Clinical Lymphoma, Myeloma & Leukemia最新文献

{"title":"Transplanting Hope: Managing Relapsed/Refractory AML.","authors":"Christopher Ronald Funk, Edmund K Waller","doi":"10.1016/j.clml.2025.04.001","DOIUrl":"https://doi.org/10.1016/j.clml.2025.04.001","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) is a heterogeneous malignant disorder of myeloid precursor cells, with high relapse rates, particularly in patients who fail to achieve morphological remission after induction therapy. Allogeneic hematopoietic cell transplantation (allo-HCT) can induce durable remissions through the graft-versus-leukemia (GVL) effect, yet current approaches of allo-HSCT often fail, with relapse rates of ∼40% within 6 months post-transplant. Outcomes following allo-HCT are inversely proportional to leukemia burden at the time of transplant. Both morphological relapse (≥5% blasts in the marrow) or minimal residual disease (MRD) positivity predict significantly reduced overall survival rates. Emerging strategies to improve outcomes in patients with high leukemic burden include aggressive bridging therapies (encompassing intensive salvage chemotherapy, hypomethylating agents, targeted inhibitors, and sequential induction-conditioning approaches such as FLAMSA), tailored conditioning regimens, post-transplant maintenance therapy, and innovative graft engineering methods. Graft engineering strategies, such as ORCA-T, which engineers stem cell grafts with a defined ratio of T-regulatory cells to effector T cells, are particularly promising and under evaluation in phase III clinical trials. These approaches aim to improve upon the poor outcomes for patients with persistent/relapsed AML undergoing allo-HCT.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143979102","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":"Comparison of Real-World Outcomes in Patients With Follicular Lymphoma Treated With BR Versus RCHOP-Like Regimens.","authors":"Kelsey Baron, Eric Anto, John Esther, Victoria A Vardell, Lisa Pappas, Allison Bock, Daniel A Ermann, Lindsey A Fitzgerald, Boyu Hu, Harsh R Shah","doi":"10.1016/j.clml.2025.03.017","DOIUrl":"https://doi.org/10.1016/j.clml.2025.03.017","url":null,"abstract":"<p><strong>Background: </strong>Patients with follicular lymphoma (FL) grade (G) 1-3A who meet treatment criteria are commonly treated with chemo-immunotherapy regimens such as bendamustine plus rituximab (BR) or rituximab plus cyclophosphamide, doxorubicin, vincristine, prednisone (RCHOP). Two prospective trials have compared BR to RCHOP-like regimens and showed improved progression free survival with BR in FL G1-2 patients. However, in real-world practice, there is clinical variability in utilization of BR versus RCHOP-like regimens. Additionally, the optimal treatment for patients with FL G3A remains unclear.</p><p><strong>Methods: </strong>We used a nationwide electronic health record-derived de-identified database to compare outcomes in 2089 patients with FL G1-3A treated with frontline BR versus RCHOP-like regimens.</p><p><strong>Results: </strong>We demonstrated clinical improvement in time to next treatment or death (TTNTD) with BR (median 96 vs. 78 months, HR 1.15, 95% CI 0.986-1.332, P = .086), albeit not statistically significant. No difference in overall survival (OS) was observed. Maintenance rituximab was associated with improved TTNTD and OS. Among G3A patients (N = 304), TTNTD was comparable between the 2 regimens, however OS was significantly improved with RCHOP-like regimens in univariate analysis (median 138 vs. 96 months, HR 0.51, 95% CI 0.313-0.834, P = .007).</p><p><strong>Conclusions: </strong>In conclusion, this study of real-world patients supports the use of BR and rituximab maintenance for front-line treatment of FL. Further prospective studies are needed to determine the ideal treatment for FL G3A.