European Journal of Haematology最新文献_第2页

Safety of Crovalimab Versus Eculizumab in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH): Pooled Results From the Phase 3 COMMODORE Studies. Crovalimab与Eculizumab对阵发性夜间血红蛋白尿症（PNH）患者的安全性对比：COMMODORE 3 期研究的汇总结果。

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-02-01 Epub Date: 2024-11-13 DOI: 10.1111/ejh.14339

Alexander Röth, Rong Fu, Guangsheng He, Hazzaa Alzahrani, Sheng-Chieh Chou, Yosr Hicheri, Maciej Kaźmierczak, Viviane Lacorte Recova, Michihiro Uchiyama, Ana-Maria Vladareanu, Leigh Beveridge, Simon Buatois, Muriel Buri, Nicolo Compagno, Dayu Shi, Nadiesh Balachandran, Sasha Sreckovic, Phillip Scheinberg

{"title":"Safety of Crovalimab Versus Eculizumab in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH): Pooled Results From the Phase 3 COMMODORE Studies.","authors":"Alexander Röth, Rong Fu, Guangsheng He, Hazzaa Alzahrani, Sheng-Chieh Chou, Yosr Hicheri, Maciej Kaźmierczak, Viviane Lacorte Recova, Michihiro Uchiyama, Ana-Maria Vladareanu, Leigh Beveridge, Simon Buatois, Muriel Buri, Nicolo Compagno, Dayu Shi, Nadiesh Balachandran, Sasha Sreckovic, Phillip Scheinberg","doi":"10.1111/ejh.14339","DOIUrl":"10.1111/ejh.14339","url":null,"abstract":"Objectives: To evaluate the tolerability of crovalimab versus eculizumab in C5 inhibitor (C5i)-naive and -experienced patients with PNH from COMMODORE 2, 3 and 1 (NCT04434092, NCT04654468 and NCT04432584).Methods: Pooled safety data were assessed in the total crovalimab and eculizumab populations and by C5i-naive versus C5i-switched status in patients receiving crovalimab. Analyses include 6.5 months of additional follow-up from the COMMODORE 2 and 1 primary analyses.Results: COMMODORE safety data (crovalimab, 393 patients [naive, 192 patients; switched, 201 patients]; eculizumab, 111 patients) were analysed. The total patient years (PY) were 503.9 and 51.1 in the total crovalimab and eculizumab populations, respectively, with 471 and 581 adverse events (AEs) per 100 PY. Serious infection rates were 8.9 and 13.7 AEs per 100 PY, respectively; no meningococcal infections were reported. Fatal AEs occurred in eight (2%) patients receiving crovalimab (naive, six patients; switched, two patients) and one (1%) receiving eculizumab, all treatment unrelated. In C5i-switched patients, 39 (19%) had transient immune complex reactions (risk when switching between C5i and crovalimab); the majority were Grades 1-2 arthralgia and rash, and 16 (8%) had Grade 3 events.Conclusions: Crovalimab's safety profile was consistent with eculizumab's and was generally comparable between C5i-naive and C5i-switched patients.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":"373-382"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11707819/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142617517","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}

引用次数: 0

Delineating Notch1 and Notch2: Receptor-Specific Significance and Therapeutic Importance of Pinpoint Targeting Strategies for Hematological Malignancies. 划分 Notch1 和 Notch2：Notch1和Notch2：受体特异性意义和血液恶性肿瘤精确靶向策略的治疗重要性。

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-02-01 Epub Date: 2024-11-12 DOI: 10.1111/ejh.14312

Priyadharshini Tamizhmani, Banumathi Balamurugan, Kishore Thirunavukarasu, Velayuthaprabhu Shanmugam, Selvakumar Subramaniam, Thirunavukkarasu Velusamy

