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

Abstract

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.

• Authors: Niall O'Connell, Matthew Goodyer, Mary Gleeson, Lorna Storey, Martina Williams, Melanie Cotter, Aengus O'Marcaigh, Owen Smith
• Journal: Pediatric transplantation (Pediatr Transplant) Vol. 18 Issue 1 Pg. E22-4 (Feb 2014) ISSN: 1399-3046 [Electronic] Denmark
• PMID: 24168326 (Publication Type: Case Reports, Journal Article)
• Copyright: © 2013 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd.

Chemical References

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

Topics

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