Leukemia research最新文献

{"title":"Donor Lymphocyte Infusion (DLI) post allogeneic stem cell transplant (allo-SCT) in Acute Myeloid Leukemia (AML) and High-Grade Myelodysplastic Syndrome (MDS). A longitudinal retrospective study using peripheral blood (PB) CD34+ and CD3+ donor chimerism (DC) monitoring","authors":"Tishya Indran ,&nbsp;Tongted Das ,&nbsp;Jenny Muirhead ,&nbsp;Maureen O’Brien ,&nbsp;Michael I. Swain ,&nbsp;Bianca Cirone ,&nbsp;Jaqueline Widjaja ,&nbsp;Sushrut Patil ,&nbsp;David J. Curtis","doi":"10.1016/j.leukres.2024.107504","DOIUrl":"10.1016/j.leukres.2024.107504","url":null,"abstract":"<div><h3>Introduction</h3><p>This longitudinal study was based on the outcomes of Donor Lymphocyte Infusion (DLI) for falling peripheral blood (PB) CD34⁺ and CD3⁺ donor chimerism (DC).</p></div><div><h3>Methods</h3><p>From 2012 to 2018, data was collected from the BMT database and electronic medical records (EMR). The primary objective was to compare the indication for DLI based on falling PB CD34⁺ or CD3⁺ DC in patients post allo-SCT for AML and MDS and their overall survival (OS).</p></div><div><h3>Results</h3><p>18/70 patients met the inclusion criteria. Indications for DLI were i) falling PB CD34⁺ DC ≤ 80 % with morphological relapse, ii) falling PB CD34⁺ DC ≤ 80 % without morphological relapse and iii) falling PB CD3⁺ DC ≤ 80 % without falling PB CD34⁺ DC. Log rank analysis showed falling PB CD34⁺ DC and morphological relapse had significantly lower OS. Linear regression demonstrated better OS post DLI if there was PB CD34⁺ and CD3⁺ chimerism response at 30 days (p = 0.029), GVHD (p = 0.032) and tapering immunosuppression at the time of falling DC (p = 0.042).</p></div><div><h3>Conclusion</h3><p>DLI for PB CD34⁺ DC values ≤ 80 % and morphological relapse had the lowest OS. In this study, full DC was achieved after DLI even with a PB CD3⁺DC value as low as 13 %, provided the PB CD34⁺ DC remained &gt; 80 %. Further research is vital in CD34⁺ DC as a biomarker for disease relapse and loss of engraftment.</p></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"142 ","pages":"Article 107504"},"PeriodicalIF":2.7,"publicationDate":"2024-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140778602","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real world outcome of B ALL with t (1; 19) (q23; p13)/TCF3::PBX1 in adolescents and adults treated with intensive regimes","authors":"Tribikram Panda,&nbsp;Sujay Rainchwar,&nbsp;Reema Singh,&nbsp;Aakanksha Singh,&nbsp;Mayank Soni,&nbsp;Disha Kakkar,&nbsp;KR Jegan,&nbsp;Reshmi Harikumar Pillai,&nbsp;Roy J. Palatty,&nbsp;Karuna Jha,&nbsp;Rayaz Ahmed,&nbsp;Rohan Halder,&nbsp;Narender Tejwani,&nbsp;Devasis Panda,&nbsp;Dinesh Bhurani,&nbsp;Narendra Agrawal","doi":"10.1016/j.leukres.2024.107506","DOIUrl":"https://doi.org/10.1016/j.leukres.2024.107506","url":null,"abstract":"<div><p>Significant heterogeneity has been reported in outcome of Acute lymphoblastic leukemia with t(1;19)(q23;p13)/TCF3::PBX1 in adolescents and adults leading to a lack of consensus on precise risk stratification. We evaluated clinical outcome of 17 adult ALL cases (≥15 years) with this genotype treated on intensive regimes.13/17 received COG0232 and 4/17 cases received UK-ALL protocol. All achieved CR (100%) with above treatment. End of induction MRD was evaluated in 14/17 cases of which 11 (78.5%) achieved MRD negativity. Total nine patients relapsed (7 marrows, 2 CNS). Overall survival at 2 years was 53.3%. The 2 year estimated PFS was 42.9%. The 2 years CIR was 54.2%. Adults with this genotype perform poorly despite early favorable response. Incorporation of novel immunotherapies and prompt HSCT should be strongly considered with this genotype. Targeted NGS panels for additional genetic aberrations can further help in risk stratifying and guiding therapy for this genotype.