European Journal of Haematology最新文献

{"title":"Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Myelodysplastic/Myeloproliferative Overlap Neoplasms.","authors":"Nihar Desai, Sergio Rodriguez-Rodriguez, Carol Chen, Tommy Alfaro Moya, Eshrak Al-Shaibani, Igor Novitzky-Basso, Ivan Pasic, Fotios V Michelis, Auro Viswabandya, Dennis Kim, Rajat Kumar, Jonas Mattsson, Arjun Datt Law","doi":"10.1111/ejh.14430","DOIUrl":"https://doi.org/10.1111/ejh.14430","url":null,"abstract":"<p><p>Myelodysplastic/myeloproliferative overlap neoplasms (MDS/MPN) are rare hematological malignancies. We analyzed the outcomes of 75 patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) for MDS/MPN. Graft-versus-host disease (GvHD) prophylaxis included post-transplantation cyclophosphamide (PTCy) in 71% of patients, with 44 (59%) receiving a combination of anti-thymocyte globulin (ATG) and PTCy. The median follow-up was 44.4 months. Primary graft failure occurred in three patients (4%). The incidence of grade III-IV acute GvHD at day 100 was 13% (95% CI: 6-22). At 2 years, the incidence of moderate-severe chronic GvHD, non-relapse mortality (NRM), relapse, GvHD-free/relapse-free survival (GRFS), and overall survival (OS) was 31.7% (95% CI 20.7-43.2), 37.9% (26-49), 17.4% (95% CI: 10-27), 24.8% (95% CI: 15-36), and 51.6% (95% CI: 39-63), respectively. PTCy-based GvHD prophylaxis seemed to be associated with improved OS (HR: 0.5, 95% CI: 0.3-0.9, p = 0.03), NRM (HR: 0.4, 95% CI: 0.2-0.9, p = 0.03), and GRFS (HR: 0.5, 95% CI: 0.3-0.8, 0.009). On multivariable analysis, the use of the PTCy-containing regimen seemed to be associated with improved NRM (HR: 0.41; 95% CI: 0.2-0.8; p = 0.03), GRFS (HR: 0.47; 95% CI: 0.3-0.8; p = 0.009), and OS (HR: 0.49; 95% CI: 0.2-0.9; p = 0.03) without an increased risk of relapse.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143991321","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"HiDAC Consolidation in AML: Comparable Outcomes With Reduced Toxicity Using a Three-Day Schedule (HiDAC-123).","authors":"Baher Krayem, Nurit Horesh, Avraham Frisch, Tsila Zuckerman, Yishai Ofran","doi":"10.1111/ejh.14432","DOIUrl":"https://doi.org/10.1111/ejh.14432","url":null,"abstract":"","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143985668","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study.","authors":"Robert Klamroth, Mahasen Al Saleh, Heidi Glosli, Michele Schiavulli, Benoît Guillet, Linda Bystrická, Anton Schönstein, Stefan Lethagen","doi":"10.1111/ejh.14427","DOIUrl":"https://doi.org/10.1111/ejh.14427","url":null,"abstract":"<p><strong>Objective: </strong>To report data from an ITI chart review study (NCT03951103) for first-time and rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in persons with haemophilia A.</p><p><strong>Methods: </strong>Retrospective and prospective real-world data are reported from a non-interventional, multicentre study of patients who had been or were currently being treated with rFVIIIFc ITI. ITI treatment outcome (defined by investigators) and regimens are reported.</p><p><strong>Results: </strong>Forty-one patients from 16 sites were included. First-time ITI was used in 24 patients; 16 had an ITI outcome at study end. Thirteen patients (81.3%) had ITI success, and three had failure. Median (range) rFVIIIFc consumption was 300 (61-2800) IU/kg/week, and most (70.8%) used ≤ 300 IU/kg/week. The vast majority of patients (87.5%) received less than daily ITI. Rescue ITI was used in 17 patients; 16 had an ITI outcome at study end. Eight patients (50.0%) had ITI success/partial success, seven had failure, and one withdrew early. Median (range) rFVIIIFc consumption was 536 (98-1435) IU/kg/week; 35.3% used ≤ 300 IU/kg/week and 52.9% used > 500 IU/kg/week. Most patients (64.7%) received daily ITI.</p><p><strong>Conclusion: </strong>ITI with rFVIIIFc is likely to be successful in first-time ITI patients and is an effective option for those who have previously experienced ITI failure.