{"title":"UNC13D和CD27基因突变的复发/难治性外周T细胞淋巴瘤相关嗜血细胞淋巴组织细胞增多症","authors":"Tingting Yang, Rongrong Chen, Mingming Zhang, Ruirui Jing, Jia Geng, Guoqing Wei, Yi Luo, Pingnan Xiao, Ruimin Hong, Jingjing Feng, Shan Fu, Houli Zhao, Jiazhen Cui, Simao Huang, He Huang, Yongxian Hu","doi":"10.1177/09636897231221887","DOIUrl":null,"url":null,"abstract":"<p><p>Hemophagocytic lymphohistiocytosis (HLH) is a severe hyperinflammatory disease characterized by familial and acquired forms. Here, we present the case of a 26-year-old male patient with relapsed/refractory peripheral T-cell lymphoma and concurrent HLH. Whole-exon sequencing revealed germline mutations associated with HLH, including those in critical genes such as <i>CD27</i> and <i>UNC13D</i> and other germline heterozygous variants (<i>NOTCH2, NOTCH3, IL2RA, TYK2, AGL, CFD</i>, and <i>F13A1</i>). CD107a analyses consistently demonstrated impaired degranulation of cytotoxic T-lymphocytes and natural killer (NK) cells. Examination of the patient's family pedigree revealed that his father and mother harbored <i>UNC13D</i> and <i>CD27</i> mutations, respectively; his brother carried the same <i>CD27</i> heterozygous mutation. However, none of them manifested the disease. Despite the missense mutation of <i>CD27</i> (c.779C>T; p.Pro260Leu) lacking previous documentation in databases, comprehensive analysis suggested non-pathogenic mutations in the <i>CD27</i> variant, indicating minimal impact on T- and NK-cell functions. These results ultimately supported the option of hematopoietic stem cell transplantation (HSCT) as a successful curative therapeutic approach. As of this report, the patient has remained free of lymphoma and quiescent HLH 15.2 months post-HSCT. This study underscores the efficacy of genetic tests in identifying significant mutations and confirming their etiologies, providing an early basis for treatment decisions and the selection of suitable transplant donors.</p>","PeriodicalId":9721,"journal":{"name":"Cell Transplantation","volume":"33 ","pages":"9636897231221887"},"PeriodicalIF":3.2000,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10771736/pdf/","citationCount":"0","resultStr":"{\"title\":\"Relapsed/Refractory Peripheral T-Cell Lymphoma-Associated Hemophagocytic Lymphohistiocytosis With UNC13D and CD27 Germline Mutations.\",\"authors\":\"Tingting Yang, Rongrong Chen, Mingming Zhang, Ruirui Jing, Jia Geng, Guoqing Wei, Yi Luo, Pingnan Xiao, Ruimin Hong, Jingjing Feng, Shan Fu, Houli Zhao, Jiazhen Cui, Simao Huang, He Huang, Yongxian Hu\",\"doi\":\"10.1177/09636897231221887\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Hemophagocytic lymphohistiocytosis (HLH) is a severe hyperinflammatory disease characterized by familial and acquired forms. Here, we present the case of a 26-year-old male patient with relapsed/refractory peripheral T-cell lymphoma and concurrent HLH. Whole-exon sequencing revealed germline mutations associated with HLH, including those in critical genes such as <i>CD27</i> and <i>UNC13D</i> and other germline heterozygous variants (<i>NOTCH2, NOTCH3, IL2RA, TYK2, AGL, CFD</i>, and <i>F13A1</i>). CD107a analyses consistently demonstrated impaired degranulation of cytotoxic T-lymphocytes and natural killer (NK) cells. Examination of the patient's family pedigree revealed that his father and mother harbored <i>UNC13D</i> and <i>CD27</i> mutations, respectively; his brother carried the same <i>CD27</i> heterozygous mutation. However, none of them manifested the disease. Despite the missense mutation of <i>CD27</i> (c.779C>T; p.Pro260Leu) lacking previous documentation in databases, comprehensive analysis suggested non-pathogenic mutations in the <i>CD27</i> variant, indicating minimal impact on T- and NK-cell functions. These results ultimately supported the option of hematopoietic stem cell transplantation (HSCT) as a successful curative therapeutic approach. As of this report, the patient has remained free of lymphoma and quiescent HLH 15.2 months post-HSCT. This study underscores the efficacy of genetic tests in identifying significant mutations and confirming their etiologies, providing an early basis for treatment decisions and the selection of suitable transplant donors.