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

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 β-thalassemia.

Keywords: HRQOL; complications; cost; management; morbidity; thalassemia.

FIGURE 1

Literature flow diagram. ^*One of the included studies was a conference abstract. HCRU, healthcare resource utilization; HRQOL, health‐related quality of life.

FIGURE 2

Complication rates (A) and treatment patterns (B) among patients with hemoglobin H disease. Each box represents the prevalence rate reported in an individual study on a 0%–100% percentage scale. Data are presented for deletional, non‐deletional, and mixed (deletional + non‐deletional, or genotype unspecified) subgroups; based on what was reported in individual studies. For transfusions, data are presented as the prevalence rate for frequent (F), historical (H), or occasional (O) transfusions; or as a probability (P) of receiving at least one transfusion by age 20 year; based on what was reported in individual studies. ^*Defined as liver iron concentration ≥7 mg Fe/g dry weight (moderate‐to‐severe). ^†Defined as liver iron concentration ≥5 mg Fe/g dry weight or serum ferritin ≥800 ng/mL. EMH, extramedullary hematopoiesis; HbH, hemoglobin H disease, IE, ineffective erythropoiesis; TRI, transfusion‐related infection.