EJHaem最新文献

{"title":"Symptomatic Ferric Carboxymaltose-Induced Hypophosphataemia: A Case Series of 11 Patients.","authors":"Marina Weerheim, Heva Korevaar, Maurice Bizino, Gijs Fortrie, Loes Vrolijk, Ted Koster, Faiz Karim","doi":"10.1002/jha2.70382","DOIUrl":"10.1002/jha2.70382","url":null,"abstract":"","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70382"},"PeriodicalIF":1.3,"publicationDate":"2026-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13500041/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148809457","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Complement-Dominant Relapse of Mixed Autoimmune Hemolytic Anemia After Rituximab Response.","authors":"Naru Tomigaki, Seiji Kakiuchi, Akimasa Sakamoto, Shutaro Fujioka, Isamu Harima, Hiroaki Akiyama, Ryotaro Niwa, Ikumi Takagi, Yoko Kozuki, Yoshiharu Miyata, Sou Tanaka, Hiroyuki Tsuji, Nobuko Iwata","doi":"10.1002/jha2.70381","DOIUrl":"10.1002/jha2.70381","url":null,"abstract":"<p><strong>Introduction: </strong>Mixed autoimmune hemolytic anemia (AIHA) may show temporal shifts in its predominant effector mechanism.</p><p><strong>Methods: </strong>We serially assessed clinical course, direct antiglobulin test (DAT) specificity, cold agglutinin activity, complement markers, marrow findings, and immunofixation after rituximab response.</p><p><strong>Results: </strong>During winter relapse, the previously identified <i>κ</i>-restricted CD20-positive population was no longer detectable by marrow flow cytometry. Hemolysis was cold-reactive and complement-dominant. Hemoglobin increased without transfusion after sutimlimab, with C4 recovery and total hemolytic complement activity (CH50) suppression despite persistent cold agglutinin activity. Immunofixation later identified IgG-<i>κ</i> and IgM-<i>κ</i> monoclonal proteins.</p><p><strong>Conclusions: </strong>Serial reassessment may identify complement-mediated relapse in mixed AIHA.</p><p><p>Trial Registration: The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70381"},"PeriodicalIF":1.3,"publicationDate":"2026-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13500038/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148809355","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sequential High-Dose Ruxolitinib and Low-Dose Splenic Irradiation for Pretransplant Management of Splenomegaly Before Allogeneic Hematopoietic Cell Transplantation With Fludarabine/Busulfan-Based Conditioning in Myelofibrosis.","authors":"Peng Ke, Chun Feng, Guoqiang Li, Xiaoyong Chen, Yixuan Cao, Lina Hu, Jiahe Zhang, Linlin Wang, Jihao Zhou","doi":"10.1002/jha2.70383","DOIUrl":"10.1002/jha2.70383","url":null,"abstract":"<p><strong>Background: </strong>Significant splenomegaly remains a major barrier to successful allogeneic hematopoietic cell transplantation (allo-HCT) in myelofibrosis (MF), contributing to delayed engraftment and an increased risk of graft failure. Systemic JAK inhibition and splenic irradiation are commonly used as independent pretransplant strategies; however, evidence regarding their sequential use and integration into contemporary conditioning platforms remains limited.</p><p><strong>Methods: </strong>We conducted a single-center retrospective study of nine patients with MF who underwent allo-HCT between 2020 and 2025. Patients received sequential high-dose ruxolitinib followed by low-dose splenic irradiation (LDSI) as pretransplant spleen-directed therapy prior to a fludarabine/busulfan-based conditioning regimen. Spleen response, engraftment kinetics, and transplant outcomes were evaluated.</p><p><strong>Results: </strong>High-dose ruxolitinib reduced median spleen length from 185 to 172 mm, followed by a further decrease to 137 mm after LDSI. All patients achieved engraftment, with median times to neutrophil and platelet recovery of 15 and 19 days, respectively. The cumulative incidence of any-grade acute graft-versus-host disease (GVHD) was 22.2%, and moderate chronic GVHD occurred in 11.1%. The estimated 2-year overall survival was 88.9%, and the estimated 2-year GVHD-free and relapse-free survival (GRFS) was 76.2%. The estimated 2-year cumulative incidence of relapse was 12.7%, and estimated non-relapse mortality was 11.1%.</p><p><strong>Conclusion: </strong>Sequential high-dose ruxolitinib followed by LDSI appeared feasible and was associated with stepwise pretransplant spleen reduction and encouraging early transplant outcomes following allogeneic HCT with fludarabine/busulfan-based conditioning. Given the small sample size and absence of a comparator group, this combined systemic and local spleen-directed strategy warrants further evaluation in prospective multicenter studies.