</p>\",\"PeriodicalId\":9721,\"journal\":{\"name\":\"Cell Transplantation\",\"volume\":\"33 \",\"pages\":\"9636897231221887\"},\"PeriodicalIF\":3.2000,\"publicationDate\":\"2024-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10771736/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Cell Transplantation\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/09636897231221887\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"CELL & TISSUE ENGINEERING\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Cell Transplantation","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/09636897231221887","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"CELL & TISSUE ENGINEERING","Score":null,"Total":0}
引用次数: 0
摘要
嗜血细胞淋巴组织细胞增多症(HLH)是一种严重的高炎症性疾病,有家族性和获得性之分。在此,我们介绍了一名 26 岁男性患者的病例,他患有复发/难治性外周 T 细胞淋巴瘤,并同时患有 HLH。全外显子测序发现了与HLH相关的种系突变,包括CD27和UNC13D等关键基因的突变以及其他种系杂合变异(NOTCH2、NOTCH3、IL2RA、TYK2、AGL、CFD和F13A1)。CD107a 分析结果一致表明,细胞毒性 T 淋巴细胞和自然杀伤细胞(NK)的脱颗粒功能受损。对患者家族血统的检查显示,他的父亲和母亲分别携带 UNC13D 和 CD27 基因突变;他的兄弟也携带相同的 CD27 杂合突变。然而,他们都没有发病。尽管 CD27 的错义突变(c.779C>T; p.Pro260Leu)以前在数据库中没有记录,但综合分析表明 CD27 变异中存在非致病性突变,表明对 T 细胞和 NK 细胞功能的影响微乎其微。这些结果最终支持将造血干细胞移植(HSCT)作为一种成功的治疗方法。截至本报告发布时,该患者在造血干细胞移植后15.2个月仍未出现淋巴瘤和静止性HLH。这项研究强调了基因检测在识别重大突变和确认病因方面的功效,为治疗决策和选择合适的移植供体提供了早期依据。
Relapsed/Refractory Peripheral T-Cell Lymphoma-Associated Hemophagocytic Lymphohistiocytosis With UNC13D and CD27 Germline Mutations.
Hemophagocytic lymphohistiocytosis (HLH) is a severe hyperinflammatory disease characterized by familial and acquired forms. Here, we present the case of a 26-year-old male patient with relapsed/refractory peripheral T-cell lymphoma and concurrent HLH. Whole-exon sequencing revealed germline mutations associated with HLH, including those in critical genes such as CD27 and UNC13D and other germline heterozygous variants (NOTCH2, NOTCH3, IL2RA, TYK2, AGL, CFD, and F13A1). CD107a analyses consistently demonstrated impaired degranulation of cytotoxic T-lymphocytes and natural killer (NK) cells. Examination of the patient's family pedigree revealed that his father and mother harbored UNC13D and CD27 mutations, respectively; his brother carried the same CD27 heterozygous mutation. However, none of them manifested the disease. Despite the missense mutation of CD27 (c.779C>T; p.Pro260Leu) lacking previous documentation in databases, comprehensive analysis suggested non-pathogenic mutations in the CD27 variant, indicating minimal impact on T- and NK-cell functions. These results ultimately supported the option of hematopoietic stem cell transplantation (HSCT) as a successful curative therapeutic approach. As of this report, the patient has remained free of lymphoma and quiescent HLH 15.2 months post-HSCT. This study underscores the efficacy of genetic tests in identifying significant mutations and confirming their etiologies, providing an early basis for treatment decisions and the selection of suitable transplant donors.
期刊介绍:
Cell Transplantation, The Regenerative Medicine Journal is an open access, peer reviewed journal that is published 12 times annually. Cell Transplantation is a multi-disciplinary forum for publication of articles on cell transplantation and its applications to human diseases. Articles focus on a myriad of topics including the physiological, medical, pre-clinical, tissue engineering, stem cell, and device-oriented aspects of the nervous, endocrine, cardiovascular, and endothelial systems, as well as genetically engineered cells. Cell Transplantation also reports on relevant technological advances, clinical studies, and regulatory considerations related to the implantation of cells into the body in order to provide complete coverage of the field.