Systematic literature review of the indirect costs and humanistic burden of β-thalassemia

Background:β-Thalassemia is an inherited blood disorder requiring lifetime management of anemia and its complications.Objective:This study aimed to determine the indirect costs and humanistic burden of β-thalassemia.Design:A systematic literature review was conducted.Data sources and methods:Searches were conducted in Embase, MEDLINE, MEDLINE In-Process, and EconLit (November 1, 2010, to November 25, 2020). Studies reporting indirect costs and health-related quality of life (HRQoL) for patients with β-thalassemia were eligible.Results:Seventy-five publications were included. Mean annual days lost due to transfusion-related absenteeism ranged from 15.6 to 35 days. Patients spent a mean of 592 min (standard deviation (SD): 349) daily on disease management on transfusion days and 91 min (SD: 221) daily on non-transfusion days. Patients with non-transfusion-dependent β-thalassemia (NTDT) showed worse HRQoL versus those with transfusion-dependent β-thalassemia (TDT) on the 36-item Short Form Health Survey (75.8 vs 66.5; p = 0.021). Caregivers of patients with TDT had more severe stress compared with patients (20.17 vs 18.95; p = 0.006), as measured by the standardized Cohen Perceived Stress Questionnaire.Conclusion:TDT is associated with substantial indirect costs and caregiver burden, and NTDT is associated with worse HRQoL. There is an unmet need for novel treatments in both TDT and NTDT that minimize patient and caregiver burden.