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-04-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143967105","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":"SOHO State of the Art Updates and Next Questions. Atypical Chronic Myeloid Leukemia: Pathogenesis, Diagnostic Challenges, and Therapeutic Strategies.","authors":"Alessandro Costa, Massimo Breccia","doi":"10.1016/j.clml.2025.03.015","DOIUrl":"https://doi.org/10.1016/j.clml.2025.03.015","url":null,"abstract":"<p><p>Atypical chronic myeloid leukemia (aCML) is a rare and challenging clonal hematopoietic disorder within the myelodysplastic/myeloproliferative neoplasm (MDS/MPN) spectrum. Over the past two decades, substantial progress has been made in understanding the genetic mechanisms driving aCML, revealing a complex and heterogeneous mutational landscape. Key ancestral mutations, such as ASXL1 and ETNK1, have been identified, providing a foundation for the pathogenesis and for the possible emergence of secondary abnormalities, particularly in epigenetic regulation (eg, SETBP1), and in splicing process (eg, SRSF2). These molecular insights have been integrated into current diagnostic classifications, refining disease characterization and offering potential targets for precision therapies. Despite these advances, significant clinical challenges persist due to the disease's rarity and the lack of randomized clinical trials. Therapeutic strategies remain inadequately defined, with allogeneic stem cell transplantation being the only curative option. This review provides an overview of the molecular, clinical, and therapeutic information that may pave the way for essential advancements in the proper management of this disease.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143967979","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":"Clinicians' Perspectives and Methodological Application of Fluorescence in situ Hybridization (FISH) to Define Cytogenetic Risk in Multiple Myeloma: An Italian, Real-World, Survey-Based Report From the European Myeloma Network (EMN) Italy.","authors":"Lorenzo Cani, Maria Teresa Petrucci, Katia Mancuso, Renato Zambello, Laura Paris, Sara Aquino, Flavia Lotti, Francesco Vassallo, Norbert Pescosta, Micol Quaresima, Patrizia Tosi, Iolanda Donatella Vincelli, Sonia Ronconi, Nicola Giuliani, Francesco Pisani, Mario Luppi, Rita Rizzi, Rita Mazza, Delia Rota-Scalabrini, Claudia Cellini, Silvia Mangiacavalli, Giuseppe Pietrantuono, Maria Luisa Pioltelli, Antonietta Pia Falcone, Elisabetta Antonioli, Angelo Belotti, Sonia Morè, Benedetto Bruno, Mario Boccadoro, Mattia D'Agostino","doi":"10.1016/j.clml.2025.03.011","DOIUrl":"https://doi.org/10.1016/j.clml.2025.03.011","url":null,"abstract":"<p><strong>Background: </strong>Fluorescence in situ hybridization (FISH) is the standard technique for the prognostic detection of cytogenetic abnormalities (CA) in multiple myeloma (MM). In Italy, the application of practical guidelines for FISH testing in clinical studies and the degree of standardization of laboratory techniques are largely unknown.</p><p><strong>Methods: </strong>We conducted a survey from April to July 2023 among 70 MM-treating centers associated with the European Myeloma Network Italy and geographically well distributed across Italy. We aimed to record laboratory and clinicians' perspectives about FISH application in Italy, with a focus on 1q alterations.</p><p><strong>Results: </strong>FISH was widely accessible across the country, with 71% of centers performing it locally, while the remaining centers (predominantly those with <30 newly diagnosed MM cases/year) sent samples to external laboratories. Variability in laboratory techniques, such as CD138⁺ cell purification and CA detection thresholds, was observed among centers. The centers analyzed del(17p) (100%), t(4;14) (100%), t(14;16) (98%), 1q+ (96%, with 70% distinguishing between gain and amplification), t(11;14) (90%), del(1p32) (88%), del(13q) (68%), and hyperdiploidy (52%). FISH emerged as a crucial prognostic technique, since 94% of centers used the Revised International Staging System (R-ISS) at diagnosis, and 69% implemented the recent R2-ISS. Most centers performed FISH at diagnosis in all patients, while others did not routinely perform FISH in some categories of patients (e.g., aged >80 years). At relapse, 53% of centers routinely repeated FISH testing, 9% did not, while others repeated it selectively.</p><p><strong>Conclusions: </strong>This overview of FISH use in Italy provides a basis for future standardization efforts.