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DISTINCT PATHOGENESIS OF CLONAL HEMATOPOIESIS REVEALED BY SINGLE-CELL MULTI-OMICS SEQUENCING. 单细胞多组学测序揭示了克隆性造血的独特发病机制。
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100435
M. Nakagawa , R. Inagaki , Y. Kuroda , Y. Nannya , M. Motomura , A. Kon , L. Zhao , Y. Ochi , J. Takeda , X. Qi , K. Okazaki , A. Yoda , N. Kakiuchi , H. Makishima , S. Matsuda , S. Ogawa
{"title":"DISTINCT PATHOGENESIS OF CLONAL HEMATOPOIESIS REVEALED BY SINGLE-CELL MULTI-OMICS SEQUENCING.","authors":"M. Nakagawa ,&nbsp;R. Inagaki ,&nbsp;Y. Kuroda ,&nbsp;Y. Nannya ,&nbsp;M. Motomura ,&nbsp;A. Kon ,&nbsp;L. Zhao ,&nbsp;Y. Ochi ,&nbsp;J. Takeda ,&nbsp;X. Qi ,&nbsp;K. Okazaki ,&nbsp;A. Yoda ,&nbsp;N. Kakiuchi ,&nbsp;H. Makishima ,&nbsp;S. Matsuda ,&nbsp;S. Ogawa","doi":"10.1016/j.lrr.2024.100435","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100435","url":null,"abstract":"<div><h3>Introduction</h3><p>Despite the significant impact of clonal hematopoiesis (CH) on leukemogenesis, the pathogenesis of CH is still not fully understood.</p></div><div><h3>Methods</h3><p>Utilizing a novel single-cell sequencing platform that allows for simultaneous detection of mutations and gene expression, we examined the gene expression profiles of hematopoietic stem and progenitor cells (HSPCs) harboring CH-related mutations from CH(+) cases, which was compared with that of wild-type (WT) cells from both CH(+) and CH(−) cases. Age-related changes in the bone marrow (BM) environment were also assessed using CH(−) cases.</p></div><div><h3>Results</h3><p>In 12 patients with CH, genes associated with cell proliferation were upregulated in mutant cells. Significantly, mutant cells showed decreased expression of genes related to inflammatory responses, which were enhanced in BM cells from aged CH(−) cases, indicating the potential contribution of aged BM environment to the positive selection of mutant cells. Unexpectedly, WT cells from 3 <em>TET2</em>-CH(+) cases demonstrated significant upregulation of genes related to interferon response and cell proliferation, compared with those from age-matched CH(−) cases, suggesting the altered BM environments. Notably, when competitively transplanted with <em>Tet2</em>-knockout (KO) cells, WT HSPCs displayed enhanced expression of genes associated with cell proliferation and interferon signalling, compared with those transplanted with WT cells, implying non-cell autonomous effects of mutant cells.</p></div><div><h3>Conclusions</h3><p>These results suggest that mutant cells in CH(+) BM may exert non-cell autonomous effects on WT cells. Alongside aged BM environments, these effects may contribute to the positive selection of CH clones, playing a pivotal role in the pathogenesis of CH.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100435"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000256/pdfft?md5=a2255fb0a12b165edb092ce07c602d42&pid=1-s2.0-S2213048924000256-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103601","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
SINGLE-CENTRE PERSPECTIVE ON ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR PAEDIATRIC MYELODYSPLASTIC SYNDROME IN THE DEVELOPING WORLD 发展中世界儿童骨髓增生异常综合征异基因造血干细胞移植的单中心视角
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100449
R. Khandelwal, S. Arora, S.P. Yadav
{"title":"SINGLE-CENTRE PERSPECTIVE ON ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR PAEDIATRIC MYELODYSPLASTIC SYNDROME IN THE DEVELOPING WORLD","authors":"R. Khandelwal,&nbsp;S. Arora,&nbsp;S.P. Yadav","doi":"10.1016/j.lrr.2024.100449","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100449","url":null,"abstract":"<div><h3>Introduction</h3><p>Pediatric Myelodysplastic Syndrome (MDS) presents complex challenges, often requiring Allogeneic Hematopoietic Stem Cell Transplantation (HSCT). This study explores the clinical profile and demographics of seven pediatric MDS patients post-HSCT, shedding light on causes, molecular abnormalities, and patient diversity.</p></div><div><h3>Methods</h3><p>Retrospective data from 2014 to 2023 were gathered from the Pediatric Hematology Oncology &amp; Bone Marrow Transplantation Unit at the Cancer Institute in North India.