Margaret Stalker, Alfred Garfall, Adam Cohen, Dan T Vogl, Mia Djulbegovic, Sandra Susanibar-Adaniya, Edward Stadtmauer, Oxana Megherea, Adam J Waxman
{"title":"Safety and Efficacy of Teclistamab in Patients With Relapsed or Refractory AL Amyloidosis.","authors":"Margaret Stalker, Alfred Garfall, Adam Cohen, Dan T Vogl, Mia Djulbegovic, Sandra Susanibar-Adaniya, Edward Stadtmauer, Oxana Megherea, Adam J Waxman","doi":"10.1111/ejh.14348","DOIUrl":"https://doi.org/10.1111/ejh.14348","url":null,"abstract":"<p><strong>Introduction: </strong>Teclistamab has demonstrated deep responses in patients with multiple myeloma in the MajesTEC-1 study. However, the safety and efficacy of teclistamab in patients with AL amyloidosis are unknown.</p><p><strong>Methods: </strong>We retrospectively analyzed patients with biopsy-proven relapsed/refractory AL amyloidosis who were treated with teclistamab from December 2022 to February 2024 at the University of Pennsylvania. The data cutoff was 2/29/24. Adverse events (AE) were extracted from the electronic medical record. Patients were assessed for hematologic and organ response per consensus guidelines.</p><p><strong>Results: </strong>Eight patients were included in this case series: median age 63 (range 59-67), 75% female, 88% White. All eight patients achieved at least very good partial response (VGPR) and had normalization of free light chains (FLC), and six (75%) patients achieved undetectable FLC levels. Of the six patients with immunofixation completed, all six (100%) achieved hematologic complete response (hCR). The median time to hematologic VGPR and hCR was 13 days (range 12-18 days) and 88 days (range 32-150 days), respectively. The median duration of follow-up was 8.5 months (range 1-14 months). Of the five patients with cardiac involvement, four (80%) achieved a cardiac response. Of the seven patients with renal involvement, two patients already achieved renal response prior to teclistamab, and of the remaining five, three (60%) achieved renal response. Six patients (75%) developed low-grade cytokine release syndrome (CRS). No patients developed ICANS. Neutropenia and AKI both occurred in 25% of patients, respectively.</p><p><strong>Conclusions: </strong>In this series of patients, teclistamab showed outstanding depth of response and was well-tolerated. Teclistamab shows promise in treating patients with relapsed AL amyloidosis.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142647133","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}
Sara Redondo, María Costa, Maria-Estela Moreno-Martinez, Miguel Arguello-Tomas, Mireia Riba, Olga Aso, Eva Iranzo, Albert Esquirol, Jorge Sierra, Javier Briones, Rodrigo Martino, Edgar Zapico, Irene García-Cadenas
{"title":"Development and Clinical Validation of Liquid Chromatography-Tandem Mass Spectrometry for Measuring Ruxolitinib in Steroid-Refractory Graft-Versus-Host Disease: A First Step Towards Optimized Treatment.","authors":"Sara Redondo, María Costa, Maria-Estela Moreno-Martinez, Miguel Arguello-Tomas, Mireia Riba, Olga Aso, Eva Iranzo, Albert Esquirol, Jorge Sierra, Javier Briones, Rodrigo Martino, Edgar Zapico, Irene García-Cadenas","doi":"10.1111/ejh.14349","DOIUrl":"https://doi.org/10.1111/ejh.14349","url":null,"abstract":"<p><strong>Objective: </strong>This non-interventional, prospective, single-center study aimed to develop a technique to measure ruxolitinib (RUX) concentrations and provide preliminary data on the distribution of plasma drug levels in patients with steroid refractory (SR) GvHD.</p><p><strong>Methods: </strong>Between April 2023 and May 2024, we analyzed 48 blood samples from 29 patients with SR-GvHD.</p><p><strong>Results: </strong>Median individual plasma concentrations varied across different RUX doses and largely overlapped: 39.2 ng/mL at 10 mg b.i.d (range: 0-73), 13.1 ng/mL at 10-5 mg (range, 6.1-35.6), and 31.6 ng/mL at 5 mg b.i.d (range: 0.7-99.9). Samples taken under non-optimal temperature conditions showed a lower median concentration of 0.77 ng/mL (range: 0-7.4 ng/mL). The four patients who did not respond at days +28 and +180 after RUX initiation (3 with lower gastrointestinal aGvHD, and 1 with ocular, hepatic, and pulmonary cGvHD) showed a median concentration of only (7.4 ng/mL (range, 0-29) ng/mL) with full dosing.