title: National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: VI. Design of Clinical Trials Working Group report.
abstract: The complexity of chronic graft-versus-host disease (GVHD) and the lack of established research methods have made it difficult to design, conduct, and analyze clinical trials involving subjects with this disease, even when promising treatment options are available. This consensus document was developed to offer an approach for overcoming these obstacles. Clinical trials in chronic GVHD should adhere to principles of good trial design and practice. Inclusion and exclusion criteria should allow as many subjects to participate as possible without compromising the interpretation of results. Pre-enrollment assessment of chronic GVHD characteristics should be standardized. The protocol should provide clear guidance about administration of study medication and other interventions. Methods of assessing response should be defined and validated in advance. Efficacy endpoints should be selected to reflect clinical benefit. Expert biostatistical support is needed to ensure the validity and reliability of trial results. The use of consistent standards in clinical trial designs to evaluate agents that have activity in pathogenic pathways could facilitate advances in the treatment of chronic GVHD.
authors.affiliation: Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, Washington 98109-1024, USA, and Hopital St. Louis, Paris, France. pmartin@fhcrc.org
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Martin
authors.forename: Daniel
authors.initials: D
authors.lastname: Weisdorf
authors.forename: Donna
authors.initials: D
authors.lastname: Przepiorka
authors.forename: Steven
authors.initials: S
authors.lastname: Hirschfeld
authors.forename: Ann
authors.initials: A
authors.lastname: Farrell
authors.forename: J Douglas
authors.initials: JD
authors.lastname: Rizzo
authors.forename: Ronan
authors.initials: R
authors.lastname: Foley
authors.forename: Gerard
authors.initials: G
authors.lastname: Socie
authors.forename: Shelly
authors.initials: S
authors.lastname: Carter
authors.forename: Daniel
authors.initials: D
authors.lastname: Couriel
authors.forename: Kirk R
authors.initials: KR
authors.lastname: Schultz
authors.forename: Mary E D
authors.initials: ME
authors.lastname: Flowers
authors.forename: Alexandra H
authors.initials: AH
authors.lastname: Filipovich
authors.forename: Rima
authors.initials: R
authors.lastname: Saliba
authors.forename: Georgia B
authors.initials: GB
authors.lastname: Vogelsang
authors.forename: Steven Z
authors.initials: SZ
authors.lastname: Pavletic
authors.forename: Stephanie J
authors.initials: SJ
authors.lastname: Lee
mesh_terms: D000305:Adrenal Cortex Hormones / Q000008:administration & dosage / Q000627:therapeutic use; D000328:Adult; D002648:Child; D002908:Chronic Disease; D002986:Clinical Trials as Topic / Q000379:methods* / Q000592:standards; D017322:Clinical Trials, Phase II as Topic / Q000379:methods; D017326:Clinical Trials, Phase III as Topic / Q000379:methods; D003625:Data Collection; D018450:Disease Progression; D005544:Forecasting; D005568:Forms and Records Control; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000517:prevention & control / Q000628:therapy*; D006801:Humans; D007165:Immunosuppression Therapy / Q000009:adverse effects / Q000379:methods; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use; D008499:Medical Records; D015337:Multicenter Studies as Topic; D018579:Patient Selection; D010361:Patients / Q000523:psychology; D012107:Research Design; D016896:Treatment Outcome; D014481:United States; D014486:United States Food and Drug Administration / Q000592:standards
chemical_list: D000305:Adrenal Cortex Hormones; D007166:Immunosuppressive Agents