</p><p><strong>Trial registration: </strong>The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70383"},"PeriodicalIF":1.3,"publicationDate":"2026-08-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13500039/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148809368","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Persistence to Multiple Myeloma Treatments Given at First Relapse-Results From the Australian Real-World OPTIMISE Study.","authors":"Felix Buescher, Steve Su, Ben Waterhouse, Cletus Pinto","doi":"10.1002/jha2.70377","DOIUrl":"10.1002/jha2.70377","url":null,"abstract":"<p><strong>Background: </strong>Daratumumab became publicly available in Australia in 2021 in combination with bortezomib and dexamethasone (DVd), as a second-line (2L) therapy for multiple myeloma (MM). We evaluated real-world persistence to any MM treatment after first relapse before and after DVd availability using the Pharmaceutical Benefits Scheme 10% sample dataset.</p><p><strong>Methods: </strong>Dispensing records for adults receiving 2L MM treatment from January 2018 to December 2020 (Period 1) and January 2021 to April 2025 (Period 2) were analyzed using Kaplan-Meier to determine treatment persistence.</p><p><strong>Results: </strong>Eighty patients were included in Period 1 and 290 in Period 2. Median treatment persistence was longer in Period 2 (15 months) than Period 1 (9.8 months) (hazard ratios [HRs]: 0.46; 99.9% CI: 0.23-0.90; <i>p</i> < 0.001). Treatment persistence in patients who progressed within 18 months on first-line therapy was longer in Period 2 (13 months) than Period 1 (9.2 months) (HR: 0.53; 95% CI: 0.34-0.81; <i>p</i> < 0.01). The Period 2 lenalidomide-refractory cohort (<i>n</i> = 78) demonstrated a median persistence of 11 months (95% CI: 8.3-23 months). The number of lenalidomide-refractory patients in Period 1 (<i>n</i> = 11) was insufficient for comparative analysis.</p><p><strong>Conclusions: </strong>The observed improvements in treatment persistence among patients with MM including those refractory to lenalidomide, following reimbursement-driven availability of DVd, indicate meaningful clinical benefit in routine practice after the introduction of novel therapies, and underscores the need for sustained access to diverse and effective treatment options for relapsed MM.</p><p><p><b>Trial Registration</b>: The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70377"},"PeriodicalIF":1.3,"publicationDate":"2026-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13495057/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148802386","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Correction to \"A Delphi Consensus on Optimising the Care Pathway for Adult Patients With Acute Myeloid Leukaemia (AML): Strategies to Enhance Transplant Accessibility and Feasibility in the United Kingdom\".","authors":"","doi":"10.1002/jha2.70379","DOIUrl":"https://doi.org/10.1002/jha2.70379","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1002/jha2.70353.].</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70379"},"PeriodicalIF":1.3,"publicationDate":"2026-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13495056/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148802345","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Practices of Antifungal Prophylaxis Among Italian Pediatric Oncology-Hematology Centers.","authors":"Martina Colli, Antonio Grasso, Manuela Spadea, Francesca Compagno, Lorenzo Chiusaroli, Paola Muggeo, Alessia Pancaldi, Rosamaria Mura, Daniela Onofrillo, Raffaella De Santis, Beatrice Mongelli, Angelica Barone, Simona Rinieri, Paola Coccia, Erica Ricci, Maura Faraci, Maria Rosaria D'Amico, Federica Galaverna, Pietro Gasperini, Letizia Brescia, Antonella Colombini, Riccardo Masetti, Francesco Baccelli, Simone Cesaro","doi":"10.1002/jha2.70337","DOIUrl":"10.1002/jha2.70337","url":null,"abstract":"<p><strong>Background: </strong>IFD is a significant cause of illness and death among pediatric patients undergoing chemotherapy or HSCT. Application of AFP in children varies widely and is frequently off-label. This survey aims to describe real-world AFP practices across centers within AIEOP and provide an estimate of IFD incidence.</p><p><strong>Methods: </strong>In the second half of 2023, AIEOP centers participated in a web-based questionnaire collecting data on their AFP practices, antifungal agents, and IFD cases from 2021 to 2022.</p><p><strong>Results: </strong>Nineteen out of 44 AIEOP centers (43%) responded. AFP practices varied based on underlying disease and associated risk of IFD: it was used in 100% of centers treating AML, ALL-Rel, and allo-HSCT recipients, in 94.5% of centers for ALL-HR patients, and in 53% of centers with ALL-IR. Usage of AFP was limited (11%-35%) in low-risk patients (ALL-SR, HL, solid tumors). The most frequently used agents were liposomal amphotericin B and posaconazole for high-risk patients, and fluconazole for lower-risk patients. The estimated incidence of IFD was 4.7% (> 10% in ALL-Rel, NHL, allo-HSCT, SAA/AA).</p><p><strong>Conclusions: </strong>This survey shows variability in AFP drug choice. IFD rates remain elevated in high-risk groups. These results underline the need for updated, pediatric-specific national guidelines and antifungal stewardship to improve prophylaxis strategies.