Today we are outlining key recommendations from the American Society of Hematology (ASH) 2026 guideline, Diagnosis and Management of Severe and Very Severe Acquired Aplastic Anemia. Using an evidence-based approach, ASH developed this guideline to support clinical decision-making in the diagnosis and management of patients with severe and very severe immune acquired aplastic anemia. The ASH panel developed 33 recommendations and four good practice statements addressing diagnostic testing, treatment, and supportive care. 

Below is a summary of the recommendations from the 2026 ASH guideline on severe and very severe acquired aplastic anemia. Refer to the full-text version for the full recommendations and good practice statements. 

Key Elements from the 2026 Guideline

Diagnostic Tests 

The guideline suggests diagnostic testing for patients with severe or very severe aplastic anemia, including those that are refractory to immunosuppressive therapy. Suggestions include paroxysmal nocturnal hemoglobinuria clone testing, somatic mutations testing, and telomere length testing. 

Frontline Therapy

The guideline suggests hematopoietic cell transplant (HCT) over immunosuppressive therapy (IST) for patients with severe or very severe aplastic anemia who have a matched sibling donor available, unless the patient is over 40 years of age, then the guideline suggests IST over HCT. 

In situations with a matched unrelated donor, the guideline suggests either matched unrelated HCT or IST, unless the patient is over 40 years of age, where the guideline suggests IST over matched unrelated HCT. 

For patients with a haploidentical donor, the guideline suggests IST over haploidentical HCT. 

For both children and adult patients undergoing IST, the guideline suggests adding eltrombopag. 

Second Line Therapy 

The guideline suggests HCT over second anti-thymocyte globulin-based therapy in patients under 60 years of age who have had no response to IST. For patients over 60 years of age, the guideline suggests either HCT or a second anti-thymocyte globulin-based therapy. 

For patients who initially respond to IST but relapse, the guideline suggests either HCT or a second anti-thymocyte globulin-based therapy for those under 60 years of age. In patients over 60 years of age, the guideline suggests second anti-thymocyte globulin-based therapy over HCT. 

The guideline suggests initiating second-line treatment within six months of anti-thymocyte globulin administration for patients who do not respond to IST. 

Medical Management 

The guideline suggests both mold-active antifungal prophylaxis and antibiotic prophylaxis for patients with a neutrophil count below 500 per µl.

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