{"title":"Delineating Notch1 and Notch2: Receptor-Specific Significance and Therapeutic Importance of Pinpoint Targeting Strategies for Hematological Malignancies.","authors":"Priyadharshini Tamizhmani, Banumathi Balamurugan, Kishore Thirunavukarasu, Velayuthaprabhu Shanmugam, Selvakumar Subramaniam, Thirunavukkarasu Velusamy","doi":"10.1111/ejh.14312","DOIUrl":"10.1111/ejh.14312","url":null,"abstract":"Notch1 and Notch2, transmembrane receptors belonging to the Notch family, are pivotal mediators of intercellular communication and have profound implications including cell fate determination, embryonic development, and tissue homeostasis in various cellular processes. Despite their structural homology, Notch1 and Notch2 exhibit discrete phenotypic characteristics and functional nuances that necessitate their individualized targeting in specific medical scenarios. Aberrant Notch signaling, often driven by the dysregulated activity of one receptor over the other, is implicated under various pathological conditions. Notch1 dysregulation is frequently associated with T-cell acute lymphoblastic leukemia, whereas Notch2 perturbations are linked to B-cell malignancies and solid tumors, including breast cancer. Hence, tailored therapeutic interventions that selectively inhibit the relevant Notch receptor need to be devised to disrupt the signaling pathways driving the specific disease phenotype. In this review, we emphasize the importance of distinct tissue-specific expression patterns, functional divergence, disease-specific considerations, and the necessity to minimize off-target effects that collectively underscore the significance of \"individualized\" targeting for Notch1 and Notch2. This comprehensive review sheds light on the receptor-specific characteristics of Notch1 and Notch2, providing insights into their roles in cellular processes and offering opportunities for developing tailored therapeutic interventions in the fields of biomedical research and clinical practice.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":"213-230"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142617492","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}

引用次数: 0

Favorable Prognosis in Patients With Multiple Myeloma and Lenalidomide-Induced Skin Rash: A Multicenter Retrospective Study. 多发性骨髓瘤和来那度胺诱发皮疹患者的良好预后：一项多中心回顾性研究

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-02-01 Epub Date: 2024-10-22 DOI: 10.1111/ejh.14333

Ayumi Nakamura, Yuta Kimura, Yuka Tanaka, Daisuke Tsuchimoto, Atsuhiko Naruse, Tetsufumi Kanematsu, Kento Takeuchi, Nobukazu Tomita, Kenji Miyazawa, Tomohiro Fukuoka, Akiya Mori, Shinya Tamaki, Aiko Fujioka, Satoshi Yokoyama, Yoshiaki Ikeda, Hirokazu Nagai

{"title":"Favorable Prognosis in Patients With Multiple Myeloma and Lenalidomide-Induced Skin Rash: A Multicenter Retrospective Study.","authors":"Ayumi Nakamura, Yuta Kimura, Yuka Tanaka, Daisuke Tsuchimoto, Atsuhiko Naruse, Tetsufumi Kanematsu, Kento Takeuchi, Nobukazu Tomita, Kenji Miyazawa, Tomohiro Fukuoka, Akiya Mori, Shinya Tamaki, Aiko Fujioka, Satoshi Yokoyama, Yoshiaki Ikeda, Hirokazu Nagai","doi":"10.1111/ejh.14333","DOIUrl":"10.1111/ejh.14333","url":null,"abstract":"Although lenalidomide is an essential treatment for multiple myeloma (MM), skin rashes are a common adverse event. This retrospective study aimed to examine the association between skin rash development during lenalidomide treatment and the prognosis of relapsed/refractory MM. All patients who received lenalidomide at 10 hospitals between July 2009 and December 2015 were included in the study. The relationship of skin rash development with disease progression and survival was evaluated. Multivariate analysis was performed to identify factors affecting disease progression or survival, including skin rash. Of the 245 patients analyzed, 70 developed skin rashes. The median progression-free survival (PFS) of patients with skin rashes was 22.4 months, whereas the median PFS for patients who did not develop skin rashes was 10.5 months (p = 0.003). The median overall survival for patients with and without skin rash was 42.6 and 24.6 months, respectively (p = 0.013). Multivariate regression analysis showed that skin rash was an independent prognostic factor for PFS (p = 0.009). In this study, patients with skin rashes during lenalidomide treatment had significantly better PFS than those without such symptoms, indicating that lenalidomide-associated skin rashes may be a predictor of clinical outcomes in patients with MM.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":"267-275"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142497484","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}

引用次数: 0

Inferior Overall Survival After Haploidentical Donor Lymphocyte Infusions in Relapsed Myeloid Neoplasms. 复发性髓系肿瘤患者接受同种异体淋巴细胞输注后的总体生存率较低。

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-02-01 Epub Date: 2024-11-05 DOI: 10.1111/ejh.14340

Tobias Matthieu Benoit, Adrian Bachofner, Nathan Wolfensberger, Yvonne Zaugg-Berger, Markus Gabriel Manz, Dominik Schneidawind

