Successful TCRαβ/CD19-Depleted Hematopoietic Cell Transplantation for a Patient With Artemis Deficiency. Issue 2 (6th March 2023)

• Record Type: Journal Article
• Authors: Tomomasa, Dan; Isoda, Takeshi; Mitsuiki, Noriko; Inoue, Kento; Nishimura, Akira; Uda, Kazuhiro; Uchiyama, Toru; Yamashita, Motoi; Kamiya, Takahiro; Endo, Akifumi; Takagi, Masatoshi; Imai, Kohsuke; Kajiwara, Michiko; Cowan, Morton J.; Morio, Tomohiro; Kanegane, Hirokazu
• Is Part Of: Journal of pediatric hematology/oncology. Volume 45:Issue 2(2023)
• Page Start: e285
• Page End: e289
• Publication Date: 2023-03-06
• Subjects: artemis deficiency -- TCRαβ/CD19-depleted hematopoietic cell transplantation -- haploidentical hematopoietic cell transplantation; Pediatric hematology -- Periodicals; Tumors in children -- Periodicals; 618.9215
• DOI: 10.1097/MPH.0000000000002522
• Languages: English
• ISSNs: 1077-4114

Abstract

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.