Review, conceptual model, and gap analysis of transfusion burden in hematological diseases

Hematological diseases affect millions of people worldwide, and many of these patients require blood transfusions. Although the potential adverse clinical outcomes associated with transfusions are well documented, the patient perspective of transfusion burden in hematological diseases is not. This perspective is critical, as health-related quality of life scores alone cannot fully capture patients' experiences.

A comprehensive review was conducted to understand the patient perspective of transfusion burden in hematological diseases. This study included a targeted literature review of peer-reviewed articles conducted in Embase and MEDLINE from January 2012-September 2024 (stage 1), a preliminary conceptual model (stage 2), a review of patient-reported outcome measures (PROMs) included in clinical trials and label claims (stage 3), and a gap analysis of existing PROMs (stage 4).

This study identified four key concepts and two existing PROMs that were relevant to transfusion burden in hematological diseases. Transfusions impact patients economically, physically, socially, and emotionally. The study identified an unmet need: existing PROMs lack specificity to transfusion burden, lack generalizability to all hematological diseases, and do not reflect all four key concepts identified. A new PROM is needed to better understand the patient perspective of transfusion burden in hematological diseases.