Use of rituximab to treat refractory Diamond-Blackfan anemia.
We report here the first case with Diamond-Blackfan anemia (DBA) who responded to rituximab. The patient is an 8-yr-old Japanese girl with refractory DBA accompanied by complex congenital heart disease. She received two doses of rituximab, 375 mg/m(2)/wk. She became transfusion independent 6 months after the treatment without any serious side effect. However, after 8 months of transfusion-free period, her condition returned to the pretreatment level with recovery of peripheral B cells. Rituximab may be a successful therapy for refractory DBA where B cells play a crucial role in the pathogenesis of the severe anemia.
Morimoto
Akira
A
Department of Pediatrics, Kyoto Prefectural University of Medicine, Graduate School of Medical Science, Kyoto, Japan. akira@koto.kpu-m.ac.jp
Kuriyama
Kikuko
K
Tsuji
Keiji
K
Isoda
Kenichi
K
Hibi
Shigeyoshi
S
Todo
Shinjiro
S
Sugimoto
Tohru
T
Imashuku
Shinsaku
S
D029503:Anemia, Diamond-Blackfan / Q000188:drug therapy*; D000911:Antibodies, Monoclonal / Q000627:therapeutic use*; D058846:Antibodies, Monoclonal, Murine-Derived; D001402:B-Lymphocytes / Q000187:drug effects / Q000276:immunology; D001803:Blood Transfusion; D002648:Child; D005260:Female; D006330:Heart Defects, Congenital / Q000150:complications; D006801:Humans; D018655:Lymphocyte Count; D000069283:Rituximab; D016896:Treatment Outcome
D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D000069283:Rituximab