Leukemia Research Reports最新文献

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Intravascular large B-cell lymphoma masquerading as stroke successfully treated with R-Hyper-CVAD 用R-Hyper-CVAD成功治疗伪装成中风的血管内大b细胞淋巴瘤
Leukemia Research Reports Pub Date : 2023-11-27 DOI: 10.1016/j.lrr.2023.100401
Christopher Popiolek , Kanchan Gupta , Mallorie L. Huff , Ranju Gupta
{"title":"Intravascular large B-cell lymphoma masquerading as stroke successfully treated with R-Hyper-CVAD","authors":"Christopher Popiolek ,&nbsp;Kanchan Gupta ,&nbsp;Mallorie L. Huff ,&nbsp;Ranju Gupta","doi":"10.1016/j.lrr.2023.100401","DOIUrl":"https://doi.org/10.1016/j.lrr.2023.100401","url":null,"abstract":"<div><p>Intravascular large B cell lymphoma (IVLBCL) is exceedingly rare and difficult to diagnose. We describe a case of IVLBCL in a 56-year-old male which was identified after recurrent strokes. Right partial nephrectomy was then performed which demonstrated renal oncocytoma and IVLBCL. Chemotherapy was initiated with standard R-Hyper-CVAD which included intrathecal methotrexate and cytarabine. R-CHOP is largely considered the treatment of choice in IVLBCL, however low doses of chemotherapy in this regimen do not cross the blood brain barrier like in R-Hyper-CVAD. The patient achieved complete remission after completion of treatment and has remained in remission for 5 years after diagnosis.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048923000419/pdfft?md5=08cf857daa5bc968c89b065b100a4623&pid=1-s2.0-S2213048923000419-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138475244","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Acute myeloid leukemia cutis with KMT2A::MLLT3 fusion presenting with leonine facies KMT2A::MLLT3融合的急性髓性白血病切面表现为腱鞘面容
Leukemia Research Reports Pub Date : 2023-11-24 DOI: 10.1016/j.lrr.2023.100400
Bret Wankel , Muhammad Afzal , Eric Y. Loo , Robert E. LeBlanc , Joi B. Carter , Erick Lansigan , Swaroopa Yerrabothala
{"title":"Acute myeloid leukemia cutis with KMT2A::MLLT3 fusion presenting with leonine facies","authors":"Bret Wankel ,&nbsp;Muhammad Afzal ,&nbsp;Eric Y. Loo ,&nbsp;Robert E. LeBlanc ,&nbsp;Joi B. Carter ,&nbsp;Erick Lansigan ,&nbsp;Swaroopa Yerrabothala","doi":"10.1016/j.lrr.2023.100400","DOIUrl":"https://doi.org/10.1016/j.lrr.2023.100400","url":null,"abstract":"<div><p>A 63-year-old woman presented with plaques covering 60 % body-surface-area and leonine facies. Blood work showed no diagnostic aberrancies. Skin biopsy contained a malignant CD4+/CD56+ mononuclear cell population concerning for blastic plasmacytoid dendritic cell neoplasm. A later bone marrow biopsy confirmed AML with <em>KMT2A::MLLT10</em> fusion detected by next-generation sequencing (NGS). This patient's LC preceded blood and marrow based symptoms of AML. NGS of the initial skin biopsy should be considered as part of diagnostic guidelines in cases with LC in the differential as this may have led to earlier diagnosis in this case and future cases.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-11-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048923000407/pdfft?md5=0b19d4c2e00f403b405770ee31b33ff3&pid=1-s2.0-S2213048923000407-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138557443","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Successful anti-tumor effects with two novel bifunctional chemotherapeutic compounds that combine a LAT1 substrate with cytotoxic moieties in aggressive T-cell lymphomas 结合了 LAT1 底物和细胞毒性分子的两种新型双功能化疗化合物在侵袭性 T 细胞淋巴瘤中成功发挥了抗肿瘤作用
Leukemia Research Reports Pub Date : 2023-11-22 DOI: 10.1016/j.lrr.2023.100398