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143984221","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":"Characterization of Indolent Chronic Myelomonocytic Leukemia Phenotypes and Dynamic Features of Disease Progression.","authors":"Luis E Aguirre, Somedeb Ball, Akriti Jain, Najla Al Ali, David A Sallman, Andrew Kuykendall, Kendra Sweet, Jeffrey E Lancet, Eric Padron, Rami S Komrokji","doi":"10.1016/j.clml.2025.03.016","DOIUrl":"https://doi.org/10.1016/j.clml.2025.03.016","url":null,"abstract":"<p><strong>Background: </strong>Chronic myelomonocytic leukemia (CMML) is a clonal hematopoietic disorder characterized by features of myeloproliferation and myelodysplasia. Various prognostic models incorporate clinical, cytogenetic, and molecular factors to assess risk and guide treatment decisions. However, there has been limited exploration of a subset of patients exhibiting more indolent disease behavior. Due to the significant heterogeneity in disease biology, clonal architecture, clinical presentation, and outcomes, identifying key markers is essential for predicting which patients may present with indolent disease and for recognizing those at greater risk of progression who may require early intervention.</p><p><strong>Patients and methods: </strong>We analyzed baseline clinical and molecular parameters in 656 CMML patients, stratifying them into 2 groups: those observed for ≥ 3 years (indolent CMML) and those needing treatment within that period.</p><p><strong>Results: </strong>14% of CMML patients exhibited indolent disease, correlating with superior outcomes (mOS: 78.5 months vs. 25 months in nonindolent cases). Indolent disease was associated with higher hemoglobin and platelet counts, JAK2 mutations, and fewer cytopenias. In contrast, features indicating the need for earlier treatment included leukocytosis, elevated lymphocyte/monocyte counts, higher percentage of circulating immature cells/blasts, increased marrow cellularity/blasts, and NRAS, ASXL1, and RUNX1 mutations. Changes in the M:E ratio, the presence of abnormally localized immature precursors (ALIP) and gain of mutations reflecting clonal evolution indicated clinical decline and need for timely treatment initiation.</p><p><strong>Conclusion: </strong>A small proportion of CMML patients exhibit indolent features linked to better outcomes, contrasting with markers indicating earlier treatment initiation, which are associated with increased risk of progression; recognizing these markers aids in predicting disease aggressiveness.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143976681","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":"Treatment Patterns and Clinical Outcomes of Patients With Systemic Light Chain Amyloidosis in the United States: Evidence From Real-World Clinical Practice.","authors":"Sophia S Li, Anita D'Souza, Suzanne Lentzsch, Faiza Zafar, Irina Pivneva, Talissa Watson, Annie Guerin, Shaji Kumar","doi":"10.1016/j.clml.2025.03.012","DOIUrl":"https://doi.org/10.1016/j.clml.2025.03.012","url":null,"abstract":"<p><strong>Background: </strong>This study described real-world treatment patterns and outcomes in patients with systemic light chain (AL) amyloidosis in the United States (US).</p><p><strong>Methods: </strong>De-identified chart review data were obtained from 117 adult patients diagnosed with systemic AL amyloidosis (01/01/2014-12/31/2021) who initiated 1 L treatment, outside of a clinical trial setting, on or after January 1, 2014, and received ≥ 1 treatment at a US medical center.</p><p><strong>Results: </strong>Among patients, (median age: 63.2 years; female: 50.4%; White: 73.5%; Black/African American: 14.5%), most patients received 2 or more lines of treatment (62.4%) with 30.8% receiving 2 lines of treatment, predominantly cyclophosphamide, bortezomib, and dexamethasone (CyBorD)-, daratumumab-, and dexamethasone-based regimens. In first-line, one third of patients had hematologic response (complete response [CR]: 31.6%; very good partial response (29.1%); for later lines, CR rates tended to decrease. By 57.5 months, ≥60% of patients were still alive (Kaplan-Meier rates - 12 months: 92.0%; 24 months: 84.4%). Median progression-free survival (41.4 months), event-free survival (24.4 months), and time to next treatment (43.2 months) were unexpectedly longest in second line.</p><p><strong>Conclusions: </strong>The study demonstrated that there was heterogeneity in the treatment of AL amyloidosis, and patients who receive treatment can survive for several years after diagnosis without progression.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143966549","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":"Outcomes of Patients With Extramedullary Disease in Triple-Class Exposed Relapsed/Refractory Multiple Myeloma From the Pooled LocoMMotion and MoMMent Studies.","authors":"Philippe Moreau, María-Victoria Mateos, Hartmut Goldschmidt, Maria Esther Gonzalez Garcia, Britta Besemer, Marta Sonia Gonzalez Perez, Mohamad Mohty, Joanne Lindsey-Hill, Suriya Kirkpatrick, Michel Delforge, Emanuele Angelucci, Francesco Di Raimondo, Ravi Vij, Margaret Doyle, Kathleen Gray, Claire Albrecht, Vadim Strulev, Imène Haddad, Silva Koskinen, Lorenzo Acciarri, Jozefien Buyze, Katja Weisel","doi":"10.1016/j.clml.2025.03.014","DOIUrl":"https://doi.org/10.1016/j.clml.2025.03.014","url":null,"abstract":"<p><strong>Background: </strong>Patients with relapsed/refractory multiple myeloma (RRMM) who develop extramedullary disease (EMD) generally have a poor prognosis, highlighting the urgent need for new therapies. We report effectiveness outcomes and safety in patients with and without EMD from the pooled analysis of LocoMMotion and MoMMent.</p><p><strong>Methods: </strong>LocoMMotion and MoMMent-1 are prospective, noninterventional, consecutive studies assessing the evolving standard of care from 20192022 in patients with triple-class exposed RRMM.</p><p><strong>Results: </strong>Of 302 patients, 29 had EMD per investigator discretion and only 15 patients were assessed as having true extramedullary plasmacytoma (EMP; defined as patients with ≥1 EMP lesion) by the response review committee. The 29 EMD patients received 21 unique regimens (most commonly chemotherapy-based regimens). Of the 29 patients with EMD, overall response rate (ORR) was 24.1%, median progression-free survival (PFS) was 2.66 months, median overall survival (OS) was 7.16 months, and median time to next treatment (TTNT) was 3.09 months. All responses were lower (ORR) and shorter (median PFS, OS, and TTNT) in patients with EMD vs patients without EMD. Nineteen (65.5%) patients with EMD received ≥1 subsequent lines of therapy. Of those, two (10.5%) patients received bispecific antibodies and achieved a partial response or better; three (15.8%) patients received antibody-drug conjugates (responses were unknown or not determined at data cut-off), and no patients received chimeric antigen receptorT cell therapy.</p><p><strong>Conclusions: </strong>These results demonstrate the urgent need for more effective novel therapies for patients with EMD and highlight the need to use clear definitions of EMD and EMD response criteria for clinical trials.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143970752","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":"Inferior Outcomes in Acute Lymphoblastic Leukemia With Translocation (14;18).","authors":"Diane Habib, Elias Jabbour, Alex Bataller, Koji Sasaki, Guilin Tang, Sanam Loghavi, Shaoying Li, Jayastu Senapati, Nicholas J Short, Nitin Jain, Hagop Kantarjian, Fadi G Haddad","doi":"10.1016/j.clml.2025.03.010","DOIUrl":"https://doi.org/10.1016/j.clml.2025.03.010","url":null,"abstract":"<p><strong>Introduction: </strong>The (14;18)(q32;q21) chromosomal translocation leading to IGH::BCL2 rearrangement, has been rarely described in B-cell acute lymphoblastic leukemia (B-ALL), mainly in association with MYC rearrangement. The outcome of B-ALL harboring t(14;18)(q32;q21) without MYC rearrangement remains unknown.</p><p><strong>Methods: </strong>We retrospectively reviewed 2778 cases of B-ALL treated at our institution and identified those harboring a t(14;18)(q32;q21) by karyotype. Cases with concomitant MYC rearrangement and cases of lymphoma were excluded. Three patients with no MYC rearrangement by karyotype but without further confirmation by Fluorescence In Situ Hybridization (FISH), were included.