ISSN 1866-8836
Клеточная терапия и трансплантация

PO-03. Efficacy of high-dose chemotherapy with autologous hematopoietic stem cell transplantation in the treatment of pediatric malignant brain tumors

Asmik G. Gevorgian1, Polina S. Tolkunova1, Elena V. Morozova1, Ilya V. Kazantsev1, Tatiana V. Iukhta1, Darya A. Drozdovskaya1, Andrey V. Kozlov1, Yury A. Punanov1, Ludmila S. Zubarovskaya1, Olga G. Zheludkova2, Boris V. Afanasyev1

1 RM Gorbacheva Research Institute of Pediatric Oncology, Hematology and Transplantation, Pavlov University, St. Petersburg, Russia
2 V. F. Voino-Yassenetsky Practical Scientific Center of Specialized Pediatric Medical Care, Moscow, Russia

Contact: Dr. Asmik Gevorgian, PhD, e-mail:

doi 10.18620/ctt-1866-8836-2020-9-3-1-152



Pediatric brain tumors are most common solid malignancies in children. Histologically characterized as undifferentiated small round cell tumors, all are uniformly aggressive, they have a tendency to disseminate throughout central nervous system and generally have unfavorable prognosis. We aimed to assess the effectiveness of high-dose chemotherapy (HDCT) with autologous hematopoietic stem cell transplantation (auto-HSCT) in two cohorts: children younger than 4 year with newly diagnosed CNS tumors and children older than 4 year with recurrent brain tumors.

Patients and methods

From 2010 to 2020, a total of 142 patients with medulloblastoma (N=93), PNET NOS (N=15), pineoblastoma (N=7), atypical teratoid rhabdoid tumor (N=4), ETMR (N=9), germ cell tumor (N=12), CNS neuroblastoma (N=2) received single or tandem HDCT with auto-HSCT after surgical resection and induction chemotherapy.


The median follow-up is 60 (range, 6-230) months. The median time to engraftment after the first auto-HSCT was 15 (range, 8-86) days. Five-year overall survival (OS) and disease free survival (DFS) in infants <4 year was 56% and 41%, accordingly, while OS and DFS in children >4 year were 37% and 35%, accordingly. Patients in complete (CR) and partial response (PR) at the moment of HDCT had 45% of 5-year OS, while children with stable disease (SD) all died due to progression within 24 months after transplantation (p=0.04). The OS was significantly better among patients with classic (47%) and desmoplastic (40%) compared to anaplastic MB (16%). Also the results are much better for molecular subgroup WNT MB (100%), Group 3 (86%), than for Group 4 (55%) and SHH (39%). All tandem HDCT recipients had better OS (75%) compared to patients receiving single HDCT (44%). Patients with MB and PNET had better prognosis with OS 69% and 51%, respectively, in compare with other embryonal tumors. Patients without MYC amplification had much better OS (92%) than MYCamp+ patients (25%; p=0.0002). Ten-year cumulative incidence of relapse was 59% (95% CI 16%-79%). Conditioning regimens had acceptable toxicity. Grade 4 (CTCAE V.5.0 2017) complications were seen in 13% of cases. Cumulative incidence of transplant-related mortality was 12% (95% CI 1%-33%).


HDCT with auto-HSCT is characterized by acceptable toxi-city and may be a feasible option for patients with high-risk brain tumors after induction treatment and conditioning regimens. It is ineffective as a salvage therapy. Molecular subgroup WNT and Group 3 patients without MYC amplification have most favorable prognosis. Tandem HDCT with auto-HSCT may improve a survival rate in infants <4 years old with newly diagnosed brain tumors sparing them from toxic cranio-spinal irradiation.


High-dose chemotherapy, autologous hematopoietic stem cell transplantation, pediatric brain tumors, medulloblastoma, PNET.

Volume 9, Number 3

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doi 10.18620/ctt-1866-8836-2020-9-3-1-152

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