Rituximab for refractory childhood autoimmune hemolytic anemia.
BACKGROUND
Chronic childhood autoimmune hemolytic anemia is an uncommon disorder that is associated with significant morbidity. Treatment with high dose steroids, splenectomy and frequent blood transfusions results in a myriad of complications including growth failure, bone demineralization, Cushing's syndrome, immunosuppression, and transfusional hemosiderosis.


OBJECTIVE
To investigate the efficacy of the monoclonal anti-CD20 antibody, rituximab, in treating children with AIHA.


METHODS
Four children with chronic AIHA, including two with prior splenectomy, who were dependent on high dose steroids and refractory to other immunosuppressive regimens were treated with four to six weekly doses of rituximab at a dose of 375 mg/m2.


RESULTS
All four patients became transfusion-independent and were taken off prednisone completely. Adverse effects included infusion-related reactions that were mild, and infectious complications of Pneumocystis carinii pneumonia and varicella pneumonia.


CONCLUSIONS
Treatment with rituximab appears promising for refractory AIHA; if may obviate the need for prednisone and may result in sustained disease remissions in some patients.
Motto
David G
DG
Department of Pediatric Hematology/Oncology, University of Michigan, Ann Arbor, MI, USA. dgmotto@umich.edu
Williams
James A
JA
Boxer
Laurence A
LA
D000293:Adolescent; D000744:Anemia, Hemolytic, Autoimmune / Q000188:drug therapy*; D000911:Antibodies, Monoclonal / Q000009:adverse effects / Q000627:therapeutic use*; D058846:Antibodies, Monoclonal, Murine-Derived; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D002675:Child, Preschool; D006801:Humans; D007262:Infusions, Intravenous; D011241:Prednisone / Q000627:therapeutic use; D000069283:Rituximab; D016896:Treatment Outcome
D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D000970:Antineoplastic Agents; D000069283:Rituximab; D011241:Prednisone