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

被引：1

作者：

Tomomasa, Dan ^{[1
]}

Isoda, Takeshi ^{[1
]}

Mitsuiki, Noriko ^{[1
]}

Inoue, Kento ^{[1
]}

Nishimura, Akira ^{[1
]}

Uda, Kazuhiro ^{[2
]}

Uchiyama, Toru ^{[3
]}

Yamashita, Motoi ^{[1
]}

Kamiya, Takahiro ^{[1
]}

Endo, Akifumi ^{[1
]}

Takagi, Masatoshi ^{[1
]}

Imai, Kohsuke ^{[4
]}

Kajiwara, Michiko ^{[5
]}

Cowan, Morton J. ^{[6
]}

Morio, Tomohiro ^{[1
]}

Kanegane, Hirokazu ^{[7
,8
]}

机构：

[1] Tokyo Med & Dent Univ TMDU, Grad Sch Med & Dent Sci, Dept Pediat & Dev Biol, Tokyo, Japan

[2] Tokyo Metropolitan Childrens Med Ctr, Div Infect Dis, Tokyo, Japan

[3] Natl Res Inst Child Hlth & Dev, Dept Human Genet, Tokyo, Japan

[4] Tokyo Med & Dent Univ TMDU, Grad Sch Med & Dent Sci, Dept Community Pediat Perinatal & Maternal Med, Tokyo, Japan

[5] Tokyo Med & Dent Univ Hosp, Ctr Transfus Med & Cell Therapy, Tokyo, Japan

[6] Univ Calif San Francisco, Benioff Childrens Hosp, Allergy Immunol & Blood & Marrow Transplant Div, San Francisco, CA USA

[7] Tokyo Med & Dent Univ TMDU, Grad Sch Med & Dent Sci, Deparment Child Hlth & Dev, Tokyo, Japan

[8] Tokyo Med & Dent Univ TMDU, Grad Sch Med & Dent Sci, Dept Child Hlth & Dev, 1-5-45 Yushima, Bunkyo ku, Tokyo 1138519, Japan

来源：

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY | 2023年 / 45卷 / 02期

关键词：

artemis deficiency; TCR alpha beta; CD19-depleted hematopoietic cell transplantation; haploidentical hematopoietic cell transplantation; SEVERE COMBINED IMMUNODEFICIENCY; EXCISION CIRCLES; QUANTIFICATION; IDENTIFICATION; ALPHA/BETA; DEPLETION; CHILDREN; RISK;

D O I：

10.1097/MPH.0000000000002522

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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]alpha beta/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, TCR alpha beta/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. TCR alpha beta/CD19-depleted HCT could be an effective treatment for patients with radiation-sensitive severe combined immunodeficiency.

引用

页码：E285 / E289

页数：5

共 22 条

[1] Single-Center Experience of Unrelated and Haploidentical Stem Cell Transplantation with TCRαβ and CD19 Depletion in Children with Primary Immunodeficiency Syndromes [J].

Balashov, Dmitry ;

Shcherbina, Anna ;

Maschan, Michael ;

Trakhtman, Pavel ;

Skvortsova, Yulia ;

Shelikhova, Larisa ;

Laberko, Alexandra ;

Livshits, Anna ;

Novichkova, Galina ;

Maschan, Alexei .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2015, 21 (11) :1955-1962

[2] HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders [J].

Bertaina, Alice ;

Merli, Pietro ;

Rutella, Sergio ;

Pagliara, Daria ;

Bernardo, Maria Ester ;

Masetti, Riccardo ;

Pende, Daniela ;

Falco, Michela ;

Handgretinger, Rupert ;

Moretta, Francesca ;

Lucarelli, Barbarella ;

Brescia, Letizia P. ;

Li Pira, Giuseppina ;

Testi, Manuela ;

Cancrini, Caterina ;

Kabbara, Nabil ;

Carsetti, Rita ;

Finocchi, Andrea ;

Moretta, Alessandro ;

Moretta, Lorenzo ;

Locatelli, Franco .

BLOOD, 2014, 124 (05) :822-826

[3] Radiation-sensitive severe combined immunodeficiency: The arguments for and against conditioning before hematopoietic cell transplantation-what to do? [J].

Cowan, Morton J. ;

Gennery, Andrew R. .

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2015, 136 (05) :1178-1185

[4] Low Exposure Busulfan Conditioning to Achieve Sufficient Multilineage Chimerism in Patients with Severe Combined Immunodeficiency [J].

