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16-01-2024 | Graft-Versus-Host Disease | Case Report

HLA-haploidentical T-cell receptor αβT/B-cell-depleted stem cell transplantation for Fanconi anemia

Authors: Akihiro Iguchi, Toru Uchiyama, Kentaro Fujimori, Yoshihiro Gocho, Hirotoshi Sakaguchi, Takao Deguchi, Daisuke Tomizawa, Ken-Ichi Imadome, Masafumi Onodera, Kimikazu Matsumoto

Published in: International Journal of Hematology | Issue 3/2024

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Abstract

HLA-haploidentical stem cell transplantation (haplo-SCT) using post-transplant high-dose cyclophosphamide (PT-CY) is an alternative choice when a suitable donors is unavailable. However, PT-CY is difficult in patients with Fanconi anemia (FA) due to their high vulnerability to alkylating agents. For FA, we prefer haplo-SCT by T-cell receptor αβT-cell and B-cell depletion (αβT/B-depleted haplo-SCT), which can reduce the risks of PT-CY-related complications and graft-versus-host disease (GVHD). An 11-year-old boy with diagnosed FA (FANCG mutation) and bone marrow failure was to receive αβT/B-depleted haplo-SCT from his father (HLA 4/8 allele matched) due to absence of an HLA-matched donors. αβT/B-depleted peripheral blood stem cells (CD34 + cell count, 1.17 × 107/kg; αβ + T-cell count, 1.3 × 105/kg) were infused following conditioning consisting of fludarabine (150 mg/m2), cyclophosphamide (40 mg/kg), anti-thymocyte globulin (5 mg/kg), rituximab (375 mg/m2), and thoraco-abdominal irradiation (3 Gy). Tacrolimus was used for GVHD prophylaxis until day + 30. Neutrophil engraftment was achieved on day + 9, and complete chimerism was confirmed on days + 28 and + 96. At 12-month post-SCT, the patient was well without GVHD or any other complications. αβT/B-depleted haplo-SCT is a good choice not only for patients unsuitable for PT-CY, but also for all pediatric recipients to reduce SCT-related complications.
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Metadata
Title
HLA-haploidentical T-cell receptor αβT/B-cell-depleted stem cell transplantation for Fanconi anemia
Authors
Akihiro Iguchi
Toru Uchiyama
Kentaro Fujimori
Yoshihiro Gocho
Hirotoshi Sakaguchi
Takao Deguchi
Daisuke Tomizawa
Ken-Ichi Imadome
Masafumi Onodera
Kimikazu Matsumoto
Publication date
16-01-2024
Publisher
Springer Nature Singapore
Published in
International Journal of Hematology / Issue 3/2024
Print ISSN: 0925-5710
Electronic ISSN: 1865-3774
DOI
https://doi.org/10.1007/s12185-023-03703-x

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