</p><p><strong>Conclusions: </strong>The introduction and validation of a liquid chromatography-tandem mass spectrometry method for quantifying plasma RUX concentrations was feasible in our center. Administering predetermined and fixed doses of RUX in patients with SR-GvHD showed highly variable and overlapping plasma drug concentrations. This underscores the potential importance of RUX- pharmacokinetics (PK) monitoring.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638613","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}
Kaiya Hassan, Robert Kinan, Ashley Casey, Miranda Dermady, Britta Mizuki, Katerina Stanilova, Heather Savage, Helen Yuan, Emma Hillis, Connor Bertaut, Taylor Guillory, Eric Coons
{"title":"Direct Oral Anticoagulants Versus Warfarin in Patients With Isolated Heparin-Induced Thrombocytopenia or Heparin-Induced Thrombocytopenia With Thrombosis.","authors":"Kaiya Hassan, Robert Kinan, Ashley Casey, Miranda Dermady, Britta Mizuki, Katerina Stanilova, Heather Savage, Helen Yuan, Emma Hillis, Connor Bertaut, Taylor Guillory, Eric Coons","doi":"10.1111/ejh.14350","DOIUrl":"https://doi.org/10.1111/ejh.14350","url":null,"abstract":"<p><p>No existing studies compare oral anticoagulants to treat heparin-induced thrombocytopenia with or without thrombosis (HIT/HITT). This retrospective study evaluated thrombotic and bleeding outcomes in adults treated for HIT/HITT with a direct oral anticoagulant (DOAC) or warfarin between 2012 and 2023 within the Ochsner Health System. Patients with mechanical heart valves, valvular atrial fibrillation, antiphospholipid syndrome, active malignancy, or venous thromboembolism (VTE) within the previous 6 months were excluded. The primary outcome was a composite of new or progressive VTE or arterial thromboembolism. Secondary outcomes included major and clinically relevant non-major bleeding, duration of hospitalization, time to platelet recovery, and incidence of skin necrosis, gangrene, and amputation. Forty-nine patients receiving a DOAC and 30 patients receiving warfarin were included. Baseline characteristics were similar between cohorts. There were non-statistically significant increased rates of both the primary outcome (8.9% vs. 4.3%, p = 0.65) and the composite bleeding outcome (32.7% vs. 23.3%, p = 0.37) in the DOAC cohort. Larger, prospective studies are needed to confirm these findings.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-11-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142617508","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}
T Richardson, G Kobbe, R Fenk, T Schroeder, M Crysandt, C Neuerburg, Tobias A W Holderried, D Schütte, P Gödel, M Hallek, C Scheid, U Holtick
{"title":"Allogeneic Hematopoietic Stem Cell Transplantation in Refractory Multiple Myeloma-A Retrospective Multicenter Analysis.","authors":"T Richardson, G Kobbe, R Fenk, T Schroeder, M Crysandt, C Neuerburg, Tobias A W Holderried, D Schütte, P Gödel, M Hallek, C Scheid, U Holtick","doi":"10.1111/ejh.14346","DOIUrl":"https://doi.org/10.1111/ejh.14346","url":null,"abstract":"<p><p>A growing list of therapies available for patients with multiple myeloma (MM) results in deep response rates, but eventually almost all patients relapse. Allogeneic hematopoietic cell transplantation (allo-SCT) is a familiar approach for MM, but responses are often short and side effects burdensome. Simultaneously, allo-SCT provides a unique platform on which novel immune therapies can be employed to improve clinical outcomes. Our work describes the characteristics and outcomes of 128 refractory myeloma patients who underwent allo-SCT at five German centers between 2010 and 2021. The median number of therapies before the transplant was 6. With a median follow-up of 6, 4 years, the median progression-free survival and overall survival were 7 and 19 months, respectively. NRM was 28% after 6 years. OS and PFS were 61% and 45% at 1 year, 49% and 34% at 2 years, and 38% and 25% at 6 years. Achieving a CR before transplant was the single most significant variable before transplant. Allo-SCT yet remains an option for fit patient's refractory to all other treatments available. It is potentially curative for a subset of patients. Finding the characteristics of patients with durable remissions is key to sparing unnecessary toxicity for those unlikely to benefit.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142617478","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}