Carlos Murga-Zamalloa , Shaun Webb , John Reneau , Alejandro Zevallos , Pierina Danos-Diaz , Vanessa Perez-Silos , Mirna Rodriguez , Guangyao Gao , Wolf-Nicolas Fischer , Bernd Jandeleit , Ryan Wilcox
{"title":"Successful anti-tumor effects with two novel bifunctional chemotherapeutic compounds that combine a LAT1 substrate with cytotoxic moieties in aggressive T-cell lymphomas","authors":"Carlos Murga-Zamalloa ,&nbsp;Shaun Webb ,&nbsp;John Reneau ,&nbsp;Alejandro Zevallos ,&nbsp;Pierina Danos-Diaz ,&nbsp;Vanessa Perez-Silos ,&nbsp;Mirna Rodriguez ,&nbsp;Guangyao Gao ,&nbsp;Wolf-Nicolas Fischer ,&nbsp;Bernd Jandeleit ,&nbsp;Ryan Wilcox","doi":"10.1016/j.lrr.2023.100398","DOIUrl":"https://doi.org/10.1016/j.lrr.2023.100398","url":null,"abstract":"<div><p>T-cell lymphomas are aggressive neoplasms characterized by poor responses to current chemotherapeutic agents. Expression of the l-type amino acid transporter 1 (LAT 1, SLC7A5) allows for the expansion of healthy T-cell counterparts, and upregulation of LAT1 has been reported in precursor T-cell acute leukemia. Therefore, the expression of LAT1 was evaluated in a cohort of cutaneous and peripheral T-cell lymphomas. The findings demonstrated that LAT1 is upregulated in aggressive variants and absent in low-grade or indolent disease such as mycosis fungoides. In addition, upregulated LAT1 expression was seen in a large proportion of aggressive peripheral T-cell lymphomas, including peripheral T-cell lymphoma not otherwise specific (PTCL-NOS) and angioimmunoblastic T-cell lymphoma (AITL). The anti-tumor effects of two novel non-cleavable and bifunctional compounds, QBS10072S and QBS10096S, that combine a potent cytotoxic chemotherapeutic domain (tertiary N-bis(2-chloroethyl)amine) with the structural features of a selective LAT1 substrate (aromatic β-amino acid) were tested in vitro and in vivo in T-cell lymphoma cell lines. The findings demonstrated decreased survival of T-cell lymphoma lines with both compounds. Overall, the results demonstrate that LAT1 is a valuable biomarker for aggressive T-cell lymphoma counterparts and QBS10072S and QBS10096S are successful therapeutic options for these aggressive diseases.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048923000389/pdfft?md5=c638ea237da29a9a7493e919985d5f1a&pid=1-s2.0-S2213048923000389-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138738945","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Venetoclax monotherapy as front-line therapy for blastic plasmacytoid dendritic cell neoplasm. 将 Venetoclax 单药疗法作为水泡性浆细胞树突状细胞肿瘤的一线疗法。
Leukemia Research Reports Pub Date : 2023-07-03 eCollection Date: 2023-01-01 DOI: 10.1016/j.lrr.2023.100380
Ankit Mitesh Shah, Denise Pereira, Julio Poveda, Trent Peng Wang
{"title":"Venetoclax monotherapy as front-line therapy for blastic plasmacytoid dendritic cell neoplasm.","authors":"Ankit Mitesh Shah, Denise Pereira, Julio Poveda, Trent Peng Wang","doi":"10.1016/j.lrr.2023.100380","DOIUrl":"10.1016/j.lrr.2023.100380","url":null,"abstract":"<p><p>Venetoclax is an approved treatment for relapsed/refractory Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). We report a unique case of venetoclax monotherapy used for front-line induction and as a bridge to allogeneic hematopoietic stem cell transplantation (HCT). Venetoclax therapy resulted in rapid complete resolution of skin lesions, however, treatment interruption due to neutropenia led to brisk cancer recurrence. Fortunately, the patient responded to re-challenge and was able to undergo HCT. Venetoclax is active in the first-line treatment setting for BPDCN, however its effect on blood counts and durability of response should be further studied.</p>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10719512/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"54768411","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Venetoclax monotherapy as front-line therapy for blastic plasmacytoid dendritic cell neoplasm Venetoclax单药治疗作为母细胞浆细胞样树突状细胞肿瘤的一线治疗