</p></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"141 ","pages":"Article 107506"},"PeriodicalIF":2.7,"publicationDate":"2024-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140644666","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A systematic review of second-generation FLT3 inhibitors for treatment of patients with relapsed/refractory acute myeloid leukemia","authors":"Alireza Mohebbi ,&nbsp;Fahimeh Shahriyary ,&nbsp;Vida Farrokhi ,&nbsp;Bita Bandar ,&nbsp;Najmaldin Saki","doi":"10.1016/j.leukres.2024.107505","DOIUrl":"10.1016/j.leukres.2024.107505","url":null,"abstract":"<div><h3>Background</h3><p>Acute myeloid leukemia (AML) is a complex disease with diverse mutations, including prevalent mutations in the FMS-like receptor tyrosine kinase 3 (FLT3) gene that lead to poor prognosis. Recent advancements have introduced FLT3 inhibitors that have improved outcomes for FLT3-mutated AML patients, however, questions remain on their application in complex conditions such as relapsed/refractory (R/R) disease. Therefore, we aimed to evaluate the clinical effectiveness of second-generation FLT3 inhibitors in treating patients with R/R AML.</p></div><div><h3>Methods</h3><p>A systematic literature search of PubMed, MEDLINE, SCOPUS and Google Scholar databases was made to identify relevant studies up to January 30, 2024. This study was conducted following the guidelines of the PRISMA.</p></div><div><h3>Results</h3><p>The ADMIRAL trial revealed significantly improved overall survival and complete remission rates with gilteritinib compared to salvage chemotherapy, with manageable adverse effects. Ongoing research explores its potential in combination therapies, showing synergistic effects with venetoclax and promising outcomes in various clinical trials. The QuANTUM-R trial suggested longer overall survival with quizartinib compared to standard chemotherapy, although concerns were raised regarding trial design and cardiotoxicity. Ongoing research explores combination therapies involving quizartinib, such as doublet or triplet regimens with venetoclax, showing promising outcomes in FLT3-mutated AML patients.</p></div><div><h3>Conclusion</h3><p>These targeted therapies offer promise for managing this subgroup of AML patients, but further research is needed to optimize their use. This study underscores the importance of personalized treatment based on genetic mutations in AML, paving the way for more effective and tailored approaches to combat the disease.</p></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"141 ","pages":"Article 107505"},"PeriodicalIF":2.7,"publicationDate":"2024-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140776496","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prediction of deep molecular response in chronic myeloid leukemia using supervised machine learning models","authors":"Zahra Zad ,&nbsp;Simone Bonecker ,&nbsp;Taiyao Wang ,&nbsp;Ilana Zalcberg ,&nbsp;Gustavo T. Stelzer ,&nbsp;Bruna Sabioni ,&nbsp;Luciana Mayumi Gutiyama ,&nbsp;Julia L. Fleck ,&nbsp;Ioannis Ch. Paschalidis","doi":"10.1016/j.leukres.2024.107502","DOIUrl":"https://doi.org/10.1016/j.leukres.2024.107502","url":null,"abstract":"","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"141 ","pages":"Article 107502"},"PeriodicalIF":2.7,"publicationDate":"2024-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140558679","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Racial and ethnic disparities in Acute Myeloid Leukemia: 15-year experience at a safety net hospital system","authors":"Sharlene Dong ,&nbsp;Naveen Premnath ,&nbsp;Navid Sadeghi ,&nbsp;Radhika Kainthla ,&nbsp;Stephen S. Chung ,&nbsp;Robert H. Collins ,&nbsp;Hsiao C. Li ,&nbsp;Yazan F. Madanat","doi":"10.1016/j.leukres.2024.107503","DOIUrl":"https://doi.org/10.1016/j.leukres.2024.107503","url":null,"abstract":"<div><p>Despite recent therapeutic advances, ethnic minorities in the U.S. continue to have disproportionately poor outcomes in many hematologic malignancies including AML. We identified 162 adult AML patients treated at a non-transplant safety net hospital from 2007 to 2022 and evaluated differences in disease characteristics, treatment and clinical outcomes based on race and ethnicity. Our cohort consisted of 82 (50.6%) Hispanic, 36 (22.2%) non-Hispanic black and 44 (27.2%) non-Hispanic white and Asian patients. Median age at diagnosis was 42.5, 49.0 and 52.5 years respectively (p=0.025). Hispanics had higher rates of intermediate and high-risk disease (p=0.699) and received high intensity induction and consolidation chemotherapy at lower rates (p=0.962), although differences did not reach statistical significance. Despite this, similar remission rates were