A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)
M.D. Anderson Cancer Center
Summary
To learn if luspatercept is more effective in helping to reduce the number of blood transfusions needed by patients with LR-MDS.
Description
Objectives: Primary Objectives: * To assess the clinical activity and safety of luspatercept in patients with transfusion independent lower risk MDS * To assess the clinical activity and safety of luspatercept in patients with transfusion dependent lower risk MDS * To study the effects on hematopoeisis of luspatercept in patients treated on this study Secondary Objectives: * To assess the duration of response of patients with treated with luspatercept in this study * To assess the overall survival and time to transformation of patients with treated with luspatercept in this study * To asse…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Age ≥ 18 years; as MDS there is no significant experience with luspatercept in pediatric patients 2. Cohorts #1 and #2: Diagnosis of MDS according to WHO 2023 criteria (5) and with low or int-1 risk by IPSS or a score of ≤ 3.5 by IPSS-R.(3, 22) 3. Cohort #2: Patient defined as transfusion dependent by documentation of receiving at least 2 units of packed red blood cells (PRBCs) for a hemoglobin of less than 8.0 g/dL during an 8-week period prior to study enrollment. 4. Cohort #1: Patients with symptomatic anemia that are transfusion independent defined as not requiring…
Interventions
- DrugLuspatercept
Given by SC or (Injection)
Location
- M D Anderson Cancer CenterHouston, Texas