Blood Adv

ASH updates playbook for severe acquired aplastic anemia

April 2, 2026

The American Society of Hematology (ASH) has published its 2026 Guidelines for the Diagnosis and Management of Severe Acquired Aplastic Anemia (SAA), providing 33 evidence‑based recommendations and four good practice statements for both adults and children with severe or very severe disease. Developed using GRADE methodology, the guidelines address ongoing variation in practice and incorporate newer therapeutic strategies, while emphasizing shared decision‑making and individualized care.

(Most recommendations are conditional, reflecting low or very low certainty evidence.)

Confirm the diagnosis carefully

• Use peripheral counts and marrow hypocellularity to define severe disease (ANC <500/µL, platelets <20,000/µL, reticulocytes <60,000/µL), and exclude inherited marrow failure syndromes and alternative causes of pancytopenia.

Upfront treatment selection

• Matched sibling hematopoietic stem cell transplantation (HSCT) is recommended as first‑line therapy for eligible younger patients.
• Immunosuppressive therapy (IST) remains standard first‑line treatment for patients without a suitable donor or with higher transplant risk.

Triple IST as contemporary standard

• Incorporate eltrombopag with antithymocyte globulin (ATG) plus cyclosporine for most patients receiving IST, reflecting improved hematologic response rates in recent studies.

Supportive and preventive care

• Use antimicrobial prophylaxis in patients at high risk for infection.
• Apply transfusion support and iron overload monitoring as part of routine management.

Monitoring and next steps

• Regularly assess response to IST and plan early for second‑line therapy or transplant referral in nonresponders.
• Emphasize shared decision‑making, recognizing patient preferences, age, comorbidities, and donor availability.

• Formal ASH guideline framework using GRADE and Evidence‑to‑Decision processes.
• Clear endorsement of eltrombopag‑containing IST regimens.
• More explicit recommendations on diagnostic testing and infection prophylaxis, areas previously driven largely by expert opinion.

Source: Scheinberg P, et al. (2026, March 24). Blood Adv. American Society of Hematology 2026 Guidelines for the Diagnosis and Management of Severe Acquired Aplastic Anemia