News and Treatment Updates

Here's where you'll find a regularly updated, broad range of articles written by the AAMDSIF team, allied health organizations and news organizations. By staying well-informed, patients and families are practicing a form of self-support that will help them be more effective self-advocates when engaging with health care providers.

Health-related quality of life and symptom profile of patients with BCR::ABL1-negative myeloproliferative neoplasms: Real-world evidence from the GIMEMA-PROPHECY observational study

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Abstract Health-related quality of life (HRQoL) of patients with myeloproliferative neoplasms (MPNs) may be impaired across several domains. In this multicenter observational study, we evaluated HRQoL and symptoms in a cohort of MPN patients with validated measures, including the…

Isolated KRAS and NRAS mutations in adults with monocytosis and/or cytopenia(s)

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Abstract NRAS and KRAS mutations, commonly identified alongside ancestral co-mutations, are generally regarded as pathogenic in adults presenting with monocytosis and/or cytopenia(s). However, their significance in isolation is not well defined. We studied a multi-institutional…

Isolated KRAS and NRAS mutations in adults with monocytosis and/or cytopenia(s)

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Abstract NRAS and KRAS mutations, commonly identified alongside ancestral co-mutations, are generally regarded as pathogenic in adults presenting with monocytosis and/or cytopenia(s). However, their significance in isolation is not well defined. We studied a multi-institutional…

Basiliximab in the Prophylaxis of aGVHD for Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Thalassemia Major: A Prospective, Multicenter, Open-Label, Randomized Controlled Study

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To the Editor, We present the first prospective, multicenter, randomized controlled trial (RCT) evaluating Basiliximab prophylaxis for acute graft-versus-host disease (GVHD) in thalassemia major (TM) patients undergoing Matched unrelated donor hematopoietic stem-cell…

Basiliximab in the Prophylaxis of aGVHD for Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Thalassemia Major: A Prospective, Multicenter, Open-Label, Randomized Controlled Study

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To the Editor, We present the first prospective, multicenter, randomized controlled trial (RCT) evaluating Basiliximab prophylaxis for acute graft-versus-host disease (GVHD) in thalassemia major (TM) patients undergoing Matched unrelated donor hematopoietic stem-cell…

Oral Decitabine/Cedazuridine Plus Venetoclax Yields 91% ORR in High-Risk MDS and CMML

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Key Takeaways The combination therapy achieved a 91% overall response rate, with 45% complete remission and 46% marrow complete remission rates. Median overall survival was 30 months, with 1- and 3-year survival rates of 68.8% and 40.7%, respectively. SHOW MORE Decitabine…

Rusfertide Continues to Show Strong Results for Polycythemia Vera

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Rusfertide sustained hematocrit control and sharply reduced phlebotomy need through week 52 in PV, showing durable efficacy and a consistent safety profile. Treatment with the hepcidin mimetic rusfertide continued to demonstrate sustained hematocrit control below 45% and high…

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