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Systematic review and evidence gap assessment of the clinical, quality of life, and economic burden of alpha-thalassemia.
Musallam, Khaled M; Viprakasit, Vip; Lombard, Louise; Gilroy, Keely; Rane, Amey; Vinals, Lydia; Tam, Candice; Rizzo, Maria; Coates, Thomas D.
Afiliação
  • Musallam KM; Center for Research on Rare Blood Disorders (CR-RBD), Burjeel Medical City Abu Dhabi United Arab Emirates.
  • Viprakasit V; Department of Pediatrics & Thalassemia Center Faculty of Medicine Siriraj Hospital Mahidol University Bangkok Thailand.
  • Lombard L; Agios Pharmaceuticals Cambridge Massachusetts USA.
  • Gilroy K; Agios Pharmaceuticals Cambridge Massachusetts USA.
  • Rane A; Agios Pharmaceuticals Cambridge Massachusetts USA.
  • Vinals L; Cytel Toronto Ontario Canada.
  • Tam C; Cytel London UK.
  • Rizzo M; Cytel London UK.
  • Coates TD; Cancer and Blood Disease Institute Children's Hospital Los Angeles and USC Keck School of Medicine Los Angeles California USA.
EJHaem ; 5(3): 541-547, 2024 Jun.
Article em En | MEDLINE | ID: mdl-38895066
ABSTRACT
A 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 ß-thalassemia.
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Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2024 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Idioma: En Ano de publicação: 2024 Tipo de documento: Article