A Pilot, Open-Label Study of Luspatercept for Patients With Lower Risk Myelodysplastic Syndromes (MDS)
To learn if luspatercept is more effective in helping to reduce the number of blood transfusions needed by patients with LR-MDS.
• Age ≥ 18 years; as MDS there is no significant experience with luspatercept in pediatric patients
• 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)
• 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.
• Cohort #1: Patients with symptomatic anemia that are transfusion independent defined as not requiring a transfusion for a hemoglobin of less than 8.0 g/dL during an 8-week period prior to study enrollment.
• MDS patients with either a platelet count of ≤100 K/uL and/or ANC of ≤1.8 K/uL
• Patient must have signed an informed consent and is willing to participate in the study.
• Adequate hepatic function with total bilirubin ≤3 x ULN, AST or ALT ≤3xULN.
• Serum creatinine clearance ≥40mL/min and no end/stage renal disease (using Cockcroft-Gault).
• ECOG performance status \</=2.