PMID- 34291195
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20210723
IS  - 2572-9241 (Electronic)
IS  - 2572-9241 (Linking)
VI  - 5
IP  - 8
DP  - 2021 Aug
TI  - 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.
PG  - e616
LID - 10.1097/HS9.0000000000000616 [doi]
LID - e616
AB  - 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%, P < 
      0.0001, for patients receiving luspatercept versus placebo, respectively. In the 
      BT trial, the percentage of responders was 21.4% versus 4.5% (P < 0.0001) for 
      luspatercept versus placebo, respectively. Treatment with luspatercept led to 
      similar incidences of adverse events (AEs), but higher incidences of grade >/=3 AEs 
      and serious AEs compared to placebo. The most frequently reported 
      treatment-emergent AEs (>/=15%) in the pooled luspatercept group were headache; 
      back pain, bone pain, and arthralgia; diarrhea; fatigue; pyrexia; and cough. The 
      aim of this article is to summarize the scientific review of the application, 
      which led to the regulatory approval in the EU.
CI  - Copyright (c) 2021 the Author(s). Published by Wolters Kluwer Health, Inc. on 
      behalf of the European Hematology Association.
FAU - Delgado, Julio
AU  - Delgado J
AD  - Oncology and Haematology Office, European Medicines Agency, Amsterdam, The 
      Netherlands.
AD  - Department of Haematology, Hospital Clinic, Barcelona, Spain.
FAU - Voltz, Caroline
AU  - Voltz C
AD  - Oncology and Haematology Office, European Medicines Agency, Amsterdam, The 
      Netherlands.
FAU - Stain, Milena
AU  - Stain M
AD  - Bundesamt fur Sicherheit im Gesundheitswesen, Vienna, Austria.
AD  - Committe for Medicinal Products for Human Use, European Medicines Agency, 
      Amsterdam, The Netherlands.
FAU - Balkowiec-Iskra, Ewa
AU  - Balkowiec-Iskra E
AD  - Committe for Medicinal Products for Human Use, European Medicines Agency, 
      Amsterdam, The Netherlands.
AD  - Urzad Rejestracji Produktow Leczniczych, Wyrobow Medycznych i Produktow 
      Biobojczych, Warsaw, Poland.
AD  - Department of Experimental and Clinical Pharmacology, Medical University of 
      Warsaw, Poland.
FAU - Mueller, Brigitte
AU  - Mueller B
AD  - Bundesamt fur Sicherheit im Gesundheitswesen, Vienna, Austria.
FAU - Wernsperger, Johanna
AU  - Wernsperger J
AD  - Bundesamt fur Sicherheit im Gesundheitswesen, Vienna, Austria.
FAU - Malinowska, Iwona
AU  - Malinowska I
AD  - Urzad Rejestracji Produktow Leczniczych, Wyrobow Medycznych i Produktow 
      Biobojczych, Warsaw, Poland.
AD  - Department of Paediatrics, Haematology and Oncology, Medical University of 
      Warsaw, Warsaw, Poland.
FAU - Gisselbrecht, Christian
AU  - Gisselbrecht C
AD  - Department of Haematology, Hopital Saint Louis, Paris, France.
FAU - Enzmann, Harald
AU  - Enzmann H
AD  - Committe for Medicinal Products for Human Use, European Medicines Agency, 
      Amsterdam, The Netherlands.
AD  - Bundesinstitut fur Arzneimittel und Medizinprodukte, Bonn, Germany.
FAU - Pignatti, Francesco
AU  - Pignatti F
AD  - Oncology and Haematology Office, European Medicines Agency, Amsterdam, The 
      Netherlands.
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20210719
PL  - United States
TA  - Hemasphere
JT  - HemaSphere
JID - 101740619
PMC - PMC8288896
EDAT- 2021/07/23 06:00
MHDA- 2021/07/23 06:01
PMCR- 2021/07/19
CRDT- 2021/07/22 06:38
PHST- 2021/05/18 00:00 [received]
PHST- 2021/06/08 00:00 [accepted]
PHST- 2021/07/22 06:38 [entrez]
PHST- 2021/07/23 06:00 [pubmed]
PHST- 2021/07/23 06:01 [medline]
PHST- 2021/07/19 00:00 [pmc-release]
AID - 10.1097/HS9.0000000000000616 [doi]
PST - epublish
SO  - Hemasphere. 2021 Jul 19;5(8):e616. doi: 10.1097/HS9.0000000000000616. eCollection 
      2021 Aug.