Leukemia Research Reports Pub Date : 2023-07-03 DOI: 10.1016/j.lrr.2023.100380
Ankit Mitesh Shah , Denise Pereira , Julio Poveda , Trent Peng Wang
{"title":"Venetoclax monotherapy as front-line therapy for blastic plasmacytoid dendritic cell neoplasm","authors":"Ankit Mitesh Shah ,&nbsp;Denise Pereira ,&nbsp;Julio Poveda ,&nbsp;Trent Peng Wang","doi":"10.1016/j.lrr.2023.100380","DOIUrl":"https://doi.org/10.1016/j.lrr.2023.100380","url":null,"abstract":"","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"49899181","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 1
Isolated bone marrow gamma-delta T-Cell lymphoma: Another challenge to the WHO lymphoid neoplasm classification 分离骨髓γ - δ t细胞淋巴瘤:对WHO淋巴样肿瘤分类的又一挑战
Leukemia Research Reports Pub Date : 2023-01-01 DOI: 10.1016/j.lrr.2023.100363
Rohit Singh , David Alejos , Casey Rankins , Katherine A. Devitt , Joanna L. Conant , Diego Adrianzen-Herrera
{"title":"Isolated bone marrow gamma-delta T-Cell lymphoma: Another challenge to the WHO lymphoid neoplasm classification","authors":"Rohit Singh ,&nbsp;David Alejos ,&nbsp;Casey Rankins ,&nbsp;Katherine A. Devitt ,&nbsp;Joanna L. Conant ,&nbsp;Diego Adrianzen-Herrera","doi":"10.1016/j.lrr.2023.100363","DOIUrl":"10.1016/j.lrr.2023.100363","url":null,"abstract":"","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/b3/b7/main.PMC10050643.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9609548","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
IgG4 plasma cell neoplasm in liver transplant biopsy masquerading as rejection 肝移植活检中伪装为排斥反应的IgG4浆细胞肿瘤
Leukemia Research Reports Pub Date : 2023-01-01 DOI: 10.1016/j.lrr.2023.100379
Amarpreet Bhalla , Qiang Liu , Yanan Fang , Jay H Lefkowitch
{"title":"IgG4 plasma cell neoplasm in liver transplant biopsy masquerading as rejection","authors":"Amarpreet Bhalla ,&nbsp;Qiang Liu ,&nbsp;Yanan Fang ,&nbsp;Jay H Lefkowitch","doi":"10.1016/j.lrr.2023.100379","DOIUrl":"10.1016/j.lrr.2023.100379","url":null,"abstract":"<div><p>IgG4 plasma cell neoplasm and myeloma are rare disease entities, not associated with systemic fibroinflammatory IgG4 related disease. We herein present a case of IgG4 plasma cell neoplasm in a liver transplant biopsy. A 55 year old female was treated with living donor transplant and had a complicated post-operative course. Three months post-transplant, she presented with small for size syndrome, biliary stricture, and inferior vena cava stenosis. Concomitant liver biopsy revealed mild acute cellular rejection with central perivenulitis pattern, and mild centrilobular fibrosis. She was treated with steroids which resulted in improvement of liver enzymes. Seven months post-transplant, she presented with subtherapeutic prograf levels and cholestatic pattern of elevated liver tests. ERCP revealed a stone which was removed. Hematological evaluation revealed an abnormal serum protein electrophoresis (SPEP). Monoclonal IgG kappa was elevated along with mildly elevated free Kappa/Lambda ratio. She was followed up and readmitted two months later for worsening liver function tests. The