Transfusion Independence Achieved with Combination Fedratinib and Luspatercept in an Elderly Man with Heavily Pretreated Intermediate-2 Risk Primary Myelofibrosis

Case Reports in Oncology

Nicole K. Yun¹  Ira J. Miller²  Taha Alrifai³  Jamile M. Shammo³ 

[1] Department of Internal Medicine, Rush University Medical Center, Chicago, IL, USA;Department of Pathology, Rush University Medical Center, Chicago, IL, USA;Division of Hematology, Oncology, and Cell Therapy, Rush University Medical Center, Chicago, IL, USA;

关键词: myeloproliferative neoplasm;    primary myelofibrosis;    fedratinib;    luspatercept;    transfusion independence;    heavily pretreated;   

DOI  :  10.1159/000521889

来源: DOAJ

【 摘 要 】

Myelofibrosis (MF)-associated anemia and transfusion dependency are associated with inferior quality of life and poor prognosis. JAK2 inhibitors and TGF-β superfamily ligand traps are being explored as treatment options for MF-associated anemia. Here, we present the case of a 66-year-old man with heavily pretreated intermediate-2 (INT-2) risk primary MF who had an exceptional response to combination fedratinib and luspatercept therapy. He achieved transfusion independence and experienced a reduction in spleen size from 20 cm to 12 cm, with remarkable improvement in performance status. Compared with other JAK inhibitors, the mechanism of action of fedratinib may explain its milder effect on anemia. It is possible that the addition of luspatercept may result in an additive or synergistic effect of one or both medications. Although the exact biological pathways have not yet been elucidated, combination fedratinib and luspatercept nevertheless is a promising therapy for anemia in patients with transfusion-dependent INT-2 risk MF.

【 授权许可】

Unknown