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ctt-journal > Makarova et al. (Abstract)

Makarova et al. (Abstract)

Cellular Therapy and Transplantation (CTT), Vol. 3, No. 12
doi: 10.3205/ctt-2011-No12-abstract64

© 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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High-dose chemotherapy with autologous hematopoietic stem-cell transplantation in the treatment of resistant pediatric sarcomas

Inna V. Makarova1, Elena V. Morozova1, Ilya V. Kazantsev1, Asmik G. Gevorgyan1, Tatjana V. Youhta2, Svetlana A. Safonova2, Yury A. Punanov2, Boris V. Afanasyev1

1R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, St. Petersburg, Russia; 2Federal State Institution N.N. Petrov Research Institute of Oncology, St. Petersburg, Russia

Correspondence: Inna V. Makarova, R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, St. Petersburg Pavlov State Medical University, 6/8, Tolstoy str., St. Petersburg, 197022, Russia, E-mail: juhimchuk85@spam is badmail.ru


Pediatric soft tissue sarcomas are comparatively rare and some cases present a therapeutic challenge due to their aggressive course and resistance to conventional chemotherapy.

The aim of this study was to assess the effectiveness of single or tandem high-dose chemotherapy (HDCT) with autologous hematopoietic stem-cell transplantation (auto-HSCT) in this patient group.

Methods: From October 2009 to April 2011, 5 pediatric patients with alveolar rhabdomyosarcoma (n=3), fibrosarcoma (n=1), and embryonal rhabdomyosarcoma (n=1) received single (n=4) or tandem (n=1) HDCT with auto-HSCT. Before auto-HSCT all patients received surgical treatment, chemotherapy (median 7 courses), and local irradiation (median 40 Gy) without an adequate response. At the moment of auto-HSCT the stage of disease was progression (n=2) or stabilization (n=3). The conditioning regimen consisted of busulfan 16 mg/kg and melphalan 140 mg/m2 in single auto-HSCT, or carboplatin 900 mg/m2, etoposide 45 mg/kg and cyclophosphamide 120 mg/kg in tandem auto-HSCT. Bone marrow was used in all pts as the source of HSC, and the mean CD34+ cell dose was 4.1 x 106/kg (1.1–7.5 x 106/kg).

Results: The conditioning regimens were characterized by moderate toxicity (WHO Grade I–II). Median follow-up time is currently 370 days. All patients achieved good partial response with a decrease in diameter of the target lesions totaling 60–90%. The disease remained stable for a median of 250 days, but all patients later experienced progression. Three of the patients are alive with stable disease, and 2 patients died of disease progression.

Conclusions: HDCT with auto-SCT is a feasible method in pediatric patients with chemoresistant soft-tissue sarcomas, achieving a good short-term response. More aggressive post-transplant local treatment or cell-based immunotherapy could improve the prognosis.

Keywords: pediatric sarcomas, autologous transplantation, high-dose chemotherapy, rhabdomyosarcoma, fibrosarcoma