European Journal of Haematology最新文献

{"title":"Gender Disparities in Health-Related Quality of Life in Patients With Chronic Lymphocytic Leukemia: Impact of Treatment Modality.","authors":"Bushra Mahameed, Giora Sharf, Tamar Tadmor, Geffen Kleinstern","doi":"10.1111/ejh.70017","DOIUrl":"https://doi.org/10.1111/ejh.70017","url":null,"abstract":"<p><p>In chronic lymphocytic leukemia (CLL), treatment initiation is deferred until clinical symptoms emerge, aiming to minimize treatment-related burden and maintain health-related quality-of-life (QoL). We examined gender differences in QoL among patients undergoing active surveillance versus targeted therapies. Participants completed the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) Questionnaire, and the score was derived from the FACT-General (FACT-G) and leukemia-specific concerns (Leu-Subscale). Comparisons between treatment groups were conducted using Student's t-test, stratified by sex. Among 210 participants, 63 were under surveillance, 115 in treatment, and 32 post-treatment. Among men, the surveillance group reported better physical well-being (mean: 25.2 vs. 19.7, p < 0.001), Leu-Subscale (mean: 51.5 vs. 42.8, p = 0.002), and FACT-Leu (mean: 135.9 vs. 120.5, p = 0.02) compared to the active treatment group. In contrast, women in surveillance reported lower social well-being (mean: 18.9 vs. 22.5, p = 0.008); emotional well-being (mean: 15.5 vs. 18.2, p = 0.003); FACT-G (mean: 74.6 vs. 83.3, p = 0.009); and FACT-Leu (mean: 116.7 vs. 128.3, p = 0.014) compared to the active treatment. A significant interaction was observed between treatment group and sex in the emotional well-being subscale. In conclusion, men in surveillance reported better overall QoL than those in active treatment, whereas women in active treatment experienced better overall QoL than those in surveillance. These findings highlight the importance of gender-specific approaches to improve health-related QoL in CLL care.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144759542","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":"Patient Characteristics, Healthcare Contacts and Drug Use in Polycythaemia Vera in Denmark.","authors":"Karoline Mathilde Lundgaard, Dustin Andersen Patel, Mette Brabrand, Christen Lykkegaard Andersen, Anton Pottegård","doi":"10.1111/ejh.70008","DOIUrl":"https://doi.org/10.1111/ejh.70008","url":null,"abstract":"<p><strong>Objective: </strong>Patients with polycythaemia vera (PV) may experience symptoms and cardiovascular complications before receiving a diagnosis. Understanding the associated patterns of healthcare and drug utilisation may help detect patients who need diagnostic workup. In this study, we described healthcare contacts and prescription drug use in patients with PV in Denmark before and after their diagnosis.</p><p><strong>Methods: </strong>We conducted a descriptive study using national Danish registries, matching patients with PV diagnosed between 2012 and 2021 (n = 1428) to the background population on age and sex.</p><p><strong>Results: </strong>Patients with PV had a median age of 70 years [61-77] with an equal distribution of men and women. The patients were comparable to the comparison group across most characteristics except for a history of cardiovascular disease. In the 3 months before and 6 months after diagnosis, patients with PV had an increased number of healthcare contacts and rate of new drugs. The drug rate returned to baseline after 6 months, while outpatient contacts remained increased.</p><p><strong>Conclusions: </strong>Patients with PV experienced an increase in healthcare and drug utilisation around the time of diagnosis, but were comparable to the background population up to 3 months before, with no indicators allowing earlier detection of PV related to drug use or healthcare contacts.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144759543","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":"Machine Learning for Prediction of High-Risk Hospitalizations in Lymphoma Patients: A Danish Population-Based Study.","authors":"Alexander Djupnes Fuglkjaer, Deniz Kenan Kilic, Mathias Holmsgaard Eskesen, Mikkel Runason Simonsen, Laurids Østergaard Poulsen, Carsten Utoft Niemann, Paw Jensen, Kirstine Kobberøe Søgaard, Mikkel Werling, Frederik Christensen, Izabela Ewa Nielsen, Tarec Christoffer El-Galaly","doi":"10.1111/ejh.70012","DOIUrl":"https://doi.org/10.1111/ejh.70012","url":null,"abstract":"<p><strong>Objective: </strong>Infections are a leading cause of hospitalization in patients treated for lymphoma and can be life-threatening. This study developed a machine learning (ML)-based risk stratification method to classify infection-related hospitalizations (IRH) into serious-IRH (S-IRH) and non-serious-IRH (NS-IRH).