- Human Genome Sciences
- Ironwood Pharmaceuticals
- KaloBios Pharmaceuticals
- Lexicon Pharmaceuticals
- Lycera Corp
- Meda Pharmaceuticals
- Nisshin Kyorin Pharmaceuticals
- Optimer Pharmaceuticals
- Orexigen Therapeutics Inc
- Procter and Gamble
- Prometheus Laboratories
- ProtAb Ltd
- Purgenesis Technologies Inc
- Relypsa Inc
- Roche
- Salient Pharmaceuticals
- Santarus
- Sirtris Pharmaceuticals
- SLA Pharma UK Ltd
- Targacept
- Therakos
- TxCell SA
- Viamet Pharmaceuticals
- Vascular Biogenics Ltd
- Warner Chilcott UK Ltd
- Wyeth
- Genentech
- Milennium Pharmaceuticals
- Novartis
- Millennium Pharmaceuticals
- Abbott Labs
- Novartis Pharmaceuticals
- Centocor Inc.
- ActoGenix
- Wyeth Pharmaceuticals Inc.
- Janssen-Ortho
- Astra Zeneca
- Serono
- Unity Pharmaceuticals
- Albireo Pharma
- Given Imaging Inc.
- Novonordisk
- Prometheus Therapeutics and Diagnostics
- Athersys
- Axcan
- Gilead
- Zealand Pharma
- Zyngenia
- GiCare Pharma Inc.
- Sigmoid Pharma
- National Institutes of Health Research (NIHR) [NIHR-CTF-2014-03-05] Funding Source: National Institutes of Health Research (NIHR)
- National Institute for Health Research [NIHR-CTF-2014-03-05] Funding Source: researchfish
BackgroundPatient-reported outcomes (PROs) have an increasingly important role in the evaluation of new therapies for inflammatory bowel disease. The US Food and Drug Administration has issued formal guidance to describe the role of PRO instruments in evaluation of claims for product labelling. However, no validated PRO exists for ulcerative colitis. AimTo investigate whether the PROs from the Mayo Clinic Score (MCS) for UC can be modified, to develop an interim PRO for use in clinical trials, alone or in combination with endoscopy. MethodsData from an induction trial of a mesalazine (mesalamine) formulation were used to compare effect sizes between mesalazine and placebo for PRO items (stool frequency and rectal bleeding) alone and in combination with endoscopy. The operating properties of the PRO were validated using data from a phase 2 trial of MLN02, a humanised antibody to the 47 integrin in patients with UC. ResultsA two-item PRO (PRO2) consisting of rectal bleeding = 0 and stool frequency 1 or 2, combined with an endoscopy subscore 1 yielded statistically significant differences between active drug and placebo. This combination yielded the most similar effect sizes and placebo rates for remission, compared to the primary trials. Use of PRO items alone yielded high placebo remission rates in both data sets, although rates were lower when the items were combined and remission defined as PRO2 = 0. ConclusionPatient-reported outcomes items derived from the Mayo Clinic Score combined with endoscopy as a co-primary endpoint may be an appropriate interim outcome measure for ulcerative colitis trials.
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