Allogeneic hematopoietic stem cell transplantation for alveolar rhabdomyosarcoma: the first clinical experience of haploidentical transplantation

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Abstract

This article describes the use of allogeneic hematopoietic stem cell transplantation (HSCT) for the treatment of a patient with metastatic alveolar rhabdomyosarcoma. The reported case is unusual in terms of its presentation, diagnosis, and choice of treatment. This study is supported by the Independent Ethics Committee and approved by the Academic Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. In the last decades, the survival rates of patients with metastatic malignant neoplasms, including those with soft-tissue sarcomas, have not improved significantly. The prognosis for these patients remains extremely poor despite the intensification of chemotherapy and the application of radiation therapy. The use of autologous HSCT has not brought about any positive changes in treatment outcomes. Experimental approaches to treatment, including immunotherapeutic techniques, aimed at improving the prognosis for very high-risk patients are currently under development. We chose to report this case because of the unconventional treatment approach that had helped to achieve long-term control over the disease and to prevent systemic progression. It demonstrates that allogeneic HSCT can be regarded as one of the promising options for treatment intensification mainly due to the “graftversus-tumour” effect. The patients' parents gave their consent to the use of their child's data, including photographs, for research purposes and in publications.

About the authors

N. A. Andreeva

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation

Author for correspondence.
Email: andreeva793@bk.ru
ORCID iD: 0000-0001-5626-218X

Natalya A. Andreeva, a pediatric oncologist at the Department of Clinical Oncology, a junior researcher at the Department of Embryonal Tumour Research and the Laboratory of Molecular Oncology

1 Samory Mashela St., Moscow 117997

Russian Federation

E. A. Druy

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation

ORCID iD: 0000-0003-0899-7266
Moscow Russian Federation

Yu. M. Mareeva

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation

ORCID iD: 0000-0003-0652-6049
Moscow Russian Federation

A. E. Druy

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation

ORCID iD: 0000-0003-1308-8622
Moscow Russian Federation

L. N. Shelikhova

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation

ORCID iD: 0000-0003-0520-5630
Moscow Russian Federation

A. I. Karachunsky

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation

ORCID iD: 0000-0002-9300-198X
Moscow Russian Federation

M. A. Maschan

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation

ORCID iD: 0000-0003-1735-0093
Moscow Russian Federation

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Copyright (c) 2025 Andreeva N.A., Druy E.A., Mareeva Y.M., Druy A.E., Shelikhova L.N., Karachunsky A.I., Maschan M.A.

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