Leonardo Javier Arcuri, Simone Pereira Lermontov, Carla de Oliveira Ribeiro
Several adaptations have been reported since the introduction of posttransplant cyclophosphamide (PTCy)-based haploidentical transplantation (Haplo). This single-center observational study compared the outcomes of PTCy-based Haplo with MMF at 30 mg/kg to a concurrent cohort of unrelated donors (URD) who received mainly ATG at 6 mg/kg proximal to transplantation. We included 174 patients (median follow-up: 33 months). Two-year OS for Haplo was 55%, which was not significantly lower than that for URD (67%, p = 0.22). Two-year RFS was 53% for Haplo, compared to 60% for URD (p = 0.31). Two-year GRFS was significantly worse for Haplo (24%) compared to URD (40%, p < 0.01). Relapse rates were similar (16% for Haplo and 21% for URD, p = 0.66), but non-relapse mortality was significantly higher for Haplo (31%) versus 19% for URD (p = 0.04). GVHD outcomes did not differ in univariable analyses. In multivariable analyses, there was a trend toward higher grades III–IV aGVHD (HR = 2.01, p = 0.08) and moderate to severe cGVHD (HR = 1.80, p = 0.05). In summary, we have shown that MMF at 30 mg/kg in Haplo transplants achieved similar PFS and OS compared to ATG-based URD. However, GRFS was lower, likely due to a significantly higher NRM associated with a trend towards higher moderate to severe cGVHD.
{"title":"Posttransplant Cyclophosphamide-Based Haploidentical Transplantation, With MMF 30 mg/kg, Compared With Unrelated Donor Transplantation With ATG 6 mg/kg, With Bone Marrow or Peripheral Blood Stem Cell Grafts","authors":"Leonardo Javier Arcuri, Simone Pereira Lermontov, Carla de Oliveira Ribeiro","doi":"10.1111/ejh.70054","DOIUrl":"https://doi.org/10.1111/ejh.70054","url":null,"abstract":"<div>\u0000 \u0000 <p>Several adaptations have been reported since the introduction of posttransplant cyclophosphamide (PTCy)-based haploidentical transplantation (Haplo). This single-center observational study compared the outcomes of PTCy-based Haplo with MMF at 30 mg/kg to a concurrent cohort of unrelated donors (URD) who received mainly ATG at 6 mg/kg proximal to transplantation. We included 174 patients (median follow-up: 33 months). Two-year OS for Haplo was 55%, which was not significantly lower than that for URD (67%, <i>p</i> = 0.22). Two-year RFS was 53% for Haplo, compared to 60% for URD (<i>p</i> = 0.31). Two-year GRFS was significantly worse for Haplo (24%) compared to URD (40%, <i>p</i> < 0.01). Relapse rates were similar (16% for Haplo and 21% for URD, <i>p</i> = 0.66), but non-relapse mortality was significantly higher for Haplo (31%) versus 19% for URD (<i>p</i> = 0.04). GVHD outcomes did not differ in univariable analyses. In multivariable analyses, there was a trend toward higher grades III–IV aGVHD (HR = 2.01, <i>p</i> = 0.08) and moderate to severe cGVHD (HR = 1.80, <i>p</i> = 0.05). In summary, we have shown that MMF at 30 mg/kg in Haplo transplants achieved similar PFS and OS compared to ATG-based URD. However, GRFS was lower, likely due to a significantly higher NRM associated with a trend towards higher moderate to severe cGVHD.</p>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"116 2","pages":"142-147"},"PeriodicalIF":2.3,"publicationDate":"2025-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145915894","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}
R. Klamroth, M. Al Saleh, H. Glosli, M. Schiavulli, B. Guillet, L. Bystrická, A. Schönstein, and S. Lethagen, “Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study,” European Journal of Haematology 115 (2025): 134–141, https://doi.org/10.1111/ejh.14427.