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier: NCT03951103.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-04-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144004214","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal Haemoglobinuria.","authors":"Regis Peffault de Latour, Austin Kulasekararaj, David Dingli, Koo Wilson, Piotr Wojciechowski, Zalmai Hakimi, Jameel Nazir, Barbara Czech, Peter Hillmen, Jeff Szer","doi":"10.1111/ejh.14422","DOIUrl":"https://doi.org/10.1111/ejh.14422","url":null,"abstract":"<p><strong>Background: </strong>Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare, acquired non-malignant haematological disorder characterised by thrombosis risk, serious complications and debilitating symptoms in untreated patients.</p><p><strong>Objective: </strong>This anchored indirect treatment comparison (ITC) evaluated efficacy data between proximal complement 3 inhibitor (C3i) pegcetacoplan and factor B inhibitor, iptacopan, in patients with PNH previously treated with complement 5 inhibitors (C5i; eculizumab, ravulizumab).</p><p><strong>Methods: </strong>Respective pivotal studies provided patient-level trial data for pegcetacoplan (16-week PEGASUS [NCT03500549]) and published data for iptacopan (24-week APPLY PNH [NCT04558918]). Differences in study design, duration and statistical methods between PEGASUS and APPLY PNH necessitated the comparative analyses to be conducted on secondary measures based on haemoglobin (Hb) levels, absolute reticulocyte count (ARC), lactate dehydrogenase (LDH) levels, and patient-reported outcomes on the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale scores. The availability of a common reference C5i treatment group in both PEGASUS and APPLY PNH studies allowed anchored ITC (Bucher method). Simulated treatment comparison (STC) assessed the robustness of the main analysis.</p><p><strong>Results: </strong>Overall, baseline characteristics of the populations in the PEGASUS and APPLY PNH studies were broadly comparable. Anchored ITC showed comparable outcomes (mean difference, [95% CI]) on change-from-baseline to end of study for pegcetacoplan versus C5i, and iptacopan versus C5i, respectively, across endpoints: Hb level (-0.49 g/dL [-1.78, 0.80]); ARC (-34.41 × 10⁹/L [-90.02, 21.21]); LDH level (-115.16 U/L [-244.40, 14.01]); FACIT-Fatigue score (3.57 [-5.60, 12.73]). Finally, the STC produced results consistent with the main Bucher analyses across all clinical endpoints and patient-reported fatigue, providing similar point estimates and confidence intervals.</p><p><strong>Conclusion: </strong>This anchored ITC, based on data from pivotal trials, did not indicate significant differences in clinical or patient-reported outcomes between pegcetacoplan and iptacopan in PNH treatment. The findings suggest that PNH treatment decisions should also consider individualised disease- and patient-related factors.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier: NCT03500549.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143998727","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Medical and Nutritional Outcomes Are Similar Among Autologous Transplant Patients on Enteral Nutrition When Compared to Parenteral Nutrition. A Randomized Pilot Study.","authors":"Janet Madill, Brenda Hartman, Heather Resvick, Mehraneh Mohebbi, Alison Andrade, Cheryl Sigfrid, Maisam Abouzeeni, Adrienne Fulford, Anargyros Xenocostas, Uday Deotare","doi":"10.1111/ejh.14428","DOIUrl":"https://doi.org/10.1111/ejh.14428","url":null,"abstract":"<p><p>Autologous hematopoietic stem cell transplantation (AHSCT) is the treatment for myeloma and lymphoma. posttreatment, significant nutritional and medical issues and malnutrition assessed by Subjective Global Assessment (SGA) arise. No established effective treatment for using either parenteral (PN) or enteral routes (EN) to improve nutritional status, reduce medical complications, and be cost-effective is available. We