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

Angelica Barone, Annunziata Lucarelli, Daniela Onofrillo, Federico Verzegnassi, Sonia Bonanomi, Simone Cesaro, Francesca Fioredda, Anna Paola Iori, Saverio Ladogana, Anna Locasciulli, Daniela Longoni, Marina Lanciotti, Alessandra Macaluso, Rosalba Mandaglio, Nicoletta Marra, Baldo Martire, Matteo Maruzzi, Giuseppe Menna, Lucia Dora Notarangelo, Giovanni PalazziMarta Pillon, Ugo Ramenghi, Giovanna Russo, Johanna Svahn, Fabio Timeus, Fabio Tucci, Chiara Cugno, Marco Zecca, Piero Farruggia, Carlo Dufour, Paola Saracco

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Abstract

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.

Original languageEnglish
Pages (from-to)40-47
Number of pages8
JournalBlood Cells, Molecules, and Diseases
Volume55
Issue number1
DOIs
Publication statusPublished - 1 Jun 2015
Externally publishedYes

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Keywords

  • Acquired aplastic anemia
  • Childhood
  • Guidelines

ASJC Scopus subject areas

  • Molecular Medicine
  • Hematology
  • Molecular Biology
  • Cell Biology

Cite this

Barone, A., Lucarelli, A., Onofrillo, D., Verzegnassi, F., Bonanomi, S., Cesaro, S., Fioredda, F., Iori, A. P., Ladogana, S., Locasciulli, A., Longoni, D., Lanciotti, M., Macaluso, A., Mandaglio, R., Marra, N., Martire, B., Maruzzi, M., Menna, G., Notarangelo, L. D., ... Saracco, P. (2015). 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, Molecules, and Diseases, 55(1), 40-47. https://doi.org/10.1016/j.bcmd.2015.03.007