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Single-center experience of unrelated and haploidentical stem cell transplantation with TCR-alpha/beta and CD19 depletion in children with primary immunodeficiency syndromes.
Biol Blood Marrow Transplant. 2015 Jul 14;
Authors: Balashov D, Shcherbina A, Maschan M, Trakhtman P, Skvortsova Y, Shelikhova L, Laberko A, Livshits A, Novichkova G, Maschan A
Abstract
The transplantation of stem cells from a matched unrelated donor (MUD) or a haploidentical mismatched related donor (MMRD) is a widely used variant of curative treatment for patients with primary immunodeficiency (PID). Currently, different strategies are used to reduce the risk of posttransplant complications and enhance immune reconstitution. We report the preliminary results of MUD and MMRD transplantation with TCRαβ/CD19 depletion in patients with PID (trial registered at www.clinicaltrials.gov as #NCT02327351). Thirty-seven PID patients, with a median age of 2.6 years (range, 0.2-17 years), were transplanted from MUD (n=27) or haploidentical MMRD (n=10) after TCRαβ+/CD19+ graft depletion. The median numbers of CD34+ and TCRαβ+ cells in the graft were 11.7 × 10(6)/kg and 10.6 x10(3)/kg, respectively. Acute graft-versus-host disease (GVHD) was observed in 8 patients (22%), without a statistically significant difference between MUD and MMRD; 7 of those patients had grade II acute GVHD and responded to first-line therapy, whereas 1 patient had grade IV acute GVHD with transformation to extensive chronic GVHD. Primary and secondary graft failure (non-engraftment or rejection) was observed in 10 patients (27%), 9 of whom were treated with one alkylating agent in the conditioning regimen. All these patients were successfully re-transplanted with different rescue protocols. The preliminary data on immune reconstitution were very encouraging. Most patients had significant numbers of T lymphocytes detected on the first assessment (day +30) and more than 500 T cells/ul, primarily bearing the CD45RA+ naïve phenotype, on day +120. Based on our preliminary data, there was no significant difference between MMRD and MUD hematopoietic stem cell transplantation (HSCT). With a median follow-up period of 15 months, the cumulative probabilities of overall patient survival and transplant-related mortality were 96.7% and 3.3%, respectively. Based on the results, the ability to control the main posttransplant complications and the immune reconstitution rates are the main factors leading to successful outcome in patients with PID after TCRαβ+-depleted HSCT.
PMID: 26187864 [PubMed – as supplied by publisher]
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