</p><p><strong>Trial registration: </strong>The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70337"},"PeriodicalIF":1.3,"publicationDate":"2026-08-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13495061/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148802361","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patient Journey, Treatment Patterns, and Disease Burden of Patients With Idiopathic Hypereosinophilic Syndrome.","authors":"Juliana Schwaab, Paul Dolin, Bo Ding, Priya Jain, Lotte Westerink, Chris Edmonds, Tia Pennant, Oliver-Thomas Carter, Fritha Hennessy, Stephanie Yanjing Chen","doi":"10.1002/jha2.70375","DOIUrl":"https://doi.org/10.1002/jha2.70375","url":null,"abstract":"<p><strong>Introduction: </strong>Idiopathic hypereosinophilic syndrome (I-HES) is a rare disorder characterized by persistent eosinophilia without an identifiable underlying cause, leading to organ damage and dysfunction. The objectives of this study were to describe the real-world demographics, patient journey, treatment patterns, and disease burden of patients with I-HES.</p><p><strong>Methods: </strong>Data were drawn from the Adelphi Real World HES Disease Specific Programme, a cross-sectional survey of physicians and their patients with I-HES in Europe (France, Germany, Italy, Spain, and the UK) and the United States from July to December 2023.</p><p><strong>Results: </strong>The overall population included 117 physicians and 451 patients. Patients were predominately male (62%), White (87%), and the mean (standard deviation [SD]) age was 44.7 (16.1) years. Mean (SD) and median (range) time between symptom onset and I-HES diagnosis was 8.2 (11.1) and 4.2 (0-87.3) months, respectively. Most patients (66%) were treated with corticosteroids, and the mean (SD) and median (range) doses of oral and/or parenteral corticosteroids were 19.1 (20.3) and 10.0 (1.0-100.0) mg/day, respectively. The use of interleukin-5/receptor alpha targeted therapies was low (23%). Patients had a mean (SD) of 7.7 (6.4) symptoms at diagnosis; 58% had organ system damage attributed to I-HES, and disease was perceived by physicians as moderate or severe in 72%. Patient-reported health-related quality of life, work productivity, and fatigue were negatively impacted in patients with deteriorating or moderate-to-severe disease.</p><p><strong>Conclusion: </strong>These findings underscore the need for greater disease awareness to support timely diagnosis with use of targeted, corticosteroid-sparing treatments to improve patient outcomes.</p><p><strong>Trial registration: </strong>The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70375"},"PeriodicalIF":1.3,"publicationDate":"2026-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13481312/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148802335","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Choroidal Leakage during Daratumumab Therapy: A Desensitization-Based Strategy for Treatment Continuation in Relapsed/Refractory Multiple Myeloma.","authors":"Metban Mastanzade, Zafer Cebeci, Kıvanç Koruk, Kerem Yiğit, Gül Beste Can, Sevgi Kalayoglu Beşışık","doi":"10.1002/jha2.70364","DOIUrl":"https://doi.org/10.1002/jha2.70364","url":null,"abstract":"<p><strong>Background: </strong>Daratumumab is widely used in relapsed/refractory multiple myeloma, but ocular adverse events are rare and not well-characterized.</p><p><strong>Case presentation: </strong>A 68-year-old woman developed acute blurred vision during the second day of her first daratumumab infusion. Multimodal imaging, including fluorescein angiography, indocyanine green angiography, and optical coherence tomography (OCT), demonstrated bilateral choroidal leakage with subretinal fluid and choroidal vascular changes.</p><p><strong>Management and outcome: </strong>Daratumumab was temporarily discontinued due to suspected drug-related ocular toxicity. Following complete symptom resolution, treatment was successfully reintroduced using a 12-step desensitization protocol with close monitoring. No recurrence of ocular symptoms was observed, and follow-up imaging showed regression of subretinal fluid.</p><p><strong>Conclusion: </strong>This case highlights a rare ocular complication associated with daratumumab and suggests that desensitization may enable safe continuation of therapy in selected patients with non-vision-threatening findings.