Successful treatment with sorafenib for primary refractory acute monoblastic leukemia with FLT3-ITD].

Noriyuki Takahashi, Takako Kawakami, Hiroko Sato, Tadashi Onoda, Miyako Kanno, Tetsuo Mitsui, Kiyoshi Hayasaka

Department of Pediatrics, Yamagata University, Japan.

[Rinshō ketsueki] The Japanese journal of clinical hematology 2012 Nov

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A 6-year-old boy was diagnosed with FLT3-ITD+acute monoblastic leukemia (AMoL). He showed resistance to 2 cycles of induction chemotherapy with etoposide, cytarabine, and mitoxantrone or idarubicin performed according to the Japan Pediatric Leukemia/Lymphoma Study Group (JPLSG) AML-05 protocol. His condition was also refractory to salvage FLAI-GO (fludarabine, cytarabine, idarubicin, and gemtuzumab ozogamicin) chemotherapy. Sequential administration of sorafenib at doses of up to 300 mg/day resulted in the first remission. He underwent bone marrow transplantation from his HLA 2-locus mismatched father. Recurrence was observed on post-transplantation day 71. A sustained partial response was observed after alternate-day readministration of sorafenib 150mg/day. In spite of a donor lymphocyte infusion, his blast cell count increased on day 245. Chemotherapy with an increased dose of sorafenib reduced the blast cell count. Although a second HLA-mismatched allogeneic peripheral blood stem cell transfusion was performed, the patient died of regimen-related toxicity. Herein, we report a pediatric case of primary refractory FLT3-ITD+ AMoL. Further prospective studies are necessary to validate the efficacy of sorafenib treatment.

Citation

Noriyuki Takahashi, Takako Kawakami, Hiroko Sato, Tadashi Onoda, Miyako Kanno, Tetsuo Mitsui, Kiyoshi Hayasaka. Successful treatment with sorafenib for primary refractory acute monoblastic leukemia with FLT3-ITD]. [Rinshō ketsueki] The Japanese journal of clinical hematology. 2012 Nov;53(11):1932-6