RT Journal Article
T1 Luspatercept in Patients with Lower-Risk Myelodysplastic Syndromes.
A1 Fenaux, Pierre
A1 Platzbecker, Uwe
A1 Mufti, Ghulam J
A1 Garcia-Manero, Guillermo
A1 Buckstein, Rena
A1 Santini, Valeria
A1 Díez-Campelo, María
A1 Finelli, Carlo
A1 Cazzola, Mario
A1 Ilhan, Osman
A1 Sekeres, Mikkael A
A1 Falantes, José F
A1 Arrizabalaga, Beatriz
A1 Salvi, Flavia
A1 Giai, Valentina
A1 Vyas, Paresh
A1 Bowen, David
A1 Selleslag, Dominik
A1 DeZern, Amy E
A1 Jurcic, Joseph G
A1 Germing, Ulrich
A1 Götze, Katharina S
A1 Quesnel, Bruno
A1 Beyne-Rauzy, Odile
A1 Cluzeau, Thomas
A1 Voso, Maria-Teresa
A1 Mazure, Dominiek
A1 Vellenga, Edo
A1 Greenberg, Peter L
A1 Hellström-Lindberg, Eva
A1 Zeidan, Amer M
A1 Adès, Lionel
A1 Verma, Amit
A1 Savona, Michael R
A1 Laadem, Abderrahmane
A1 Benzohra, Aziz
A1 Zhang, Jennie
A1 Rampersad, Anita
A1 Dunshee, Diana R
A1 Linde, Peter G
A1 Sherman, Matthew L
A1 Komrokji, Rami S
A1 List, Alan F
AB Patients with anemia and lower-risk myelodysplastic syndromes in whom erythropoiesis-stimulating agent therapy is not effective generally become dependent on red-cell transfusions. Luspatercept, a recombinant fusion protein that binds transforming growth factor β superfamily ligands to reduce SMAD2 and SMAD3 signaling, showed promising results in a phase 2 study. In a double-blind, placebo-controlled, phase 3 trial, we randomly assigned patients with very-low-risk, low-risk, or intermediate-risk myelodysplastic syndromes (defined according to the Revised International Prognostic Scoring System) with ring sideroblasts who had been receiving regular red-cell transfusions to receive either luspatercept (at a dose of 1.0 up to 1.75 mg per kilogram of body weight) or placebo, administered subcutaneously every 3 weeks. The primary end point was transfusion independence for 8 weeks or longer during weeks 1 through 24, and the key secondary end point was transfusion independence for 12 weeks or longer, assessed during both weeks 1 through 24 and weeks 1 through 48. Of the 229 patients enrolled, 153 were randomly assigned to receive luspatercept and 76 to receive placebo; the baseline characteristics of the patients were balanced. Transfusion independence for 8 weeks or longer was observed in 38% of the patients in the luspatercept group, as compared with 13% of those in the placebo group (P Luspatercept reduced the severity of anemia in patients with lower-risk myelodysplastic syndromes with ring sideroblasts who had been receiving regular red-cell transfusions and who had disease that was refractory to or unlikely to respond to erythropoiesis-stimulating agents or who had discontinued such agents owing to an adverse event. (Funded by Celgene and Acceleron Pharma; MEDALIST ClinicalTrials.gov number, NCT02631070; EudraCT number, 2015-003454-41.).
YR 2020
FD 2020
LK http://hdl.handle.net/10668/14931
UL http://hdl.handle.net/10668/14931
LA en
DS RISalud
RD Apr 8, 2025