</p><p><strong>Trial registration: </strong>The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70364"},"PeriodicalIF":1.3,"publicationDate":"2026-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13472061/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148765486","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Real-World Study Characterizing a US Population of Patients Receiving Rituximab With Gemcitabine and Oxaliplatin for Relapsed or Refractory Large B-Cell Lymphoma.","authors":"L Elizabeth Budde, Adam J Olszewski, Bei Hu, Nikesh N Shah, Connie Lee Batlevi, Shen Yin, Iris To, Michael C Wei, Martine Joanna Kallemeijn, Lisa Musick, Linda Lundberg, Natasha Shamas, Mark Dixon, Navdeep Pal, Ashwini Shewade, Jason Westin","doi":"10.1002/jha2.70351","DOIUrl":"https://doi.org/10.1002/jha2.70351","url":null,"abstract":"<p><strong>Introduction: </strong>Data for the use of rituximab, gemcitabine, and oxaliplatin (R-GemOx) for relapsed/refractory (R/R) large B-cell lymphoma (LBCL) in the United States are limited. This retrospective observational study characterizes R-GemOx treatment patterns and outcomes among patients with R/R LBCL from the nationwide, longitudinal Flatiron Health electronic health record-derived database comprising patient-level data primarily from US community oncology practices.</p><p><strong>Methods: </strong>Patients diagnosed with LBCL who initiated treatment with R-GemOx in the second or later line of therapy between 2011 and 2023, were included. Patient characteristics, treatment patterns, and efficacy outcomes were assessed.</p><p><strong>Results: </strong>Of 281 patients, most (66.2%) were treated at community centers. Overall response rate was 45.4% (95% confidence interval [CI]: 38.1-52.9); complete response (CR) rate was 22.2% (95% CI: 16.4-28.8). Median progression-free and overall survival (OS) were 2.8 months (95% CI: 2.6-3.7) and 12.7 months (95% CI: 10.6-17.1), respectively. In patients who received R-GemOx in the second line, the CR rate was higher and the median OS was longer versus those who received it in later lines (CR rate, 26.3% vs. 17.4%; median OS, 19.6 vs. 10.0 months, respectively).</p><p><strong>Conclusion: </strong>These data offer a real-world benchmark for patterns of use and outcomes of R-GemOx in patients with R/R LBCL in the United States. <b>Trial Registration</b>: The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70351"},"PeriodicalIF":1.3,"publicationDate":"2026-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13472062/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148763754","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Anemia and Patient-Reported Outcomes in Patients With Paroxysmal Nocturnal Hemoglobinuria: A Real-World Observational Study.","authors":"Esther Natalie Oliva, Antonio M Risitano, Eros Di Bona, Alessandro Maria Vannucchi, Ilaria Maria Dellfino, Antonio De Vivo, Valentina Giai, Rosario Notaro, Giuseppe Iannì, Eloise Beggiato, Simona Sica, Gaetano La Barba, Sneha Balakrishnan, Joanna Large, Tanuja Thavarajah, Simona Raso, Raffaele Fontana, Wilma Barcellini, Bruno Fattizzo, Roochi Trikha, Luana Marano, Jens Panse, Austin G Kulasekararaj","doi":"10.1002/jha2.70376","DOIUrl":"10.1002/jha2.70376","url":null,"abstract":"<p><strong>Introduction: </strong>Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder characterized by complement-mediated hemolysis, thrombosis, bone marrow failure, and anemia. Although current treatments improve outcomes, patients still experience hemolysis and could remain anemic with a negative impact on quality of life (QoL). The aim of this study was to assess whether patient-reported outcomes (PROs) distinguish PNH patients with/without hemolysis and evaluate factors associated with PROs.</p><p><strong>Methods: </strong>In this international, multicenter, observational study, 97 PNH patients witfrom Italy and the UK completed the EORTC QLQ-C30 and QLQ-AA/PNH measures at baseline and after 2 weeks. Clinical, biochemical, and treatment data were collected. Hemolysis was defined as LDH ≥ 1.5 × upper normal limit. PRO scores were compared between patients above and below this threshold. Associations of PROs with patient and disease factors were analyzed.</p><p><strong>Results: </strong>Of 97 patients (mean age 49 ± 15), 24.7% were anemic (Hb < 10 g/dL) and 16% had hemolysis. QoL did not differ significantly by hemolysis status. However, anemia was associated with significantly lower EORTC QLQ-C30 global health and functional scores (<i>p</i> < 0.05), particularly in physical, role, and social domains, and with worse QLQ-AA/PNH emotional, cognitive, and physical functioning, as well as greater illness intrusiveness and stigmatization. Fatigue, dyspnea, and pain were common symptoms in anemic patients. QLQ-AA/PNH scores showed high internal consistency (Cronbach's <i>α</i> > 0.7) and test-retest reliability (ICC: 0.72-0.95).</p><p><strong>Conclusions: </strong>Anemia, but not residual hemolysis, was associated with impaired QoL across multiple domains. Addressing anemia alongside hemolysis may improve PROs in the management of PNH.</p><p><strong>Trial registration: </strong>The authors have confirmed clinical trial registration is not needed for this submission.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"7 4","pages":"e70376"},"PeriodicalIF":1.3,"publicationDate":"2026-08-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13460258/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148714851","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}