Novel drug showcased consistent favorable findings across various cohorts of patients with myelofibrosis (MF), according to a scientific poster presented at the Society Hematologic Oncology Eleventh Annual Meeting and published in Clinical Lymphoma, Myeloma & Leukemia.

“Luspatercept demonstrated a safety profile consistent with previous studies and improved anemia and transfusion burden in all cohorts,” the authors wrote.

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This phase 2 clinical trial, known as the ACE-536-MF-001 study, aimed to assess the safety and efficacy of luspatercept, an investigational drug, in patients with MF-associated anemia.

The study enrolled participants into 4 distinct cohorts based on their transfusion dependence (TD) status and whether they were receiving stable ruxolitinib treatment, a standard therapy for MF.

Cohort 1 included individuals without TD and no ruxolitinib treatment, cohort 2 comprised patients with TD but no ruxolitinib, cohort 3A consisted of subjects without TD but receiving ruxolitinib, and cohort 3B included patients with TD and ruxolitinib.

Participants received subcutaneous injections of luspatercept with dose titration up to 1.75 mg/kg in 21-day cycles. The primary endpoint was anemia response, defined as an increase in hemoglobin levels of at least 1.5 g/dL without requiring a red blood cell transfusion.

Notably, cohort 3B showcased the highest anemia response rate at 26.3%. Furthermore, in cohorts 2 and 3B, nearly half of the patients achieved a significant reduction (≥50%) in transfusion burden. In cohorts 1 and 3A, 27.3% and 50.0% of the subjects, respectively, achieved a mean hemoglobin increase of at least 1.5 g/dL throughout the entire treatment period.

Results 3 years after the last dose showed that luspatercept had a favorable safety profile consistent with previous trials. Most patients experienced at least 1 treatment-related adverse event, with hypertension being the most common (17.9%). Serious treatment-related adverse events were rare, leading to luspatercept discontinuation in only 1 case.


Gerds A, Harrison C, Kiladjian J, et al. Safety and efficacy of luspatercept for the treatment of anemia in patients with myelofibrosis: results from the ACE‐536‐MF‐001 study. Clin Lymphoma Myeloma Leuk. Published online September 2023. doi: 10.1016/S2152-2650(23)01240-5