30-Second Takeaway
- ASH 2026 aplastic anemia guidelines refine transplant versus IST decisions and endorse eltrombopag and antimicrobial prophylaxis.
- Gene therapy guidance in sickle cell disease standardizes eligibility, conditioning, manufacturing, and life-long surveillance as programs scale up.
- HMA–venetoclax is emerging as a competitive induction option in selected younger AML, enabling high MRD-negative remissions and transplant.
Week ending April 4, 2026
Practice-shaping updates in marrow failure, myeloid disease, CLL, plasma cell disorders, and cellular therapies
ASH 2026 guideline standardizes diagnosis and frontline management of severe acquired aplastic anemia
These ASH guidelines issue 33 recommendations and 4 good practice statements for severe and very severe immune acquired aplastic anemia. They prioritize upfront allogeneic hematopoietic cell transplantation for younger patients with an available matched sibling or unrelated donor. For non-transplant candidates, the panel recommends immunosuppressive therapy with the addition of eltrombopag to enhance hematologic response. The guideline supports antibiotic and antifungal prophylaxis in neutropenic high-risk patients to reduce infectious complications.
Consensus playbook for implementing ex vivo gene therapy in sickle cell disease
This ASTCT/ISCT consensus document offers practical recommendations for clinical implementation of ex vivo gene therapies in sickle cell disease. It addresses patient eligibility, including comparative considerations with allogeneic transplant when selecting curative strategies. The authors outline approaches to stem cell mobilization, apheresis, and myeloablative conditioning, emphasizing fertility preservation and psychosocial support. They specify manufacturing quality attributes and recommend lifelong surveillance for late effects to promote durable, safe, and equitable delivery.
HMA–venetoclax induction achieves high MRD-negative remissions and transplant rates in younger AML
This meta-analysis pooled eight studies including 429 younger AML patients (mean age 54) treated with hypomethylating agents plus venetoclax. The combined complete remission/CRi rate was 66%, with 69% of responders attaining measurable residual disease negativity. One-year overall survival was 75%, exceeding Surveillance, Epidemiology, and End Results historical cohorts at 62%. Event-free survival at 1 year was 59%, and 66% of patients proceeded to allogeneic transplantation.
Five-year CLL13/GAIA data support venetoclax–obinutuzumab–based fixed-duration regimens in fit TP53‑wildtype CLL
In this phase 3 trial, 926 fit TP53‑wildtype CLL patients were randomized to chemoimmunotherapy or three venetoclax-based fixed-duration combinations. Five-year progression-free survival was 81.3% with GIV, 69.8% with GV, 57.4% with RV, and 50.7% with chemoimmunotherapy. GV and GIV significantly outperformed chemoimmunotherapy and RV for progression-free survival, with GIV also superior to GV. Severe infections were most common with chemoimmunotherapy, while cardiac events were more frequent with GIV, informing regimen choice.
References
Numbered in order of appearance. Click any reference to view details.
Additional Reads
Optional additional studies from this edition.