Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association (AIEOP)

Blood Cells Mol Dis. 2015 Jun;55(1):40-7. doi: 10.1016/j.bcmd.2015.03.007. Epub 2015 Mar 31.


Acquired aplastic anemia (AA) is a rare heterogeneous disease characterized by pancytopenia and hypoplastic bone marrow. The incidence is 2-3/million inhabitants/year, in Europe, but higher in East Asia. Survival in severe aplastic anemia (SAA) has markedly improved in the past 2 decades because of advances in hematopoietic stem cell transplantation, immunosuppressive and biologic drugs, and supportive care. In SAA hematopoietic stem cell transplant (HSCT) from a matched sibling donor (MSD) is the treatment of choice. If a MSD is not available, the options include immunosuppressive therapy (IST) or unrelated donor HSCT. The objective of this guideline is to provide healthcare professionals with clear guidance on the diagnosis and management of pediatric patients with AA. A preliminary, evidence-based document issued by a group of pediatric hematologists was discussed, modified and approved during a series of "Consensus Conferences" according to procedures previously validated by the AIEOP Board. The guidelines highlight the importance of referring pediatric patients with AA to pediatric centers with long experience in diagnosis, differential diagnosis, management, supportive care and follow-up of AA.

Keywords: Acquired aplastic anemia; Childhood; Guidelines.

Publication types

  • Guideline

MeSH terms

  • Anemia, Aplastic / chemically induced
  • Anemia, Aplastic / diagnosis*
  • Anemia, Aplastic / immunology
  • Anemia, Aplastic / therapy*
  • Anti-Bacterial Agents / adverse effects
  • Anti-Inflammatory Agents / adverse effects
  • Antilymphocyte Serum / therapeutic use
  • Antirheumatic Agents / adverse effects
  • Bone Marrow / drug effects
  • Bone Marrow / immunology
  • Bone Marrow / pathology
  • Child
  • Cyclosporine / therapeutic use
  • Disease Management
  • Hematopoietic Stem Cell Transplantation / methods*
  • Histocompatibility Testing
  • Humans
  • Immunosuppressive Agents / therapeutic use*
  • Organophosphates / toxicity
  • Pancytopenia / chemically induced
  • Pancytopenia / diagnosis*
  • Pancytopenia / immunology
  • Pancytopenia / therapy*
  • Siblings
  • Unrelated Donors


  • Anti-Bacterial Agents
  • Anti-Inflammatory Agents
  • Antilymphocyte Serum
  • Antirheumatic Agents
  • Immunosuppressive Agents
  • Organophosphates
  • Cyclosporine