Dvorak, Christopher C. ;

Long-Boyle, Janel ;

Dara, Jasmeen ;

Melton, Alexis ;

Shimano, Kristin A. ;

Huang, James N. ;

Puck, Jennifer M. ;

Dorsey, Morna J. ;

Facchino, Janelle ;

Chang, Catherine K. ;

Cowan, Morton J. .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2019, 25 (07) :1355-1362

[5] New graft manipulation strategies improve the outcome of mismatched stem cell transplantation in children with primary immunodeficiencies [J].

Elfeky, Reem ;

Shah, Ravi M. ;

Unni, Mohamed N. M. ;

Ottaviano, Giorgio ;

Rao, Kanchan ;

Chiesa, Robert ;

Amrolia, Persis ;

Worth, Austen ;

Flood, Terry ;

Abinun, Mario ;

Hambleton, Sophie ;

Cant, Andrew J. ;

Gilmour, Kimberly ;

Adams, Stuart ;

Ahsan, Gul ;

Barge, Dawn ;

Gennery, Andrew R. ;

Qasim, Waseem ;

Slatter, Mary ;

Veys, Paul .

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2019, 144 (01) :280-293

[6] Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: a PIDTC natural history study [J].

Heimall, Jennifer ;

Logan, Brent R. ;

Cowan, Morton J. ;

Notarangelo, Luigi D. ;

Griffith, Linda M. ;

Puck, Jennifer M. ;

Kohn, Donald B. ;

Pulsipher, Michael A. ;

Parikh, Suhag ;

Martinez, Caridad ;

Kapoor, Neena ;

O'Reilly, Richard ;

Boyer, Michael ;

Pai, Sung-Yun ;

Goldman, Frederick ;

Burroughs, Lauri ;

Chandra, Sharat ;

Kletzel, Morris ;

Thakar, Monica ;

Connelly, James ;

Cuvelier, Geoff ;

Saldana, Blachy J. Davila ;

Shereck, Evan ;

Knutsen, Alan ;

Sullivan, Kathleen E. ;

DeSantes, Kenneth ;

Gillio, Alfred ;

Haddad, Elie ;

Petrovic, Aleksandra ;

Quigg, Troy ;

Smith, Angela R. ;

Stenger, Elizabeth ;

Yin, Ziyan ;

Shearer, William T. ;

Fleisher, Thomas ;

Buckley, Rebecca H. ;

Dvorak, Christopher C. .

BLOOD, 2017, 130 (25) :2718-2727

[7]

Kanakry CG, 2016, NAT REV CLIN ONCOL, V13, P10, DOI [10.1038/nrclinonc.2015.234, 10.1038/nrclinonc.2015.128]

[8] Newborn Screening for Primary Immunodeficiency Diseases: History, Current and Future Practice [J].

King, Jovanka R. ;

Hammarstrom, Lennart .

JOURNAL OF CLINICAL IMMUNOLOGY, 2018, 38 (01) :56-66

[9] Mismatched related vs matched unrelated donors in TCRαβ/CD19-depleted HSCT for primary immunodeficiencies [J].

Laberko, Alexandra ;

Sultanova, Elvira ;

Gutovskaya, Elena ;

Shipitsina, Irina ;

Shelikhova, Larisa ;

Kurnikova, Elena ;

Muzalevskii, Yakov ;

Kazachenok, Alexei ;

Pershin, Dmitriy ;

Voronin, Kirill ;

Shcherbina, Anna ;

Maschan, Michael ;

Maschan, Alexey ;

Balashov, Dmitry .

BLOOD, 2019, 134 (20) :1755-1763

[10] Risk Factors for and the Clinical Impact of Cytomegalovirus and Epstein-Barr Virus Infections in Pediatric Recipients of TCR-α/β- and CD19-Depleted Grafts [J].

Laberko, Alexandra ;

Bogoyavlenskaya, Anna ;

Shelikhova, Larisa ;

Shekhovtsova, Zhanna ;

Balashov, Dmitriy ;

Voronin, Kirill ;

Kurnikova, Elena ;

Boyakova, Elena ;

Raykina, Elena ;

Brilliantova, Varvara ;

Pirumova, Valentina ;

Novichkova, Galina ;

Maschan, Alexei ;

Maschan, Michael .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2017, 23 (03) :483-490

← 1 2 3 →