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Delgado J, Voltz C, Stain M, et al. The European Medicines Agency Review of Luspatercept for the Treatment of Adult Patients With Transfusion-dependent Anemia Caused by Low-risk Myelodysplastic Syndromes With Ring Sideroblasts or Beta-thalassemia. Hemasphere. 2021;5(8):e616doi: 10.1097/HS9.0000000000000616.
Delgado, J., Voltz, C., Stain, M., Balkowiec-Iskra, E., Mueller, B., Wernsperger, J., Malinowska, I., Gisselbrecht, C., Enzmann, H., & Pignatti, F. (2021). The European Medicines Agency Review of Luspatercept for the Treatment of Adult Patients With Transfusion-dependent Anemia Caused by Low-risk Myelodysplastic Syndromes With Ring Sideroblasts or Beta-thalassemia. HemaSphere, 5(8), e616. https://doi.org/10.1097/HS9.0000000000000616
Delgado, Julio, et al. "The European Medicines Agency Review of Luspatercept for the Treatment of Adult Patients With Transfusion-dependent Anemia Caused by Low-risk Myelodysplastic Syndromes With Ring Sideroblasts or Beta-thalassemia." HemaSphere vol. 5,8 (2021): e616. doi: https://doi.org/10.1097/HS9.0000000000000616
Delgado J, Voltz C, Stain M, Balkowiec-Iskra E, Mueller B, Wernsperger J, Malinowska I, Gisselbrecht C, Enzmann H, Pignatti F. The European Medicines Agency Review of Luspatercept for the Treatment of Adult Patients With Transfusion-dependent Anemia Caused by Low-risk Myelodysplastic Syndromes With Ring Sideroblasts or Beta-thalassemia. Hemasphere. 2021 Jul 19;5(8):e616. doi: 10.1097/HS9.0000000000000616. eCollection 2021 Aug. PMID: 34291195; PMCID: PMC8288896.
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Hemasphere. 2021 Jul 19;5(8):e616. doi: 10.1097/HS9.0000000000000616. eCollection 2021 Aug.

The European Medicines Agency Review of Luspatercept for the Treatment of Adult Patients With Transfusion-dependent Anemia Caused by Low-risk Myelodysplastic Syndromes With Ring Sideroblasts or Beta-thalassemia.

HemaSphere

Julio Delgado, Caroline Voltz, Milena Stain, Ewa Balkowiec-Iskra, Brigitte Mueller, Johanna Wernsperger, Iwona Malinowska, Christian Gisselbrecht, Harald Enzmann, Francesco Pignatti

Affiliations

  1. Oncology and Haematology Office, European Medicines Agency, Amsterdam, The Netherlands.
  2. Department of Haematology, Hospital Clinic, Barcelona, Spain.
  3. Bundesamt fur Sicherheit im Gesundheitswesen, Vienna, Austria.
  4. Committe for Medicinal Products for Human Use, European Medicines Agency, Amsterdam, The Netherlands.
  5. Urz?d Rejestracji Produktów Leczniczych, Wyrobow Medycznych i Produktów Biobójczych, Warsaw, Poland.
  6. Department of Experimental and Clinical Pharmacology, Medical University of Warsaw, Poland.
  7. Department of Paediatrics, Haematology and Oncology, Medical University of Warsaw, Warsaw, Poland.
  8. Department of Haematology, Hopital Saint Louis, Paris, France.
  9. Bundesinstitut für Arzneimittel und Medizinprodukte, Bonn, Germany.

PMID: 34291195 PMCID: PMC8288896 DOI: 10.1097/HS9.0000000000000616

Abstract

Luspatercept is a recombinant fusion protein that selectively binds to ligands belonging to the transforming growth factor-beta superfamily, resulting in erythroid maturation and differentiation. On June 25, 2020, a marketing authorization valid through the European Union (EU) was issued for luspatercept for the treatment of adult patients with transfusion-dependent anemia caused by very low-, low-, and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, or those with transfusion-dependent beta thalassemia (BT). Luspatercept was evaluated in 2 separate phase 3, double-blind, placebo-controlled multicentre trials. The primary endpoints of these trials were the percentage of patients achieving transfusion independence over ≥8 weeks or longer for patients with MDS, and the percentage of patients achieving a ≥33% reduction in transfusion burden from baseline to week 13-24 for patients with BT. In the MDS trial, the percentage of responders was 37.91% versus 13.16%,

Copyright © 2021 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Hematology Association.

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