{"title":"Inferior Overall Survival After Haploidentical Donor Lymphocyte Infusions in Relapsed Myeloid Neoplasms.","authors":"Tobias Matthieu Benoit, Adrian Bachofner, Nathan Wolfensberger, Yvonne Zaugg-Berger, Markus Gabriel Manz, Dominik Schneidawind","doi":"10.1111/ejh.14340","DOIUrl":"10.1111/ejh.14340","url":null,"abstract":"Objectives: Allogeneic hematopoietic stem cell transplantation (HSCT) effectively treats high-risk myeloid neoplasms, but relapses post-HSCT, particularly in acute myeloid leukemia (AML) and myelodysplastic neoplasms (MDS), pose significant challenges. Donor lymphocyte infusion (DLI) has been utilized, but its effectiveness, especially in haploidentical settings, remains insufficiently clarified, and graft-versus-host disease (GvHD) poses a substantial risk.Methods: In this retrospective cohort study, 57 patients with AML or MDS who received DLI after allogeneic HSCT at our center from 2002 to 2023 were analyzed. Herein, only preemptively or therapeutically applied DLI were included, and endpoints included overall survival (OS), progression-free survival (PFS), and GvHD incidence post-DLI.Results: Median OS after DLI was 517 days, with a 1-year OS of 62.5%. Factors associated with longer OS included patient age, HLA-identical donor, post-HSCT treatment naivety, and preemptive DLI indication. Haploidentical DLI was associated with inferior OS compared to HLA-identical DLI; however, PFS and GvHD incidence post-DLI did not differ significantly.Conclusions: Our study findings indicate that OS rate is inferior in patients with relapsed AML or MDS treated with haploidentical DLI in comparison to those who received HLA-identical DLI. Given the limitations of haploidentical DLI, alternative strategies, such as higher cell doses or combination treatment approaches, warrant further investigation.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":"315-324"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11707813/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142582382","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}

引用次数: 0

Pacritinib Response Is Associated With Overall Survival in Myelofibrosis: PERSIST-2 Landmark Analysis of Survival. 帕克替尼反应与骨髓纤维化患者的总生存期有关：PERSIST-2生存期标志性分析。

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-02-01 Epub Date: 2024-10-14 DOI: 10.1111/ejh.14321

Helen Ajufo, Jan Philipp Bewersdorf, Claire Harrison, Francesca Palandri, John Mascarenhas, Jeanne Palmer, Aaron Gerds, Jean-Jacques Kiladjian, Sarah Buckley, Andriy Derkach, Karisse Roman-Torres, Raajit K Rampal

{"title":"Pacritinib Response Is Associated With Overall Survival in Myelofibrosis: PERSIST-2 Landmark Analysis of Survival.","authors":"Helen Ajufo, Jan Philipp Bewersdorf, Claire Harrison, Francesca Palandri, John Mascarenhas, Jeanne Palmer, Aaron Gerds, Jean-Jacques Kiladjian, Sarah Buckley, Andriy Derkach, Karisse Roman-Torres, Raajit K Rampal","doi":"10.1111/ejh.14321","DOIUrl":"10.1111/ejh.14321","url":null,"abstract":"Spleen volume reduction (SVR) is a key endpoint in inhibitors of Janus kinase (JAK) inhibitor studies. Retrospective analyses have demonstrated an association between SVR and improved overall survival (OS) among patients treated with ruxolitinib with a platelet count > 100 × 109/L. Whether this association occurs in patients with thrombocytopenia is unclear. Pacritinib, a JAK2/IRAK1/ACVR1 inhibitor, demonstrated improved SVR versus best available therapy (BAT [best available therapy]; including ruxolitinib) in patients with myelofibrosis and platelet counts ≤ 100 × 109/L in the PERSIST-2 study. Patients on study at the start of the 12-week SVR window on pacritinib 200 mg twice daily or BAT were included. OS was evaluated among SVR responders versus non-responders using different SVR thresholds (≥ 35%, ≥ 20%, ≥ 10%, and > 0%). Among patients on pacritinib (n = 89), SVR ≥ 10% demonstrated the greatest separation in OS curves between responders and non-responders (HR, 0.00; 95% CI, 0.00-0.14; p < 0.01), though SVR ≥ 0% and SVR ≥ 20% were also associated with improved OS. No SVR threshold conferred OS benefit on BAT (n = 84), including ruxolitinib (n = 39). In patients with myelofibrosis and platelets ≤ 100 × 109/L, achieving SVR on pacritinib, but not BAT (including ruxolitinib), was associated with significant OS benefit, suggesting that pacritinib may offer a unique survival advantage in patients with myelofibrosis and thrombocytopenia who achieve any SVR. Trial Registration: ClinicalTrials.gov number: NCT02055781.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":"238-247"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11707811/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461086","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}

