Autoimmune Hematological Diseases After Allogeneic Hematopoietic Stem Cell Transplantation in Children: An Italian Multicenter Experience

Biol Blood Marrow Transplant. 2014 Feb;20(2):272-8. doi: 10.1016/j.bbmt.2013.11.014. Epub 2013 Nov 23.


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.

Keywords: Autoimmune hematological disease; Children; Hematopoietic stem cell transplantation; Rituximab.

Publication types

  • Multicenter Study

MeSH terms

  • Child
  • Child, Preschool
  • Female
  • Hematologic Diseases / drug therapy
  • Hematologic Diseases / etiology*
  • Hematopoietic Stem Cell Transplantation / adverse effects*
  • Hematopoietic Stem Cell Transplantation / methods
  • Humans
  • Italy
  • Male
  • Remission Induction
  • Risk Factors
  • Transplantation Conditioning / adverse effects*
  • Transplantation Conditioning / methods