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Analysis of the results of hematopoietic stem cell transplantation (HSCT) derived from alternative donors in patients with primary immunodeficiency syndromes. 110 HSCTs for patients with PIDs derived from alternative donors (unrelated, n = 85, haploidentical, t = 25) were performed at the Dmitry Rogachev National Medical Research Centre within 2012-2017 timeframe. In all cases, there were used conditioning regimes with reduced toxicity based on threosulfan TCRotP+/CD19+ depletion with immunomagnetic method were used as the basic cell transplant preparation. The cumulative probability of acute GVHD was 17% (95% CI 10-25) (n = 18); however, it should be noted that in 16 of 18 cases, an acute GVHD, stage II, was observed, showing a good response to the first line therapy; but acute GVHD, stage III, was documented only in 2 patients. Reactivation of cytomegalovirus infection remained one of the serious issues, with a cumulative probability of its reactivation reaching up to 50% and CMV visceral infection rate found in 15.4% cases. The conditioning regimen in patients with Wiskott-Aldrich syndrome by using granulocyte colony-stimulating factor and plerixafor demonstrated a full control over transplant dysfunction compared to control group.

Rate of immunological reconstitution upon inoculation of HSCT on the platform TCRotP+/CD19+ deletion did not differ in dynamics from that one after using undepleted HSCT together with native hematopoietic stem cell sources in a historical cohort. The overall survival probability for entire PID patient cohort was 84% (95% CI 77-92). No differences in patients transplanted from unrelated and haploidentical donors were revealed by assessing any of the studied parameters.

Introduction of new HSCT technologies allows us to dramatically minimize adverse outcomes of PIDs and opens new avenues for further research in cellular regulation of autoinflammatory oncological and infectious disorders resulting in lethal outcome in PID patients.

About the authors

D. N. Balashov

D. Rogachev National Medical Research Centre for Pediatric Hemtology, Oncology and Immunology

Author for correspondence.
Email: bala8@yandex.ru

Balashov Dmitry N. D. PhD, MD (Medicine), Professor, Head, Hematopoietic Stem Cell Transplantation Department No. 2

Phone: 7 (495) 221-66-40. 117997, Moscow, Samori Mashela str., 1

Russian Federation

M. A. Maschan

D. Rogachev National Medical Research Centre for Pediatric Hemtology, Oncology and Immunology

Email: fake@neicon.ru
Maschan M.A., PhD, MD (Medicine), Professor, Director, High School of Molecular and Experimental Medicine Russian Federation

A. Yu. Shcherbina

D. Rogachev National Medical Research Centre for Pediatric Hemtology, Oncology and Immunology

Email: fake@neicon.ru
Shcherbina A.Yu., PhD, MD (Medicine), Professor, Head, Сlinical Immunology Department, Deputy Director Russian Federation

A. G. Rumyantsev

D. Rogachev National Medical Research Centre for Pediatric Hemtology, Oncology and Immunology

Email: fake@neicon.ru
Rumyantsev A.G., PhD, MD (Medicine), Professor, President Russian Federation


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Copyright (c) 2020 Balashov D.N., Maschan M.A., Shcherbina A.Y., Rumyantsev A.G.

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