achieved. Hispanics with high-risk disease had longer overall survival (OS) than the combined non-Hispanic cohort (mOS 14 m vs 7 m, p=0.030). Multivariate regression analysis showed that OS was negatively associated with age (HR 1.023, p=0.006), intermediate (HR 3.431, p=0.0003) and high-risk disease (HR 4.689, p&lt;0.0001) and positively associated with Hispanic ethnicity (HR 0.614, p=0.026). This report suggests that contrary to other studies, Hispanics, particularly those with high-risk AML, may have improved OS compared to other ethnic groups. These results are unique to our safety net hospital setting where common barriers to medical care and healthcare disparities are largely mitigated.</p></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"141 ","pages":"Article 107503"},"PeriodicalIF":2.7,"publicationDate":"2024-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140650010","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Poor prognosis of SRSF2 gene mutations in patients treated with VEN-AZA for newly diagnosed acute myeloid leukemia","authors":"Guillaume Berton ,&nbsp;Bochra Sedaki ,&nbsp;Erwann Collomb ,&nbsp;Sami Benachour ,&nbsp;Michael Loschi ,&nbsp;Bilal Mohty ,&nbsp;Colombe Saillard ,&nbsp;Yosr Hicheri ,&nbsp;Camille Rouzaud ,&nbsp;Valerio Maisano ,&nbsp;Ferdinand Villetard ,&nbsp;Evelyne D.'Incan Corda ,&nbsp;Aude Charbonnier ,&nbsp;Jerome Rey ,&nbsp;Marie-Anne Hospital ,&nbsp;Antoine Ittel ,&nbsp;Norman Abbou ,&nbsp;Raphaelle Fanciullino ,&nbsp;Bérengère Dadone-Montaudié ,&nbsp;Norbert Vey ,&nbsp;Sylvain Garciaz","doi":"10.1016/j.leukres.2024.107500","DOIUrl":"https://doi.org/10.1016/j.leukres.2024.107500","url":null,"abstract":"<div><p>Mutations in spliceosome genes (<em>SRSF2</em>, <em>SF3B1</em>, <em>U2AF1</em>, <em>ZRSR2)</em> correlate with inferior outcomes in patients treated with intensive chemotherapy for Acute Myeloid Leukemia. However, their prognostic impact in patients treated with less intensive protocols is not well known. This study aimed to evaluate the impact of Spliceosome mutations in patients treated with Venetoclax and Azacitidine for newly diagnosed AML. 117 patients treated in 3 different hospitals were included in the analysis. 34 harbored a mutation in at least one of the spliceosome genes (splice-mut cohort). K/NRAS mutations were more frequent in the splice-mut cohort (47% vs 19%, p=0.0022). Response rates did not differ between splice-mut and splice-wt cohorts. With a median follow-up of 15 months, splice mutations were associated with a lower 18-month LFS (p=0.0045). When analyzing splice mutations separately, we found SRSF2 mutations to be associated with poorer outcomes (p=0.034 and p=0.037 for OS and LFS respectively). This negative prognostic impact remained true in our multivariate analysis. We believe this finding should warrant further studies aimed at overcoming this negative impact.</p></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"141 ","pages":"Article 107500"},"PeriodicalIF":2.7,"publicationDate":"2024-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S0145212624000663/pdfft?md5=4d4e114f1e8bcfb3607ca62b5c9babbc&pid=1-s2.0-S0145212624000663-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140558678","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Donor matters: Donor selection impact on hematopoietic stem cell transplantation outcomes in Hispanic patients with B-cell acute lymphocytic leukemia: Insights from a myeloablative HSCT study","authors":"Karam Ashouri ,&nbsp;Anush A. Ginosyan ,&nbsp;Mollee Chu ,&nbsp;Brian Hom ,&nbsp;Jennifer Hwang ,&nbsp;Karen Resnick ,&nbsp;Yekta Rahimi ,&nbsp;Preet Chaudhary ,&nbsp;Karrune Woan ,&nbsp;Imran Siddiqi ,&nbsp;Abdullah Ladha ,&nbsp;Amir Ali ,&nbsp;Eric Leon Tam ,&nbsp;George Yaghmour","doi":"10.1016/j.leukres.2024.107501","DOIUrl":"https://doi.org/10.1016/j.leukres.2024.107501","url":null,"abstract":"<div><h3>Background</h3><p>Hematopoietic stem cell transplantation (HSCT) is a pivotal treatment for high-risk acute lymphocytic leukemia (ALL), although limited by suitable human leukocyte antigen (HLA)-matched sibling donors (MSD). This study evaluates the impact of donor selection on outcomes in post-HSCT Hispanic B-cell ALL patients.