Luspatercept in Preventing Poor Erythroid Engraftment for Hematological Malignancies With Moderate to Severe Myelofibrosis

Summary

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an important treatment for hematological malignancies. Poor erythroid engraftment after transplantation is a serious complication, especially in patients with moderate to severe myelofibrosis (MF). Currently, there is a lack of effective prevention strategies for poor erythroid engraftment after transplantation. Luspatercept, a novel TGF-β superfamily signaling pathway modulator, has shown potential in small-sample studies for the treatment and prevention of post-transplant anemia. Given the high proportion and poor prognosis of poor engraftment function in hematological malignancies with moderate to severe myelofibrosis after transplantation, we plan to conduct a prospective, multicenter, randomized controlled study to explore the efficacy and safety of luspatercept in preventing poor erythroid engraftment after allo-HSCT in hematological malignancies with moderate to severe myelofibrosis.

Timeline

Start

2026-05-01

Primary completion

2030-01-01

Completion

2030-12-31

Publications

• Results Wobus M, Mies A, Asokan N, Oelschlagel U, Mobus K, Winter S, Cross M, Weidner H, Rauner M, Hofbauer LC, Bornhauser M, Platzbecker U. Luspatercept restores SDF-1-mediated hematopoietic support by MDS-derived mesenchymal stromal cells. Leukemia. 2021 Oct;35(10):2936-2947. doi: 10.1038/s41375-021-01275-5. Epub 2021 May 17.
• Results Fenaux P, Santini V, Spiriti MAA, Giagounidis A, Schlag R, Radinoff A, Gercheva-Kyuchukova L, Anagnostopoulos A, Oliva EN, Symeonidis A, Berger MH, Gotze KS, Potamianou A, Haralampiev H, Wapenaar R, Milionis I, Platzbecker U. A phase 3 randomized, placebo-controlled study assessing the efficacy and safety of epoetin-alpha in anemic patients with low-risk MDS. Leukemia. 2018 Dec;32(12):2648-2658. doi: 10.1038/s41375-018-0118-9. Epub 2018 Mar 30.
• Results Platzbecker U, Della Porta MG, Santini V, Zeidan AM, Komrokji RS, Shortt J, Valcarcel D, Jonasova A, Dimicoli-Salazar S, Tiong IS, Lin CC, Li J, Zhang J, Giuseppi AC, Kreitz S, Pozharskaya V, Keeperman KL, Rose S, Shetty JK, Hayati S, Vodala S, Prebet T, Degulys A, Paolini S, Cluzeau T, Fenaux P, Garcia-Manero G. Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial. Lancet. 2023 Jul 29;402(10399):373-385. doi: 10.1016/S0140-6736(23)00874-7. Epub 2023 Jun 10.
• Results de Witte T, Bowen D, Robin M, Malcovati L, Niederwieser D, Yakoub-Agha I, Mufti GJ, Fenaux P, Sanz G, Martino R, Alessandrino EP, Onida F, Symeonidis A, Passweg J, Kobbe G, Ganser A, Platzbecker U, Finke J, van Gelder M, van de Loosdrecht AA, Ljungman P, Stauder R, Volin L, Deeg HJ, Cutler C, Saber W, Champlin R, Giralt S, Anasetti C, Kroger N. Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an international expert panel. Blood. 2017 Mar 30;129(13):1753-1762. doi: 10.1182/blood-2016-06-724500. Epub 2017 Jan 17.
• Results Gratwohl A, Baldomero H, Aljurf M, Pasquini MC, Bouzas LF, Yoshimi A, Szer J, Lipton J, Schwendener A, Gratwohl M, Frauendorfer K, Niederwieser D, Horowitz M, Kodera Y; Worldwide Network of Blood and Marrow Transplantation. Hematopoietic stem cell transplantation: a global perspective. JAMA. 2010 Apr 28;303(16):1617-24. doi: 10.1001/jama.2010.491.

Drugs

Indications