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Tandem autologous hematopoietic stem cell transplantation for embryonal brain tumors in infants and very young children

Bone Marrow Transplantation volume 57, pages 607–612 (2022)Cite this article

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Abstract

Withdrawal of radiotherapy in patients with brain tumors under four years decreases chance for cure. AutoHSCT in a series of pilot studies demonstrated a potential to improve outcomes in these patients. The study included 50 patients with median age of 39 months (7–53). Medulloblastoma (n = 28, 56%), ETMR (n = 9, 18%) and other histological types (n = 13, 26%) were most commonly diagnosed. Forty two patients (84%) received tandem autoHSCT by HIT-MED protocol, and single autoHSCT was performed in eight children (16%). Adjuvant radiotherapy was administered in 25 (50%) children and treatment of relapse included radiotherapy in 6 (12%). Median follow-up was 39.6 months (6–121). Long-term CIR was 37%, and TRM – 6%. Five-year OS was 71% in medulloblastoma, 37% in ETMR and in other tumors – 51% (p = 0.07). Irradiation-free OS at 5 years for children with medulloblastoma was 24%. For the whole cohort of CNS tumors, independently of histology, OS and PFS at five years were 60% and 46%, respectively Young children with medulloblastoma, following tandem autoHSCT, demonstrate OS comparable to older children. Patients with other histological types demonstrate suboptimal long-term survival rates after autoHSCT and one should assess whether these patients benefit from autoHSCT.

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Fig. 1: Outcome of young children with embryonal brain tumors after autoHSCT.

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Authors and Affiliations

  1. RM Gorbacheva Research Institute, Pavlov University, St. Petersburg, Russian Federation

    A. G. Gevorgian, A. V. Kozlov, P. S. Tolkunova, I. V. Kazantzev, T. V. Yukhta, E. V. Morozova, A. D. Kulagin, Y. A. Punanov & L. S. Zubarovskaya

  2. V.F. Voyno-Yasenetsky Scientific and Practical Center of Specialized Medical Care for Children, Moscow, Russian Federation

    O. G. Zheludkova

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  1. A. G. Gevorgian
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Contributions

Conceptualization, AGG and LSZ; methodology, OGZ; resources, ADK; software, PST; formal analysis, IVK; investigation, TVY; data curation, EVM; writing—original draft preparation, AVK and AGG; writing—review and editing, YAP and OGZ; supervision, LSZ.

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Correspondence to A. V. Kozlov.

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Gevorgian, A.G., Kozlov, A.V., Tolkunova, P.S. et al. Tandem autologous hematopoietic stem cell transplantation for embryonal brain tumors in infants and very young children. Bone Marrow Transplant 57, 607–612 (2022). https://doi.org/10.1038/s41409-022-01593-x

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