</p><p><strong>Methods: </strong>S-IRH was defined based on death, positive blood culture, blood stream infection, and sepsis during hospitalization. Clinical data from health records and registries were used to construct the feature matrix for an XGBoost model. The study included 727 adult lymphoma patients diagnosed 2013-2023 and treated with first-line therapies including CHOP (or CHOP-like), ABVD, BEACOPP, CVP, and Bendamustine.</p><p><strong>Results: </strong>A total of 591 IRHs were identified, of which 119 were categorized as S-IRH. The developed model achieved a ROC-AUC of 71.0% for predicting S-IRH. At a prediction threshold of 0.3, the average sensitivity and specificity were 63.0% and 63.0%, respectively. The negative and positive predictive values were 87.5% and 30.3%, respectively.</p><p><strong>Conclusion: </strong>The developed model demonstrated acceptable clinical performance in predicting S-IRH. However, despite the wealth of data points entered in the model, performance was not sufficient for a stand-alone decision tool, but it should rather be seen as a decision support tool for clinicians.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144728988","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":"Quality of Patient-Reported Outcomes in Multiple Myeloma Randomized Controlled Trial Protocols and Publications According to the SPIRIT-PRO and CONSORT-PRO guidelines: A Systematic Review.","authors":"Darshi Shah, Francesco Sparano, Veer Shah, Daniela Krepper, Thomas Baldi, Rajshekhar Chakraborty, Fabio Efficace","doi":"10.1111/ejh.70002","DOIUrl":"https://doi.org/10.1111/ejh.70002","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Patient-reported outcomes (PROs) are now frequently incorporated into multiple myeloma (MM) randomized controlled trials (RCTs) to help inform clinical decision making. Although the quality of PRO components in cancer RCT protocols is generally recognized as suboptimal, there are limited data on adherence to international quality standards in MM RCT protocols.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;We performed a systematic review to identify MM RCTs published between January 2014 and June 2023 that utilized the EORTC QLQ-C30 questionnaire. The quality of PRO-specific protocol content was evaluated using the SPIRIT-PRO guidelines, which establish key requirements for PRO inclusion in protocols. The framework consists of 16 items: 5 elaborations of the existing SPIRIT checklist and 11 PRO-specific extensions items. The quality of PRO reporting was evaluated using CONSORT-PRO Extension, which comprises 14 items to improve the reporting of PRO data in clinical trials.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;Our systematic review identified 35 RCTs encompassing 20,612 patients, with 24 trials (69%) having publicly accessible protocols. The median protocol compliance was 7.5 SPIRIT-PRO items. Analysis of 24 protocols showed strong compliance in assessment schedules (96%), PRO domains/instrument justification (79%), and PRO objectives (71%). Intervention deviation procedures were described in 62% of protocols. Half included comprehensive data collection plans, while 58% addressed missing data methods and 42% outlined PRO research questions or multiplicity controls. Notably weak areas included strategies for preventing missing data (38%), and only 8% of protocols detailed PRO monitoring plans, personnel specifications, or eligibility criteria. CONSORT-PRO scores varied across all 35 RCTs (median 11, range 0.5-14). Among the 35 RCTs, while trials showed strong reporting of statistical elements (94% precision estimates, 91% subgroup analyses, 89% intention-to-treat, 86% timepoint completion) and results (80% hypothesized domains, 71% PRO validity, 69% clinical implications, 66% limitations), key PRO-specific components were reported by less than two-thirds of RCTs: only 11% stated PRO hypotheses, 31% specified administration mode, 43% identified domains, 60% addressed missing data, and 63% provided PRO rationale. Two factors were associated with higher reporting quality, both potentially reflecting publication bias: having a secondary PRO-focused paper (p &lt; 0.001) and finding statistically significant EORTC QLQ-C30 differences (p = 0.024). Our multivariable analysis showed no significant association between SPIRIT-PRO scores and CONSORT-PRO scores.