Leukemia Research Reports最新文献

{"title":"MITOCHONDRIAL FRAGMENTATION IS A NEW THERAPEUTIC TARGET AND DIAGNOSTIC CRITERIA FOR MYELODYSPLASTIC SYNDROMES.","authors":"Y. Kamimura-Aoyagi ,&nbsp;Y. Harada ,&nbsp;K. Nomura ,&nbsp;N. Matsunuma ,&nbsp;K. Yuki ,&nbsp;H. Kobayashi ,&nbsp;Y. Hayashi ,&nbsp;A. Hijikata ,&nbsp;H. Harada","doi":"10.1016/j.lrr.2024.100424","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100424","url":null,"abstract":"<div><h3>Introduction</h3><p>Myelodysplastic syndromes (MDS) are characterized by persistent of cytopenia, gene abnormalities, and dysplasia. Although the correlation between chronic inflammation and ineffective hematopoiesis is demonstrated in forming MDS pathogenesis, the detailed mechanisms remain unclear.</p></div><div><h3>Methods</h3><p>Recently, we established a new MDS with low blasts (MDS-LB) model (<em>CBL^ΔE8/9-RUNX1^S291fs</em> mice) , which recapitulates MDS-LB pathogenesis such as pancytopenia and chronic inflammation, by introducing these two mutations into murine hematopoietic stem and progenitor cells (HSC/Ps) .</p></div><div><h3>Results</h3><p>MDS model mice exhibited excessive mitochondrial fragmentation due to Drp1 activation in HSC/Ps. Importantly, pharmacological inhibition of mitochondrial fragmentation rescued leukocytopenia and dysplasia formation in MDS mice by suppressing inflammatory signaling activation, suggesting that mitochondrial fragmentation could be a new therapeutic target of MDS. Given that mitochondrial fragmentation is related to MDS pathogenesis, we hypothesized that mitochondrial fragmentation can be used for morphological diagnosis of MDS. Differential diagnosis of patients with non-MDS cytopenia and MDS-LB has been challenging. We assessed mitochondrial morphology in bone marrow samples from 10 healthy individuals and 141 patients before disease-modifying therapy. The percentage of cells with mitochondrial fragmentation was significantly increased in patients with MDS-LB, compared with that in patients with cytopenia without dysplasia and gene abnormality (mean 50.7% vs 22.4%, P&lt;0.001, cutoff value 30.8%). The calculated cutoff value clearly distinguishes patients with MDS-LB and non-MDS cytopenia.</p></div><div><h3>Conclusions</h3><p>These data suggest that mitochondrial fragmentation can be not only a new therapeutic target of MDS-LB but also one category of dysplasia that can diagnose MDS-LB.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100424"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000141/pdfft?md5=eaf437a90e97a820f57941330d565a3f&pid=1-s2.0-S2213048924000141-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103289","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}

{"title":"KINOME EXPRESSION PROFILING FOR RISK STRATIFICATION OF MYELODYSPLASTIC SYNDROMES","authors":"C.-Y. Yao ,&nbsp;C.-C. Lin ,&nbsp;Y.-H. Wang ,&nbsp;C.-J. Kao ,&nbsp;C.-H. Tsai ,&nbsp;H.-A. Hou ,&nbsp;H.-F. Tien ,&nbsp;C.-L. Hsu ,&nbsp;W.-C. Chou","doi":"10.1016/j.lrr.2024.100433","DOIUrl":"https://doi.org/10.1016/j.lrr.2024.100433","url":null,"abstract":"<div><h3>Introduction</h3><p>Myelodysplastic syndrome (MDS) is a heterogeneous constellation of myeloid neoplasms originating from the clonal proliferation of aberrant hematopoietic stem cells (HSC). The human kinome, which comprises over five hundred kinases, plays a critical role in regulating numerous cellular functions. Although the dysregulation of kinases has been observed in various human cancers, the characterization and clinical implications of kinase expressions in MDS have not been investigated before.</p></div><div><h3>Methods</h3><p>Overall, 341 patients diagnosed with primary MDS according to the 2016 WHO classification, who had adequate cryopreserved diagnostic unsorted bone marrow (BM) samples for DNA and RNA sequencing, were recruited. The normalized gene expressions of a total of 517 kinase gene were studied. We first identified those kinases whose expressions were higher in MDS patients than in healthy controls, and then used LASSO-regularized Cox proportional hazards regression to identify prognostically significant kinases to construct the KInase Stratification Score (KISS).</p></div><div><h3>Results</h3><p>We discovered that the expression levels of seven kinases (<em>PTK7, KIT, MAST4, NTRK1, PAK6, CAMK1D, PRKCZ</em>) could predict patient outcome, and we used these kinases to construct the KISS; we further validated its prognostic significance in two external MDS cohorts. A higher KISS was associated with older age, higher BM blast percentage, higher IPSS-R risk, complex karyotype, and mutations in several adverse-risk genes in MDS. In the multivariate analysis, a higher KISS was proved to be an independent unfavorable risk factor.</p></div><div><h3>Conclusions</h3><p>Altogether, our findings suggest that KISS holds the potential to improve the current prognostic scheme of MDS, and inform novel therapeutic opportunities.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100433"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000232/pdfft?md5=c610a93c5930abf41034806f87be75a7&pid=1-s2.0-S2213048924000232-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140103599","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}

{"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}

{"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}

{"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}

{"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}

{"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}

{"title":"Acute leukemia with KMT2A rearrangement: A master of disguise","authors":"Sawyer J. Bawek ,&nbsp;Eunice S. Wang ,&nbsp;Steven D. Green","doi":"10.1016/j.lrr.2024.100464","DOIUrl":"10.1016/j.lrr.2024.100464","url":null,"abstract":"<div><p>Mixed-phenotype acute leukemia (MPAL) is a rare form of leukemia with ambiguous lineage, and there are challenges in accurately diagnosing this entity according to formal criteria. Here we report a case which was initially diagnosed as “AML” based on atypical peripheral blood flow cytometry that was subsequently determined to be B-ALL with <em>KMT2A</em> rearrangement based on marrow results. Although <em>KMT2A</em> rearrangements represent a defining genetic abnormality for acute leukemia of ambiguous lineage, this case did not meet the criteria for MPAL based on WHO 2022 criteria. This case highlights the diagnostic challenges of MPAL and the potential limitations of the current classification. We discuss the most appropriate workup and management of these patients and identify areas for future study.</p></div>","PeriodicalId":38435,"journal":{"name":"Leukemia Research Reports","volume":"21 ","pages":"Article 100464"},"PeriodicalIF":0.0,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2213048924000542/pdfft?md5=f23917a73bb86c9fd905a42e6e9f90f9&pid=1-s2.0-S2213048924000542-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141132466","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}

{"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}

{"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 × 10³/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 × 10³/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}