The second sentence of Paragraph 3.4.2 | Rescue ITI of the Safety section (3.4): “Seven (41.2%) of these patients had SAEs, and one (5.9%) event was adjudicated as a serious adverse drug reaction by the investigator (Table S5).” was incorrect. This should have read: “Seven (41.2%) of these patients had SAE. One (5.9%) SAE was assigned as a serious adverse drug reaction (SADR) by the Sponsor based on predefined conservative criteria, as relatedness was not assigned by the Investigator (Table S5).”
*This SAE (haematoma) was also classified as a SADR (†) by the Sponsor based on predefined conservative criteria, as relatedness was not assigned by the Investigator. ITI: immune tolerance induction; SADR: serious adverse drug reaction; SAE: serious adverse event.
The conclusions of the paper are not affected. We apologize for these errors.
R. Klamroth， M. Al Saleh， H. Glosli， M. Schiavulli， B. Guillet， L. bystrick, a . Schönstein， S. Lethagen，“重组因子VIII Fc诱导血友病a的免疫耐受：来自图表回顾研究的数据”，欧洲血液病杂志115 (2025):134-141,https://doi.org/10.1111/ejh.14427.The第3.4.2 |营救ITI安全部分（3.4）第二句：“这些患者中有7例（41.2%）发生SAEs， 1例（5.9%）事件被研究者判定为严重药物不良反应（表S5）”是不正确的。结果应该是：“这些患者中有7例（41.2%）发生SAE。1例（5.9%）SAE被发起人根据预先定义的保守标准指定为严重药物不良反应（SADR），因为研究者没有指定相关性（表S5）。*该血肿也被主办方根据预先定义的保守标准分类为SADR（†），因为研究者没有指定相关性。ITI：免疫耐受诱导；SADR：严重药物不良反应；SAE：严重不良事件。本文的结论不受影响。我们为这些错误道歉。
{"title":"Correction to “Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study”","authors":"","doi":"10.1111/ejh.70043","DOIUrl":"10.1111/ejh.70043","url":null,"abstract":"<p>R. Klamroth, M. Al Saleh, H. Glosli, M. Schiavulli, B. Guillet, L. Bystrická, A. Schönstein, and S. Lethagen, “Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study,” <i>European Journal of Haematology</i> 115 (2025): 134–141, https://doi.org/10.1111/ejh.14427.</p><p>The second sentence of Paragraph 3.4.2 | Rescue ITI of the Safety section (3.4): “Seven (41.2%) of these patients had SAEs, and one (5.9%) event was adjudicated as a serious adverse drug reaction by the investigator (Table S5).” was incorrect. This should have read: “Seven (41.2%) of these patients had SAE. One (5.9%) SAE was assigned as a serious adverse drug reaction (SADR) by the Sponsor based on predefined conservative criteria, as relatedness was not assigned by the Investigator (Table S5).”</p><p>*This SAE (haematoma) was also classified as a SADR (<sup>†</sup>) by the Sponsor based on predefined conservative criteria, as relatedness was not assigned by the Investigator. ITI: immune tolerance induction; SADR: serious adverse drug reaction; SAE: serious adverse event.</p><p>The conclusions of the paper are not affected. We apologize for these errors.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"115 6","pages":"618-619"},"PeriodicalIF":2.3,"publicationDate":"2025-10-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.70043","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145291450","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}
Julia Z. Xu, Brian J. Philips, Nelly K. Kiriza, Selma K. Bendtsen, Jesper B. Petersen, Jens Helby, Manzoor Mohideen, Enrico M. Novelli, Andreas Glenthøj
{"title":"Tebapivat Improves Red Cell Deformability and Decreases Sickling in Patients With Sickle Cell Disease: A Phase 1 Trial Substudy","authors":"Julia Z. Xu, Brian J. Philips, Nelly K. Kiriza, Selma K. Bendtsen, Jesper B. Petersen, Jens Helby, Manzoor Mohideen, Enrico M. Novelli, Andreas Glenthøj","doi":"10.1111/ejh.70048","DOIUrl":"10.1111/ejh.70048","url":null,"abstract":"","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"116 1","pages":"98-100"},"PeriodicalIF":2.3,"publicationDate":"2025-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145285955","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}
Blas Holger Mogensen, Torsten Holm Nielsen, Mette Ølgod Pedersen, Lise Mette Rahbek Gjerdrum, Tarec Christoffer El-Galaly, Lars Møller Pedersen
In the diagnostic evaluation of suspected lymphoma, both core needle biopsy (CNB) and surgical excisional biopsy (SEB) are routinely employed for tissue sampling. While SEB remains the gold standard, CNB is a less invasive and more cost-effective alternative. However, concerns persist regarding its diagnostic yield. This study aimed to assess the diagnostic performance of CNB in comparison to SEB in routine clinical practice. We included 179 patients with newly diagnosed lymphoma referred to Zealand University Hospital in 2023, of whom 84 underwent CNB based on PET/CT findings. A definitive lymphoma diagnosis was established in 74% of CNB cases, compared to 98% with SEB, underscoring a lower diagnostic yield of CNB. The diagnostic accuracy of CNB varied by biopsy location, lymphoma subtype, and the volume of tissue obtained. Inconclusive diagnoses were more frequent in indolent lymphoma subtypes, biopsies yielding a limited number of tissue cylinders, and samples taken from the axillary and inguinal regions or extranodal sites. Grade 3–4 complications were observed in 1% of CNBs and in 5% of SEBs. Although CNB was a safe procedure, further refinement of biopsy techniques and improved patient selection are needed to enhance its diagnostic accuracy and clinical utility.
{"title":"Core Needle Biopsies in Patients With Lymphoproliferative Malignancies: An Observational Single-Center Cohort Study","authors":"Blas Holger Mogensen, Torsten Holm Nielsen, Mette Ølgod Pedersen, Lise Mette Rahbek Gjerdrum, Tarec Christoffer El-Galaly, Lars Møller Pedersen","doi":"10.1111/ejh.70045","DOIUrl":"10.1111/ejh.70045","url":null,"abstract":"<p>In the diagnostic evaluation of suspected lymphoma, both core needle biopsy (CNB) and surgical excisional biopsy (SEB) are routinely employed for tissue sampling. While SEB remains the gold standard, CNB is a less invasive and more cost-effective alternative. However, concerns persist regarding its diagnostic yield. This study aimed to assess the diagnostic performance of CNB in comparison to SEB in routine clinical practice. We included 179 patients with newly diagnosed lymphoma referred to Zealand University Hospital in 2023, of whom 84 underwent CNB based on PET/CT findings. A definitive lymphoma diagnosis was established in 74% of CNB cases, compared to 98% with SEB, underscoring a lower diagnostic yield of CNB. The diagnostic accuracy of CNB varied by biopsy location, lymphoma subtype, and the volume of tissue obtained. Inconclusive diagnoses were more frequent in indolent lymphoma subtypes, biopsies yielding a limited number of tissue cylinders, and samples taken from the axillary and inguinal regions or extranodal sites. Grade 3–4 complications were observed in 1% of CNBs and in 5% of SEBs. Although CNB was a safe procedure, further refinement of biopsy techniques and improved patient selection are needed to enhance its diagnostic accuracy and clinical utility.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"116 1","pages":"85-92"},"PeriodicalIF":2.3,"publicationDate":"2025-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.70045","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145274251","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}
Mike Laffan, Heena Howitt, Cheryl Jones, Sarah Brighton, Rosa Willock, Anna Sanigorska, Oliver Heard
Objectives
We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) receiving recombinant von Willebrand factor (rVWF) in surgical settings in the United Kingdom.
Methods
Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first-time rVWF for the prevention/treatment of surgery-related bleeds, or the on-demand treatment of spontaneous/traumatic bleeds, between 1 October 2020 and 30 June 2022 at seven hospitals. Treatment, outcome and VWD-related HCRU data associated with the prevention/treatment of surgery-related bleeds were collected at first (index) event, for 12 months pre-index and 3–12 months post-index.
Results
Twenty patients (55.0% female, 90.0% White/Caucasian, mean age 56.7 years) received rVWF to prevent/treat surgery-related bleeds at index: normal haemostasis following abnormal bleeding was achieved for all applicable bleeds with limited requirement for additional treatments. Only four patients (all VWD type 2) received exogenous factor VIII in addition to rVWF at index. There were no treatment switches and no likely treatment-related complications. Physician-rated treatment satisfaction was ‘excellent’ (36.7%) or ‘good’ (63.3%) for all rVWF-treated surgery-related bleeds (n = 30).
Conclusions
Results support the effectiveness and safety profile of rVWF in a surgical setting in adults with VWD, supplementing the growing body of evidence for rVWF.
{"title":"Outcomes and Healthcare Resource Utilisation in Adults With von Willebrand Disease Treated With Recombinant von Willebrand Factor in Surgical Settings in the United Kingdom","authors":"Mike Laffan, Heena Howitt, Cheryl Jones, Sarah Brighton, Rosa Willock, Anna Sanigorska, Oliver Heard","doi":"10.1111/ejh.70033","DOIUrl":"10.1111/ejh.70033","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) receiving recombinant von Willebrand factor (rVWF) in surgical settings in the United Kingdom.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first-time rVWF for the prevention/treatment of surgery-related bleeds, or the on-demand treatment of spontaneous/traumatic bleeds, between 1 October 2020 and 30 June 2022 at seven hospitals. Treatment, outcome and VWD-related HCRU data associated with the prevention/treatment of surgery-related bleeds were collected at first (index) event, for 12 months pre-index and 3–12 months post-index.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Twenty patients (55.0% female, 90.0% White/Caucasian, mean age 56.7 years) received rVWF to prevent/treat surgery-related bleeds at index: normal haemostasis following abnormal bleeding was achieved for all applicable bleeds with limited requirement for additional treatments. Only four patients (all VWD type 2) received exogenous factor VIII in addition to rVWF at index. There were no treatment switches and no likely treatment-related complications. Physician-rated treatment satisfaction was ‘excellent’ (36.7%) or ‘good’ (63.3%) for all rVWF-treated surgery-related bleeds (<i>n</i> = 30).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Results support the effectiveness and safety profile of rVWF in a surgical setting in adults with VWD, supplementing the growing body of evidence for rVWF.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"116 1","pages":"75-84"},"PeriodicalIF":2.3,"publicationDate":"2025-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.70033","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145274272","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}
Mike Laffan, Heena Howitt, Cheryl Jones, Sarah Brighton, Rosa Willock, Anna Sanigorska, Oliver Heard
Objectives
We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) treated on demand with recombinant von Willebrand factor (rVWF) in the United Kingdom.
Methods
Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first-time rVWF for the on-demand treatment of spontaneous/traumatic bleeds, or the prevention/treatment of surgical bleeds, between 1 October 2020 and 30 June 2022 at seven hospitals. Treatment, outcome and VWD-related HCRU data associated with the on-demand treatment of all recorded bleeds were collected at first (index) treatment, for 12 months pre-index and 3–12 months post-index.
Results
Twelve patients (91.7% female and White/Caucasian, mean age 34.2 years) were treated on demand with rVWF at index: bleed resolution was achieved for all bleeds with limited requirement for additional treatments, no treatment switches, and no complications reported. Physician-rated treatment satisfaction was ‘excellent’ (82.6%) or ‘good’ (17.4%) for all recorded on-demand rVWF-treated bleeds (n = 23). Patients receiving any on-demand treatment required an inpatient admission and ≥ 1 outpatient visit in 33.7% and 66.7% of cases, respectively.
Conclusions
Results support the effectiveness and safety profile of on-demand rVWF treatment for spontaneous/traumatic bleeds in adults with VWD, adding to the growing body of evidence for rVWF.
{"title":"Outcomes and Healthcare Resource Utilisation in Adults With von Willebrand Disease Receiving On-Demand Recombinant von Willebrand Factor in the United Kingdom","authors":"Mike Laffan, Heena Howitt, Cheryl Jones, Sarah Brighton, Rosa Willock, Anna Sanigorska, Oliver Heard","doi":"10.1111/ejh.70032","DOIUrl":"10.1111/ejh.70032","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objectives</h3>\u0000 \u0000 <p>We describe treatment outcomes and healthcare resource utilisation (HCRU) in adults with von Willebrand disease (VWD) treated on demand with recombinant von Willebrand factor (rVWF) in the United Kingdom.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Retrospective chart review of adults (≥ 18 years) with congenital VWD receiving first-time rVWF for the on-demand treatment of spontaneous/traumatic bleeds, or the prevention/treatment of surgical bleeds, between 1 October 2020 and 30 June 2022 at seven hospitals. Treatment, outcome and VWD-related HCRU data associated with the on-demand treatment of all recorded bleeds were collected at first (index) treatment, for 12 months pre-index and 3–12 months post-index.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Twelve patients (91.7% female and White/Caucasian, mean age 34.2 years) were treated on demand with rVWF at index: bleed resolution was achieved for all bleeds with limited requirement for additional treatments, no treatment switches, and no complications reported. Physician-rated treatment satisfaction was ‘excellent’ (82.6%) or ‘good’ (17.4%) for all recorded on-demand rVWF-treated bleeds (<i>n</i> = 23). Patients receiving any on-demand treatment required an inpatient admission and ≥ 1 outpatient visit in 33.7% and 66.7% of cases, respectively.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Results support the effectiveness and safety profile of on-demand rVWF treatment for spontaneous/traumatic bleeds in adults with VWD, adding to the growing body of evidence for rVWF.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"116 1","pages":"66-74"},"PeriodicalIF":2.3,"publicationDate":"2025-10-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.70032","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145274265","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}
Baseline cardiovascular (CV) risk stratification is an essential component in managing patients undergoing potential cardiotoxic anticancer therapies. Chimeric antigen receptor (CAR)-T cell therapy, a groundbreaking treatment for hematologic malignancies, is associated with a non-negligible risk of cardiovascular adverse events (CVAE). This study aimed to identify predictors of CAR-T cell-related CVAE and to develop a corresponding risk stratification score.
Methods
We conducted a meta-analysis of studies comparing baseline clinical, biomarker, echocardiographic findings, and pharmaceutical treatments between patients who developed CAR-T cell-related CVAE and those who did not. We subsequently used the pooled relative risks (RR) of significant predictors to construct a risk stratification score.
Results
We identified 12 relevant studies encompassing a total of 1354 patients with haematologic malignancies, the majority of which were treated with CD19-directed CAR-T cell therapy, of whom 228 (16.8%) developed CVAE. Significant predictors of CAR-T cell-related CVAE included coronary artery disease [RR = 2.27 (95% confidence interval, 1.46–3.51)], hyperlipidaemia [1.57 (1.14–2.15)], diabetes [1.59 (1.13–2.24)], hypertension [1.45 (1.18–1.77)], atrial fibrillation [2.42 (1.51–3.88)], heart failure [2.74 (1.62–4.61)], and smoking [1.40 (1.10–1.79)]. The resulting risk prediction score, incorporating the above seven factors, named CART-7, ranges from 0 to 33, with a score of 0–8 indicating low risk, 9–17 moderate risk, 18–25 high risk, and 26–33 very high risk.
Conclusion
Seven baseline CV conditions were significantly associated with CAR-T cell-related CVAE. Further validation of the resulting CART-7 score is warranted to support its clinical use in baseline CV risk stratification for patients undergoing CAR-T cell therapy.
{"title":"Predictive Factors and Baseline Risk Stratification for CAR-T Cell Therapy-Related Cardiovascular Toxicity","authors":"Dimitrios Farmakis, Konstantinos I. Karampinos, Raul Cordoba, Teresa López-Fernández, Gerasimos Filippatos","doi":"10.1111/ejh.70040","DOIUrl":"10.1111/ejh.70040","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Aims</h3>\u0000 \u0000 <p>Baseline cardiovascular (CV) risk stratification is an essential component in managing patients undergoing potential cardiotoxic anticancer therapies. Chimeric antigen receptor (CAR)-T cell therapy, a groundbreaking treatment for hematologic malignancies, is associated with a non-negligible risk of cardiovascular adverse events (CVAE). This study aimed to identify predictors of CAR-T cell-related CVAE and to develop a corresponding risk stratification score.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted a meta-analysis of studies comparing baseline clinical, biomarker, echocardiographic findings, and pharmaceutical treatments between patients who developed CAR-T cell-related CVAE and those who did not. We subsequently used the pooled relative risks (RR) of significant predictors to construct a risk stratification score.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>We identified 12 relevant studies encompassing a total of 1354 patients with haematologic malignancies, the majority of which were treated with CD19-directed CAR-T cell therapy, of whom 228 (16.8%) developed CVAE. Significant predictors of CAR-T cell-related CVAE included coronary artery disease [RR = 2.27 (95% confidence interval, 1.46–3.51)], hyperlipidaemia [1.57 (1.14–2.15)], diabetes [1.59 (1.13–2.24)], hypertension [1.45 (1.18–1.77)], atrial fibrillation [2.42 (1.51–3.88)], heart failure [2.74 (1.62–4.61)], and smoking [1.40 (1.10–1.79)]. The resulting risk prediction score, incorporating the above seven factors, named CART-7, ranges from 0 to 33, with a score of 0–8 indicating low risk, 9–17 moderate risk, 18–25 high risk, and 26–33 very high risk.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Seven baseline CV conditions were significantly associated with CAR-T cell-related CVAE. Further validation of the resulting CART-7 score is warranted to support its clinical use in baseline CV risk stratification for patients undergoing CAR-T cell therapy.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"116 1","pages":"54-65"},"PeriodicalIF":2.3,"publicationDate":"2025-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145244017","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}
Herpes zoster (HZ), resulting from reactivation of latent varicella-zoster virus (VZV), imposes a significant burden on immunocompromised patients, particularly those with hematological malignancies and recipients of hematopoietic stem cell transplants (HSCT). These populations face markedly increased risks of severe complications, including disseminated disease and postherpetic neuralgia.
Objective
This review examines the pathogenesis, epidemiology, risk factors, and evolving preventive strategies for HZ in patients with hematological disorders, with an emphasis on antiviral prophylaxis and vaccination.
Methods
Relevant data from recent clinical trials, observational studies, and international guidelines were critically reviewed to evaluate the burden of HZ and the effectiveness of prophylactic interventions in immunocompromised populations.
Results
Immunosuppression—whether due to the underlying disease or post-transplant immune reconstitution—compromises VZV-specific cellular immunity, thereby increasing HZ susceptibility. Incidence rates in high-risk groups, such as HSCT recipients or patients treated with proteasome inhibitors, JAK2 inhibitors, or CAR-T therapies, may exceed 30–60 per 1000 person-years. Antiviral prophylaxis remains a fundamental preventive approach. The adjuvanted recombinant zoster vaccine (aRZV) demonstrates 68%–87% efficacy even in heavily immunosuppressed individuals. Emerging vaccine platforms, including mRNA-based formulations, offer promising improvements in immunogenicity and scalability.
Conclusion
Patients with hematological conditions are particularly vulnerable to HZ. Effective prevention requires a tailored combination of antiviral prophylaxis and aRZV immunization. The development of mRNA-based vaccines may further enhance preventive strategies for this at-risk population.
{"title":"Herpes Zoster in Hematological Disorders: Pathogenesis, Risk Stratification, and Emerging Strategies for Prevention and Immunization","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.70049","DOIUrl":"10.1111/ejh.70049","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Herpes zoster (HZ), resulting from reactivation of latent varicella-zoster virus (VZV), imposes a significant burden on immunocompromised patients, particularly those with hematological malignancies and recipients of hematopoietic stem cell transplants (HSCT). These populations face markedly increased risks of severe complications, including disseminated disease and postherpetic neuralgia.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>This review examines the pathogenesis, epidemiology, risk factors, and evolving preventive strategies for HZ in patients with hematological disorders, with an emphasis on antiviral prophylaxis and vaccination.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Relevant data from recent clinical trials, observational studies, and international guidelines were critically reviewed to evaluate the burden of HZ and the effectiveness of prophylactic interventions in immunocompromised populations.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Immunosuppression—whether due to the underlying disease or post-transplant immune reconstitution—compromises VZV-specific cellular immunity, thereby increasing HZ susceptibility. Incidence rates in high-risk groups, such as HSCT recipients or patients treated with proteasome inhibitors, JAK2 inhibitors, or CAR-T therapies, may exceed 30–60 per 1000 person-years. Antiviral prophylaxis remains a fundamental preventive approach. The adjuvanted recombinant zoster vaccine (aRZV) demonstrates 68%–87% efficacy even in heavily immunosuppressed individuals. Emerging vaccine platforms, including mRNA-based formulations, offer promising improvements in immunogenicity and scalability.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Patients with hematological conditions are particularly vulnerable to HZ. Effective prevention requires a tailored combination of antiviral prophylaxis and aRZV immunization. The development of mRNA-based vaccines may further enhance preventive strategies for this at-risk population.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"116 1","pages":"14-22"},"PeriodicalIF":2.3,"publicationDate":"2025-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/ejh.70049","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145244004","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}
Sabrina de Souza, Mari D Takashima, Areum Hyun, Victoria Gibson, Thiago Lopes Silva, Patricia Kuerten Rocha, Siriporn Vetcho, Sophie C H Wen, Amanda J Ullman
Objective: To examine global practices for identifying and managing central venous access device (CVAD) complications-catheter-associated bloodstream infection (CABSI), thrombosis, and occlusion-in paediatric cancer care, comparing patterns between high- and other-income countries.
Methods: A cross-sectional international survey was conducted from 2022 to 2023 and analysed 2024 to 2025. Clinicians involved in paediatric CVAD cancer care were recruited through global networks.
Results: A total of 161 respondents from 38 countries completed the complication section, including 102 (63.4%) from high-income and 59 (36.6%) from other-income countries (lower- and upper-middle income). For CABSI, blood culture was the main diagnostic method (122 [75.8%]; high-income: 87 [85.3%], other-income: 35 [59.3%]). Differential time to positivity was more often reported in other-income settings (33 [55.9%] vs. 35 [34.3%]), who also more frequently initiated antibiotics based on nonspecific or immediate criteria. CABSI treatment varied, with intravenous antibiotics (68 [60.7%]) and catheter removal (47 [42.0%]) most reported. For thrombosis, anticoagulation before line removal was common (88 [54.7%]), and alteplase use was higher in high-income countries (76 [74.5%] vs. 19 [32.2%]). Thrombolytic agents were the most reported treatment for occlusion (103 [64.0%]), especially in high-income countries (77 [75.5%] vs. 26 [44.1%]).
Conclusions: CVAD complication management varies by country income level, highlighting the need for context-adapted guidelines, training, and equitable access to key resources.