</p></div><div><h3>Results</h3><p><strong>Patient Demographics:</strong> Seven pediatric patients (4 males, 3 females), median age 12, underwent HSCT (Five matched related donor HSCT and two haploidentical). <strong>Underlying Causes of MDS:</strong> Varied etiological factors identified, including Fanconi anemia (n=2), Emberger syndrome (n=1), therapy-related (n=1), GATA-2 insufficiency (n=1), and de novo cases in 2 patients, highlighting population heterogeneity. <strong>Molecular Abnormalities:</strong> Analysis revealed Monosomy-7 in two patients, NPM-1 in one, and GATA-2 mutation in two cases, providing crucial insights into the genetic landscape of MDS in this specific patient group. <strong>Post-transplant Outcome</strong>: Successful engraftment for all, with median neutrophil engraftment at 15.5 days and platelet engraftment at 13.5 days. GVHD and viral reactivation observed, yet 85.7% survived with complete donor chimerism. Unfortunately, one FA patient succumbed on day +72 due to E.coli sepsis with grade-IV GVHD. Mean follow-up is 33.5 months (2.7 years), ranging from 2.5 to 60 months.</p></div><div><h3>Conclusions</h3><p>HSCT emerges as an effective therapy for pediatric MDS patients, yielding promising results in this developing world context.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100449"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000396/pdfft?md5=39272a82a1ebca6b69548d406f8f4e5e&pid=1-s2.0-S2213048924000396-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103877","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
Efficacy and safety of different chemotherapy regimens combined with thalidomide in the treatment of diagnosed HIV-associated diffuse large B-cell lymphoma 不同化疗方案联合沙利度胺治疗确诊的艾滋病相关弥漫大B细胞淋巴瘤的疗效和安全性
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100450
Peng fei Tao, Chuan Qian, Qi wen zhou, Sen Lin, Dan qing Wang, Xi Wang, Shi fen Chen, Hai yan Min
{"title":"Efficacy and safety of different chemotherapy regimens combined with thalidomide in the treatment of diagnosed HIV-associated diffuse large B-cell lymphoma","authors":"Peng fei Tao,&nbsp;Chuan Qian,&nbsp;Qi wen zhou,&nbsp;Sen Lin,&nbsp;Dan qing Wang,&nbsp;Xi Wang,&nbsp;Shi fen Chen,&nbsp;Hai yan Min","doi":"10.1016/j.lrr.2024.100450","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100450","url":null,"abstract":"<div><h3>Objective</h3><p>To investigate the short-term efficacy and safety of different chemotherapy regimens combined with thalidomide, in the treatment of low-income patients with newly diagnosed HIV-associated diffuse large B-cell lymphoma.</p></div><div><h3>Methods</h3><p>A retrospective analysis was performed on 42 patients with HIV-DLBCL who were admitted to the Infectious Diseases Department of Yunnan Provincial Infectious Diseases Hospital from January 2018 to December 2020. 14 cases (including 1 case in stage II and 13 cases in stage III/IV) were treated with R-CHOP, 24 cases (including 1 case in stage II and 23 cases in stage III/IV) were treated with R-DAEPOCH, and 4 cases (including 1 case in stage II and 3 cases in stage III/IV) were treated with EPOCH. All patients were treated with thalidomide. The ART regimen was adjusted. At least 1 and up to 6 intrathecal injections were given during chemotherapy, and cotrimoxazole was taken orally to prevent infection. The clinical efficacy was evaluated after 4 cycles of chemotherapy, and adverse events were evaluated at each cycle of chemotherapy.</p></div><div><h3>Results</h3><p>All patients received 1–8 cycles of chemotherapy. CR (64.2 %) was achieved in 9 patients in R-CHOP group, and 5 patients died. In the R-DAEPOCH group, 17 patients achieved CR (70.8 %) and 7 died. In the EPOCH group, 2 patients reached CR (50 %) and 2 died. The main adverse reactions were grade II and above myelosuppression.</p></div><div><h3>Conclusion</h3><p>Combined treatment with thalidomide can improve the prognosis of low-income patients with newly diagnosed HIV-DLBCL.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100450"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000402/pdfft?md5=939dd388c2eea78951775be803e597f5&pid=1-s2.0-S2213048924000402-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140122079","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
A rare case of Philadelphia-positive (P210BCR-ABL1) T-cell acute lymphoblastic leukemia/lymphoma associated with minimal residual disease persistence after intensive chemotherapeutic approaches 一例罕见的费城阳性(P210BCR-ABL1)T 细胞急性淋巴细胞白血病/淋巴瘤病例,强化化疗后仍有极小残留病灶存在