</p></div><div><h3>Methodology</h3><p>This single-center retrospective study evaluates outcomes in 88 adult Hispanic B-cell ALL patients who underwent haploidentical, MSD, or MUD myeloablative HSCT between 2013 and 2023.</p></div><div><h3>Results</h3><p>Compared to Haploidentical transplants, MSD exhibited worse cumulative incidence of relapse (CIR) (HR = 3.39; P = 0.014) and disease-free survival (DFS) (HR = 2.44; P = 0.048) whereas MUD outcomes did not differ. This effect persisted even when controlling for pre-HSCT stage and Minimal residual disease (MRD) status. In addition, Ph-like was a significant predictor of worse DFS (HR = 3.60; P=0.014) and CIR (HR = 2.97; P=0.035) on multivariate analysis. Older donor age correlated with worse GVHD-free, relapse-free survival (GRFS) in haploidentical transplants (HR = 1.05; P=0.036).</p></div><div><h3>Conclusion</h3><p>Our data highlights improved outcomes with younger, haploidentical donors among Hispanic B-cell ALL patients undergoing myeloablative HSCT. This underscores the importance of donor selection in optimizing outcomes for ALL patients.</p></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"141 ","pages":"Article 107501"},"PeriodicalIF":2.7,"publicationDate":"2024-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140558677","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Alternative polyadenylation quantitative trait loci contribute to acute myeloid leukemia risk genes regulation","authors":"Xi Hu ,&nbsp;Panxiang Cao ,&nbsp;Fang Wang ,&nbsp;Tong Wang ,&nbsp;Junbo Duan ,&nbsp;Xue Chen ,&nbsp;Xiaoli Ma ,&nbsp;Yang Zhang ,&nbsp;Jiaqi Chen ,&nbsp;Hongxing Liu ,&nbsp;Huqin Zhang ,&nbsp;Xiaoming Wu","doi":"10.1016/j.leukres.2024.107499","DOIUrl":"https://doi.org/10.1016/j.leukres.2024.107499","url":null,"abstract":"<div><p>Acute myeloid leukemia (AML) is a hematopoietic malignancy with a high relapse rate and progressive drug resistance. Alternative polyadenylation (APA) contributes to post-transcriptional dysregulation, but little is known about the association between APA and AML. The APA quantitative trait locus (apaQTL) is a powerful method to investigate the relationship between APA and single nucleotide polymorphisms (SNPs). We quantified APA usage in 195 Chinese AML patients and identified 4922 cis-apaQTLs related to 1875 genes, most of which were newly reported. Cis-apaQTLs may modulate the APA selection of 115 genes through poly(A) signals. Colocalization analysis revealed that cis-apaQTLs colocalized with cis-eQTLs may regulate gene expression by affecting miRNA binding sites or RNA secondary structures. We discovered 207 cis-apaQTLs related to AML risk by comparing genotype frequency with the East Asian healthy controls from the 1000 Genomes Project. Genes with cis-apaQTLs were associated with hematological phenotypes and tumor incidence according to the PHARMGKB and MGI databases. Collectively, we profiled an atlas of cis-apaQTLs in Asian AML patients and found their association with APA selection, gene expression, AML risk, and complex traits. Cis-apaQTLs may provide insights into the regulatory mechanisms related to APA in AML occurrence, progression, and prognosis.</p></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"141 ","pages":"Article 107499"},"PeriodicalIF":2.7,"publicationDate":"2024-04-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140619983","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Safety and efficacy of chimeric antigen receptor T-cell therapy for acute myeloid leukemia: A subgroup based meta-analysis","authors":"Mahmoud M. Morsy ,&nbsp;Ahmed Y. Azzam ,&nbsp;Osman Elamin ,&nbsp;Adam Elswedy ,&nbsp;Abdulqadir J. Nashwan","doi":"10.1016/j.leukres.2024.107498","DOIUrl":"https://doi.org/10.1016/j.leukres.2024.107498","url":null,"abstract":"<div><h3>Introduction</h3><p>Acute myeloid leukemia (AML) is a significant hematological malignancy in the United States, with a high mortality rate and limited treatment options. CAR T-cell therapy, a new and promising treatment, is being investigated for its efficacy and safety in AML. This meta-analysis aims to assess the safety and efficacy of CAR T-cell therapy in AML, considering various subgroups such as study location, study design, prior transplantation status, conditioning regimen, and CAR T-cell source.