Musallam Khaled M, Viprakasit Vip, Lombard Louise, Gilroy Keely, Rane Amey, Vinals Lydia, Tam Candice, Rizzo Maria, Coates Thomas D
Center for Research on Rare Blood Disorders (CR-RBD), Burjeel Medical City Abu Dhabi United Arab Emirates.
Department of Pediatrics & Thalassemia Center Faculty of Medicine Siriraj Hospital Mahidol University Bangkok Thailand.
EJHaem. 2024 Apr 2;5(3):541-547. doi: 10.1002/jha2.882. eCollection 2024 Jun.
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 β-thalassemia.
目前缺乏对与α地中海贫血相关的临床、健康相关生活质量和经济负担的证据差距评估。我们按照系统评价和Meta分析的首选报告项目以及Cochrane系统评价手册的方法和报告要求,对过去十年的现有文献进行了系统文献综述(SLR)。该SLR发现,从过去10年发表的研究数量稀少可以明显看出,在了解α地中海贫血当前负担方面存在相当大的证据差距。现有的有限数据仍然表明,α地中海贫血患者经历了严重的发病率以及生活质量/经济负担,这通常与β地中海贫血患者相当。