ISSN 1866-8836
Клеточная терапия и трансплантация

NM-09. Allogeneic transplantation of hematopoietic stem cells in cases of congenital immunity disorders in the Republic of Belarus

Lyubov V. Zherko, Yulia E. Mareyko, Natalya P. Kirsanova, Olga A. Mishkova, Nina V. Minakovskaya

Republican Scientific and Practical Center for Pediatric Oncology, Hematology and Immunology, Minsk, Republic of Belarus


Contact: Lyubov V. Zherko, phone: +375296147225, e-mail: lubazherko@gmail.com

doi 10.18620/ctt-1866-8836-2023-12-3-1-176

Summary

Allogeneic hematopoietic stem cell transplantation (HSCT) is a modern method of treating patients with congenital immune errors. 48 allogeneic hematopoietic stem cell transplantations were performed in the Republic of Belarus over the period of 2000 to 2023.

Patients and methods

Transplantation of hematopoietic stem cells for children with congenital immunity disorders (CID) was performed on the basis of the Belarussian Research Center of Pediatric Oncology, Hematology and Immunology. The study group included patients aged from 0 to 23 years who received allogeneic HSCT before July 2023. The diagnosis of CID was made on the basis of clinical data in accordance with the criteria of the European Immunodeficiency Society, being genetically confirmed. For molecular genetics assessment, Sanger sequencing or next generation sequencing (NGS) with a target panel of CID genes was used for most patients.

Results

The most common types of CID were combined immunodeficiencies with syndromal manifestations (31.25%), immunodeficiencies with damage to cellular and humoral immunity (27%), immunodeficiencies with congenital defects in the number or function of phagocytes (25%). The most common indication of HSCT for children under 1 year of age was severe combined immunodeficiency (SCID) (60%); from 1 year to 3 years, SCID (31.8%) and Wiskott-Aldrich syndrome (27.2%); from 3 years to 7 years, congenital neutropenia (33%); from 7 to 12 years, Nijmegen syndrome (40%). The median time to make a diagnosis of CID was 440 days (range 32 days-21.7 years); median time between the CID diagnosis and HSCT was 369.5 days (range 22 days-10.6 years). HSCT was performed at the median age of 1060 days (ranging from 0.3 to 22.2 years old). The boy-to-girl ratio among children with CID subjected to HSCT was 4:1. In 31% of cases, transplantation was performed from a compatible related donor; in 67.0%, from a compatible unrelated donor; in 2%, haplocompatible transplantation was performed.

Conclusion

The overall survival of patients with CID after HSCT was 0.75±0.07.

Keywords

Congenital immune disorders, hematopoietic stem cell transplantation.


Supplement 12-3
09/30/2023

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doi 10.18620/ctt-1866-8836-2023-12-3-1-176

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