Overview
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 assess transfusion free survival period in patients that were transfusion independent treated with luspatercept in this study
- To measure trends in neutrophil and platelet counts in patients treated in this study
Eligibility
Inclusion Criteria:
- 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.
Exclusion Criteria:
- Active infection not adequately responding to appropriate antibiotics.
- Prior treatment with luspatercept or sotarcetept
- Female patients who are pregnant or lactating.
- Patients with reproductive potential who are unwilling to following contraception requirements (including condom use for males with sexual partners, and for females: prescription oral contraceptives [birth control pills], contraceptive injections, intrauterine devices [IUD], double-barrier method [spermicidal jelly or foam with condoms or diaphragm], contraceptive patch, or surgical sterilization) throughout the study. Reproductive potential is defined as no previous surgical sterilization or females that are not post-menopausal for 12 months.
- Female patients with reproductive potential who do not have a negative urine or blood beta-human chorionic gonadotropin (beta HCG) pregnancy test at screening.
- History of an active malignancy within the past 2 years prior to study entry, with the exception of: a. Adequately treated in situ carcinoma of the cervix uteri b. Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin or any other malignancy with a life expectancy of more than 2 years.
- Patients receiving any other concurrent investigational agent or chemotherapy, radiotherapy, or immunotherapy (within 14 days of initiating study treatment).