title: Autoimmune complications after hematopoietic stem cell transplantation in children with nonmalignant disorders.
abstract: BACKGROUND
  Hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for many nonmalignant disorders, such as autoimmune disorders, inborn metabolic disorders, hemoglobinopathies, and immunodeficiency disorders. Autoimmune complications (AICs) after HSCT, such as autoimmune cytopenias, autoimmune hepatitis, primary biliary cirrhosis, and autoimmune cutaneous manifestations, are still neither well defined nor characterized.
  
  
  METHODS
  Between 2000 and 2012, 92 patients (47 males, 45 females) were treated with HSCT in our hospital, 51 with congenital hemoglobinopathies, 19 with primary immunodeficiency disease, 10 with metabolic disorders, five with Fanconi anemia, three with aplastic anemia, and four with familial hemophagocytic lymphohistiocytosis.
  
  
  RESULTS
  Mean age at HSCT was 6.4 years (range, 0.2-32 years) and mean duration of followup after HSCT was 6.81 years (range, 1-11 years). Sixteen (17.4%) patients developed chronic GVHD and five (5.4%) showed sclerodermatous features. Five (5.4%) patients were diagnosed with scleroderma manifestations, six (6.5%) with vitiligo, six (6.5%) with autoimmune hemolytic anemia (AIHA), six (6.5%) with idiopathic thrombocytopenia, three (3.3%) with mild leucopenia, two (2.2%) with aplastic anemia, two (2.2%) (one boy, one girl) with autoimmune thyroid disease, and one (1.1%) with autoimmune hepatitis.
  
  
  CONCLUSIONS
  It was concluded that AICs are clinically significant complications after HSCT that contribute to morbidity but not to mortality. AICs are more frequent after HSCT for metabolic disorders, and sclerodermatous GVHD is more significant in children who underwent allogeneic HSCT for hemoglobinopathies. The potential to identify risk factors for AICs could lead to less morbidity and mortality and to maintain the patient's quality of life.
authors.affiliation: Department of Pediatric Hematology Oncology, Meyer Children's Hospital, Rambam Health Care Campus, P.O. Box 9602, 31096 Haifa, Israel.
authors.forename: Abdalla
authors.initials: A
authors.lastname: Khalil
authors.affiliation: Department of Pediatric Hematology Oncology, Meyer Children's Hospital, Rambam Health Care Campus, P.O. Box 9602, 31096 Haifa, Israel.
authors.forename: Irena
authors.initials: I
authors.lastname: Zaidman
authors.affiliation: Department of Dermatology, Rambam Health Care Campus, Haifa, Israel ; Faculty of Medicine, Technion-Israel Institute of Technology, Haifa, Israel.
authors.forename: Reuven
authors.initials: R
authors.lastname: Bergman
authors.affiliation: Pediatric Hematology Oncology Department, Dana Children's Hospital, Tel Aviv, Israel.
authors.forename: Ronit
authors.initials: R
authors.lastname: Elhasid
authors.affiliation: Department of Pediatric Hematology Oncology, Meyer Children's Hospital, Rambam Health Care Campus, P.O. Box 9602, 31096 Haifa, Israel ; Faculty of Medicine, Technion-Israel Institute of Technology, Haifa, Israel.
authors.forename: Myriam Weyl
authors.initials: MW
authors.lastname: Ben-Arush
mesh_terms: D000293:Adolescent; D000328:Adult; D001327:Autoimmune Diseases / Q000453:epidemiology* / Q000209:etiology* / Q000473:pathology / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D020405:Dermis / Q000473:pathology; D004817:Epidermis / Q000473:pathology; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D015994:Incidence; D007223:Infant; D008297:Male; D012307:Risk Factors; D014184:Transplantation, Homologous; D055815:Young Adult