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;Despite some foundational strengths of existing MM RCT protocols, gaps exist in PRO methodological specifications, statistical analysis, and clinical interpretation. Our findings may help to better inform PRO implementation in future MM RCT protocols. For exam","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144741715","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":"Contraception in Myeloproliferative Neoplasms: A Pharmacological Review.","authors":"Awni Alshurafa, Maria Benkhadra, Anas M Babiker, Wafa M Mohammed, Mohamed A Yassin","doi":"10.1111/ejh.70014","DOIUrl":"https://doi.org/10.1111/ejh.70014","url":null,"abstract":"<p><p>Myeloproliferative neoplasms (MPNs) are chronic hematologic disorders characterized by clonal proliferation of myeloid lineage cells, commonly driven by JAK2 mutations, and associated with significant thrombotic and hemorrhagic complications. Women of reproductive age with MPNs face unique challenges in selecting safe and effective contraception, as these disorders inherently increase thrombotic risk and may interact with hormonal contraceptive therapies. Estrogen-containing contraceptives are contraindicated due to their association with venous and arterial thrombosis, while progestin-only and non-hormonal methods are generally safer but must be tailored to individual risks such as thrombocytopenia, prior thrombotic events, and disease-modifying therapies. This review provides a comprehensive analysis of contraceptive options for women with MPNs, exploring the thrombotic and bleeding risks associated with different methods and the potential impact of MPN treatments, including JAK2 inhibitors, on contraceptive efficacy. By synthesizing current evidence, the review aims to guide clinicians in optimizing reproductive health for women with MPNs while minimizing the risks of their underlying hematologic condition.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144728987","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":"Vaccination in Multiple Myeloma: Challenges and Strategies.","authors":"Enrica Antonia Martino, Ernesto Vigna, Antonella Bruzzese, Nicola Amodio, Eugenio Lucia, Virginia Olivito, Caterina Labanca, Santino Caserta, Francesco Mendicino, Fortunato Morabito, Massimo Gentile","doi":"10.1111/ejh.70013","DOIUrl":"https://doi.org/10.1111/ejh.70013","url":null,"abstract":"<p><strong>Background: </strong>Multiple myeloma (MM) is a hematological malignancy characterized by profound immunosuppression resulting from both disease-related mechanisms and treatment-induced immune dysfunction. This compromised immune status markedly increases susceptibility to infections, a leading cause of morbidity and mortality in MM patients. While vaccination represents a cornerstone of infection prevention, standard immunization strategies often yield suboptimal responses in this population.</p><p><strong>Objectives: </strong>This review synthesizes current evidence on the immunological barriers and clinical effectiveness of vaccination in MM. We evaluate vaccines targeting influenza, Streptococcus pneumoniae, SARS-CoV-2, and other relevant pathogens, and explore determinants influencing vaccine efficacy, including optimal timing, formulation, and patient-specific immune parameters.</p><p><strong>Methods: </strong>A comprehensive literature review was conducted, encompassing clinical trials, retrospective cohort studies, expert consensus guidelines, and population-based data. Extracted outcomes included serological responses, infection-related events, and vaccine safety in MM patients.</p><p><strong>Results: </strong>Patients with MM exhibit impaired vaccine responses due to hypogammaglobulinemia, T- and B-cell dysfunction, and therapy-induced lymphodepletion. Despite modest immunogenicity, influenza and pneumococcal vaccines reduce respiratory infections and hospitalizations. Sequential administration of PCV13 followed by PPSV23, as well as post-autologous stem cell transplantation (ASCT) three-dose regimens, is associated with reduced pneumonia incidence. COVID-19 vaccines elicit variable responses, particularly in patients on anti-CD38 or BCMA-targeted therapies, highlighting the need for booster doses and, in selected cases, prophylactic monoclonal antibodies. Vaccines against herpes zoster, hepatitis B, and Haemophilus influenzae type B are also recommended, particularly around ASCT. Immunophenotypic markers such as CD19+ B-cell and CD4+ T-cell counts are predictive of vaccine responsiveness, supporting immune profiling as a tool for individualized vaccination planning.</p><p><strong>Conclusions: </strong>Vaccination remains a critical component of infection prevention in MM. Although immunogenicity may be attenuated, clinical benefits-namely, reduced infection burden and healthcare utilization-support broad vaccine implementation. A personalized approach, considering the treatment phase, disease control, and immune status, is essential to optimize vaccine effectiveness. Ongoing research into high-dose, adjuvanted, and next-generation vaccines is critical to enhance protection in this vulnerable population.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144728989","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":"Second- and Third-Line Salvage Chemotherapy Followed by Allogeneic Stem Cell Transplantation Leads to High Survival Rates in Primary Refractory AML-A Population-Based Study.","authors":"Markus Liew-Littorin, Linn Deleskog-Spångberg, Stefan Deneberg, Judit Janosi, Vladimir Lazarevic, Gustav Nilsson, Aristea Papageorgiou, Anna Robelius, Lovisa Vennström, Gunnar Juliusson, Bertil Uggla, Martin Höglund, Sören Lehmann","doi":"10.1111/ejh.70009","DOIUrl":"https://doi.org/10.1111/ejh.70009","url":null,"abstract":"<p><p>Refractory acute myeloblastic leukemia (AML) is poorly studied. In this study, we characterized primary refractory AML and investigated treatment and outcome in a population-based setting. Based on all AML patients receiving intensive induction therapy at 12 Swedish hospitals from 2011 to 2018 (N = 1221), we identified 306 patients that failed to achieve composite complete remission (CRc) after first-line therapy. Two-hundred-sixteen (71%) of these patients received salvage treatment with intensive chemotherapy (ICT), of which 126 (58%) achieved CRc and 85 (39%) underwent allogeneic stem cell transplantation (HSCT). One- and 3-year overall survival (OS) in patients receiving salvage ICT were 56.8% and 28.9%, respectively. Secondary AML and adverse ELN risk were associated with worse OS after salvage ICT, while fludarabine-based FAIDA versus amsacrine-based ACE salvage and HSCT were associated with better OS. Three-year OS after first or second salvage chemotherapy, followed by HSCT were 55% and 71%, respectively. Refractory patients responding to salvage ICT showed only a nonsignificant trend toward inferior OS compared to patients in CRc after the first cycle. In conclusion, refractory AML patients eligible for further intensive therapy have a reasonable chance of obtaining remission and long-term survival when followed by HSCT. The results can serve as a basis for evaluation of new treatments in refractory AML.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144697903","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":"Standard Versus Reduced-Doses of Post-Transplant Cyclophosphamide in HLA Matched (10/10) Peripheral Blood Stem Cell Transplantation.","authors":"Sara Redondo, Irene García-Cadenas, Albert Esquirol, Eva Iranzo, Miguel Arguello-Tomas, Silvana Saavedra, Guadalupe Oñate, Ana-Carolina Caballero, Ana Garrido, Jordi López, Ana Muntañola, Annalisa Paviglianiti, Jose Manuel Portos, Maria Estela Moreno-Martinez, Mireia Riba, Jorge Sierra, Javier Briones, Rodrigo Martino","doi":"10.1111/ejh.70010","DOIUrl":"https://doi.org/10.1111/ejh.70010","url":null,"abstract":"<p><strong>Objective: </strong>This retrospective, single-center study aims to compare the efficacy and safety of a reduced dose of post-transplantation cyclophosphamide (PTCy) (60 mg/kg total dose) versus the standard dose (100 mg/kg total dose), both combined with tacrolimus, in adult patients undergoing allogeneic hematopoietic stem cell transplantation from HLA-matched related or unrelated donors.</p><p><strong>Methods: </strong>We report clinical outcomes of 59 consecutive patients who received standard doses of PTCy and 48 with the new reduced dose.</p><p><strong>Results: </strong>Neutrophil and platelet recovery occurred later in the standard-dose group (p < 0.01). The incidence of febrile neutropenia was higher in the standard-dose group (p < 0.01). No significant differences were found in the incidence of acute cardiac events, early bacteremia, CMV reactivation within the first 100 days post-HSCT, or the development of hemorrhagic cystitis. There were no significant differences in the incidence of grade II-IV and III-IV acute GvHD, moderate-to-severe chronic GvHD, or in 20-month overall survival and progression-free survival (p > 0.05). Non-relapse mortality at 100 days and at 1 year was comparable between groups, as was 1-year GVHD-free/relapse-free and immunosuppression-free survival (p > 0.05).</p><p><strong>Conclusions: </strong>Reduced doses of CyPT combined with a single immunosuppressive agent are emerging as a safe and effective alternative for adult patients undergoing allo-HSCT from HLA-matched donors. Our findings support that this approach preserves key clinical outcomes while potentially reducing acute hematological and infectious toxicity.