investigated the effectiveness of EN versus PN in terms of nutritional path of supplementation. AHSCT patients were randomized to either EN or PN and were followed at baseline, 15 and 30 days posttransplant. Age, body mass index, SGA, length of stay (LOS), medical complications, severity of complications, infections, overall survival (Day 100), albumin, random blood glucose, and C-reactive protein were evaluated. Descriptive statistics, Spearman's, chi square, correlations, and uni- and multivariate by type of feed, using SPSS v 29. Thirty-six patients with complete medical and laboratory data were followed. No significance in any of the medical or nutritional parameters between the two groups was found. No correlations between SGA at any time point and type of feeding were identified. No relationship between SGA, LOS, complications, albumin, CRP, or random blood glucose at all three time points was seen. EN is a safe, convenient, and cost-effective option for AHSCT patients since medical and nutritional outcomes were similar between those receiving EN compared to PN.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143970917","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Rilzabrutinib for the Treatment of Immune Thrombocytopenia.","authors":"Caterina Labanca, Enrica Antonia Martino, Ernesto Vigna, Antonella Bruzzese, Francesco Mendicino, Giulio Caridà, Eugenio Lucia, Virginia Olivito, Veronica Manicardi, Nicola Amodio, Antonino Neri, Fortunato Morabito, Massimo Gentile","doi":"10.1111/ejh.14425","DOIUrl":"https://doi.org/10.1111/ejh.14425","url":null,"abstract":"<p><p>Advancements in the understanding of ITP pathogenesis have led to significant improvements in disease management through the use of both traditional immunosuppressive strategies and novel targeted therapies. However, a subset of patients remains refractory to treatment or achieves only transient benefits, underscoring the need for alternative therapeutic approaches. Bruton's tyrosine kinase (BTK) inhibitors have emerged as a promising strategy for autoimmune cytopenias, including ITP, due to their ability to modulate key immune pathways. Rilzabrutinib, an oral, reversible BTK inhibitor, represents a novel therapeutic approach for ITP. Rilzabrutinib, an oral, reversible BTK inhibitor, offers a novel mechanism of action by preserving platelet aggregation while reducing macrophage-mediated platelet clearance, distinguishing it from irreversible BTK inhibitors. This review provides an updated and comprehensive analysis of the Phase 1/2 LUNA 2 trial and its long-term extension, contextualizing rilzabrutinib within the broader treatment landscape. We also offer a comparative assessment of other BTK inhibitors investigated for ITP and discuss rilzabrutinib's potential positioning relative to existing therapies, including thrombopoietin receptor agonists (TPO-RAs), rituximab, fostamatinib, and immunosuppressants. Results from the phase 1/2 LUNA 2 trial and its long-term extension demonstrated that Rilzabrutinib induced a durable platelet response in 40% of patients, with a median time to response of 11.5 days. The treatment exhibited a favorable safety profile, with predominantly grade 1 or 2 adverse events and no significant safety concerns commonly associated with BTK inhibitors, such as increased bleeding risk, hepatic toxicity, or cardiac arrhythmias. Preliminary data presented at ASH 2024 from the ongoing Phase 3 LUNA 3 trial, a randomized, double-blind study, further support rilzabrutinib's efficacy and long-term safety. If confirmed, these findings suggest that rilzabrutinib could represent a valuable therapeutic option for patients with refractory ITP, addressing a critical unmet need and potentially redefining treatment paradigms.