liver biopsy showed monotypic Kappa-and IgG4-restricted plasma cell infiltrates in portal, periportal, sinusoidal and centrilobular regions, compatible with plasma cell neoplasm. In the clinical context of positivity for a serum M-spike, the monoclonal hepatic infiltrates were deemed consistent with a Kappa-and IgG4-restricted plasma cell neoplasm. Patient was treated with pulsed steroids, and liver function tests subsequently downtrended. She was followed up by Hemoncology, and the treatment plan included carfilzomib-based induction therapy and dexamethasone to prevent end-organ damage from evolving myeloma. In the meanwhile, she developed acute appendicitis, underwent appendectomy, and passed away in the post-operative period.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/87/99/main.PMC10371806.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9910996","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Impact of cytogenetic abnormalities in symptomatic multiple myeloma; a Japanese real-world analysis from Kansai Myeloma Forum 细胞遗传学异常对症状性多发性骨髓瘤的影响关西骨髓瘤论坛对日本现实世界的分析
Leukemia Research Reports Pub Date : 2023-01-01 DOI: 10.1016/j.lrr.2023.100395
Aya Nakaya , Hirohiko Shibayama , Nobuhiko Uoshima , Ryosuke Yamamura , Satoshi Yoshioka , Kazunori Imada , Yuji Shimura , Masaaki Hotta , Toshimitsu Matsui , Satoru Kosugi , Hitoshi Hanamoto , Hitoji Uchiyama , Satoshi Yoshihara , Shin-ichi Fuchida , Yoshiyuki Onda , Yasuhiro Tanaka , Kensuke Ohta , Mitsuhiro Matsuda , Junya Kanda , Adachi Yoko , Masayuki Hino
{"title":"Impact of cytogenetic abnormalities in symptomatic multiple myeloma; a Japanese real-world analysis from Kansai Myeloma Forum","authors":"Aya Nakaya ,&nbsp;Hirohiko Shibayama ,&nbsp;Nobuhiko Uoshima ,&nbsp;Ryosuke Yamamura ,&nbsp;Satoshi Yoshioka ,&nbsp;Kazunori Imada ,&nbsp;Yuji Shimura ,&nbsp;Masaaki Hotta ,&nbsp;Toshimitsu Matsui ,&nbsp;Satoru Kosugi ,&nbsp;Hitoshi Hanamoto ,&nbsp;Hitoji Uchiyama ,&nbsp;Satoshi Yoshihara ,&nbsp;Shin-ichi Fuchida ,&nbsp;Yoshiyuki Onda ,&nbsp;Yasuhiro Tanaka ,&nbsp;Kensuke Ohta ,&nbsp;Mitsuhiro Matsuda ,&nbsp;Junya Kanda ,&nbsp;Adachi Yoko ,&nbsp;Masayuki Hino","doi":"10.1016/j.lrr.2023.100395","DOIUrl":"https://doi.org/10.1016/j.lrr.2023.100395","url":null,"abstract":"<div><p>To evaluate the specific prognostic value of CAs, we conducted an analysis of 923 symptomatic multiple myeloma patients. Among this cohort, 480 patients had complete data set of high-risk CAs by interphase fluorescent in situ hybridization at diagnosis. In the high-risk group analysis, the median OS of patients without CAs (<em>n</em> = 338, 72 %) was 6.5 years, patients with del(17p) (<em>n</em> = 42, 9 %) was 4.4 years, patients with t(4;14) or t(14;16) (<em>n</em> = 72, 15 %) was 4.4 years, and patients with double-positive CAs(del(17p) and t(4;14) or t(14;16)) (<em>n</em> = 18, 4 %) was 2.1 years (<em>p</em> = 0.032). Patients with double-positive CAs had a significantly worse prognosis.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048923000353/pdfft?md5=da41e0ae4551170b1083dfbc3d685562&pid=1-s2.0-S2213048923000353-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138435663","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Testicular involvement in mantle cell lymphoma: An analysis of 16 patients. 套细胞淋巴瘤累及睾丸16例分析。
Leukemia Research Reports Pub Date : 2023-01-01 DOI: 10.1016/j.lrr.2023.100397
Samer Alkhalili , Dharmini Manogna , Hana Safah , Elizabeth Ellent , Walter Beversdorf , Ruby Arora , Nakhle S. Saba
