Li Zhu, Ting Shi, Yi Liu, Shuqi Zhao, Huanping Wang, Zhimei Chen, Yungui Wang, Jie Jin, Hongyan Tong, Liangshun You, Hong-Hu Zhu
{"title":"首例患有 TFG-RARA 的急性早幼粒细胞白血病患者在接受 Venetoclax 和全反式维甲酸治疗后获得完全缓解。","authors":"Li Zhu, Ting Shi, Yi Liu, Shuqi Zhao, Huanping Wang, Zhimei Chen, Yungui Wang, Jie Jin, Hongyan Tong, Liangshun You, Hong-Hu Zhu","doi":"10.1080/16078454.2024.2430044","DOIUrl":null,"url":null,"abstract":"<p><p>Variant acute promyelocytic leukemia (vAPL) represents a certain type of APL case whose specific fusion proteins, which are relevant but atypical variants, may fail to be identified by polymerase chain reaction (PCR) and fluorescence in situ hybridization (FISH) and requires identification through next-generation sequencing (NGS) or RNA sequencing (RNA-seq). These patients often show insensitivity to arsenic trioxide (ATO) or all trans-retinoic acid (ATRA) and therefore exhibit unclear prognosis. Venetoclax (VEN), an oral small-molecule B-cell lymphoma 2 (BCL-2) inhibitor, demonstrates effectiveness and safety as a cytoreduction therapy for pediatric APL and has shown some promising effect on relapsed or refractory APL. However, only a few cases have been reported on the treatment of vAPL with a single drug or multiple drugs combined with VEN. Therefore, this study reported the first vAPL case with the TFG-RARA fusion gene, who achieved complete remission (CR) with oral administration of VEN and ATRA, and remained CR till submission. Our study indicated that VEN may have a good therapeutic effect and contribute to a better prognosis of vAPL and warranted further application among APL patients.</p>","PeriodicalId":13161,"journal":{"name":"Hematology","volume":"29 1","pages":"2430044"},"PeriodicalIF":2.0000,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"First case of acute promyelocytic leukemia with TFG-RARA achieved complete remission treated with venetoclax and all-trans retinoic acid.\",\"authors\":\"Li Zhu, Ting Shi, Yi Liu, Shuqi Zhao, Huanping Wang, Zhimei Chen, Yungui Wang, Jie Jin, Hongyan Tong, Liangshun You, Hong-Hu Zhu\",\"doi\":\"10.1080/16078454.2024.2430044\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Variant acute promyelocytic leukemia (vAPL) represents a certain type of APL case whose specific fusion proteins, which are relevant but atypical variants, may fail to be identified by polymerase chain reaction (PCR) and fluorescence in situ hybridization (FISH) and requires identification through next-generation sequencing (NGS) or RNA sequencing (RNA-seq). These patients often show insensitivity to arsenic trioxide (ATO) or all trans-retinoic acid (ATRA) and therefore exhibit unclear prognosis. Venetoclax (VEN), an oral small-molecule B-cell lymphoma 2 (BCL-2) inhibitor, demonstrates effectiveness and safety as a cytoreduction therapy for pediatric APL and has shown some promising effect on relapsed or refractory APL. However, only a few cases have been reported on the treatment of vAPL with a single drug or multiple drugs combined with VEN. Therefore, this study reported the first vAPL case with the TFG-RARA fusion gene, who achieved complete remission (CR) with oral administration of VEN and ATRA, and remained CR till submission. Our study indicated that VEN may have a good therapeutic effect and contribute to a better prognosis of vAPL and warranted further application among APL patients.</p>\",\"PeriodicalId\":13161,\"journal\":{\"name\":\"Hematology\",\"volume\":\"29 1\",\"pages\":\"2430044\"},\"PeriodicalIF\":2.0000,\"publicationDate\":\"2024-12-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Hematology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1080/16078454.2024.2430044\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/11/28 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Hematology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/16078454.2024.2430044","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/11/28 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
变异型急性早幼粒细胞白血病(vAPL)是APL病例中的一种,其特异性融合蛋白是相关但不典型的变体,可能无法通过聚合酶链式反应(PCR)和荧光原位杂交(FISH)鉴定,需要通过新一代测序(NGS)或RNA测序(RNA-seq)进行鉴定。这些患者通常对三氧化二砷(ATO)或全反式维甲酸(ATRA)不敏感,因此预后不明确。Venetoclax (VEN)是一种口服小分子B细胞淋巴瘤2(BCL-2)抑制剂,作为小儿APL的细胞减少疗法具有有效性和安全性,并对复发或难治性APL显示出一定的疗效。然而,关于使用单一药物或多种药物联合 VEN 治疗 vAPL 的报道却寥寥无几。因此,本研究报告了首例带有 TFG-RARA 融合基因的 vAPL 病例,该患者通过口服 VEN 和 ATRA 获得了完全缓解(CR),并且直到提交病例时仍保持完全缓解。我们的研究表明,VEN可能具有良好的治疗效果,有助于改善vAPL的预后,值得在APL患者中进一步应用。
First case of acute promyelocytic leukemia with TFG-RARA achieved complete remission treated with venetoclax and all-trans retinoic acid.
Variant acute promyelocytic leukemia (vAPL) represents a certain type of APL case whose specific fusion proteins, which are relevant but atypical variants, may fail to be identified by polymerase chain reaction (PCR) and fluorescence in situ hybridization (FISH) and requires identification through next-generation sequencing (NGS) or RNA sequencing (RNA-seq). These patients often show insensitivity to arsenic trioxide (ATO) or all trans-retinoic acid (ATRA) and therefore exhibit unclear prognosis. Venetoclax (VEN), an oral small-molecule B-cell lymphoma 2 (BCL-2) inhibitor, demonstrates effectiveness and safety as a cytoreduction therapy for pediatric APL and has shown some promising effect on relapsed or refractory APL. However, only a few cases have been reported on the treatment of vAPL with a single drug or multiple drugs combined with VEN. Therefore, this study reported the first vAPL case with the TFG-RARA fusion gene, who achieved complete remission (CR) with oral administration of VEN and ATRA, and remained CR till submission. Our study indicated that VEN may have a good therapeutic effect and contribute to a better prognosis of vAPL and warranted further application among APL patients.
期刊介绍:
Hematology is an international journal publishing original and review articles in the field of general hematology, including oncology, pathology, biology, clinical research and epidemiology. Of the fixed sections, annotations are accepted on any general or scientific field: technical annotations covering current laboratory practice in general hematology, blood transfusion and clinical trials, and current clinical practice reviews the consensus driven areas of care and management.