Rituximab for the treatment of refractory autoimmune hemolytic anemia in children.
Autoimmune hemolytic anemia (AIHA) in children is sometimes characterized by a severe course, requiring prolonged administration of immunosuppressive therapy. Rituximab is able to cause selective in vivo destruction of B lymphocytes, with abrogation of antibody production. In a prospective study, we have evaluated the use of rituximab for the treatment of AIHA resistant to conventional treatment. Fifteen children with AIHA were given rituximab, 375 mg/m(2)/dose for a median of 3 weekly doses. All patients had previously received 2 or more courses of immunosuppressive therapy; 2 patients had undergone splenectomy. After completing treatment, all children received intravenous immunoglobulin for 6 months. Treatment was well tolerated. With a median follow-up of 13 months, 13 patients (87%) responded, whereas 2 patients did not show any improvement. Median hemoglobin levels increased from 7.7 g/dL to a 2-month posttreatment level of 11.8 g/dL (P <.001). Median absolute reticulocyte counts decreased from 236 to 109 x 10(9)/L (P <.01). An increase in platelet count was observed in patients with concomitant thrombocytopenia (Evans syndrome). Three responder patients had relapse, 7, 8, and 10 months after rituximab infusion, respectively. All 3 children received a second course of rituximab, again achieving disease remission. Our data indicate that rituximab is both safe and effective in reducing or even abolishing hemolysis in children with AIHA and that a sustained response can be achieved in the majority of cases. Disease may recur, but a second treatment course may be successful in controlling the disease.
Zecca
Marco
M
Oncoematologia Pediatrica, IRCCS Policlinico San Matteo, Pavia, Italy.
Nobili
Bruno
B
Ramenghi
Ugo
U
Perrotta
Silverio
S
Amendola
Giovanni
G
Rosito
Pasquale
P
Jankovic
Momcilo
M
Pierani
Paolo
P
De Stefano
Piero
P
Bonora
Mario Regazzi
MR
Locatelli
Franco
F
D000293:Adolescent; D000743:Anemia, Hemolytic / Q000097:blood / Q000188:drug therapy* / Q000276:immunology; D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D058846:Antibodies, Monoclonal, Murine-Derived; D001327:Autoimmune Diseases / Q000188:drug therapy*; D001402:B-Lymphocytes / Q000187:drug effects / Q000276:immunology*; D002648:Child; D005260:Female; D006454:Hemoglobins / Q000378:metabolism; D006801:Humans; D007223:Infant; D008297:Male; D017701:Reticulocyte Count; D000069283:Rituximab; D016896:Treatment Outcome
D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D006454:Hemoglobins; D000069283:Rituximab