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

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.