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100456
Shruti Shah , Rupayan Kundu , Rahul Mishra , Sudipto Mukherjee , Abhay Singh
{"title":"A rare case of Philadelphia-positive (P210BCR-ABL1) T-cell acute lymphoblastic leukemia/lymphoma associated with minimal residual disease persistence after intensive chemotherapeutic approaches","authors":"Shruti Shah ,&nbsp;Rupayan Kundu ,&nbsp;Rahul Mishra ,&nbsp;Sudipto Mukherjee ,&nbsp;Abhay Singh","doi":"10.1016/j.lrr.2024.100456","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100456","url":null,"abstract":"<div><p>T-cell acute lymphoblastic leukemia/lymphoma (T-ALL/LBL) is a rare and aggressive leukemia. Philadelphia chromosome-positive cytogenetic abnormality is most common in CML. It is difficult to differentiate between de novo Ph+ T-ALL/LBL and T-cell lymphoblastic crises of CML. We present a case of adult Ph+ T-ALL/LBL with a likely history of antecedent CML. Initially thought to be a case of chronic-phase CML, a diagnostic quandary led to the pursuit of a lymph node biopsy that established the diagnosis of Ph+ T-LBL or T lymphoblastic blast crisis of CML, a clinical presentation extremely rare and only the second of its kind from our review of the literature. The patient was treated with an intensive chemotherapy regimen for over a year due to persistent minimal residual disease (MRD) positivity indicating aggressive disease.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100456"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000463/pdfft?md5=0d958dcf58cf22d79c23839ac45e5d35&pid=1-s2.0-S2213048924000463-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140327641","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
IGLL5 controlled by super-enhancer affects cell survival and MYC expression in mature B-cell lymphoma 由超级增强子控制的 IGLL5 影响成熟 B 细胞淋巴瘤中细胞的存活和 MYC 的表达
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100451
Hiroki Hosoi , Shotaro Tabata , Hideki Kosako , Yoshikazu Hori , Tadashi Okamura , Yusuke Yamashita , Kota Fujimoto , Daiki Kajioka , Kentaro Suzuki , Motomi Osato , Gen Yamada , Takashi Sonoki
{"title":"IGLL5 controlled by super-enhancer affects cell survival and MYC expression in mature B-cell lymphoma","authors":"Hiroki Hosoi ,&nbsp;Shotaro Tabata ,&nbsp;Hideki Kosako ,&nbsp;Yoshikazu Hori ,&nbsp;Tadashi Okamura ,&nbsp;Yusuke Yamashita ,&nbsp;Kota Fujimoto ,&nbsp;Daiki Kajioka ,&nbsp;Kentaro Suzuki ,&nbsp;Motomi Osato ,&nbsp;Gen Yamada ,&nbsp;Takashi Sonoki","doi":"10.1016/j.lrr.2024.100451","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100451","url":null,"abstract":"<div><p><em>IGLL5</em> is shown to be located near super-enhancer (SE) in B-cell tumors, and this gene is frequently mutated and a target of translocation in B-cell tumors. These results suggest roles of the <em>IGLL5</em> in tumorigenesis; however, its functional properties have been unclear. We found that two mature B-cell lymphoma cell lines expressed <em>IGLL5</em> mRNA with <em>Cλ1</em> segment. JQ1 treatment resulted in down-expression of <em>IGLL5</em>, indicating that <em>IGLL5</em> is controlled by SE. <em>IGLL5</em> knockdown induced cell death with down-expression of <em>MYC</em>. Our results suggested that <em>IGLL5</em> might have a role in survival of mature B-cell tumors and involvement in <em>MYC</em> expression. (100 words)</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100451"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000414/pdfft?md5=1e4f34f4af678dbfb8cd62fea439eaed&pid=1-s2.0-S2213048924000414-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139999116","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
Very late relapse of Burkitt's lymphoma in an EBV-negative patient after 20 years of complete remission 一名 EBV 阴性患者的伯基特淋巴瘤在完全缓解 20 年后极晚期复发
IF 0.7
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100470
Kmar Mrad , Nader Slama , Nouha Ben Abdeljalil , Zaineb Mlayah , Wiem Boufrikha , Abdelfattah Zakhama , Sarra Boukhris , Mohamed Adnene Laatiri
