Quality of Life in Low-Risk MDS: An Undervalued Endpoint

Article Medicine, General & Internal

Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Phase 3 Trial

Esther Natalie Oliva et al.

Summary: Patients with myelodysplastic syndromes often experience chronic anemia and long-term red blood cell transfusion dependence, leading to significant burden on clinical and health-related quality of life outcomes. In the MEDALIST trial, luspatercept significantly reduced transfusion burden in lower-risk MDS patients. However, there were no clinically meaningful changes in HRQoL observed. Patients treated with luspatercept reported improvements in daily life owing to reduced transfusion burden. These results suggest that luspatercept may offer a treatment option for patients that reduces transfusion burden while providing stability in HRQoL.

JOURNAL OF CLINICAL MEDICINE (2022)

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Article Oncology

Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion-Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study

David P. Steensma et al.

Summary: Imetelstat treatment shows a meaningful and durable transfusion independence rate in heavily transfused LR-MDS patients, reducing malignant clone count and demonstrating disease modification activity. The most common adverse events are reversible cytopenias.

JOURNAL OF CLINICAL ONCOLOGY (2021)

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Article Oncology

Phase III, Randomized, Placebo-Controlled Trial of CC-486 (Oral Azacitidine) in Patients With Lower-Risk Myelodysplastic Syndromes

Guillermo Garcia-Manero et al.

Summary: CC-486 significantly improved RBC-TI rate and induced durable bilineage improvements in patients with LR-MDS and high-risk disease features. However, more early deaths occurred in the CC-486 arm, most related to infections in patients with significant pretreatment neutropenia. Further evaluation of CC-486 in MDS is needed.

JOURNAL OF CLINICAL ONCOLOGY (2021)

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Article Hematology