title: Factors influencing outcome and incidence of late complications in children who underwent allogeneic hematopoietic stem cell transplantation for hemoglobinopathy.
abstract: BACKGROUND
  Hematopoietic stem cell transplantation (HSCT) remains the only potentially curative treatment for severe hemoglobinopathy (HGP). Late complications (LCs) are all events occurring beyond two years post-HSCT. We retrospectively analyzed prevalence, factors influencing occurrence, and prognosis of LCs post-HSCT for HGP.
  
  
  METHODS
  Between 2000 and 2011, 47 patients (21 males, 26 females; 43 with beta thalassemia major, four with sickle cell disease) who had survived more than two years post-HSCT for HGP were retrospectively reviewed. Mean age at HSCT was 7.7 years (1.1-32 years); mean follow-up was 7.1 years (2-11.6 years); 11 patients were splenectomized; mean ferritin level was 3022 ng/mL (350-10900); and seven patients underwent a second HSCT.
  
  
  RESULTS
  Endocrinological complications were observed with primary gonadal failure in 16/20 mature females and 4/11 mature males, in five patients with primary hypothyroidism and in four with insulin-dependent diabetes mellitus (DM). Skeletal complications were observed in 10 with secondary osteoporosis; 22 patients had elevated transaminase levels; two had hepatitis B reactivation. Neurological, cardiac and ocular manifestations were relatively rare. A higher incidence of LCs was observed in splenectomized than in nonsplenectomized patients: cGVHD -64% versus 13% (P = .003); endocrine abnormalities -91% versus 30.5%, (P = .001); elevated transaminase levels -73% versus 33% (P = .043); mortality -18% versus 2.7% (NS).
  
  
  CONCLUSIONS
  LCs post-HSCT for HGP are common and heterogeneous. Etiology is multifactorial with iron overload (IO), class, splenectomy, age, chronic GVHD, and corticosteroid (CS) treatment. Our data may help build follow-up guidelines to limit, detect, and treat any LCs and improve quality of life.
authors.affiliation: Department of Pediatric Hematology Oncology, Meyer Children's Hospital, Rambam Health Care Campus, Haifa, Israel.
authors.forename: Abdalla
authors.initials: A
authors.lastname: Khalil
authors.forename: Irena
authors.initials: I
authors.lastname: Zaidman
authors.forename: Ronit
authors.initials: R
authors.lastname: Elhasid
authors.forename: Monique
authors.initials: M
authors.lastname: Peretz-Nahum
authors.forename: Boris
authors.initials: B
authors.lastname: Futerman
authors.forename: Myriam
authors.initials: M
authors.lastname: Ben-Arush
mesh_terms: D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000453:epidemiology / Q000628:therapy; D002648:Child; D002675:Child, Preschool; D002908:Chronic Disease; D004700:Endocrine System Diseases / Q000097:blood / Q000453:epidemiology* / Q000209:etiology; D005128:Eye Diseases / Q000097:blood / Q000453:epidemiology* / Q000209:etiology; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000097:blood / Q000453:epidemiology* / Q000209:etiology; D006331:Heart Diseases / Q000097:blood / Q000453:epidemiology* / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006509:Hepatitis B / Q000097:blood / Q000453:epidemiology* / Q000209:etiology; D006801:Humans; D015994:Incidence; D007223:Infant; D008297:Male; D009422:Nervous System Diseases / Q000097:blood / Q000453:epidemiology* / Q000209:etiology; D012189:Retrospective Studies; D012307:Risk Factors; D014184:Transplantation, Homologous; D017086:beta-Thalassemia / Q000453:epidemiology / Q000628:therapy