引用次数: 0

Superior Prophylactic Effectiveness of a Recombinant FVIIIFc Over Standard Half-Life FVIII in Hemophilia A: A-SURE Study. 重组 FVIIIFc 对 A 型血友病的预防效果优于标准半衰期 FVIII：A-SURE 研究。

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-02-01 Epub Date: 2024-10-21 DOI: 10.1111/ejh.14309

Johannes Oldenburg, Charles Hay, Flora Peyvandi, Annarita Tagliaferri, Pål Andrè Holme, María Teresa Álvarez-Román, Christine Biron-Andréani, Håkan Malmström, Linda Bystrická, Stefan Lethagen

{"title":"Superior Prophylactic Effectiveness of a Recombinant FVIIIFc Over Standard Half-Life FVIII in Hemophilia A: A-SURE Study.","authors":"Johannes Oldenburg, Charles Hay, Flora Peyvandi, Annarita Tagliaferri, Pål Andrè Holme, María Teresa Álvarez-Román, Christine Biron-Andréani, Håkan Malmström, Linda Bystrická, Stefan Lethagen","doi":"10.1111/ejh.14309","DOIUrl":"10.1111/ejh.14309","url":null,"abstract":"Objectives: The 24-month, prospective, non-interventional, European multicenter A-SURE study evaluated the real-world effectiveness of prophylaxis using an extended half-life recombinant factor VIII (FVIII) Fc fusion protein, efmoroctocog alfa (hereinafter rFVIIIFc), compared with prophylaxis using standard half-life (SHL) FVIII products in patients with hemophilia A.Methods: Primary endpoints were annualized bleeding rate (ABR), annualized injection frequency, and annualized factor consumption. A comparative study design unique for an observational hemophilia study was implemented to reduce potential confounding in effectiveness estimates, wherein each patient prescribed rFVIIIFc was matched with one receiving SHL FVIII. Propensity scores were used for adjustment in statistical analyses.Results: Outcomes for all primary endpoints were significantly better in the rFVIIIFc group (n = 184) compared with the SHL FVIII group (n = 170): mean ABR 1.5 versus 2.3 (difference of -0.8; p = 0.0147); mean annualized injection frequency 114.4 versus 169.2 (difference of -54.8; p < 0.0001); and mean annualized factor consumption 243 024.2 versus 288 718.6 International Units (difference of 45 694.5; p = 0.0003). rFVIIIFc was well tolerated, with no inhibitor development.Conclusions: rFVIIIFc has superior prophylactic effectiveness versus SHL FVIII, providing higher bleed protection with fewer injections and lower factor consumption.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":"248-257"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11707817/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142461100","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}

引用次数: 0

Real-World Evidence of Crizanlizumab Showing Reductions in Vaso-Occlusive Crises and Opioid Usage in Sickle Cell Disease. Crizanlizumab在镰状细胞病中减少血管闭塞性危象和阿片类药物用量的真实世界证据。

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-02-01 Epub Date: 2024-10-29 DOI: 10.1111/ejh.14323

Laurie DeBonnett, Vikas Joshi, Ana Cristina Silva-Pinto, Raffaella Colombatti, Annamaria Pasanisi, Francesco Arcioni, Rodolfo D Cançado, Séverine Sarp, Rajendra Sarkar, Wesam Soliman

