A successful second allogeneic hematopoietic stem cell transplantation from an alternative donor in a patient with loss of HLA heterozygosity relapse of juvenile myelomonocytic leukemia: case series analysis

L. A. Tsvetkova; Цветкова Л. А.; A. A. Osipova; Осипова А. А.; A. V. Evdokimov; Евдокимов А. В.; P. V. Kozhokar; Кожокарь П. В.; Zh. Z. Rakhmanova; Рахманова Ж. З.; I. M. Barkhatov; Бархатов И. М.; O. V. Paina; Паина О. В.; O. S. Epifanovskaya; Епифановская О. С.; E. V. Babenko; Бабенко Е. В.; N. E. Ivanova; Иванова Н. Е.; D. V. Kozlov; Козлов Д. В.; T. L. Gindina; Гиндина Т. Л.; T. А. Bykova; Быкова Т. А.; E. V. Semenova; Семенова Е. В.; L. S. Zubarovskaya; Зубаровская Л. С.

doi:10.24287/1726-1708-2023-22-3-28-35

A successful second allogeneic hematopoietic stem cell transplantation from an alternative donor in a patient with loss of HLA heterozygosity relapse of juvenile myelomonocytic leukemia: case series analysis

Authors: Tsvetkova L.A.¹, Osipova A.A.¹, Evdokimov A.V.¹, Kozhokar P.V.¹, Rakhmanova Z.Z.¹, Barkhatov I.M.¹, Paina O.V.¹, Epifanovskaya O.S.¹, Babenko E.V.¹, Ivanova N.E.¹, Kozlov D.V.¹, Gindina T.L.¹, Bykova T.А.¹, Semenova E.V.¹, Zubarovskaya L.S.¹
Affiliations:
1. The R.M. Gorbacheva Research Institute for Pediatric Oncology, Hematology and Transplantation of the I.P. Pavlov First Saint Petersburg State Medical University of Ministry of Healthcare of the Russian Federation
Issue: Vol 22, No 3 (2023)
Pages: 28-35
Section: ORIGINAL ARTICLES
Submitted: 07.07.2023
Accepted: 29.08.2023
Published: 30.09.2023
URL: https://hemoncim.com/jour/article/view/747
DOI: https://doi.org/10.24287/1726-1708-2023-22-3-28-35
ID: 747

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Abstract

Juvenile myelomonocytic leukemia (JMML) is a rare and aggressive myeloproliferative/myelodysplastic neoplasm of early childhood characterized by activation of the Ras signaling pathway. Allogeneic hematopoietic stem cell transplantation (alloHSCT) is the only proven curative treatment for JMML. However, the 5-year overall survival is about 52–64%. In this work, we analyzed 4 clinical cases of patients with relapses of JMML with loss of heterozygosity in HLA (LoH) after allo-HSCT. The patients' parents gave their consent to the use of their children's data, including photographs, for research purposes and in publications. Two patients received a second allo-HSCT from an alternative donor, two patients – from the same donor. A positive result in the form of a durable remission was observed in one patient who underwent a second allo-HSCT from an alternative donor and restored HLA genetic heterozygosity. At the same time, immunotherapy with infusions of donor lymphocytes led to the development of graft-versus-host disease without potentiating the antileukemic effect. Thus, a second allo-HSCT from an alternative donor for the treatment of relapsed JMML with HLA LoH is necessary to restore the “graft-versus-JMML” response. The study was approved by the Independent Ethics Committee and the Scientific Council of the I.P. Pavlov First Saint Petersburg State Medical University of Ministry of Healthcare of the Russian Federation.

Keywords

juvenile myelomonocytic leukemia, allogeneic hematopoietic stem cell transplantation, HLA loss

About the authors

L. A. Tsvetkova

The R.M. Gorbacheva Research Institute for Pediatric Oncology, Hematology and Transplantation of the I.P. Pavlov First Saint Petersburg State Medical University of Ministry of Healthcare of the Russian Federation

Author for correspondence.
Email: tsvetluibov@mail.ru
ORCID iD: 0000-0003-4952-0704