Promacta (eltrombopag) - Novartis
Oncostatin M deficiency: A newly recognized inherited bone marrow failure syndrome with potential predisposition to myeloid malignancy. (PubMed, Hematol Oncol Stem Cell Ther) - Oct 15, 2026 - "In patients with biallelic variants in OSM, hematologic improvement was observed following eltrombopag in two reported patients, while transfusion dependency and high-risk cytogenetics required hematopoietic stem cell transplantation. OSM deficiency is rare; its mechanistic and clinical parallels with other IBMFS underscore its importance. This review outlines the emerging evidence on OSM's function in hematopoiesis and its deficiency as a newly recognized IBMFS with a potential predisposition to MDS, highlighting the need for early genetic diagnosis and tailored treatment strategies." 
Journal • Review • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • IL6 • LIF • LIFR
http://www.ncbi.nlm.nih.gov/pubmed/42870724
 
Abdullah H Alfalah; Alfadil Haroon; Mahmoud Aljurf; Riad El Fakih; Sateesh Maddirevula; Syed Osman Ahmed
 
Oct 15, 2026
 
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