{"title":"Testicular involvement in mantle cell lymphoma: An analysis of 16 patients.","authors":"Samer Alkhalili ,&nbsp;Dharmini Manogna ,&nbsp;Hana Safah ,&nbsp;Elizabeth Ellent ,&nbsp;Walter Beversdorf ,&nbsp;Ruby Arora ,&nbsp;Nakhle S. Saba","doi":"10.1016/j.lrr.2023.100397","DOIUrl":"https://doi.org/10.1016/j.lrr.2023.100397","url":null,"abstract":"<div><p>Mantle cell lymphoma (MCL) with testicular involvement is a rare presentation and only a few cases have been described in the literature. We present a case of MCL with testicular involvement and the first analysis of all previously reported cases assessing trends in immunohistochemical features, prognostic indicators, and survival. Our data suggest that among all MCL, testicular MCL is more likely to present with aggressive features: blastoid/pleomorphic morphology, high Ki-67 proliferative index, and CNS involvement. Testicular MCL is also associated with shorter overall survival.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048923000377/pdfft?md5=73df1b095f12d850b0ac6900aaef7a6d&pid=1-s2.0-S2213048923000377-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"136695257","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Avapritinib treatment of KIT D816V-mutant atypical chronic myeloid leukemia 阿伐替尼治疗KIT d816v突变的非典型慢性髓系白血病
Leukemia Research Reports Pub Date : 2023-01-01 DOI: 10.1016/j.lrr.2023.100371
Lyndsey Sandow , Michael Heinrich
{"title":"Avapritinib treatment of KIT D816V-mutant atypical chronic myeloid leukemia","authors":"Lyndsey Sandow ,&nbsp;Michael Heinrich","doi":"10.1016/j.lrr.2023.100371","DOIUrl":"10.1016/j.lrr.2023.100371","url":null,"abstract":"<div><h3>Background</h3><p>Atypical chronic myeloid leukemia (aCML) is a rare myelodysplastic/myeloproliferative neoplasm. There is no proven standard of care treatment and the only curative option available is hematopoietic stem cell transplant. In addition to traditional chemotherapy, targeted therapy has shown to be a promising. Avapritinib is a selective type 1 tyrosine kinase inhibitor with high potency for KIT D816V and was recently approved for treatment of systemic mastocytosis. Here we present a case of aCML with novel D816V mutation treated with avapritinib for 17 months leading to clonal extinction of the driver mutation.</p></div><div><h3>Case presentation</h3><p>An 80 year old man initially presented for evaluation of aCML. A bone marrow biopsy was completed, and next generation sequencing was notable for a novel KIT D816V mutation. Patient was started on avapritinib leading to significant improvement in leukocytosis and extinction of the D816V mutation over 17 months of treatment. The extinction was followed with serial next generation sequencing.</p></div><div><h3>Conclusion</h3><p>We present the first case of aCML with KIT D816V driver mutation. We also demonstrate two novel management strategies. First, we show that treatment with avapritinib does not need to be limited to cases of systemic mastocytosis and could be useful in other hematologic malignancies with this driver mutation. Furthermore, with the use of serial next generation sequencing we were able to identify new emerging clones. While none of the clones noted in this study were targetable, they could be in other patients with aCML and help guide treatment.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2023-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/79/95/main.PMC10248858.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9976419","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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