ASH Publishes Guidelines on Diagnosis and Management of Severe and Very Severe Acquired Aplastic Anemia

The American Society of Hematology (ASH)’s new Clinical Practice Guidelines on Diagnosis and Management of Severe and Very Severe Acquired Aplastic Anemia (AA) outline 33 recommendations and 4 good practice statements for children and adults.1

The guidelines focus on tools to improve diagnosis and treatment decisions, including expanded use of specialized blood and genetic tests, personalized initial treatment based on age and availability of a fully matched donor, the addition of eltrombopag to standard immunosuppressive therapy to improve blood cell counts, and earlier transition to second-line treatments after initial immunosuppression for patients who do not respond or relapse.1

“It’s critical to definitively diagnose individuals with aplastic anemia to ensure that they are promptly connected to appropriate treatment,” said Phil Scheinberg, MD, head of the division of hematology at Hospital Beneficência Portuguesa Hospital in São Paulo, and chair of the ASH 2026 Guidelines for the Diagnosis and Management of Severe and Very Severe Acquired Aplastic Anemia. “We hope these guidelines will not only improve patient outcomes but also expand access to diagnostic testing and catalyze broader global access to transplant and immunosuppressive therapies.”

The rare and serious hematologic disorder, AA, is characterized by the failure of bone marrow to produce enough blood cells, which leads to pancytopenia, a serious blood condition characterized by a deficiency in the cellular components of red blood cells, white blood cells, and platelets. Often, the severe complications of aplastic anemia include profound fatigue, increased susceptibility to infections, and an increased hemorrhage risk.2

In the US and Europe, the incidence of acquired AA is 2 to 3 per million people per year, and has increased rates in Asia, reported at 5–6 per million in some regions. At diagnosis, about 79.6% to 83.8% of these patients have severe or very severe disease. If treated with supportive care alone, mortality can be > 80% within 2 years due to severe neutropenia (low neutrophil count) and thrombocytopenia, leading to infection or hemorrhage.3

Modern treatment strategies such as allogeneic hematopoietic cell transplantation (alloHCT), stem cell bone marrow transplants, blood transfusions or Immunosuppressive Therapy (IST) have improved these 2-year survival rates to >80%.3

To create the guidelines, a multidisciplinary expert panel, including a patient with liver experience, focused on severe cases of acquired aplastic anemia, characterized by especially reduced counts of neutrophils (white blood cells, <500 per microliter), anemia with reduced reticulocytes (immature red blood cells, <60,000 per microliter), and platelets (cells necessary for clotting, <20,000 per microliter) in the blood.1

The updated 2026 guidelines included eltombopag, a small molecule thrombopoetin (TPO) mimetic, as a first-line treatment option. It is approved by the US Food and Drug Administration (FDA) to treat severe AA in combination with standard immunosuppressive therapy for newly diagnosed adults and pediatric patients ≥2 years of age and for patients who have exhibited an insufficient response to immunosuppressive therapy.1,2

“The Society is proud to provide this robust and much-needed resource for clinicians caring for individuals with aplastic anemia,” said Robert Negrin, MD, ASH president. “These guidelines offer important consensus for the field while helping raise awareness of a serious, yet often overlooked, disorder.”

References

1. Scheinberg P, O’Neal DA, Basquiera AL, et al. American Society of Hematology 2026 Guidelines for the Diagnosis and Management of Severe Acquired Aplastic Anemia. Blood Advances. Published March 24, 2026. Accessed April 2, 2026. https://ashpublications.org/bloodadvances/article/doi/10.1182/bloodadvances.2025019051/567383/American-Society-of-Hematology-2026-Guidelines-for

2. Schoettler ML, Nathan DG. The Pathophysiology of Acquired Aplastic Anemia. Hematology/Oncology Clinics of North America. 2018;32(4):581-594. doi:https://doi.org/10.1016/j.hoc.2018.03.001

3. Moore CA, Krishnan K. Aplastic Anemia. PubMed. Published 2022. https://pubmed.ncbi.nlm.nih.gov/30480951/

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