Kazantsev et al. (Abstract)
Cellular Therapy and Transplantation (CTT), Vol. 3, No. 9
doi: 10.3205/ctt-2010-No9-abstract18
© The Authors. This abstract is provided under the following license: Creative Commons Attribution 3.0 Unported
Abstract accepted for "4th Raisa Gorbacheva Memorial Meeting on Hematopoietic Stem Cell Transplantation",
Saint Petersburg, Russia, September 18–20, 2010
High-dose chemotherapy and autologous stem-cell transplantation in children with Ewing’s Sarcoma/PNET
Ilya V. Kazantsev1, Tatjana V. Youhta2, Elena V. Morozova1, Svetlana A. Safonova2, Yury A. Punanov2, Lyudmila S. Zubarovskaya1, Boris V. Afanasyev1
1R. M. Gorbacheva Memorial Institute of Children Hematology and Transplantation of Pavlov State Medical University, Saint-Petersburg, Russia; 2Federal State Institute N.N. Petrov Research Institute of Oncology, Saint-Petersburg, Russia
Correspondence: Ilya V. Kazantsev, R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, Pavlov State Medical University, 6/8, L. Tolstoy str., Saint-Petersburg, 197022, Russia, E-mail: ilya-kazantsev@ inbox.ru
Abstract
The five-year event-free survival (EFS) for patients with localized pediatric Ewing’s sarcoma/PNET is 70–80%, but in high-risk groups (metastatic disease, unfavorable localization, or large initial tumor mass) it is only 20–30%. Some research data shows evidence that high-dose chemotherapy (HDCT) with autologous hematopoietic stem-cell transplantation (auto-HSCT) can be a promising option for this group.
Patients and methods: From 2003 to December 2009 we treated 23 pediatric patients with histologically proven high-risk Ewing’s sarcoma/PNET. The male-female ratio was 2.8:1, and the median age at diagnosis 12.6 years. All patients received 6 courses of polychemotherapy (ifosfamide, vincristine, doxorubicin, and etoposide), surgical treatment, or local irradiation (48–56 Gy). 8 patients then received maintenance chemotherapy, and for 15 patients this was substituted with HDCT with auto-HSCT. Conditioning regimens were busulfan 16 mg/kg and melphalan 140 mg/m2 (n=13), and melphalan 140–200 mg/m2 (n=2). The stem-cell sources were bone marrow (n=7), peripheral blood stem cells (n=3), and both (n=5). The mean CD34+ cell dose was 3.15 x 106/kg.
Results: In the maintenance therapy group (n=8) 7 patients relapsed (5 patients with metastatic relapse died, 2 patients with local relapse are alive after salvage therapy), and 1 patient died of sepsis.
In the HDCT group (n=15) 12 of the patients are alive (6–41 months after HDCT), and 8 of them remain in remission. All children transplanted during partial remission or disease progression later relapsed. Only 2 patients who achieved complete remission at the end of induction chemotherapy developed late relapses.
The toxicity of HDCT regimens was moderate: grade II–III mucositis (n=15), febrile neutropenia (n=12), and toxic hepatitis (n=3).
Conclusions: HDCT with auto-SCT significantly lowers the relapse rate in pediatric patients with high-risk Ewing’s sarcoma/PNET, who achieved CR after induction chemotherapy.
Keywords: Ewing sarcoma, PNET, autologous transplantation, high-dose chemotherapy, maintenance therapy