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Use of rituximab to treat refractory Diamond-Blackfan anemia.

Morimoto A, Kuriyama K, Tsuji K, Isoda K, Hibi S, Todo S, Sugimoto T, Imashuku S

Department of Pediatrics, Kyoto Prefectural University of Medicine, Graduate School of Medical Science, Kyoto, Japan. akira@koto.kpu-m.ac.jp

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.

Published 7 April 2005 in Eur J Haematol, 74(5): 442-4.
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