</p><p><strong>Results: </strong>Five patients were included in this analysis, with a median age of 35 years (range, 19-58); 1 patient had central nervous system involvement at diagnosis. Induction therapy consisted of hyper-CVAD in 3 patients, hyper-CVAD with inotuzumab ozogamicin in 1 patient, and asparaginase-based regimen in 1 patient. Four patients (80%) responded, with a median duration of response of 12.9 months (range, 5.1-32.9); 1 patient had primary refractory disease. None of the patients proceeded to an allogeneic hematopoietic stem cell transplantation (HSCT). Four patients received salvage chemotherapy and eventually progressed and died. One patient received salvage therapy with subcutaneous blinatumomab and achieved complete remission with negative measurable residual disease (MRD) by next-generation sequencing (NGS) after 3 courses of therapy. After a median follow-up of 13.4 months, the 2-year event-free survival and overall survival rates were 20% and 25%, respectively.</p><p><strong>Conclusion: </strong>The outcome of B-ALL with t(14;18)(q32;q21) is poor. Incorporating immune-therapies, chimeric antigen receptor (CAR)-T cell therapy, with or without HSCT, into the treatment regimens, might further improve outcomes.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143956011","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":"Outcomes of Adolescent LBCL Patients Treated With Adult Type Chemotherapy: A Single Center Experience.","authors":"Riad El Fakih, Taimoor Hussain, Oudai Sahwan, Ghaith Meir, Abdulwahab A Albabtain, Saud Alhayli, Abdullah Alamer, Mhd Anas Alsibai, Shamayel Mohammed, Ali Nasser, Muhammad Shahzad Rauf, Haifa Abdulrahman Alsuwaine, Irfan Maghfoor, Saad Akhtar, Alfadel Alshaibani, Walid Rasheed, Mahmoud Aljurf","doi":"10.1016/j.clml.2025.03.009","DOIUrl":"https://doi.org/10.1016/j.clml.2025.03.009","url":null,"abstract":"<p><strong>Background: </strong>Lymphoma is the most common cancer in adolescents. Outcome disparities based on the locus of care are increasingly recognized in this age group.</p><p><strong>Materials and methods: </strong>We report outcomes of LBCL patients between 14 and 21 who were treated at an adult center using adult type chemotherapy.</p><p><strong>Results: </strong>Sixty-four patients, median age 19 were analyzed. 82.8% were DLBCL, 17.2% were PMBCL; 21.9% stage I, 31.3% stage II, 7.8% stage III, and 39 % stage IV; 18.8% \"very good\", 62.5% \"good\" and 18.8% \"poor\" RIPI score. The median follow-up period was 68.8 months. The 5 years OS was 94.3%, and the 5 years EFS was 78.3%. High RIPI patients had a worse OS. High RIPI, B symptoms, ABC cell of origin and PMBCL had worse EFS.</p><p><strong>Conclusion: </strong>Pediatric protocol use should be restricted to patients at higher risk of events rather than to all these patients.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143967917","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":"SOHO State of the Art Updates and Next Questions | Atypical Chronic Myeloid Leukemia: Pathogenesis, Diagnostic Challenges and Therapeutic Strategies.","authors":"Alessandro Costa, Massimo Breccia","doi":"10.1016/j.clml.2025.03.007","DOIUrl":"https://doi.org/10.1016/j.clml.2025.03.007","url":null,"abstract":"<p><p>Atypical chronic myeloid leukemia (aCML) is a rare and challenging clonal hematopoietic disorder within the myelodysplastic/myeloproliferative neoplasm (MDS/MPN) spectrum. Over the past two decades, substantial progress has been made in understanding the genetic mechanisms driving aCML, revealing a complex and heterogeneous mutational landscape. Key ancestral mutations, such as ASXL1 and ETNK1, have been identified, providing a foundation for the pathogenesis and for the possible emergence of secondary abnormalities, particularly in epigenetic regulation (eg, SETBP1), and in splicing process (eg, SRSF2). These molecular insights have been integrated into current diagnostic classifications, refining disease characterization and offering potential targets for precision therapies. Despite these advances, significant clinical challenges persist due to the disease's rarity and the lack of randomized clinical trials. Therapeutic strategies remain inadequately defined, with allogeneic stem cell transplantation being the only curative option. This review provides an overview of the molecular, clinical, and therapeutic information that may pave the way for essential advancements in the proper management of this disease.</p>","PeriodicalId":10348,"journal":{"name":"Clinical Lymphoma, Myeloma & Leukemia","volume":" ","pages":""},"PeriodicalIF":2.7,"publicationDate":"2025-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143794370","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}