title: Long-term follow up after allogeneic stem cell transplantation in patients with severe aplastic anemia after cyclophosphamide plus antithymocyte globulin conditioning.
abstract: BACKGROUND
  Due to increased rates of secondary solid organ cancer in patients with severe aplastic anemia who received an irradiation-based conditioning regimen, we decided some years ago to use the combination of cyclophosphamide and antithymocyte globulin. We report the long-term follow up of patients who underwent hematopoietic stem cell transplantation from an HLA-matched sibling donor after this conditioning regimen.
  
  
  METHODS
  We analyzed 61 consecutive patients transplanted from June 1991 to February 2010, following conditioning with cyclophosphamide (200 mg/kg) and antithymocyte globulin (2.5 mg/kg/day × 5 days).
  
  
  RESULTS
  Median age was 21 years (range 4-43); 41 of the 61 patients were adults. Median duration of the disease before hematopoietic stem cell transplantation was 93 days. All but 2 patients received bone marrow as the source of stem cells and all but 2 engrafted. Cumulative incidence of acute grade II-IV graft-versus-host disease was 23% (95%CI 13-34) and 18 developed chronic graft-versus-host disease (cumulative incidence 32% at 72 months, 95% CI 20-46). In multivariate analysis, a higher number of infused CD3 cells was associated with an increased risk of developing chronic graft-versus-host disease (P = 0.017). With a median follow up of 73 months (range 8-233), the estimated 6-year overall survival was 87% (95% CI 78-97). At 72 months, the cumulative incidence of avascular necrosis was 21% and 12 patients presented with endocrine dysfunction (cumulative incidence of 19%). Only one patient developed a secondary malignancy (Hodgkin's lymphoma) during follow up.
  
  
  CONCLUSIONS
  Cyclophosphamide and antithymocyte globulin is an effective conditioning regimen for patients with severe aplastic anemia and is associated with low treatment-related mortality. Long-term complications include avascular necrosis and endocrine dysfunction.
authors.affiliation: Hématologie Greffe, Hôpital Saint Louis, Paris, France.
authors.forename: Johanna
authors.initials: J
authors.lastname: Konopacki
authors.forename: Raphaël
authors.initials: R
authors.lastname: Porcher
authors.forename: Marie
authors.initials: M
authors.lastname: Robin
authors.forename: Sabine
authors.initials: S
authors.lastname: Bieri
authors.forename: Jean-Michel
authors.initials: JM
authors.lastname: Cayuela
authors.forename: Jérôme
authors.initials: J
authors.lastname: Larghero
authors.forename: Aliénor
authors.initials: A
authors.lastname: Xhaard
authors.forename: Anna Lisa
authors.initials: AL
authors.lastname: Andreoli
authors.forename: Nathalie
authors.initials: N
authors.lastname: Dhedin
authors.forename: Anna
authors.initials: A
authors.lastname: Petropoulou
authors.forename: Paula
authors.initials: P
authors.lastname: Rodriguez-Otero
authors.forename: Patricia
authors.initials: P
authors.lastname: Ribaud
authors.forename: Hélène
authors.initials: H
authors.lastname: Moins-Teisserenc
authors.forename: Maryvonnick
authors.initials: M
authors.lastname: Carmagnat
authors.forename: Antoine
authors.initials: A
authors.lastname: Toubert
authors.forename: Yves
authors.initials: Y
authors.lastname: Chalandon
authors.forename: Gérard
authors.initials: G
authors.lastname: Socie
authors.forename: Régis
authors.initials: R
authors.lastname: Peffault de Latour
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000209:etiology / Q000401:mortality / Q000517:prevention & control*; D000961:Antilymphocyte Serum / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000209:etiology / Q000401:mortality / Q000517:prevention & control*; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D011379:Prognosis; D015996:Survival Rate; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D055815:Young Adult
chemical_list: D000961:Antilymphocyte Serum; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide