ISSN 1866-8836
Клеточная терапия и трансплантация

NM-06. Results of allogenic hematopoietic stem cell transplantation for bone marrow failure syndrome in non-malignant diseases in children: single center experience

Tatyana I. Potemkina1, Irina O. Kostareva1, Yuriy V. Lozovan1, Nataliya A. Burlaka1, Timur Z. Aliev1, Karina A. Sergeenko1, Elena B. Machneva1,2, Kirill I. Kirgizov1

1 L. Durnov Research Institute of Pediatric Oncology and Hematology at the N. N. Blokhin National Medical Research Centre of Oncology, Moscow, Russia
2 N. Pirogov Russian National Research Medical University, Moscow, Russia


Contact: Dr. Tatyana I. Potemkina, e-mail: tanyabukharova0802@yandex.ru

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

Summary

Bone marrow failure syndrome is a pathological condition of various etiologies, in which hematopoiesis is partially or completely impaired. In children, this syndrome accompanies some non-malignant diseases, such as: dyskeratosis congenita (DC) and Fanconi anemia (FA). Currently, allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents the only curative option for the treatment of bone marrow failure in DC and FA. Such patients should be transplanted as soon as possible, because the incidence of malignant diseases can reach 90%. Our purpose was to assess overall survival (OS) of patients with non-malignant blood diseases, i.e., with Fanconi anemia and dyskeratosis congenita, who underwent allo-HSCT over the period of 2021-2022.

Results

Five patients were included into the study. The patients received allo-HSCT between September 2021 and September 2022. Median age was 8.4 years old. (5 months to 13 y.o). M/F ratio was 4:1. Diagnosis: FA, 2 cases; DC, 3 patients. Matched related donors (MRD) were used in 3 cases (60%); matched unrelated donors (MUD), in 2 cases (40%). Graft sources were as follows: BM in 3 cases, and PBSC in 2 patients. Conditioning regimens included Busulfan+ Cyclophosphamide+ Fludarabine+ ATG. The patients with MUD/MRD received combined IST with abatacept and calcineurin blockers. Grade I–II GvHD developed in 3 patients (60%), and severe acute GvHD was not documented in any case. Reconstitution of hematopoiesis was recorded in all patients, on average of 16 days after allo-HSCT. The OS rate of patients with bone marrow failure syndrome who underwent allo-HSCT was 100%, failed HSCT (graft rejection) was registered in one patient, 120 days after allo-HSCT.

Conclusions

Our study showed that allo-HSCT is an effective and safe treatment for bone marrow failure in FA and DC. Absence of severe GvHD conditions makes it possible to consider the applied aGvHD prevention regimens to be successful.

Keywords

Fanconi anemia, dyskeratosis congenita, hemopoietic stem cell transplantation, allogeneic, graft-versus-host disease, children.


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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