treatment

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…

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…

Abstract Improved ex vivo expansion of human haematopoietic stem cells (HSCs) would considerably advance transplantation and genome-engineered therapies, yet existing culture methods still allow substantial HSC loss. Here we show that this attrition is driven largely by…

Aged individuals with somatic TP53 mutations manifest clonal hematopoiesis (CH) and are at high risk of developing myeloid neoplasms. However, the underlying mechanisms are not fully understood. Here we show that inflammatory stress confers a competitive advantage to p53 mutant…

Abstract Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for aplastic anemia (AA), especially in younger patients with matched sibling donors (MSD). However, in low-resource settings, delayed access to HSCT and pre-transplant complications can…

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…

NIH clinical trial shows promise for people with treatment-limited, life-threatening disorder. A clinical trial at the National Institutes of Health (NIH) has shown that an experimental treatment for patients with severe aplastic anemia resulted in a 94% survival rate without…

Chemotherapy-free approaches could offer comparable efficacy with less toxicity (ORLANDO, Dec. 7, 2025) — Targeted therapies and immunotherapies are increasingly offering viable alternatives to the chemotherapies that have stood for decades as a mainstay of treatment for…

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…