Quality of life assessment and transfusion-free survival

During the MDS Hub Steering Committee meeting held on May 11, 2023, Theo de Witte chaired a discussion on quality of life (QoL) assessment and transfusion-free survival in patients with myelodysplastic syndromes (MDS), with contributions from Moshe Mittelman, Mikkael Sekeres, Rena Buckstein, and Yasushi Miyazaki.

de Witte discusses the impact of red blood cell transfusion dose density on progression-free survival in lower-risk MDS, then describes the novel patient-centric metric, time without transfusion reliance (TWiTR). He highlights the use of this metric in the P-2001 study, where TWiTR analysis demonstrated that transfusion independence is a critical endpoint for improving health-related QoL (HRQoL) in patients with higher-risk MDS. The committee discusses the association between acquired prolonged transfusion independence, and better survival and lack of disease progression. They also discuss the time period for transfusion independence assessment, endpoints for QoL studies, and the impact of frailty and disability on QoL in patients with MDS. They raise the need to repeat studies with TWiTR analysis and QoL assessment in large populations, and in patients with lower-risk MDS.