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Successful Long-Term Enzyme Replacement Therapy in a Patient with Delayed-Onset ADA Deficiency.

作者信息

Toskov Vasil, Bali Pawan, Hershfield Michael S, Ehl Stephan, Speckmann Carsten

机构信息

Division of Pediatric Hematology and Oncology, Department of Pediatrics and Adolescent Medicine, Medical Center, University of Freiburg, Faculty of Medicine, Freiburg, Germany.

Department of Medicine and Biochemistry, Duke University Medical Center, Durham, NC, USA.

出版信息

J Clin Immunol. 2024 Sep 12;45(1):8. doi: 10.1007/s10875-024-01794-7.

DOI:10.1007/s10875-024-01794-7
PMID:39264481
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11393103/
Abstract
摘要

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本文引用的文献

1
Safety and efficacy of elapegademase in patients with adenosine deaminase deficiency: A multicenter, open-label, single-arm, phase 3, and postmarketing clinical study.腺苷脱氨酶缺乏症患者使用依拉糖苷酶的安全性和有效性:一项多中心、开放标签、单臂、3 期和上市后临床研究。
Immun Inflamm Dis. 2023 Jul;11(7):e917. doi: 10.1002/iid3.917.
2
Long-Term Immune Reconstitution in ADA-Deficient Patients Treated With Elapegademase: A Real-World Experience.ADA 缺陷患者接受 Elapegademase 治疗的长期免疫重建:真实世界经验。
J Allergy Clin Immunol Pract. 2023 Jun;11(6):1725-1733. doi: 10.1016/j.jaip.2023.01.028. Epub 2023 Feb 1.
3
Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC.
ADA 缺陷型严重联合免疫缺陷症治疗后的结果:PIDTC 的报告。
Blood. 2022 Aug 18;140(7):685-705. doi: 10.1182/blood.2022016196.
4
Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study.青少年和成人遗传性免疫缺陷患者的造血干细胞移植:一项欧洲血液与骨髓移植学会免疫缺陷工作组的研究
Blood. 2022 Oct 6;140(14):1635-1649. doi: 10.1182/blood.2022015506.
5
Outcomes of 3 patients with adenosine deaminase deficiency on long-term enzyme replacement therapy.3例腺苷脱氨酶缺乏症患者长期酶替代治疗的结果。
Ann Allergy Asthma Immunol. 2021 May;126(5):593-595. doi: 10.1016/j.anai.2020.12.008. Epub 2021 Jan 30.
6
Morbidity in an adenosine deaminase-deficient patient during 27 years of enzyme replacement therapy.腺苷脱氨酶缺乏症患者接受酶替代治疗 27 年期间的发病率。
Clin Immunol. 2020 Feb;211:108321. doi: 10.1016/j.clim.2019.108321. Epub 2019 Dec 5.
7
Delayed-onset adenosine deaminase deficiency: strategies for an early diagnosis.迟发性腺苷脱氨酶缺乏症:早期诊断策略
J Allergy Clin Immunol. 2012 Oct;130(4):991-4. doi: 10.1016/j.jaci.2012.04.004. Epub 2012 May 10.
8
Clinical and immunological manifestations of patients with atypical severe combined immunodeficiency.不典型严重联合免疫缺陷患者的临床和免疫学表现。
Clin Immunol. 2011 Oct;141(1):73-82. doi: 10.1016/j.clim.2011.05.007. Epub 2011 May 30.
9
Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).腺苷脱氨酶(ADA)缺乏的重症联合免疫缺陷(SCID)的酶替代疗法的长期疗效。
Clin Immunol. 2005 Nov;117(2):133-43. doi: 10.1016/j.clim.2005.07.006. Epub 2005 Aug 22.
10
Immunophenotyping of blood lymphocytes in childhood. Reference values for lymphocyte subpopulations.儿童期血液淋巴细胞的免疫表型分析。淋巴细胞亚群的参考值。
J Pediatr. 1997 Mar;130(3):388-93. doi: 10.1016/s0022-3476(97)70200-2.