{"title":"Very late relapse of Burkitt's lymphoma in an EBV-negative patient after 20 years of complete remission","authors":"Kmar Mrad ,&nbsp;Nader Slama ,&nbsp;Nouha Ben Abdeljalil ,&nbsp;Zaineb Mlayah ,&nbsp;Wiem Boufrikha ,&nbsp;Abdelfattah Zakhama ,&nbsp;Sarra Boukhris ,&nbsp;Mohamed Adnene Laatiri","doi":"10.1016/j.lrr.2024.100470","DOIUrl":"10.1016/j.lrr.2024.100470","url":null,"abstract":"<div><p>Burkitt's lymphoma (BL) is an aggressive B-cell lymphoma that occurs in children and adults. It is a chemosensitive lymphoma with very exceptional cases of late relapse.</p><p>We report the case of a 32-year-old male, originally from a nonendemic area for BL, who was successfully treated for abdominal BL 20 years ago. He described a two-month history of cervical swelling and a one-week history of dyspnea. Physical examination was unremarkable except for a left submandibular mass that extended to the collarbone. An ultrasound of the neck revealed cervical lymphadenopathy. The patient was submitted to a lymph node biopsy with an immunohistochemical analysis, which concluded to the diagnosis of BL. Screening for recent Epstein-Barr-Virus (EBV) infection was negative. We considered this a very late relapse (VLR) of the original disease, and the patient was treated according to the same initial protocol. Unfortunately, he suffered a second relapse and died.</p><p>We report an unusual case of a VLR of nonendemic BL in an EBV-negative patient, occurring 20 years after achieving complete remission following the initial chemotherapy.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100470"},"PeriodicalIF":0.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000608/pdfft?md5=0286b3a4f369b332834a1bb0a8c7febe&pid=1-s2.0-S2213048924000608-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141951265","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
Avatrombopag for severe refractory thrombocytopenia in a pediatric patient with ALL following allogeneic hematopoietic stem cell transplantation: A case report 阿伐曲波帕治疗一名异基因造血干细胞移植后患有 ALL 的儿童患者的严重难治性血小板减少症:病例报告
IF 0.7
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100472
Emilie J. Lynch, Autumn Citta, Constance Alford, John A. Ligon, Mansi Dalal, Paul Castillo, Biljana Horn, Natalie Dotson, Giselle Moore-Higgs, Jordan Milner
{"title":"Avatrombopag for severe refractory thrombocytopenia in a pediatric patient with ALL following allogeneic hematopoietic stem cell transplantation: A case report","authors":"Emilie J. Lynch,&nbsp;Autumn Citta,&nbsp;Constance Alford,&nbsp;John A. Ligon,&nbsp;Mansi Dalal,&nbsp;Paul Castillo,&nbsp;Biljana Horn,&nbsp;Natalie Dotson,&nbsp;Giselle Moore-Higgs,&nbsp;Jordan Milner","doi":"10.1016/j.lrr.2024.100472","DOIUrl":"10.1016/j.lrr.2024.100472","url":null,"abstract":"<div><p>Patients who receive allogeneic hematopoietic stem cell transplantation (alloHSCT) are at risk for developing persistent thrombocytopenia. Here, we describe treatment with avatrombopag, a thrombopoietin receptor agonist, in a pediatric patient with chronic, severe, transfusion-dependent thrombocytopenia (&lt;10 × 10<sup>3</sup>/µL) post-alloHSCT that was persistent despite treatment with romiplostim, another thrombopoietin receptor agonist. Following the granting of a compassionate use investigational new drug authorization, avatrombopag treatment was initiated, and the patient's platelet count increased. To date, the patient has maintained a platelet count &gt;100 × 10<sup>3</sup>/µL. No adverse events or medication toxicities have been reported, and he has resumed his pre-alloHSCT activities.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100472"},"PeriodicalIF":0.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000621/pdfft?md5=3bf11fb769b63e12128df91f0b55035b&pid=1-s2.0-S2213048924000621-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141839903","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
Cup-like nuclei in adult B-cell acute lymphoblastic leukemia with the translocation (4;11)(q21;q23) 伴有 (4;11)(q21;q23) 易位的成人 B 细胞急性淋巴细胞白血病中的杯状核
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100463
Yuyang Lu, Xinran Feng, Fengyu Chen