{"title":"Real-World Evidence of Crizanlizumab Showing Reductions in Vaso-Occlusive Crises and Opioid Usage in Sickle Cell Disease.","authors":"Laurie DeBonnett, Vikas Joshi, Ana Cristina Silva-Pinto, Raffaella Colombatti, Annamaria Pasanisi, Francesco Arcioni, Rodolfo D Cançado, Séverine Sarp, Rajendra Sarkar, Wesam Soliman","doi":"10.1111/ejh.14323","DOIUrl":"10.1111/ejh.14323","url":null,"abstract":"Objective: Access to crizanlizumab, a disease-modifying therapy for sickle cell disease (SCD), was provided through a managed access program (MAP, NCT03720626). The present analysis evaluated the impact of 12 months of crizanlizumab treatment on vaso-occlusive crises (VOCs), and on the use of opioids for VOC-related pain relief, in patients with SCD from the MAP.Methods: From June 2018 to January 2023, 112 patients with a history of recurrent VOCs completed 12 months of crizanlizumab (5 mg/kg) treatment and were monitored for adverse events (AEs).Results: Crizanlizumab led to reductions of 18.0% and 36.2% in the proportions of patients having ≥ 1 home- and ≥ 1 healthcare-managed VOCs. Median absolute changes (interquartile range) from baseline in the rates of home- and healthcare-managed VOCs were -3.0 (-6.0, -1.0) and -2.0 (-4.0, 0), respectively. Data stratified by genotype and prior hydroxyurea use showed similar reductions in VOC rates. A 35.5% reduction in the proportion of patients requiring opioids was noted. AEs were consistent with earlier reports, and no new safety concerns were identified.Conclusions: Crizanlizumab demonstrated potential benefits in attenuating VOC episodes, irrespective of SCD genotype and prior hydroxyurea use, and in lowering opioid usage. The safety of crizanlizumab was consistent with earlier reports.Trial registration: The MAP has been registered at ClinicalTrials.gov with the ID, NCT03720626.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":"293-302"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11707818/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142544521","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}

引用次数: 0

Daratumumab in the Management of Red Cell Aplasia Following Allogeneic Hematopoietic Stem Cell Transplantation. 达拉单抗用于治疗异基因造血干细胞移植后的红细胞增生症。

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-02-01 Epub Date: 2024-11-04 DOI: 10.1111/ejh.14341

Nihar Desai, Auro Viswabandya, Dennis Dong Hwan Kim, Jeffrey H Lipton, Jonas Mattsson, Arjun Datt Law

{"title":"Daratumumab in the Management of Red Cell Aplasia Following Allogeneic Hematopoietic Stem Cell Transplantation.","authors":"Nihar Desai, Auro Viswabandya, Dennis Dong Hwan Kim, Jeffrey H Lipton, Jonas Mattsson, Arjun Datt Law","doi":"10.1111/ejh.14341","DOIUrl":"10.1111/ejh.14341","url":null,"abstract":"Pure red cell aplasia (PRCA) is a rare but significant complication following major ABO-incompatible allogeneic hematopoietic stem cell transplantation (HSCT). The persistence of recipient B lymphocytes producing anti-donor isohemagglutinins leads to reticulocytopenia and anemia, often resulting in transfusion dependence. Current treatment options for post-HSCT PRCA are limited and frequently yield suboptimal responses, complicating patient management. Herein, we report three cases of post-HSCT PRCA successfully managed with daratumumab, a monoclonal antibody targeting CD38-expressing plasma cells. All patients demonstrated rapid reticulocyte recovery and transfusion independence after daratumumab treatment, despite prior treatment failures. These findings suggest that daratumumab may provide a more effective therapeutic approach, with a favorable safety profile compared to traditional therapies. Given its demonstrated efficacy and safety, daratumumab warrants consideration as a first-line treatment for post-HSCT PRCA, potentially improving patient quality of life and reducing transfusion-related complications. Further studies should explore optimal dosing and long-term outcomes.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":"310-314"},"PeriodicalIF":2.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142567828","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}

引用次数: 0

Adherence to 6-Mercaptopurine (6-MP) and Habit Strength in Pediatric Acute Lymphoblastic Leukemia (ALL). 儿童急性淋巴细胞白血病（ALL）的6-巯基嘌呤（6-MP）依从性和习惯强度

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-01-20 DOI: 10.1111/ejh.14386

Mira Muxi Yang, Revika Singh, Maureen Haugen, Ashley Duff, Jenny Shoop, Elaine R Morgan, Jenna E Rossoff, Joanna L Weinstein, Mallorie B Heneghan, Sherif M Badawy