{"title":"Complications in Central Venous Access Devices in Paediatric Cancer Care: A Global Cross-Sectional Survey.","authors":"Sabrina de Souza, Mari D Takashima, Areum Hyun, Victoria Gibson, Thiago Lopes Silva, Patricia Kuerten Rocha, Siriporn Vetcho, Sophie C H Wen, Amanda J Ullman","doi":"10.1111/ejh.70041","DOIUrl":"https://doi.org/10.1111/ejh.70041","url":null,"abstract":"<p><strong>Objective: </strong>To examine global practices for identifying and managing central venous access device (CVAD) complications-catheter-associated bloodstream infection (CABSI), thrombosis, and occlusion-in paediatric cancer care, comparing patterns between high- and other-income countries.</p><p><strong>Methods: </strong>A cross-sectional international survey was conducted from 2022 to 2023 and analysed 2024 to 2025. Clinicians involved in paediatric CVAD cancer care were recruited through global networks.</p><p><strong>Results: </strong>A total of 161 respondents from 38 countries completed the complication section, including 102 (63.4%) from high-income and 59 (36.6%) from other-income countries (lower- and upper-middle income). For CABSI, blood culture was the main diagnostic method (122 [75.8%]; high-income: 87 [85.3%], other-income: 35 [59.3%]). Differential time to positivity was more often reported in other-income settings (33 [55.9%] vs. 35 [34.3%]), who also more frequently initiated antibiotics based on nonspecific or immediate criteria. CABSI treatment varied, with intravenous antibiotics (68 [60.7%]) and catheter removal (47 [42.0%]) most reported. For thrombosis, anticoagulation before line removal was common (88 [54.7%]), and alteplase use was higher in high-income countries (76 [74.5%] vs. 19 [32.2%]). Thrombolytic agents were the most reported treatment for occlusion (103 [64.0%]), especially in high-income countries (77 [75.5%] vs. 26 [44.1%]).</p><p><strong>Conclusions: </strong>CVAD complication management varies by country income level, highlighting the need for context-adapted guidelines, training, and equitable access to key resources.</p>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145191446","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}
Jing Zhong, Yemei Gao, Chunping Mo, Yan Zhang, Yan Luo, Lingbo Li
Objective
To analyze the results of Rh blood group antigen detection and irregular antibody identification of 5050 patients in China, and to explore the importance of Rh blood group typing before blood transfusion and to propose a new concept of “Transfusionomics”.
Methods
Use microcolumn gel detection card to type Rh blood group antigen of 5050 patients, and to screen and identify antibody. Use PCR amplification and Sanger sequencing techniques to detect RHD genotype.
Results
A total of 5030 D-positive cases were detected in 5050 patients; the remaining 20 cases were detected as D-negative. Totally, 49 cases of Rh blood group system antibodies were detected, which accounted for 70% of all detected antibodies. Of the 20 initial screening D-negative patients, 11 cases had RHD gene deletion, and 9 cases had retained all of 10 exons.
Conclusion
Large-scale testing and research on patients' Rh blood group antigen and antibody is aimed at avoiding red blood cell alloimmunization caused by blood transfusion, realizing individualized and precise blood transfusion. The large amount of data generated by blood transfusion laboratories every day will form an important basis for a new concept of “Transfusionomics”.
{"title":"From Rh Blood Group Antigen Antibody Testing to “Transfusionomics”","authors":"Jing Zhong, Yemei Gao, Chunping Mo, Yan Zhang, Yan Luo, Lingbo Li","doi":"10.1111/ejh.70042","DOIUrl":"10.1111/ejh.70042","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>To analyze the results of Rh blood group antigen detection and irregular antibody identification of 5050 patients in China, and to explore the importance of Rh blood group typing before blood transfusion and to propose a new concept of “Transfusionomics”.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Use microcolumn gel detection card to type Rh blood group antigen of 5050 patients, and to screen and identify antibody. Use PCR amplification and Sanger sequencing techniques to detect <i>RHD</i> genotype.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 5030 D-positive cases were detected in 5050 patients; the remaining 20 cases were detected as D-negative. Totally, 49 cases of Rh blood group system antibodies were detected, which accounted for 70% of all detected antibodies. Of the 20 initial screening D-negative patients, 11 cases had <i>RHD</i> gene deletion, and 9 cases had retained all of 10 exons.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Large-scale testing and research on patients' Rh blood group antigen and antibody is aimed at avoiding red blood cell alloimmunization caused by blood transfusion, realizing individualized and precise blood transfusion. The large amount of data generated by blood transfusion laboratories every day will form an important basis for a new concept of “Transfusionomics”.</p>\u0000 </section>\u0000 </div>","PeriodicalId":11955,"journal":{"name":"European Journal of Haematology","volume":"116 1","pages":"46-53"},"PeriodicalIF":2.3,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145191462","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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