Results of hematopoietic stem cell transplantation in children with acute leukemia: a single-center experience

I. O. Kostareva; Костарева И. О.; K. I. Kirgizov; Киргизов К. И.; E. B. Machneva; Мачнева Е. Б.; T. Z. Aliev; Алиев Т. З.; Yu. V. Lozovan; Лозован Ю. В.; K. A. Sergeenko; Сергеенко К. А.; N. A. Burlaka; Бурлака Н. А.; T. I. Potemkina; Потемкина Т. И.; K. V. Mitrakov; Митраков К. В.; A. Yu. Yelfimova; Елфимова А. Ю.; A. S. Slinin; Слинин А. С.; M. D. Malova; Малова М. Д.; R. R. Fatkhullin; Фатхуллин Р. Р.; N. G. Stepanyan; Степанян Н. Г.; N. A. Batmanova; Батманова Н. А.; T. T. Valiev; Валиев Т. Т.; S. R. Varfolomeeva; Варфоломеева С. Р.

doi:10.24287/1726-1708-2023-22-2-16-23

Results of hematopoietic stem cell transplantation in children with acute leukemia: a single-center experience

Authors: Kostareva I.O.¹, Kirgizov K.I.¹, Machneva E.B.¹^,2, Aliev T.Z.¹, Lozovan Y.V.¹, Sergeenko K.A.¹, Burlaka N.A.¹, Potemkina T.I.¹, Mitrakov K.V.¹, Yelfimova A.Y.¹, Slinin A.S.³, Malova M.D.¹, Fatkhullin R.R.¹, Stepanyan N.G.¹, Batmanova N.A.¹, Valiev T.T.¹, Varfolomeeva S.R.¹
Affiliations:
1. N. N. Blokhin National Medical Research Center of Oncology of Ministry of Healthcare of the Russian Federation
2. Russian Children's Clinical Hospital of the N. I. Pirogov Russian National Research Medical University of Ministry of Healthcare of the Russian Federation
3. Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
Issue: Vol 22, No 2 (2023)
Pages: 16-23
Section: ORIGINAL ARTICLES
Submitted: 12.10.2022
Accepted: 22.05.2023
Published: 08.07.2025
URL: https://hemoncim.com/jour/article/view/720
DOI: https://doi.org/10.24287/1726-1708-2023-22-2-16-23
ID: 720

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Abstract

Currently, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment option for relapsed / refractory (R / R) acute leukemia (AL) and high-risk AL. In this article, we present our own experience of allo-HSCT in children with R / R AL. The study was approved by the Independent Ethics Committee and the Scientific Council of the N. N. Blokhin National Medical Research Center of Oncology. Fifty-one patients with R / R AL were included in the study: 32 patients had acute lymphoblastic leukemia (ALL), 17 patients had acute myeloid leukemia (AML) and 2 patients had biphenotypic leukemia (BL). All patients underwent allo-HSCT from January 2021 to October 2022. The median age was 8.7 years (5 months – 17 years). At the time of allo-HSCT, 26 patients were in the second (and further) remission, the rest were in the first clinical and hematologic remission (high-risk AML and refractory ALL). Twenty-one (41.2 %) patients received allo-HSCT from a haploidentical donor, 19 (37.2 %) patients underwent allo-HSCT from an HLA-matched related donor and 11 (21.6 %) patients – from an HLA-matched unrelated donor. Pre-transplant conditioning in ALL: 27 patients received regimens based on total body irradiation at a dose of 12 Gy, 4 patients received busulfan-based conditioning regimens, and in 1 patient we used treosulfan. In AML and BL, we used conditioning regimens based on treosulfan/thiotepa (n = 10), treosulfan/melphalan (n = 8) or busulfan / melphalan (n = 1). Bone marrow (in 14 patients) and peripheral blood stem cells (in 37 patients) were used as a source of hematopoietic stem cells. In haploidentical allo-HSCTs in order to prevent graft-versus-host disease (GVHD) we performed TCRab/CD19 depletion followed by additional administration of abatacept / tocilizumab / rituximab on day –1 in 15 patients, 6 patients received post-transplant cyclophosphamide. In transplantations from HLA-matched related and unrelated donors, patients received combined immunosuppressive therapy with abatacept and rituximab on day –1, and calcineurin inhibitors were used as basic immunosuppressive therapy. All patients engrafted with a median time to engraftment of 13 (range, 9 to 24) days after allo-HSCT. Eight (15.7 %) patients developed a relapse of AL at different times after HSCT (five of them are alive). At the median follow-up of 9 (5–25) months, the overall and disease-free survival survival rates were 76.4 % and 68.8 %, respectively, for patients with AL. Acute GVHD was observed in 72.5 % of children, grade 3–4 GVHD was observed in 5.3 % of patients, and 13.7 % of children developed chronic GVHD. Most patients developed infectious complications in the early post-transplant period: febrile neutropenia (96.0 %), reactivation of viremia (47.3 %,) oropharyngeal mucositis (78.4 %), acute cystitis (12.3 %). The overall mortality rate was 17.6 %. Late mortality was associated with a relapse of AL.

Keywords

children, anti-relapse therapy, hematopoietic stem cell transplantation, acute leukemias, graft-versus-host disease

About the authors

I. O. Kostareva

N. N. Blokhin National Medical Research Center of Oncology of Ministry of Healthcare of the Russian Federation

Author for correspondence.
Email: kostareva_92@mail.ru
ORCID iD: 0000-0003-0179-2479

Irina O. Kostareva, a pediatric oncologist

Research Institute of Pediatric Oncology and Hematology

Department of Pediatric Bone Marrow Transplantation

N. N. Blokhin National Medical Research Center of Oncology of Ministry of Healthcare of the Russian Federation

ORCID iD: 0000-0001-6131-1783

Moscow

Russian Federation

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