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Improved benefit of continuing luspatercept therapy: sub-analysis of patients with lower-risk MDS in the MEDALIST study

University Clinic, Department of Hematology, Oncology and Clinical Immunology, Heinrich-Heine University, Düsseldorf, Germany. germing@med.uni-duesseldorf.de. Service d'Hématologie Séniors, Hôpital Saint-Louis, Université Paris, Paris, France. Hematology and Cellular Therapy, Medical Clinic and Policlinic 1, University Hospital Leipzig, Leipzig, Germany. Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, ON, Canada. MDS Unit, Azienda Ospedaliero Universitaria Careggi, University of Florence, Florence, Italy. Hematology Department, Institute of Biomedical Research of Salamanca, University Hospital of Salamanca, Salamanca, Spain. Bristol Myers Squibb, Princeton, NJ, USA. Bristol Myers Squibb, Braine-L'Alleud, Belgium. Moffitt Cancer Center, Tampa, FL, USA. Department of Internal Medicine, Yale University, New Haven, CT, USA. Yale Cancer Center, New Haven, CT, USA. Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.

Annals of Hematology. 2023;102(2):311-321
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Abstract
Red blood cell transfusion independence (RBC-TI) is an important goal in treating lower-risk myelodysplastic syndromes with ring sideroblasts. In the phase 3 MEDALIST study, RBC-TI of ≥ 8 weeks was achieved by significantly more luspatercept- versus placebo-treated patients in the first 24 weeks of treatment. In this post hoc analysis, we evaluated RBC transfusion units and visits based on patients' baseline transfusion burden level and the clinical benefit of luspatercept treatment beyond week 25 in initial luspatercept nonresponders (patients who did not achieve RBC-TI ≥ 8 weeks by week 25) but continued luspatercept up to 144 weeks. RBC transfusion burden, erythroid response, serum ferritin levels, and hemoglobin levels relative to baseline were evaluated. Through week 25, fewer RBC transfusion units and visits were observed in luspatercept-treated patients versus placebo, regardless of baseline transfusion burden. This continued through 144 weeks of luspatercept treatment, particularly in patients with low baseline transfusion burden. Sixty-eight patients were initial nonresponders at week 25 but continued treatment; most (81%) received the maximum dose of luspatercept (1.75 mg/kg). Sixteen percent achieved RBC-TI for ≥ 8 weeks during weeks 25-48, 26% had reduced RBC transfusion burden, 10% achieved an erythroid response, 44% had reduced serum ferritin, and hemoglobin levels increased an average of 1.3 g/dL from baseline. These data have implications for clinical practice, as transfusion units and visits are less in luspatercept-treated patients through week 25 regardless of baseline transfusion burden, and continuing luspatercept beyond week 25 can potentially provide additional clinical benefits for initial nonresponders. Trial registration: NCT02631070.
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Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine