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Treatment of an Indolent T-Cell Lymphoma of the Gastrointestinal Tract Harboring STAT3::JAK2 With Jakafi (Ruxolitinib) With Significant Clinical Improvements Jakafi (Ruxolitinib)治疗含有STAT3::JAK2的胃肠道惰性t细胞淋巴瘤临床疗效显著
EJHaem Pub Date : 2025-05-08 DOI: 10.1002/jha2.70047
Xi Cao, Carolyn Mulroney, Huan-You Wang
{"title":"Treatment of an Indolent T-Cell Lymphoma of the Gastrointestinal Tract Harboring STAT3::JAK2 With Jakafi (Ruxolitinib) With Significant Clinical Improvements","authors":"Xi Cao,&nbsp;Carolyn Mulroney,&nbsp;Huan-You Wang","doi":"10.1002/jha2.70047","DOIUrl":"https://doi.org/10.1002/jha2.70047","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Indolent T-cell lymphoma of the gastrointestinal tract (ITCL-GI) is a rare primary T-cell lymphoma from the gastrointestinal (GI) tract, there has been no documented successful treatment at the present time.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A CD4(+)/CD8(−) ITCL-GI with <i>STAT3::JAK2</i> fusion was treated with Jakafi (Ruxolitinib), which resulted in significant clinical improvements.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Next generation sequencing is highly recommended for any new diagnosis of CD4(+)/CD8(−) ITCL-GI in order to detect if <i>STAT3::JAK2</i> can be found in order to treat the patient with JAK2 inhibitor such as Jakafi (Ruxolitinib).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Clinical trial registration</h3>\u0000 \u0000 <p>The authors have confirmed clinical trial registration is not needed for this submission.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70047","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143925966","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Hematopoietic Stem Cell Transplantation Outcomes in Diamond–Blackfan Anemia Patients Based on Myeloablative Conditioning Regimen With or Without Total Body Irradiation: A Systematic Review and Meta-Analysis Diamond-Blackfan贫血患者在有或没有全身照射的清髓调理方案基础上的造血干细胞移植结果:一项系统回顾和荟萃分析
EJHaem Pub Date : 2025-05-08 DOI: 10.1002/jha2.70043
Parsa Fathi, Leila Jafari, Afshin Fathi, Ayat Ahmadi, Yalda Karamlou, Maryam Behfar, Amir Ali Hamidieh
{"title":"Hematopoietic Stem Cell Transplantation Outcomes in Diamond–Blackfan Anemia Patients Based on Myeloablative Conditioning Regimen With or Without Total Body Irradiation: A Systematic Review and Meta-Analysis","authors":"Parsa Fathi,&nbsp;Leila Jafari,&nbsp;Afshin Fathi,&nbsp;Ayat Ahmadi,&nbsp;Yalda Karamlou,&nbsp;Maryam Behfar,&nbsp;Amir Ali Hamidieh","doi":"10.1002/jha2.70043","DOIUrl":"https://doi.org/10.1002/jha2.70043","url":null,"abstract":"<p>Diamond–Blackfan anemia (DBA) is a rare, congenital bone marrow failure syndrome characterized by hypoplastic anemia. Earliest descriptions of this disease date back to 1936, and since then, a plethora of treatment strategies have been used to control or treat the disease. In recent decades, hematopoietic stem cell transplantation (HSCT) has been declared the only curative treatment. Despite the time elapsing from the first time HSCT has been used in this setting, no unified standard preparative and prophylactic protocol has been established. In this article, for the first time, the published articles concerning the efficacy of the most verified conditioning regimens established for these patients, the myeloablative conditioning regimen (MAC), were systematically reviewed. A comparison of two groups, based on the presence or absence of radiation in their protocol, was performed. Electronic and manual searches were conducted on PubMed, Scopus, and Web of Science. The primary study domains, selection, and outcome were assessed using the JBI Scale quality assessment for cohort and case series studies. Cohorts were categorized into treatment groups, and the characteristics of patients and donors, in addition to intervention characteristics and outcomes, were synthesized. Among a total of 196 studies reviewed, we included five cohorts in our systematic review. The studies were heterogeneous in various aspects. In conclusion, our analysis suggests that DBA patients who underwent a MAC non-total body irradiation (TBI) conditioning regimen may experience better post-HSCT outcomes; however, the findings are inconclusive.</p>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70043","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143925965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Epidemiology, Survival, and Second Primary Malignancies in T-Cell Large Granular Lymphocytic Leukemia t细胞大颗粒淋巴细胞白血病的流行病学、生存率和第二原发恶性肿瘤
EJHaem Pub Date : 2025-05-06 DOI: 10.1002/jha2.70036
Arya Mariam Roy, Sawyer Bawek, Richa Parikh, Muhammad Salman Faisal, Paola Ghione
{"title":"Epidemiology, Survival, and Second Primary Malignancies in T-Cell Large Granular Lymphocytic Leukemia","authors":"Arya Mariam Roy,&nbsp;Sawyer Bawek,&nbsp;Richa Parikh,&nbsp;Muhammad Salman Faisal,&nbsp;Paola Ghione","doi":"10.1002/jha2.70036","DOIUrl":"https://doi.org/10.1002/jha2.70036","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Large granular lymphocytic leukemia (LGL) is a rare lymphoproliferative disorder, with limited literature available about the epidemiology, survival, and development of secondary primary malignancies (SPMs) in T-LGL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The Surveillance, Epidemiology, and End Results (SEER) 17 registry was used to identify all cases of T-LGL diagnosed between 2000 and 2019, and patients with primary T-LGL were analyzed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Patients with primary T-LGL were found to have a higher incidence of SPMs compared to the general population, with increased risk for hematological malignancies seen within the first 10 years and solid tumors seen after 10 years of T-LGL diagnosis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Patients with primary T-LGL were found to have a higher incidence of SPMs. Further research is needed to better understand the reason for this increased risk.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Trial Registration</h3>\u0000 \u0000 <p>The authors have confirmed clinical trial registration is not needed for this submission</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70036","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143909059","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Sickle Cell Disease in Africa: SickleInAfrica Registry in Ghana, Nigeria and Tanzania 非洲镰状细胞病:加纳、尼日利亚和坦桑尼亚的非洲镰状细胞病登记处
EJHaem Pub Date : 2025-05-06 DOI: 10.1002/jha2.70044
Jack Morrice, Wilson Mupfururirwa, Reuben I. Chianumba, Evans Xorse Amuzu, Daniel Kandonga, Victoria Nembaware, Mario Jonas, Jade Hotchkiss, Upendo Masamu, Arthemon Nguweneza, Bruno P. Mmbando, Irene Minja, Agnes Jonathan, Nicola Mulder, Emmanuel Balandya, Alex Osei-Akoto, Vivian Paintsil, Julie Makani, Obiageli Nnodu, Members of SPARCO Nigeria, Raphael Z. Sangeda, Andre Pascal Kengne, Gaston Kuzamunu, Ambroise Wonkam
{"title":"Sickle Cell Disease in Africa: SickleInAfrica Registry in Ghana, Nigeria and Tanzania","authors":"Jack Morrice,&nbsp;Wilson Mupfururirwa,&nbsp;Reuben I. Chianumba,&nbsp;Evans Xorse Amuzu,&nbsp;Daniel Kandonga,&nbsp;Victoria Nembaware,&nbsp;Mario Jonas,&nbsp;Jade Hotchkiss,&nbsp;Upendo Masamu,&nbsp;Arthemon Nguweneza,&nbsp;Bruno P. Mmbando,&nbsp;Irene Minja,&nbsp;Agnes Jonathan,&nbsp;Nicola Mulder,&nbsp;Emmanuel Balandya,&nbsp;Alex Osei-Akoto,&nbsp;Vivian Paintsil,&nbsp;Julie Makani,&nbsp;Obiageli Nnodu,&nbsp;Members of SPARCO Nigeria,&nbsp;Raphael Z. Sangeda,&nbsp;Andre Pascal Kengne,&nbsp;Gaston Kuzamunu,&nbsp;Ambroise Wonkam","doi":"10.1002/jha2.70044","DOIUrl":"https://doi.org/10.1002/jha2.70044","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Sickle cell disease (SCD) is most prevalent in Sub-Saharan Africa (SSA), where incomplete patient profiles and limited management strategies hinder research and healthcare standards.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We describe the first large-scale and multinational assessment of 13,403 SCD patients enrolled from 2017–2021 across 31 facilities in Ghana, Nigeria, and Tanzania into the SickleInAfrica consortium registry. We used hierarchical regression models to estimate and analyze the demographics, adoption levels of SCD diagnosis and therapies.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The average age at diagnosis was 3 months, 19 months and 3 years in Ghana, Nigeria and Tanzania respectively, reflecting differences in country-specific newborn screening programs and policies. Hydroxyurea (HU) use was highest in Ghana (21%), followed by Nigeria (12%) and Tanzania (6%), with significant variability across facilities. Sex differences in SCD management were observed, with males more likely to receive HU and blood transfusions. At the consortium level, HU initiation correlated with enrolment age rather than age at diagnosis, highlighting the need for earlier intervention.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Our findings highlight the potential of the SickleInAfrica registry toward enhancing understanding of regional disparities in SCD care and potential gender inequalities, emphasizing the need for enabling policies toward strengthened SCD research and improved quality of life and care of patients in Africa.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70044","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143908975","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Outcomes With Venetoclax 50 mg, Hypomethylating Agents, and Voriconazole or Posaconazole in Acute Myeloid Leukemia Venetoclax 50mg,低甲基化药物,Voriconazole或泊沙康唑治疗急性髓系白血病
EJHaem Pub Date : 2025-05-06 DOI: 10.1002/jha2.70049
Kendall Diebold, Devan Parker, Sarah Worth, Manuel Espinoza-Gutarra, Pankit Vachhani, Kimo Bachiashvili, Sravanti Rangaraju, Razan Mohty, Ravi Bhatia, Omer Jamy
{"title":"Outcomes With Venetoclax 50 mg, Hypomethylating Agents, and Voriconazole or Posaconazole in Acute Myeloid Leukemia","authors":"Kendall Diebold,&nbsp;Devan Parker,&nbsp;Sarah Worth,&nbsp;Manuel Espinoza-Gutarra,&nbsp;Pankit Vachhani,&nbsp;Kimo Bachiashvili,&nbsp;Sravanti Rangaraju,&nbsp;Razan Mohty,&nbsp;Ravi Bhatia,&nbsp;Omer Jamy","doi":"10.1002/jha2.70049","DOIUrl":"https://doi.org/10.1002/jha2.70049","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Real-world evidence for hypomethylating agent (HMA) + venetoclax 50 mg (VEN50) with voriconazole and posaconazole in acute myeloid leukemia (AML), is limited.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We evaluated outcomes of patients with newly-diagnosed AML treated with HMA + VEN50 with either posaconazole (<i>n</i> = 23) or voriconazole (<i>n</i> = 95).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>We report that treatment with HMA + VEN50 with either azole elicits a response rate similar to that described in the VIALE-A trial. Reducing the VEN dose to 50 mg with either strong CYP3A4 inhibitor did not compromise on the efficacy of the combination.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>HMA + VEN50 with either posaconazole or voriconazole yields comparable responses to higher doses of VEN reported previously.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Clinical trial registration</h3>\u0000 \u0000 <p>The authors have confirmed clinical trial registration is not needed for this submission.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70049","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143908915","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Prevalence of CCR7-Positive CD8 T Cells as a Prognostic Factor in B-Cell Maturation Antigen -Targeted Chimeric Antigen Receptor T Cell Therapy ccr7阳性CD8 T细胞的流行作为b细胞成熟抗原靶向嵌合抗原受体T细胞治疗的预后因素
EJHaem Pub Date : 2025-05-05 DOI: 10.1002/jha2.70040
Yoshiaki Marumo, Masaki Ri, Toru Ebina, Tomoyuki Nakamura, Yoshiko Oshima, Takahiro Nakashima, Shiori Kinoshita, Tomotaka Suzuki, Tomoko Narita, Takaomi Sanda, Hirokazu Komatsu, Shinsuke Iida
{"title":"Prevalence of CCR7-Positive CD8 T Cells as a Prognostic Factor in B-Cell Maturation Antigen -Targeted Chimeric Antigen Receptor T Cell Therapy","authors":"Yoshiaki Marumo,&nbsp;Masaki Ri,&nbsp;Toru Ebina,&nbsp;Tomoyuki Nakamura,&nbsp;Yoshiko Oshima,&nbsp;Takahiro Nakashima,&nbsp;Shiori Kinoshita,&nbsp;Tomotaka Suzuki,&nbsp;Tomoko Narita,&nbsp;Takaomi Sanda,&nbsp;Hirokazu Komatsu,&nbsp;Shinsuke Iida","doi":"10.1002/jha2.70040","DOIUrl":"https://doi.org/10.1002/jha2.70040","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Chimeric antigen receptor T (CAR-T) cell therapy is effective for relapsed or refractory multiple myeloma (RRMM); however, relapse after the B-cell maturation antigen (BCMA) CAR-T cell therapy is associated with poor outcome. Hence, appropriate biomarkers that can predict the outcome are needed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Patients who received idecabtagene vicleucel, a BCMA-targeted CAR-T cell therapy, were divided into two groups according to a cut-off value of 180 days for the progression-free survival (PFS) event.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Patients in the short responder group were older at diagnosis, had a shorter time from diagnosis to apheresis, and more frequently had prior bispecific antibody treatment or alkylator-containing chemotherapies, while they received less immunomodulatory drugs-based chemotherapy just prior to apheresis. Apheresis samples collected from the long responder group had significantly higher proportion of CD8-positive naïve or stem cell memory (CCR7<sup>+</sup>CD45RO<sup>−</sup>) or central memory (CCR7<sup>+</sup>CD45RO<sup>+</sup>) T cells. When these two T cell subsets were combined into CCR7-positive CD8 T cells, the patients with high levels of CCR7-positive CD8 T cells showed significantly better PFS.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>In the future, our results will help us to select specific patients that are likely to have a more favorable outcome and should contribute to establishing an optimal application strategy for CAR-T cell therapies in RRMM.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70040","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143904985","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Safe and Efficacious Use of Low-Dose Rituximab in Postpubertal Paediatric Patients With Immune Thrombocytopenia 低剂量利妥昔单抗在青春期后儿童免疫性血小板减少症患者中的安全有效应用
EJHaem Pub Date : 2025-05-01 DOI: 10.1002/jha2.70010
Jennifer Darlow, Gerard Gurumurthy, Nathan Jeffreys, Lianna Reynolds, Vismay Deshani, John Grainger
{"title":"Safe and Efficacious Use of Low-Dose Rituximab in Postpubertal Paediatric Patients With Immune Thrombocytopenia","authors":"Jennifer Darlow,&nbsp;Gerard Gurumurthy,&nbsp;Nathan Jeffreys,&nbsp;Lianna Reynolds,&nbsp;Vismay Deshani,&nbsp;John Grainger","doi":"10.1002/jha2.70010","DOIUrl":"https://doi.org/10.1002/jha2.70010","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Rituximab, a CD20 monoclonal antibody, is used in chronic/refractory immune thrombocytopenia (ITP). Standard dosing is 375 mg/m<sup>2</sup> weekly for 4 weeks alongside dexamethasone. A lower dose of Rituximab at 100 mg weekly demonstrates comparable efficacy that is well tolerated. This study evaluates lower-dose Rituximab with dexamethasone in postpubertal pediatric ITP patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Patients treated with 100 mg weekly Rituximab for 4 weeks alongside dexamethasone at 10 mg/m<sup>2</sup> on days 1–5 and 21–25 were assessed for response and safety.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of the 10 patients treated, six responded completely, one partially and three showed no response. Four responders maintained their response over 2 years. One Rituximab-related infusion reaction and one Dexamethasone-related adverse event were reported.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Rituximab 100 mg weekly may be non-inferior to 375 mg/m<sup>2</sup> weekly for paediatric patients.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70010","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143892862","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Belantamab Mafodotin Monotherapy for Multiply-Relapsed Myeloma: A Retrospective Study From the United Kingdom and the Republic of Ireland Belantamab Mafodotin单药治疗多发性复发骨髓瘤:来自英国和爱尔兰共和国的回顾性研究
EJHaem Pub Date : 2025-04-30 DOI: 10.1002/jha2.70039
Edmund C. R. Watson, Faouzi Djebbari, Fotios Panitsas, Grant Vallance, Samir Asher, Malahat Saeed, Mairi Walker, Matthew Powell, Alexandros Rampotas, Heather Leary, Akhil Khera, Angharad Atkinson, Ni Ni Aung, Gillian Brearton, Joseph Froggatt, Ezzat El Hassadi, Ellen Gokkel, Sarah Lawless, Beena Salhan, Salim Shafeek, Anand Lokare, Carol Stirling, Udo Oppermann, Richard Soutar, Rakesh Popat, Charalampia Kyriakou, Karthik Ramasamy
{"title":"Belantamab Mafodotin Monotherapy for Multiply-Relapsed Myeloma: A Retrospective Study From the United Kingdom and the Republic of Ireland","authors":"Edmund C. R. Watson,&nbsp;Faouzi Djebbari,&nbsp;Fotios Panitsas,&nbsp;Grant Vallance,&nbsp;Samir Asher,&nbsp;Malahat Saeed,&nbsp;Mairi Walker,&nbsp;Matthew Powell,&nbsp;Alexandros Rampotas,&nbsp;Heather Leary,&nbsp;Akhil Khera,&nbsp;Angharad Atkinson,&nbsp;Ni Ni Aung,&nbsp;Gillian Brearton,&nbsp;Joseph Froggatt,&nbsp;Ezzat El Hassadi,&nbsp;Ellen Gokkel,&nbsp;Sarah Lawless,&nbsp;Beena Salhan,&nbsp;Salim Shafeek,&nbsp;Anand Lokare,&nbsp;Carol Stirling,&nbsp;Udo Oppermann,&nbsp;Richard Soutar,&nbsp;Rakesh Popat,&nbsp;Charalampia Kyriakou,&nbsp;Karthik Ramasamy","doi":"10.1002/jha2.70039","DOIUrl":"https://doi.org/10.1002/jha2.70039","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Belantamab mafodotin (belamaf) was the first BCMA-targeting immunotherapy licensed in myeloma and was available as monotherapy for a fifth or greater line of treatment. Outcomes for patients in the United Kingdom and the Republic of Ireland potentially differ from those of other regions and may illuminate factors predicting response to therapy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods and Results</h3>\u0000 \u0000 <p>We performed a retrospective study of patients treated with belamaf monotherapy in the United Kingdom and the Republic of Ireland. In our cohort of 88 patients, we saw an overall response rate (ORR) of 60%, a median progression-free survival (PFS) of 8.7 months and a median duration of response (DoR) of 15.8 months. The spectrum of adverse events was as expected, with 84% (71/85) of patients experiencing toxicity. Eye-related adverse events were the most common, affecting 66% (56/85), leading to dose reduction or delay in 41% (35/85) and discontinuation in 6% (5/85). We specifically assessed physician decision-making in the context of ocular side effects and found a relatively high frequency of the drug being administered despite moderate levels of toxicity.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Our cohort's ORR is significantly different from those of the DREAMM-2 and -3 trials and other real-world studies, though a long-duration response has been reported in other cohorts. Comparative analysis with other real-world studies did not reveal any significant factors predictive of ORR. The frequent administration of belamaf to patients with eye disease may well reflect a more pragmatic approach than was originally prescribed in the landmark trials.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Trial Registration</h3>\u0000 \u0000 <p>The authors have confirmed clinical trial registration is not needed for this submission.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70039","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143892849","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Balancing the Risk of Cardiotoxicity Outcomes in Treatment Selection for Multiple Myeloma: A Retrospective Multicenter Evaluation of Ixazomib, Lenalidomide, and Dexamethasone (IRd) Versus Carfilzomib, Lenalidomide, and Dexamethasone (KRd) 在多发性骨髓瘤治疗选择中平衡心脏毒性结局的风险:伊沙唑米、来那度胺和地塞米松(IRd)与卡非佐米、来那度胺和地塞米松(KRd)的回顾性多中心评价
EJHaem Pub Date : 2025-04-28 DOI: 10.1002/jha2.70038
Benjamin J. Lee, Michael Sayer, Ali A. Naqvi, Karen T. Mai, Pranav M. Patel, Lisa X. Lee, Aya F. Ozaki
{"title":"Balancing the Risk of Cardiotoxicity Outcomes in Treatment Selection for Multiple Myeloma: A Retrospective Multicenter Evaluation of Ixazomib, Lenalidomide, and Dexamethasone (IRd) Versus Carfilzomib, Lenalidomide, and Dexamethasone (KRd)","authors":"Benjamin J. Lee,&nbsp;Michael Sayer,&nbsp;Ali A. Naqvi,&nbsp;Karen T. Mai,&nbsp;Pranav M. Patel,&nbsp;Lisa X. Lee,&nbsp;Aya F. Ozaki","doi":"10.1002/jha2.70038","DOIUrl":"https://doi.org/10.1002/jha2.70038","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Carfilzomib use has been extensively associated with cardiovascular toxicity; the risk with ixazomib, a novel oral proteasome inhibitor, is underreported and no large comparative analysis is available.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted a retrospective cohort study utilizing the TriNetX platform to compare toxicity outcomes among multiple myeloma patients who received lenalidomide, dexamethasone, and ixazomib (IRd) or carfilzomib (KRd).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>After propensity-score-matching 478 patients from each cohort, the onset of new heart failure (HR 0.25; <i>p </i>&lt; 0.001) and arrhythmias (HR 0.57; <i>p</i> = 0.014) at 6 months were significantly lower with IRd while overall survival at 3 years was similar (<i>p</i> = 0.50).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>IRd is associated with a significantly lower risk of cardiac toxicities compared to KRd.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70038","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143883873","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Management of Short Saphenous Vein Thrombosis Close to the Saphenopopliteal Junction: A Survey of the Membership of HaemSTAR, British Society for Haemostasis and Thrombosis and VTE Exemplar Centres in the United Kingdom 靠近隐静脉连接处的短隐静脉血栓形成的处理:对HaemSTAR成员的调查,英国止血和血栓形成协会和VTE范例中心在英国
EJHaem Pub Date : 2025-04-26 DOI: 10.1002/jha2.70037
Kirollos Salah Kamel, Lucy Wood, Nicola Curry
{"title":"Management of Short Saphenous Vein Thrombosis Close to the Saphenopopliteal Junction: A Survey of the Membership of HaemSTAR, British Society for Haemostasis and Thrombosis and VTE Exemplar Centres in the United Kingdom","authors":"Kirollos Salah Kamel,&nbsp;Lucy Wood,&nbsp;Nicola Curry","doi":"10.1002/jha2.70037","DOIUrl":"https://doi.org/10.1002/jha2.70037","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>The optimal management of superficial thrombophlebitis (STP) close to the saphenopopliteal junction (SPJ) is not known.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted an online survey of members of the HaemSTAR network, British society of haemostasis and thrombosis and UK VTE exemplar network over a 6-week period.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Fifty-three respondents participated in the survey (estimated 22% response rate). Note that 89% of respondents indicated they would manage all STP at the SPJ with anticoagulation, with 70% indicating they would offer 3 months of therapeutic anticoagulation. The most common threshold for instigating anticoagulation was being within 3 cm off the SPJ (68%). Factors most associated with the decision to anticoagulate included previous thrombosis, active malignancy, persistent immobilisation and severe symptoms (with hospitalisation, hyperestrogenaemic states, thrombophilia and recent surgery being additionally identified in the non-treatment group).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Despite lack of evidence, most UK practitioners surveyed offered intermediate to treatment doses of anticoagulation in the case of STP within 3 cm of the SPJ. Further research is needed to assess the validity of this approach.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Trial Registration</h3>\u0000 \u0000 <p>The authors have confirmed clinical trial registration is not needed for this submission.</p>\u0000 </section>\u0000 </div>","PeriodicalId":72883,"journal":{"name":"EJHaem","volume":"6 3","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-04-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/jha2.70037","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143875551","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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