title: Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato-Oncology Italian Association (AIEOP).
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.
authors.affiliation: Department of Pediatric Onco-Hematology, University Hospital, Parma, Italy.
authors.forename: Angelica
authors.initials: A
authors.lastname: Barone
authors.affiliation: Pediatric Onco-Hematology Unit, Vito Fazzi Hospital, Lecce, Italy.
authors.forename: Annunziata
authors.initials: A
authors.lastname: Lucarelli
authors.affiliation: Hematology Unit, Hospital of Pescara, Italy.
authors.forename: Daniela
authors.initials: D
authors.lastname: Onofrillo
authors.affiliation: Institute of Maternal and Child Health IRCCS Burlo Garofalo, Trieste, Italy.
authors.forename: Federico
authors.initials: F
authors.lastname: Verzegnassi
authors.affiliation: Department of Pediatric Hematology, San Gerardo Hospital, Monza, Italy.
authors.forename: Sonia
authors.initials: S
authors.lastname: Bonanomi
authors.affiliation: Pediatric Hematology Oncology Unit, Borgo Roma Hospital, Verona, Italy.
authors.forename: Simone
authors.initials: S
authors.lastname: Cesaro
authors.affiliation: Clinical and Experiment Hematology Unit, G. Gaslini Children's Hospital, Genoa, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Fioredda
authors.affiliation: Hematology and HSCT Unit, University La Sapienza, Rome, Italy.
authors.forename: Anna Paola
authors.initials: AP
authors.lastname: Iori
authors.affiliation: Pediatric Onco-Hematology Unit, Casa Sollievo della Sofferenza Hospital, San Giovanni Rotondo, Italy.
authors.forename: Saverio
authors.initials: S
authors.lastname: Ladogana
authors.affiliation: Department of Pediatric Hematology, S. Camillo Hospital, Rome, Italy.
authors.forename: Anna
authors.initials: A
authors.lastname: Locasciulli
authors.affiliation: Department of Pediatric Hematology, San Gerardo Hospital, Monza, Italy.
authors.forename: Daniela
authors.initials: D
authors.lastname: Longoni
authors.affiliation: Clinical and Experiment Hematology Unit, G. Gaslini Children's Hospital, Genoa, Italy.
authors.forename: Marina
authors.initials: M
authors.lastname: Lanciotti
authors.affiliation: Pediatric Onco-Hematology Unit, A.R.N.A.S. Civico Hospital, Palermo, Italy.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Macaluso
authors.affiliation: Pediatric Onco-Hematology and Transfusional Department, Pugliese Ciaccio Hospital, Catanzaro, Italy.
authors.forename: Rosalba
authors.initials: R
authors.lastname: Mandaglio
authors.affiliation: Department of Pediatric Haemato-Oncology, Santobono-Pausilipon Hospital, Naples, Italy.
authors.forename: Nicoletta
authors.initials: N
authors.lastname: Marra
authors.affiliation: Department of Pediatric, University of Bari, Bari, Italy.
authors.forename: Baldo
authors.initials: B
authors.lastname: Martire
authors.affiliation: Pediatric Onco-Hematology Unit, Casa Sollievo della Sofferenza Hospital, San Giovanni Rotondo, Italy.
authors.forename: Matteo
authors.initials: M
authors.lastname: Maruzzi
authors.affiliation: Department of Pediatric Haemato-Oncology, Santobono-Pausilipon Hospital, Naples, Italy.
authors.forename: Giuseppe
authors.initials: G
authors.lastname: Menna
authors.affiliation: Oncology-Haematology and Bone Marrow Transplantation Unit, Children's Hospital, Spedali Civili, Brescia, Italy.
authors.forename: Lucia Dora
authors.initials: LD
authors.lastname: Notarangelo
authors.affiliation: Department of Mother and Child, University Hospital of Modena, Modena, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Palazzi
authors.affiliation: Pediatric Onco-Hematology Unit, University Hospital of Padua, Padua, Italy.
authors.forename: Marta
authors.initials: M
authors.lastname: Pillon
authors.affiliation: University of Turin, Turin, Italy.
authors.forename: Ugo
authors.initials: U
authors.lastname: Ramenghi
authors.affiliation: Division of Pediatric Hematology/Oncology, University of Catania, Catania, Italy.
authors.forename: Giovanna
authors.initials: G
authors.lastname: Russo
authors.affiliation: Clinical and Experiment Hematology Unit, G. Gaslini Children's Hospital, Genoa, Italy.
authors.forename: Johanna
authors.initials: J
authors.lastname: Svahn
authors.affiliation: Pediatric Onco-Hematology, Regina Margherita Children's Hospital, Turin, Italy.
authors.forename: Fabio
authors.initials: F
authors.lastname: Timeus
authors.affiliation: Department of Pediatric Onco-Hematology, Meyer Children's Hospital, Florence, Italy.
authors.forename: Fabio
authors.initials: F
authors.lastname: Tucci
authors.affiliation: Pediatric Hematology/Oncology Department, IRCCS San Matteo Hospital, Pavia, Italy.
authors.forename: Chiara
authors.initials: C
authors.lastname: Cugno
authors.affiliation: Pediatric Hematology/Oncology Department, IRCCS San Matteo Hospital, Pavia, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.affiliation: Pediatric Onco-Hematology Unit, A.R.N.A.S. Civico Hospital, Palermo, Italy.
authors.forename: Piero
authors.initials: P
authors.lastname: Farruggia
authors.affiliation: Clinical and Experiment Hematology Unit, G. Gaslini Children's Hospital, Genoa, Italy. Electronic address: carlodufour@ospedale-gaslini.ge.it.
authors.forename: Carlo
authors.initials: C
authors.lastname: Dufour
authors.affiliation: University of Turin, Turin, Italy.
authors.forename: Paola
authors.initials: P
authors.lastname: Saracco
mesh_terms: D000741:Anemia, Aplastic / Q000139:chemically induced / Q000175:diagnosis* / Q000276:immunology / Q000628:therapy*; D000900:Anti-Bacterial Agents / Q000009:adverse effects; D000893:Anti-Inflammatory Agents / Q000009:adverse effects; D000961:Antilymphocyte Serum / Q000627:therapeutic use; D018501:Antirheumatic Agents / Q000009:adverse effects; D001853:Bone Marrow / Q000187:drug effects / Q000276:immunology / Q000473:pathology; D002648:Child; D016572:Cyclosporine / Q000627:therapeutic use; D019468:Disease Management; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006650:Histocompatibility Testing; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D010755:Organophosphates / Q000633:toxicity; D010198:Pancytopenia / Q000139:chemically induced / Q000175:diagnosis* / Q000276:immunology / Q000628:therapy*; D035781:Siblings; D061349:Unrelated Donors
chemical_list: D000900:Anti-Bacterial Agents; D000893:Anti-Inflammatory Agents; D000961:Antilymphocyte Serum; D018501:Antirheumatic Agents; D007166:Immunosuppressive Agents; D010755:Organophosphates; D016572:Cyclosporine
keywords: Acquired aplastic anemia; Childhood; Guidelines