Successful TCRαβ/CD19-Depleted Hematopoietic Cell Transplantation for a Patient With Artemis Deficiency-Reference-Cited by-同舟云学术

Successful TCRαβ/CD19-Depleted Hematopoietic Cell Transplantation for a Patient With Artemis Deficiency

Published:2023-02-06 Issue:2 Volume:45 Page:e285-e289
ISSN:1077-4114
Container-title:Journal of Pediatric Hematology/Oncology
language:en
Short-container-title:

Author:

Tomomasa Dan¹,Isoda Takeshi¹^ORCID,Mitsuiki Noriko¹^ORCID,Inoue Kento¹^ORCID,Nishimura Akira¹,Uda Kazuhiro²^ORCID,Uchiyama Toru³^ORCID,Yamashita Motoi¹^ORCID,Kamiya Takahiro¹,Endo Akifumi¹^ORCID,Takagi Masatoshi¹^ORCID,Imai Kohsuke⁴^ORCID,Kajiwara Michiko⁵,Cowan Morton J.⁶^ORCID,Morio Tomohiro¹^ORCID,Kanegane Hirokazu⁷^ORCID

Affiliation:

1. Department of Pediatrics and Developmental Biology, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan

2. Division of Infectious Diseases, Tokyo Metropolitan Children’s Medical Center, Tokyo, Japan

3. Department of Human Genetics, National Research Institute for Child Health and Development, Tokyo, Japan

4. Department of Community Pediatrics, Perinatal and Maternal Medicine, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan

5. Center for Transfusion Medicine and Cell Therapy, Tokyo Medical and Dental University Hospital, Tokyo, Japan

6. Allergy Immunology and Blood and Marrow Transplant Division, Benioff Children’s Hospital, University of California San Francisco, San Francisco, California

7. Deparment of Child Health and Development, Graduate School of Medical and Dental Sciences, Tokyo Medical and Dental University (TMDU), Tokyo, Japan

Abstract

Artemis deficiency is characterized by DNA double-strand breaks repairing dysfunction and increased sensitivity to ionizing radiation and alkylating reagents. We describe the first successful case of T-cell receptor [TCR]αβ/CD19-depleted hematopoietic cell transplantation [HCT] for Artemis deficiency in Japan. A 6-month-old Korean boy was diagnosed with Artemis-deficient severe combined immunodeficiency. He had no human leukocyte antigen (HLA)-matched sibling or unrelated donor. Therefore, TCRαβ/CD19-depleted HCT from his haploidentical mother was performed. Despite mixed chimerism in whole blood, T cells achieved complete donor chimerism 6 months after HCT. TCRαβ/CD19-depleted HCT could be an effective treatment for patients with radiation-sensitive severe combined immunodeficiency.

Publisher

Ovid Technologies (Wolters Kluwer Health)

Subject

Oncology,Hematology,Pediatrics, Perinatology and Child Health

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