Eric J. Niesor, Anne Perez, Serge Rezzi, Andrew Hodgson, Stephane Canarelli, Gregoire Millet, Tadej Debevec, Claire Bordat, Elie Nader, Philippe Connes
{"title":"Featured Cover","authors":"Eric J. Niesor, Anne Perez, Serge Rezzi, Andrew Hodgson, Stephane Canarelli, Gregoire Millet, Tadej Debevec, Claire Bordat, Elie Nader, Philippe Connes","doi":"10.1111/ejh.14344","DOIUrl":"https://doi.org/10.1111/ejh.14344","url":null,"abstract":"<p>The cover image is based on the Article <i>Plasma monomeric ApoA1 and high-density lipoprotein bound ApoA1 are markedly decreased and associated with low levels of lipophilic antioxidants in sickle cell disease: A potential new pathway for therapy</i> by Eric J. Niesor et al., https://doi.org/10.1111/ejh.14288\u0000 \u0000 <figure>\u0000 <div><picture>\u0000 <source></source></picture><p></p>\u0000 </div>\u0000 </figure></p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"113 6","pages":"i"},"PeriodicalIF":2.3,"publicationDate":"2024-11-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14344","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142588138","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Fortunato Morabito, Enrica Antonia Martino, Maria Elena Nizzoli, Annalisa Talami, Stefano Pozzi, Massimo Martino, Antonino Neri, Massimo Gentile
{"title":"Comparative Analysis of Bispecific Antibodies and CAR T-Cell Therapy in Follicular Lymphoma","authors":"Fortunato Morabito, Enrica Antonia Martino, Maria Elena Nizzoli, Annalisa Talami, Stefano Pozzi, Massimo Martino, Antonino Neri, Massimo Gentile","doi":"10.1111/ejh.14335","DOIUrl":"10.1111/ejh.14335","url":null,"abstract":"<p>The treatment landscape for relapsed/refractory follicular lymphoma (RR-FL) is marked by a pivotal debate between chimeric antigen receptor T-cell (CAR-T) therapy and bispecific antibodies (BsAbs). While both CAR-T therapy and BsAbs target similar immunobiology and molecular markers, their efficacy comparisons are hindered by the lack of direct clinical trial comparisons. Key trials, such as the ZUMA-5 study, underscore axicabtagene ciloleucel (axi-cel)'s efficacy in treating RR-FL, achieving a 79% complete response rate with a median duration of response exceeding 3 years. Similarly, lisocabtagene maraleucel (liso-cel) in the TRANSCEND FL study reports a 94% complete response rate, emphasizing robust outcomes in heavily pretreated patients. Among BsAbs, mosunetuzumab showed promise in the GO29781 trial, with a 62% overall response rate in heavily pretreated RR-FL patients. Thus, CAR-T therapy offers potential curative benefits with a single infusion. However, its efficacy is tempered by significant adverse events such as cytokine release syndrome (CRS), neurotoxicity, and cytopenias, requiring specialized management and patient monitoring. In contrast, BsAbs provide a more tolerable treatment option counterbalancing by lower response rates and frequent dosing requirements. Personalized treatment strategies are crucial because of these distinct efficacy and safety profiles. When considering cost-effectiveness, both therapies need to be evaluated in the context of their clinical outcomes and quality of life improvements. Cost-effectiveness considerations are essential; while CAR-T therapies incur higher initial costs, their potential for long-term remission may mitigate expenses associated with repeated treatments or hospitalizations. Future research into resistance mechanisms and optimal therapeutic sequencing will further refine RR-FL management strategies.