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. 2025 Sep;100(9):1651-1655.
doi: 10.1002/ajh.27758. Epub 2025 Jul 1.

Luspatercept for Transfusion-Dependent Beta-Thalassemia: Real-World Experience in a Large Cohort of Patients From Italy

Raffaella Origa  1 , Barbara Gianesin  2 , Antonietta Zappu  3 , Anna Rita Denotti  3 , Mary Ann Di Giorgio  4 , Roberta Sciortino  4 , Irene Motta  5   6 , Daniele Lello Panzieri  5   6 , Rosamaria Rosso  7 , Anna Bulla  7 , Martina Culcasi  8   9 , Anna Maria Pasanisi  10 , Lucia De Franceschi  11   12 , Rosario Di Maggio  13 , Valeria Maria Pinto  14 , Paola Maria Grazia Sanna  15 , Paolo Ricchi  16 , Giovan Battista Ruffo  17 , Francesca Schieppati  18 , Domenico Roberti  19 , Giovanni Battista Ferrero  20 , Elisa De Michele  21 , Francesco Arcioni  22 , Ilaria Fotzi  23 , Sarah Marktel  24 , Antonella Poloni  25 , Giulia Soverini  26 , Epifania Rita Testa  27 , Giusy Cabiddu  28 , Carmelo Fortugno  29 , Antonia Gigante  2 , Francesca Polese  30 , Davide Rapezzi  31 , Antonella Sau  32 , Gian Luca Forni  2   33 , Maria Domenica Cappellini  5 , Filomena Longo  8
Affiliations

Luspatercept for Transfusion-Dependent Beta-Thalassemia: Real-World Experience in a Large Cohort of Patients From Italy

Raffaella Origa et al. Am J Hematol. 2025 Sep.

Abstract

Keywords: extramedullary hematopoiesis; luspatercept; real‐world setting; thromboembolism; transfusion‐dependent thalassemia.

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Conflict of interest statement

R.O. has received speaker honoraria from BMS, Vertex, and Chiesi and received consultancy fees from BMS, Vertex, and Agios. V.M.P. was a member of the advisory board, part of the speaker's bureau for BMS, and a consultant for Vertex. F.L. was a consultant, a member of the advisory board, part of the speaker's bureau, and participated in educational events for BMS and Vertex. I.M. was a member of the advisory board of BMS, Vertex, and Sanofi, received honoraria for lectures from Bristol Myers Squibb and Sanofi, and research support from Sanofi. M.D.C. was a member of the advisory board for Sanofi Genzyme, BMS/Celgene, Vertex/CRISPR, Novo Nordisk, Pharmacosmos, and Agios. P.R. received honoraria from Agios Pharmaceuticals and BMS (Celgene) and was a member of the safety monitoring board/advisory board for Agios Pharmaceuticals and BMS (Celgene). R.R. received speaker honoraria and research support from Vertex and BMS. The other authors declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
Erythroid response parameters.
FIGURE 2
FIGURE 2
Iron overload changes in patients treated with Luspatercept. Serum ferritin (A) and Liver Iron Concentration (B) trend during treatment with Luspatercept. (C) Variation of iron chelation therapy during treatment with Luspatercept. Responders: From good to excellent; non‐responders: Satisfactory and non‐responders. ICT, Iron chelation therapy; LIC, Liver iron concentration; MRI, Magnetic resonance imaging.
See this image and copyright information in PMC

References

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