Systematic review and evidence gap assessment of the clinical, quality of life, and economic burden of alpha‐thalassemia

Author:

Musallam Khaled M.1ORCID,Viprakasit Vip2,Lombard Louise3,Gilroy Keely3,Rane Amey3,Vinals Lydia4,Tam Candice5,Rizzo Maria5,Coates Thomas D.6

Affiliation:

1. Center for Research on Rare Blood Disorders (CR‐RBD), Burjeel Medical City Abu Dhabi United Arab Emirates

2. Department of Pediatrics & Thalassemia Center Faculty of Medicine Siriraj Hospital Mahidol University Bangkok Thailand

3. Agios Pharmaceuticals Cambridge Massachusetts USA

4. Cytel Toronto Ontario Canada

5. Cytel London UK

6. Cancer and Blood Disease Institute Children's Hospital Los Angeles and USC Keck School of Medicine Los Angeles California USA

Abstract

AbstractA recent evidence gaps assessment of the clinical, health‐related quality of life, and economic burden associated with α‐thalassemia is lacking. We conducted a systematic literature review (SLR) following the methodological and reporting requirements of the Preferred Reporting Items for Systematic Reviews and Meta‐Analyses and the Cochrane Handbook for Systematic Reviews, using available literature over the past decade. This SLR identified a considerable evidence gap with regard to understanding the current burden of α‐thalassemia as evident from paucity of studies published in the past 10 years. The limited data available still indicate that patients with α‐thalassemia experience substantial morbidity and quality of life/economic burden that is generally comparable to patients with β‐thalassemia.

Funder

Agios Pharmaceuticals

Publisher

Wiley

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