</p></div><div><h3>Methods</h3><p>We conducted a comprehensive literature review across multiple databases, adhering to PRISMA guidelines and focusing on studies concerning CAR T-cell therapy in AML. We included original articles in English and excluded non-original reviews, abstracts, and non-English studies. The risk of bias was assessed using the Cochrane ROBINS-I tool. Statistical analysis involved meta-analysis with Cochrane’s Q-test and I² statistic, using both fixed-effect and random-effects models, and assessed for publication bias.</p></div><div><h3>Results</h3><p>Our search yielded studies encompassing 57 AML patients treated with CAR T-cell therapy. The meta-analysis revealed a 48% incidence of complete remission with CAR T-cell therapy, varying significantly across subgroups based on study design, location, prior transplantation, conditioning regimen, and CAR T-cell source. The highest complete remission rates were observed in patients from China, those who had undergone prior hematopoietic cell transplantation, and those treated with fludarabine and cyclophosphamide conditioning regimen. Adverse events included graft-versus-host disease (7%) and cytokine release syndrome (53%).</p></div><div><h3>Conclusions</h3><p>This meta-analysis highlights the potential of CAR T-cell therapy in AML treatment, especially when integrated with certain prior treatments and conditioning regimens. The findings suggest a higher efficacy in patients with previous hematopoietic cell transplantation and specific conditioning regimens. Further large-scale, randomized trials are essential to confirm these findings and establish CAR T-cell therapy as a standard treatment for AML.</p></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"140 ","pages":"Article 107498"},"PeriodicalIF":2.7,"publicationDate":"2024-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S014521262400064X/pdfft?md5=73e40562cdec7186190138a5405e3458&pid=1-s2.0-S014521262400064X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140351082","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Single agent vemurafenib or rituximab-vemurafenib combination for the treatment of relapsed/refractory hairy cell leukemia, a multicenter experience","authors":"Süreyya Yiğit Kaya ,&nbsp;Yaşa Gül Mutlu ,&nbsp;Ümit Yavuz Malkan ,&nbsp;Özgür Mehtap ,&nbsp;Fatma Keklik Karadağ ,&nbsp;Gülten Korkmaz ,&nbsp;Tuğrul Elverdi ,&nbsp;Güray Saydam ,&nbsp;Gülsüm Özet ,&nbsp;Muhlis Cem Ar ,&nbsp;Elif Melek ,&nbsp;Senem Maral ,&nbsp;Leylagül Kaynar ,&nbsp;Ömür Gökmen Sevindik","doi":"10.1016/j.leukres.2024.107495","DOIUrl":"10.1016/j.leukres.2024.107495","url":null,"abstract":"<div><h3>Background</h3><p>Hairy cell leukemia (HCL) is a rare mature B-cell malignancy that is primarily treated with purine analogues. However, relapse remains a significant challenge, prompting the search for alternative therapies. The BRAF V600E mutation prevalent in HCL patients provides a target for treatment with vemurafenib.</p></div><div><h3>Patients and methods</h3><p>This multicenter retrospective study included nine patients with relapsed/refractory (R/R) HCL from six different centers. Patient data included demographics, prior treatments, clinical outcomes, and adverse events.</p></div><div><h3>Results</h3><p>Patients received different treatment regimens between centers, including vemurafenib alone or in combination with rituximab. Despite the differences in protocols, all patients achieved at least a partial response, with seven patients achieving a complete response. Adverse events were generally mild with manageable side effects. The absence of myelotoxic effects and manageable side effects make BRAF inhibitors attractive, especially for patients ineligible for purine analogues or those with severe neutropenia.</p></div><div><h3>Conclusion</h3><p>Single agent vemurafenib or in combination with rituximab appears to be a promising therapeutic option for R/R HCL. Further research is needed to establish standardized treatment protocols and to investigate long-term outcomes.</p></div>","PeriodicalId":18051,"journal":{"name":"Leukemia research","volume":"140 ","pages":"Article 107495"},"PeriodicalIF":2.7,"publicationDate":"2024-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140399027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}