{"title":"Cup-like nuclei in adult B-cell acute lymphoblastic leukemia with the translocation (4;11)(q21;q23)","authors":"Yuyang Lu,&nbsp;Xinran Feng,&nbsp;Fengyu Chen","doi":"10.1016/j.lrr.2024.100463","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100463","url":null,"abstract":"<div><p>Cuplike Nuclei(CLN) cells, particularly rare in Acute Lymphoblastic Leukemia(ALL), have been documented in only few cases to date. A recent study has revealed a correlation between CLN and IKZF1 deletions in pediatric B-ALL. This study introduces a case of CLN in adult B-ALL with a translocation (4;11)(q21;q23), while discussing the current relevant literature on the subject. Through this examination, several unique characteristics of CLN cells in both ALL and Acute Myeloid Leukemia are highlighted. It is essential to accumulate further data to confirm these distinctive traits, and investigate the potential association between CLN and cytogenetic/molecular abnormalities.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100463"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000530/pdfft?md5=5858aaae09370ebe47b10a263fd4f1b3&pid=1-s2.0-S2213048924000530-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141084644","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
FIRST SINGLE-CENTER EXPERIENCE WITH LUSPATERCEPT THERAPY IN LOW-RISK MYELODYSPLASTIC SYNDROME (LR-MDS) PATIENTS WITH TRANSFUSION DEPENDENCE REFRACTORY TO ERYTHROPOIETIN THERAPY 对红细胞生成素治疗难治的输血依赖性低风险骨髓增生异常综合征(LR-MDS)患者使用 Luspatercept 治疗的首次单中心经验
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100444
A. Jonasova , L. Minarik
{"title":"FIRST SINGLE-CENTER EXPERIENCE WITH LUSPATERCEPT THERAPY IN LOW-RISK MYELODYSPLASTIC SYNDROME (LR-MDS) PATIENTS WITH TRANSFUSION DEPENDENCE REFRACTORY TO ERYTHROPOIETIN THERAPY","authors":"A. Jonasova ,&nbsp;L. Minarik","doi":"10.1016/j.lrr.2024.100444","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100444","url":null,"abstract":"<div><h3>Introduction</h3><p>Luspatercept is a recent breakthrough in the therapy of anemia in low-risk MDS.</p></div><div><h3>Methods</h3><p>From January 2021 to October 2023, 44 patients (median age 77, M/F 25/19, WHO 2016 classification: MDS-RS-MLD 28, MDS-MLD -4, RARS-T 8, CMML- 0 2, 5q- + RS 2, IPSS-R: very low 2, low 33, Intermediate 9, IPSS-M (35 pts): very low + low 18, moderate low 11, moderate high 2, high 2, very high 2) were treated with luspatercept. Median follow-up was 13 months (range 1-42). The median number of cycles was 15 (2-42). Transfusion dependency (TD) before luspatercept initiation ranged from 2 transfusion units (TU) to 12 TU/8 weeks. All patients were tested for <em>SF3B1</em> mutation.</p></div><div><h3>Results</h3><p>We evaluated 42 patients. Twenty-four (57 %) patients reached TI (&gt;12weekes), 6 (14 %) patients have had a reduction in transfusion need (HI, according to IWG criteria 2006). There were differences in response according to transfusion burden. Significant more responders belonged to lower IPSS-R, IPSS-M categories. In 17 patients, we added ESA (± <u>prednisone)</u>, which led to the improvement of response in 12 cases with 9 TI. Four patients died (2-disease progression, 2 for comorbidity). There were no adverse effects of Grade II or more.</p></div><div><h3>Conclusions</h3><p>We did observed better responses in patients bearing single mutation in <em>SF3B1</em>, in lower IPSS-R and IPSS-M risk categories, patients with LTB and lower initial baseline EPO levels. The higher response rate in our follow-up may be influenced by the combination with ESA and rapid dose escalation.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100444"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000347/pdfft?md5=1b7441e2a9653431cdf33e47754dc6b8&pid=1-s2.0-S2213048924000347-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103893","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
Diagnostic challenges in classification of plasmacytoid dendritic cell proliferation associated with acute myeloid leukemia in the context of secondary-type mutations 与急性髓性白血病相关的浆细胞树突状细胞增殖在次生型突变背景下的分类诊断难题
IF 0.7
Leukemia Research Reports Pub Date : 2024-01-01 DOI: 10.1016/j.lrr.2024.100480
Iran Rashedi , Yasmeen Abulkhair , Eric Diehl , Hong Chang
{"title":"Diagnostic challenges in classification of plasmacytoid dendritic cell proliferation associated with acute myeloid leukemia in the context of secondary-type mutations","authors":"Iran Rashedi ,&nbsp;Yasmeen Abulkhair ,&nbsp;Eric Diehl ,&nbsp;Hong Chang","doi":"10.1016/j.lrr.2024.100480","DOIUrl":"10.1016/j.lrr.2024.100480","url":null,"abstract":"","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"22 ","pages":"Article 100480"},"PeriodicalIF":0.7,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000700/pdfft?md5=3172dfc2a3e647cdf8499ed2843b484b&pid=1-s2.0-S2213048924000700-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142271782","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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