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"4-16"},"PeriodicalIF":2.3,"publicationDate":"2024-10-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14335","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497482","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Therapy-Related Acute Promyelocytic Leukemia: Case Series and Current Insights","authors":"Antonella Bruzzese, Enrica Antonia Martino, Caterina Labanca, Francesco Mendicino, Eugenio Lucia, Virginia Olivito, Rosellina Morelli, Teresa Rossi, Antonino Neri, Fortunato Morabito, Massimo Gentile, Ernesto Vigna","doi":"10.1111/ejh.14327","DOIUrl":"10.1111/ejh.14327","url":null,"abstract":"<p>Therapy-related acute promyelocytic leukemia (t-APL) is rare and often linked to previous treatment with alkylating agents or topoisomerase II inhibitors. This report describes three cases of t-APL treated at the Haematology Department of Cosenza Hospital between 2022 and 2024, which occurred after alkylating agents and exemestane, alkylating agents and radiation therapy, alkylating agents, taxane, and checkpoint inhibitor, respectively. Each case was managed with a different therapeutic approach. The first case involved a 71-year-old man with colorectal and breast cancer, who developed low-risk t-APL and achieved complete remission (CR) with ATRA alone. A second 71-year-old man case with colorectal cancer developed high-risk t-APL with PML/RARA and FLT3-ITD fusion transcripts; he achieved CR with idarubicin and ATRA despite severe sepsis and acute heart failure. The third case involved a 74-year-old man with lung squamous cell carcinoma who developed intermediate-risk t-APL following chemoimmunotherapy but unfortunately succumbed to pseudotumor cerebri complications during induction therapy.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"195-198"},"PeriodicalIF":2.3,"publicationDate":"2024-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14327","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461101","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
R. Liévin, B. Burroni, E. Balducci, P. Palmic, J. Decroocq, B. Deau-Fischer, P. Franchi, M. Vignon, J. Zerbit, A. S. Cottereau, A. Touzart, P. Villarese, S. Kaltenbach, L. Lhermitte, V. Asnafi, D. Bouscary, L. Willems
{"title":"Biological Findings and Clinical Outcomes in Patients Treated With R-CHOP Plus High-Dose Methotrexate as First-Line Therapy in Large B-Cell Lymphoma With Testis Involvement","authors":"R. Liévin, B. Burroni, E. Balducci, P. Palmic, J. Decroocq, B. Deau-Fischer, P. Franchi, M. Vignon, J. Zerbit, A. S. Cottereau, A. Touzart, P. Villarese, S. Kaltenbach, L. Lhermitte, V. Asnafi, D. Bouscary, L. Willems","doi":"10.1111/ejh.14325","DOIUrl":"10.1111/ejh.14325","url":null,"abstract":"<p>Primary testicular lymphoma (PTL) is a rare occurrence of diffuse large B-cell lymphoma (DLBCL) that accounts for 1%–2% of all cases. Nodal DLBCL with testis involvement (DLBCL-T) and PTL are associated with poor prognosis, with high incidence of central nervous system relapse. Fifteen patients (median age 60 years) with PTL (<i>n</i> = 5) or DLBCL-T (<i>n</i> = 10) received high-dose methotrexate + R-CHOP. Overall, complete response (CR) rate was 73% and overall response rate 86%. With a 3.9-year median follow-up, 100% of patients with PTL had CR and none relapsed. On the contrary, 55% of DLBCL-T patients achieved CR among which only one was still in remission at the end of follow-up. Molecular parallels between PTL and Primary CNS Lymphoma (PCNSL) suggest shared origins, urging further research for tailored treatments and enhanced understanding of these lymphomas' biology.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"186-194"},"PeriodicalIF":2.3,"publicationDate":"2024-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14325","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461083","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Maja Dam Andersen, Katharina Wolter, Marie Beck Hairing Enemark, Mette Abildgaard Pedersen, Lars Christian Gormsen, Kristina Lystlund Lauridsen, Jørn Starklint, Stephen Jacques Hamilton-Dutoit, Francesco d'Amore, Maja Ludvigsen, Bent Honoré, Peter Kamper
