Aplasie midollari acquisite in età pediatrica: Raccomandazioni diagnostico-terapeutiche

Translated title of the contribution: Acquired aplastic anaemia in children: Diagnostic-therapeutic recommendations

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

Research output: Contribution to journalArticlepeer-review

Abstract

Acquired Aplastic Anaemia (AA) is a rare heterogeneous disease characterized by pancytopoenia and hypoplastic bone marrow. The incidence is 2-3 millions per year (all age groups) in Europe, but is higher in East Asia. The pathogenesis of AA is complex and involves haematopoietic stem cell/progenitor cell deficiencies and autoimmune mechanism. Survival in severe aplastic anaemia (SAA) has markedly improved in the past 2 decades because of advances in haematopoietic stem cell transplantation, immunosuppressive and biologic drugs, and supportive care. In SAA haematopoietic 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 paediatric patients with AA. A preliminary, evidence-based document issued by a group of paediatric haematologists 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 paediatric patients with AA to paediatric centres with long experience in diagnosis, differential diagnosis, management, supportive care and follow-up of AA; haematopoietic stem cell transplantation as first line therapy if an MSD is identified; the use of first-line IST containing horse anti-thymocyte globulin and cyclosporine A (CsA) if an MSD is not identified.

Translated title of the contributionAcquired aplastic anaemia in children: Diagnostic-therapeutic recommendations
Original languageItalian
Pages (from-to)383-389
Number of pages7
JournalMedico e Bambino
Volume33
Issue number6
Publication statusPublished - 2014

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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