title: Autoimmune hematological diseases after allogeneic hematopoietic stem cell transplantation in children: an Italian multicenter experience.
abstract: Autoimmune hematological diseases (AHDs) may occur after allogeneic hematopoietic stem cell transplantation (HSCT), but reports on these complications in large cohorts of pediatric patients are lacking. Between 1998 and 2011, 1574 consecutive children underwent allogeneic HSCT in 9 Italian centers. Thirty-three children (2.1%) developed AHDs: 15 autoimmune hemolytic anemia (45%), 10 immune thrombocytopenia (30%), 5 Evans' syndrome (15%), 2 pure red cell aplasia (6%), and 1 immune neutropenia (3%). The 10-year cumulative incidence of AHDs was 2.5% (95% confidence interval, 1.7 to 3.6). In a multivariate analysis, the use of alternative donor and nonmalignant disease was statistically associated with AHDs. Most patients with AHDs (64%) did not respond to steroids. Sustained complete remission was achieved in 87% of cases with the anti-CD20 monoclonal antibody (rituximab). Four patients (9%) (1 autoimmune hemolytic anemia, 1 Evans' syndrome, 2 immune thrombocytopenia) died at a median of 87 days after AHD diagnosis as a direct or indirect consequence of their disorder. Our data suggest that AHDs are a relatively rare complication occurring after HSCT that usually respond to treatment with rituximab.
authors.affiliation: Haematopoietic Stem Cell Transplantation Unit, Haematology-Oncology Department, G. Gaslini Children's Research Institute, Genova, Italy. Electronic address: maurafaraci@ospedale-gaslini.ge.it.
authors.forename: Maura
authors.initials: M
authors.lastname: Faraci
authors.affiliation: Paediatric Haematology/Oncology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Zecca
authors.affiliation: Paediatric Oncology-Haematology Unit, University Hospital of Padova, Padova, Italy.
authors.forename: Marta
authors.initials: M
authors.lastname: Pillon
authors.affiliation: Paediatric Haematopoietic Stem Cell Transplantation Unit, MBBM Foundation, University of Milano-Bicocca, Monza, Italy.
authors.forename: Attilio
authors.initials: A
authors.lastname: Rovelli
authors.affiliation: Haematopoietic Stem Cell Transplantation Unit, Paediatric Clinic of University of Pisa, Pisa, Italy.
authors.forename: Maria Cristina
authors.initials: MC
authors.lastname: Menconi
authors.affiliation: Haematopoietic Stem Cell Transplantation Unit, Haematology-Oncology Department, Pausilipon Hospital, Napoli, Italy.
authors.forename: Mimmo
authors.initials: M
authors.lastname: Ripaldi
authors.affiliation: Haematopoietic Stem Cell Transplantation Unit, Department of Paediatrics, Regina Margherita Children's Hospital, University of Turin, Turin, Italy.
authors.forename: Franca
authors.initials: F
authors.lastname: Fagioli
authors.affiliation: Haematopoietic Stem Cell Transplantation Unit, Paediatric Clinic, Burlo Garofolo Research Institute, Trieste, Italy.
authors.forename: Marco
authors.initials: M
authors.lastname: Rabusin
authors.affiliation: Paediatric Haematology Oncology, Ospedale dei Bambini "G. Di Cristina, Palermo", Palermo, Italy.
authors.forename: Ottavio
authors.initials: O
authors.lastname: Ziino
authors.affiliation: Haematopoietic Stem Cell Transplantation Unit, Haematology-Oncology Department, G. Gaslini Children's Research Institute, Genova, Italy.
authors.forename: Edoardo
authors.initials: E
authors.lastname: Lanino
authors.affiliation: Department of Paediatric Haematology-Oncology, IRCCS Bambino Gesù Children's Hospital, Rome, University of Pavia, Pavia, Italy.
authors.forename: Franco
authors.initials: F
authors.lastname: Locatelli
authors.affiliation: Department of Rheumatology, University Hospital Basel, Basel, Switzerland.
authors.forename: Thomas
authors.initials: T
authors.lastname: Daikeler
authors.affiliation: Paediatric Oncology and Haematology Unit Lalla Seràgnoli, Department of Paediatrics, University of Bologna Sant'Orsola-Malpighi Hospital, Bologna, Italy.
authors.forename: Arcangelo
authors.initials: A
authors.lastname: Prete
mesh_terms: D002648:Child; D002675:Child, Preschool; D005260:Female; D006402:Hematologic Diseases / Q000188:drug therapy / Q000209:etiology*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000379:methods; D006801:Humans; D007558:Italy; D008297:Male; D012074:Remission Induction; D012307:Risk Factors; D019172:Transplantation Conditioning / Q000009:adverse effects* / Q000379:methods
keywords: Autoimmune hematological disease; Children; Hematopoietic stem cell transplantation; Rituximab