{"title":"Proteomic Profiling of Lymph Nodes Differentiates Classic Hodgkin Lymphoma With and Without Skeletal Involvement","authors":"Maja Dam Andersen, Katharina Wolter, Marie Beck Hairing Enemark, Mette Abildgaard Pedersen, Lars Christian Gormsen, Kristina Lystlund Lauridsen, Jørn Starklint, Stephen Jacques Hamilton-Dutoit, Francesco d'Amore, Maja Ludvigsen, Bent Honoré, Peter Kamper","doi":"10.1111/ejh.14326","DOIUrl":"10.1111/ejh.14326","url":null,"abstract":"<p>Classic Hodgkin lymphoma (CHL) is a highly curable disease, even in advanced stages. Controversy remains over whether bone involvement negatively affects overall and progression-free survival in patients treated with intensive chemotherapy regimens. Whether cases that present with bone lesions harbor specific tumor microenvironmental features is unknown. We investigated protein expression in diagnostic lymph node biopsies from CHL patients with and without skeletal involvement at diagnosis to identify potential markers of skeletal disease. Protein expression patterns in diagnostic formalin-fixed paraffin-embedded lymphoma lymph node samples from CHL patients were analyzed by nano-liquid chromatography–tandem mass spectrometry. Patients were grouped according to skeletal involvement, which was defined as the presence of one or more FDG-avid lesions on a diagnostic FDG-PET/CT scan. Protein profiles identified patients with skeletal disease at diagnosis and showed disrupted cellular pathways, including immune system processes, cell adhesion, and cell growth/survival. Immunohistochemical evaluation also demonstrated differential expressions of angiotensin-converting enzyme (ACE), intercellular adhesion molecule 3 (ICAM3), integrin alpha-X (ITGAX), and calreticulin (CALR). In conclusion, proteomics identified altered protein expression profiles in lymph nodes among CHL cases presenting with disease disseminated to the skeletal system, which implies altered disease pathogenesis for these patients.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"173-185"},"PeriodicalIF":2.3,"publicationDate":"2024-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14326","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461099","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Cecilie Maria Lüthje Clausen, Eva Funding, Anders Tolver, Mary Jarden
{"title":"Adherence and Quality of Life in Adult Patients With Haemophilia A, Haemophilia B and Von Willebrand Disease: A National Cross-Sectional Survey","authors":"Cecilie Maria Lüthje Clausen, Eva Funding, Anders Tolver, Mary Jarden","doi":"10.1111/ejh.14324","DOIUrl":"10.1111/ejh.14324","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>New treatments for patients with bleeding disorders (PWB) have emerged, including products with extended half-life and subcutaneous administration. These less frequent treatments can potentially enhance quality of life (QoL), but adherence becomes critically important.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To investigate adherence and QoL among PWB and explore the correlation between treatment adherence and QoL in adult patients with haemophilia A (HA), haemophilia B (HB) and Von Willebrand disease (vWD) in Denmark.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Method</h3>\u0000 \u0000 <p>This survey used disease-specific patient-reported questionnaires: Veritas-PRO and Veritas-PRN to measure adherence, and Haemo-A-QoL and VWD-QoL to assess QoL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Responses were obtained from 149 patients with HA, 32 with HB and 118 with vWD. Adherence was reported by 87.1% of patients on prophylaxis and 71.2% of patients treated on demand, according to Veritas-PRO and Veritas-PRN cut-off scores. High QoL was generally reported, decreasing with age in HA and HB, but not in vWD.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Danish patients with HA, HB and vWD reported high QoL and high adherence to prescribed treatments. There was no correlation between treatment adherence and QoL among the different patient groups. These findings highlight the need for further research to better understand adherence behaviours and identify opportunities to further improve QoL in PWB.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"114 1","pages":"164-172"},"PeriodicalIF":2.3,"publicationDate":"2024-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.14324","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142399771","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}