Giugno 2014 - Volume XXXIII - numero 6

Medico e Bambino


Linee guida

Aplasie midollari acquisite in età pediatrica: raccomandazioni diagnostico-terapeutiche

Angelica Barone1, Annunziata Lucarelli1, Daniela Onofrillo1, Federico Verzegnassi1, Sonia Bonanomi, Simone Cesaro, Chiara Cugno, 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 Palazzi, Marta Pillon, Ugo Ramenghi, Giovanna Russo, Johanna Svahn, Fabio Timeus, Fabio Tucci, Marco Zecca, Piero Farruggia2, Carlo Dufour2, Paola Saracco2

Gruppo di Lavoro “Insufficienze Midollari” dell’AIEOP - Associazione Italiana di Ematologia e Oncologia Pediatrica
1Questi Autori hanno fornito il medesimo contributo alla stesura delle presenti Linee Guida e condividono la posizione di primo Autore; 2Questi Autori condividono la posizione di ultimo Autore

Indirizzo per corrispondenza: ugo.ramenghi@unito.it

Acquired aplastic anaemia in children: diagnostic-therapeutic recommendations

Key words: Acquired aplastic anemia, Childhood

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.

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Gruppo di Lavoro “Insufficienze Midollari” dell’AIEOP Aplasie midollari acquisite in età pediatrica: raccomandazioni diagnostico-terapeutiche. Medico e Bambino 2014;33(6):383-389 https://www.medicoebambino.com/?id=1406_383.pdf


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