The American Society of Hematology (ASH) released guidelines on the diagnosis and management of severe acquired aplastic anemia, a rare and life-threatening bone marrow failure disorder. The guidelines, developed by a multidisciplinary expert panel including a patient with lived experience, outline evidence-based best practices to improve outcomes for individuals living with the disorder. They were published in Blood Advances.
"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."
In aplastic anemia, a person's bone marrow stops producing enough blood cells, leaving them vulnerable to profound fatigue, life-threatening infections, and dangerous bleeding. The disorder affects one to two million people per year in western countries, with higher incidence reported in Asia (up to five to seven million people per year). Most cases of aplastic anemia are immune-mediated (caused by an overactive immune system response) or idiopathic (with no identifiable cause); however, the disorder can also be acquired, or triggered by infections, medications, toxic substances, chemicals, and other environmental exposures. These guidelines focus on severe cases of acquired aplastic anemia, which are characterized by especially low counts of neutrophils (white blood cells, <500 per microliter), anemia with low reticulocytes (immature red blood cells, <60,000 per microliter), and platelets (cells necessary for clotting, <20,000 per microliter) in the blood.
The guidelines outline 33 distinct recommendations and four good practice statements for improving the diagnosis and treatment of severe acquired aplastic anemia in children and adults. Recommendations include:
- Expanded use of advanced diagnostic testing to improve diagnosis and guide treatment decision, including specialized blood and genetic tests
- Personalized initial treatment based on age and availability of a fully matched donor
- Addition of eltrombopag to standard immunosuppressive therapy to improve blood cell counts in both adults and children
- Guidance for patients who do not respond or relapse after initial immunosuppression, including earlier transition to second-line treatments
The guidelines also highlight the importance of shared decision making between patients and their providers and a need for additional research to develop higher-certainty recommendations.
"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."
Additional guidelines resources, including infographics, visual summaries, and teaching slides, can be accessed at hematology.org/aplasticanemia .
