Outcomes for studies assessing the efficacy of hemostatic therapies in persons with congenital bleeding disorders

Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada. Department of Clinical Neurosciences, University of Calgary, Calgary, Canada. Hotchkiss Brain Institute, Calgary, Canada. Department of Anesthesiology, McMaster University, Hamilton, ON, Canada. Michael G. DeGroote Institute for Pain Research and Care, McMaster University, Hamilton, ON, Canada. Department of Medicine, McMaster University, Hamilton, ON, Canada. Department of Hematology and Medical Oncology, Emory University School of Medicine, Atlanta, GA, USA. Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory, Emory University School of Medicine, Atlanta, GA, USA. Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA. Department of Pediatrics, Emory University School of Medicine, Aflac Cancer and Blood Disorders, Atlanta, GA, USA. Boston Children's Hospital, Boston Hemophilia Center, Harvard Medical School, Boston, MA, USA. Hemophilia Treatment Center, Nationwide Children's Hospital and The Ohio State University College of Medicine, Columbus, OH, USA. Division of Hematology and Blood Research Center, University of North Carolina School of Medicine, University of North Carolina, Chapel Hill, NC, USA.

Haemophilia : the official journal of the World Federation of Hemophilia. 2021
Abstract
INTRODUCTION Management strategies and hemostatic treatments to achieve control of bleeding are relevant across many disease areas. Identification of primary outcomes for studies assessing hemostatic intervention was the objective of a National Heart, Lung and Blood Institute (NHLBI) sponsored multidisciplinary initiative. The aim of this report is to summarize the evidence reviewed, and the outcomes identified by the subgroup tasked to assess outcomes for inherited bleeding disorders. METHODS The subgroup decided to focus on haemophilia, the prototypal congenital bleeding disorder and the one with the largest available body of evidence. MEDLINE, EMBASE and PsycINFO, The Cochrane Review, CINAHL, and Web of Science were searched for systematic and narrative reviews on outcomes used in haemophilia clinical trials. Three different clinical goals were identified as typical objectives of future research. RESULTS Out of 1322 unique citations, 24 reviews published in the period 2002-2019 were included. We identified 113 outcome measures, categorized in 6 domains: health-related quality of life (HRQoL), comorbidities and mortality, overall physical functioning and participation, bleeding and hemostasis, joint health, and costs and resource use. Three different clinical goals were identified as typical objectives of future research: Episodic 'on demand' replacement therapy, prevention of bleeding (Prophylaxis), and long-term and overall impact of bleeding. For each of these scenarios, specific outcomes were recommended. CONCLUSIONS Primary outcomes for clinical trials assessing the efficacy of hemostatic treatment in achieving control, prevention and limiting long-term consequences of bleeding in inherited bleeding disorders are suggested, and their strength and limitations discussed.
Study details
Study Design : Systematic Review
Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine