Outcomes for studies assessing the efficacy of hemostatic therapies in persons with congenital bleeding disorders
Camila C. Aquino
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
Search for more papers by this authorVictoria Borg Debono
Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada
Department of Anesthesiology, McMaster University, Hamilton, ON, Canada
Michael G. DeGroote Institute for Pain Research and Care, McMaster University, Hamilton, ON, Canada
Search for more papers by this authorFederico Germini
Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada
Department of Medicine, McMaster University, Hamilton, ON, Canada
Search for more papers by this authorDrashti Pete
Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada
Search for more papers by this authorChristine L. Kempton
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
Search for more papers by this authorGuy Young
Hemostasis and Thrombosis Center, Children’s Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA
Search for more papers by this authorRobert Sidonio
Department of Pediatrics, Emory University School of Medicine, Aflac Cancer and Blood Disorders, Atlanta, GA, USA
Search for more papers by this authorStacy E. Croteau
Boston Children’s Hospital, Boston Hemophilia Center, Harvard Medical School, Boston, MA, USA
Search for more papers by this authorAmy L. Dunn
Hemophilia Treatment Center, Nationwide Children’s Hospital and The Ohio State University College of Medicine, Columbus, OH, USA
Search for more papers by this authorNigel S. Key
Division of Hematology and Blood Research Center, University of North Carolina School of Medicine, University of North Carolina, Chapel Hill, NC, USA
Search for more papers by this authorCorresponding Author
Alfonso Iorio
Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada
Department of Medicine, McMaster University, Hamilton, ON, Canada
Correspondence
Alfonso Iorio, CRL 140, 1200 Main Street West, Hamilton, ON L8S 4K1, Canada.
Email: [email protected]
Search for more papers by this authorCamila C. Aquino
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
Search for more papers by this authorVictoria Borg Debono
Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada
Department of Anesthesiology, McMaster University, Hamilton, ON, Canada
Michael G. DeGroote Institute for Pain Research and Care, McMaster University, Hamilton, ON, Canada
Search for more papers by this authorFederico Germini
Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada
Department of Medicine, McMaster University, Hamilton, ON, Canada
Search for more papers by this authorDrashti Pete
Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada
Search for more papers by this authorChristine L. Kempton
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
Search for more papers by this authorGuy Young
Hemostasis and Thrombosis Center, Children’s Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA
Search for more papers by this authorRobert Sidonio
Department of Pediatrics, Emory University School of Medicine, Aflac Cancer and Blood Disorders, Atlanta, GA, USA
Search for more papers by this authorStacy E. Croteau
Boston Children’s Hospital, Boston Hemophilia Center, Harvard Medical School, Boston, MA, USA
Search for more papers by this authorAmy L. Dunn
Hemophilia Treatment Center, Nationwide Children’s Hospital and The Ohio State University College of Medicine, Columbus, OH, USA
Search for more papers by this authorNigel S. Key
Division of Hematology and Blood Research Center, University of North Carolina School of Medicine, University of North Carolina, Chapel Hill, NC, USA
Search for more papers by this authorCorresponding Author
Alfonso Iorio
Health Information Research Unit, Department of Health Research Methods, Evidence, and Impact (HEI), McMaster University, Hamilton, ON, Canada
Department of Medicine, McMaster University, Hamilton, ON, Canada
Correspondence
Alfonso Iorio, CRL 140, 1200 Main Street West, Hamilton, ON L8S 4K1, Canada.
Email: [email protected]
Search for more papers by this authorAbstract
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.
CONFLICT OF INTEREST
Some authors have conflict of interest. Please see Appendix 2.
Supporting Information
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| hae14247-sup-0001-TableS1-S5.docxWord document, 294.5 KB | Tables S1-S5 |
Please note: The publisher is not responsible for the content or functionality of any supporting information supplied by the authors. Any queries (other than missing content) should be directed to the corresponding author for the article.
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