Successful treatment with rituximab and mycophenolate mofetil of refractory autoimmune hemolytic anemia post-hematopoietic stem cell transplant for dyskeratosis congenita due to TINF2 mutation

Pediatr Transplant. 2014 Feb;18(1):E22-4. doi: 10.1111/petr.12172. Epub 2013 Oct 29.


AIHA following allogeneic HSCT is appearing more frequently in the literature. It occurs as a result of donor cell-derived antibodies targeting donor red cell antigens. Little guidance exists on the management of such patients, particularly in the pediatric setting. First-line conventional treatment is corticosteroids and/or immunoglobulin therapy with monoclonal antibody therapy reserved for treatment failure. We report our experience of a child refractory to immunoglobulin and steroid therapy who required several infusions of rituximab and immunomodulatory therapy to obtain a clinically significant response.

Keywords: anemia; hematopoietic stem cell transplant; mycophenolate mofetil; rituximab.

Publication types

  • Case Reports

MeSH terms

  • Anemia, Hemolytic, Autoimmune / drug therapy*
  • Antibodies, Monoclonal, Murine-Derived / administration & dosage*
  • Child, Preschool
  • Dyskeratosis Congenita / genetics
  • Dyskeratosis Congenita / therapy
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Immunologic Factors / administration & dosage*
  • Immunosuppressive Agents / administration & dosage*
  • Male
  • Mutation
  • Mycophenolic Acid / administration & dosage
  • Mycophenolic Acid / analogs & derivatives*
  • Rituximab
  • Telomere-Binding Proteins / genetics
  • Treatment Outcome


  • Antibodies, Monoclonal, Murine-Derived
  • Immunologic Factors
  • Immunosuppressive Agents
  • TINF2 protein, human
  • Telomere-Binding Proteins
  • Rituximab
  • Mycophenolic Acid