{"title":"Adherence to 6-Mercaptopurine (6-MP) and Habit Strength in Pediatric Acute Lymphoblastic Leukemia (ALL).","authors":"Mira Muxi Yang, Revika Singh, Maureen Haugen, Ashley Duff, Jenny Shoop, Elaine R Morgan, Jenna E Rossoff, Joanna L Weinstein, Mallorie B Heneghan, Sherif M Badawy","doi":"10.1111/ejh.14386","DOIUrl":"https://doi.org/10.1111/ejh.14386","url":null,"abstract":"Background: Low 6-mercaptopurine (6-MP) adherence (< 95%) is associated with increased relapse in pediatric acute lymphoblastic leukemia (ALL). Stronger habit has been associated with higher adherence. We examined the relationship of 6-MP adherence to habit strength and health-related quality of life in pediatric ALL.Methods: A single-center, cross-sectional study of 52 participants: 11 patients (mean age 16 ± 4) and 41 parents/caregivers (age 37 ± 5). Participants completed Visual Analogue Scale (VASdose), Patient Reported Outcomes Measurement Information System Medication Adherence Scale, and the Self-Regulated Habit Index (SRHI). Twelve semi-structured participant interviews were analyzed using thematic analysis.Results: 81% (42/52) of participants reported high 6-MP adherence (VASdose ≥ 95%): patients 91% (10/11), parents 78% (32/41). No significant correlation was found between adherence and habit strength. Reported adherence facilitators included reminders, care team communications, personalized tools, administration experience, self-efficacy, and social support. Conversely, financial burden, scheduling conflicts, and medication access were cited as barriers.Conclusions: One-fifth of participants reported low 6-MP adherence, with habit strength not associated with adherence. Variability of 6-MP routines may prohibit automaticity. While 6-MP adherence may not correlate with habit strength, interventions promoting and strengthening habit formation may overcome barriers to 6-MP adherence and improve outcomes.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143002504","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}

引用次数: 0

Increased Non-Relapse Mortality in Older People With Allogeneic Hematopoietic Stem Cell Transplantation Using Fludarabine and Myeloablative Dose of Busulfan-Based Regimen. 异基因造血干细胞移植老年人使用氟达拉滨和清髓剂量布苏凡方案的非复发死亡率增加

IF 2.3 3区医学

European Journal of Haematology Pub Date : 2025-01-20 DOI: 10.1111/ejh.14374

Akihito Shinohara, Michiho Shindo, Nobuaki Nakano, Emiko Sakaida, Naoyuki Uchida, Kentaro Fukushima, Hideyuki Nakazawa, Kentaro Serizawa, Yoshinobu Kanda, Toshiro Kawakita, Takashi Ikeda, Hiroyuki Ohigashi, Ayumu Ito, Toshio Wakayama, Ken-Ichi Matsuoka, Takahiro Fukuda, Junji Tanaka, Yoshiko Atsuta, Hideki Nakasone

{"title":"Increased Non-Relapse Mortality in Older People With Allogeneic Hematopoietic Stem Cell Transplantation Using Fludarabine and Myeloablative Dose of Busulfan-Based Regimen.","authors":"Akihito Shinohara, Michiho Shindo, Nobuaki Nakano, Emiko Sakaida, Naoyuki Uchida, Kentaro Fukushima, Hideyuki Nakazawa, Kentaro Serizawa, Yoshinobu Kanda, Toshiro Kawakita, Takashi Ikeda, Hiroyuki Ohigashi, Ayumu Ito, Toshio Wakayama, Ken-Ichi Matsuoka, Takahiro Fukuda, Junji Tanaka, Yoshiko Atsuta, Hideki Nakasone","doi":"10.1111/ejh.14374","DOIUrl":"https://doi.org/10.1111/ejh.14374","url":null,"abstract":"Fludarabine and myeloablative busulfan (FluBu4) in allogeneic hematopoietic stem cell transplantation (HSCT) for older people have not been adequately examined. This retrospective study analyzed data from a large-scale, nationwide database in Japan. Adult patients (> 15 years old, y/o) who received their first HSCT with FluBu4 for hematological malignancies were included. They were categorized into the younger (< 60 y/o, N = 1295) and the older group (≥ 60 y/o, N = 993). The 3-year overall survival (OS) rate after HSCT was significantly worse in the older group than in the other (p < 0.01, 39.9% vs. 48.5%). The 3-year non-relapse mortality (NRM) was significantly higher in the older group than in the other (p < 0.01, 30.9% vs. 23.0%), and the 3-year cumulative incidence of relapse was comparable between them. According to the multivariate analysis, age ≥ 60 years was significantly associated with poor OS and high NRM. In a subgroup analysis of the older group, the use of additional chemotherapeutic drugs to FluBu4 was significantly associated with poor OS and high NRM. Total body irradiation was significantly associated with high NRM and 1-year incidence of sinusoidal obstruction syndrome but not with OS. Thus, FluBu4 should be used with caution in older people.","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143002508","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}

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