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143970920","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Home-Based Daratumumab in Patients With Multiple Myeloma.","authors":"Tine Rosenberg, Jannie Kirkegaard, Michael Tveden Gundesen, Maja Kjær Rasmussen, Karin Brochstedt Dieperink, Thomas Lund","doi":"10.1111/ejh.14409","DOIUrl":"https://doi.org/10.1111/ejh.14409","url":null,"abstract":"<p><strong>Objective: </strong>Multiple myeloma is an incurable cancer with lifelong treatment needs. This, together with a global nursing shortage, calls for new approaches for future treatment. In this study, we therefore investigated the feasibility of home-based subcutaneous daratumumab administered by primary care nurses outside the hospital.</p><p><strong>Methods: </strong>Applying a mixed-methods prospective design, we included 30 patients; 18 had completed ≥ 6 cycles of daratumumab treatment, and 12 were newly started. New patients were followed for six 28-day cycles, with every second treatment administered outside the hospital. Patients already on treatment were followed for seven cycles with 2/3 treatments administered outside the hospital.</p><p><strong>Results: </strong>Of 123 administrations planned at the hospital, 122 (97.6%) were administered and three were cancelled. Of 144 administrations planned outside the hospital, 133 (92.4%) were administered, six were redirected to the hospital, and five were cancelled. No significant difference between numbers of cancellations/redirections was observed. Patients spent significantly longer time on treatment at the hospital, even when deducting travel time. Reducing patients' visits to the hospital did not cause additional unplanned contacts with the healthcare system.</p><p><strong>Conclusion: </strong>This study thus concludes that administration of daratumumab outside the hospital is safe, feasible, and time saving.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov ID: NCT05306587.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751795","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dual Targeting of Pim and PI3 Kinases in Mature T-Cell Lymphoma.","authors":"M Lohrberg, M Heber, L Ries, K Markus, N Ksionsko, N Schmidt, G Nothnick, L Thielking, M O'Neill, S Martínéz-González, C Blanco-Aparicio, J Pastor, D Cunningham, R Koch","doi":"10.1111/ejh.14420","DOIUrl":"https://doi.org/10.1111/ejh.14420","url":null,"abstract":"<p><p>Provirus Integration site for Moloney leukemia virus (Pim) family members are well-known oncogenes, with an expression that is restricted to few cell types including hematopoietic cells in adult organisms, making it a promising target for lymphoma treatment. Indeed, previous studies in mature T-cell lymphoma (mTCL) cells revealed frequent upregulation of Pim expression. Nevertheless, inhibition of Pim kinases showed only minor effects on the viability of mTCL cells so far. Thus, we here addressed cellular responses to therapeutic inhibition of Pim kinases and identified a PI3K/Akt-driven activation of mTOR as a significant escape mechanism mitigating the anti-lymphoma effects of Pim inhibition. Indeed, dual inhibition of Pim and PI3 kinases showed synergistic anti-lymphoma effects in vitro through downregulation of mTOR-induced protein translation and mitigation of BCL-xL-mediated anti-apoptotic mechanisms. Based on this finding, we next explored the therapeutic potential of the dual Pim/PI3K inhibitor IBL-202 in mTCL cell lines. Strikingly, IBL-202 strongly induced cell-cycle-dependent cell death in cell lines of different mTCL subtypes. Together, our study provides mechanistic evidence supporting a therapeutic strategy of dual Pim- and PI3-kinase inhibition in mature T-cell lymphoma.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751791","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparison of Hetrombopag and Eltrombopag Added to First-Line Immunosuppressive Therapy in Severe Aplastic Anemia.","authors":"Baohang Zhang, Wenrui Yang, Rui Kang, Yimeng Shi, Xiangrong Hu, Li Zhang, Liping Jing, Weiping Yuan, Jun Shi, Fengkui Zhang, Xin Zhao","doi":"10.1111/ejh.14418","DOIUrl":"https://doi.org/10.1111/ejh.14418","url":null,"abstract":"<p><p>The addition of thrombopoietin receptor agonists (TPO-RAs) to immunosuppressive therapy (IST) improves the hematologic response rate and quality in patients with severe aplastic anemia (SAA). However, no studies have yet reported on whether there are differences in the efficacy of TPO-RAs. Here, we retrospectively analyzed the clinical data of SAA patients who received hetrombopag (HPAG) or eltrombopag (EPAG) as part of first-line standard IST to compare the efficacy. Sixty-seven patients were enrolled in the HPAG group and 42 patients in the EPAG group. The overall hematologic response (OR) rates of the HPAG group at 3 and 6 months after IST were 50.7% and 65.6%, respectively, close to that of the EPAG group (50%, p = 0.973; 73.8%, p = 0.494). The rates of complete response (CR) at 3 and 6 months were 13.4% and 31.3% in the HPAG group, respectively, which were like those in the EPAG group (11.9% and 28.6%), with no statistical difference (p = 1.00 and 0.59). The median time to first response (3.0 vs. 3.2 months, p = 0.79) was similar in HPAG and EPAG. The overall survival (OS) rates were 91.0% and 92.8% in the HPAG group and EPAG group (p = 0.53), respectively. Monosomy 7 was detected in one patient in the EPAG group and her disease transformed into acute myelocytic leukemia (AML) at 25 months after ATG treatment. One patient in HPAG had trisomy 8 at 9 months of ATG treatment, and bone marrow examination showed no disease progression. CONCLUSION: The addition of HPAG to standard IST in SAA patients showed similar response rates and response quality to that of EPAG. HPAG could be an alternative to EPAG for the first-line treatment of SAA patients.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-03-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143751788","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinicopathologic Characteristics and Prognostic Profile of Chronic Myeloid Neoplasms With Somatic NF1 Mutations in Adult Patients.","authors":"Hamza Tariq, Margarita Loxas, Mir B Alikhan, Juehua Gao, Xinyan Lu, Qing Ching Chen, Yi-Hua Chen, Jessica K Altman, Lawrence J Jennings, Madina Sukhanova","doi":"10.1111/ejh.14419","DOIUrl":"https://doi.org/10.1111/ejh.14419","url":null,"abstract":"<p><strong>Objectives: </strong>The clinicopathologic and prognostic features of somatic NF1 mutations have been well studied in pediatric myeloid neoplasms and adult acute myeloid leukemia (AML) but not in adult chronic myeloid neoplasms (CMNs), including myelodysplastic syndrome (MDS), myeloproliferative neoplasms (MPNs), and myelodysplastic/myeloproliferative neoplasms (MDS/MPNs).</p><p><strong>Methods: </strong>A retrospective review was performed to identify adult patients diagnosed with NF1-mutated CMNs between 1/2010 and 8/2023. Patients with NF1 wildtype (NF1-WT) CMNs concurrently diagnosed during the same time were used as a comparative group. Clinicopathologic and genetic characteristics and overall survival (OS) were compared between the two groups.</p><p><strong>Results: </strong>A total of 36 NF1-mutated CMNs were identified (4.7% of all CMNs), including 19 MDS, 4 MPNs, and 13 MDS/MPNs (all CMML). NF1-mutated CMMLs showed significantly higher absolute monocyte counts (AMC), more frequent complex karyotypes, and higher frequencies of SRSF2 and KRAS mutations compared to NF1-WT CMMLs. NF1-mutated MDS also showed significantly higher AMC, lower frequency of SF3B1, and higher frequencies of SRSF2 and KRAS mutations compared to NF1-WT MDS. The OS of NF1-mutated CMNs was significantly inferior to NF1-WT CMNs (median survival: 2.05 vs. 4.8 years; log-rank p = 0.03).</p><p><strong>Conclusions: </strong>Adult CMNs with mutated NF1 show higher AMC, high-risk molecular cytogenetic features, and inferior survival. Therefore, testing for NF1 mutations could be considered part of risk assessment for patients with CMNs.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143729498","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}