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144689596","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":"Interstitial 11q Deletions and Terminal 11q Duplications Cause a Bleeding Tendency due to Platelet Dysfunction That Is Similar to 11q Deletions Causing Jacobsen Syndrome.","authors":"Elise J Huisman, Virgil A S H Dalm, Marieke Joosten, Frans J Smiers, Leendert Porcelijn, Arjan Hoogendijk, Hans Janssen, Nicole N van der van der Wel, Idske C L Kremer Hovinga, Masja de Haas, Marjon H Cnossen","doi":"10.1111/ejh.14447","DOIUrl":"https://doi.org/10.1111/ejh.14447","url":null,"abstract":"<p><strong>Introduction: </strong>Jacobsen syndrome, resulting from a terminal deletion of chromosome 11 (11q), may lead to an increased bleeding tendency due to low platelet counts or platelet dysfunction. Currently, information on bleeding tendency and platelet function in patients with nonterminal 11q-aberrations such as larger deletions, interstitial 11q-deletions, or 11q-duplications is lacking.</p><p><strong>Methods: </strong>We investigated the bleeding symptoms in relation to platelet numbers and function in 14 patients: 10 patients with a terminal 11q-deletion, one terminal 11q-duplication, two interstitial 11q-deletions, and one with an interstitial 11q-duplication.</p><p><strong>Results: </strong>Twelve patients reported bleeding complications (12/14, 86%), most frequently perioperative bleeding (21%), hematomas (17%), epistaxis (13%), prolonged bleeding, and severe bruising (11%) and heavy menstrual bleeding in 4/5 postmenarcheal women (80%). Seven (50%) had platelet counts below normal values (median 62 × 10⁹/L). Strikingly, the seven patients with a normal platelet count reported more frequent perioperative bleeding complications, as well as atypical bleeding such as intramuscular hematoma after injection than the group with low platelet counts. Platelet dysfunction was detected in all (100%) patients by light transmission aggregometry (LTA). Mass-spectrometry (MS) analysis showed an overrepresentation of NUBP2 and ATP5S, both involved in mitochondrial ATP synthesis. Electron microscopy (EM) confirmed abnormal granules in some.</p><p><strong>Conclusions: </strong>We demonstrated increased bleeding tendency due to platelet disorders in the majority of patients with terminal 11q-deletions but also in nonterminal 11q-aberrations. This is confirmed by abnormal platelet function tests and MS. We therefore recommend screening of patients with all forms of 11q-disorders and, when indicated, preventive preoperative measurements and counseling for heavy menstrual bleeding in females.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144689595","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":"Eltrombopag for Bone Marrow Failure in Fanconi Anemia: Results From the Phase II Clinical Trial FANCREV.","authors":"June Iriondo, Josune Zubicaray, Paula Río, Albert Català, María Solsona, Alejandro Sanz, Ana Gómez, Elena Sebastián, Ana de la Cruz, Almudena Galán, Susana Navarro, Lise Larcher, Nagore García de Andoin, Jose Javier Uriz, José Manuel Vagace, Jesús Gonzalez de Pablo, Maria Roser Pujol, Eileen Nicoletti, Jordi Surrallés, Silvia Martín-Prado, Jonathan D Schwartz, Jean Soulier, Juan Antonio Bueren, Julián Sevilla","doi":"10.1111/ejh.70007","DOIUrl":"https://doi.org/10.1111/ejh.70007","url":null,"abstract":"<p><p>Eltrombopag stimulates hematopoiesis in aplastic anemia, and preclinical studies suggest it promotes DNA repair in Fanconi Anemia (FA) hematopoietic stem cells. We conducted a clinical trial to explore its safety and efficacy in bone marrow failure due to FA. Eltrombopag was administered to 8 pediatric patients with one or more significant cytopenias. After 6 months, those who achieved at least a partial response in peripheral blood continued treatment for 6 additional months. Median age was 7 years (4-12), one patient had somatic mosaicism in bone marrow, and two had been treated by gene therapy (GT). At 6 months, three patients (37.5%) achieved a response, persisting in one at 12 months. The three patients with either somatic mosaicism or with GT-corrected cells showed an enhanced increase of corrected cells. One patient required dose modifications due to gastrointestinal intolerance and two because of hepatobiliary laboratory toxicity. No clonal evolution was observed by conventional cytogenetics, but a patient developed a transient somatic variant in RUNX1. In conclusion, eltrombopag did not mediate clinically relevant responses, although a possible impact of dose reductions and treatment duration must be considered. The increase of gene-corrected cells suggests it promoted a proliferative advantage over uncorrected ones. Www.clinicaltrials.gov #NCT06045052.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144642154","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}