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ctt-journal > Boyarshinov et al. (Abstract)

Boyarshinov et al. (Abstract)

Cellular Therapy and Transplantation (CTT), Vol. 3, No. 12
doi: 10.3205/ctt-2011-No12-abstract11

© The Authors. This abstract is provided under the following license: Creative Commons Attribution 3.0 Unported

Abstract accepted for "5th Raisa Gorbacheva Memorial Meeting Hematopoietic Stem Cell Transplantation in Children and Adults", Saint Petersburg, Russia, September 18–20, 2011

Preliminary Program

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The efficacy of Treosulfan as a single agent in newly diagnosed neuroblastoma stage IV patients

Vasiliy K. Boyarshinov, Igor S. Dolgopolov, Roman I. Pimenov, Georgy L. Mentkevich

Institute of Pediatric Oncology/Hematology, CRC, Moscow, Russia

Correspondence: Vasiliy K. Boyarshinov, Institute for Pediatric Oncology and Hematology, 24, Kashirskoye sh., 115478, Moscow, Russia, E-mail: vasiliy287@spam is badyandex.ru


Treosulfan (Treo) is a structural analogue of busulfan currently used for high-dose chemotherapy of advanced Ewing sarcomas, neuroblastomas (NB) and high-risk leukemias. There is no clinical data supporting the hypothesis that Treo is active as a single agent in pediatric malignancies. Therefore, from March 2009 to January 2011, 13 pts (M/F 8/5) with NB stage IV, >2 years of age at the time of diagnosis were included in our window study. The median age was 8.2 (3–15). Еleven pts had been newly diagnosed with stage IV of NB, and two were in relapse (one 3 yrs after haploidentical PBSCT). Treo was applied twice at a dose of 10g/m2 on days 1 and 7. After evaluation of the response, the high-risk protocol including 4 courses of CT, surgery, and High Dose CT with Auto PBSC rescue, followed by a biological phase was delivered. PBSC harvest was performed after the 4th course. MIBG I123 positive lesions retained after transplant were irradiated.

Efficacy of Treo was as follows: the bone marrow was morphologically clean after 2 courses in 8 out of 11 pts (in 1 pt with relapse). Seven pts achieved PR and 6 SD (3 of them without tumor clearance in BM) after 2 courses of Treo. Toxicity was minor with hematological toxicity of stage 1 in 2 pts. No other toxicity was registered. Ten pts received bone marrow transplantation. Five pts relapsed, and two patients died. The EFS of the whole group was 38%, and 50% in the transplant group.

In conclusion Treo is an effective agent in newly diagnosed NB pts and further evaluation of the doses, location, and schedule are warranted.

Keywords: treosulfan, neuroblastoma, chemotherapy