British Journal of Haematology最新文献_第9页

Long-term efficacy and safety of avatrombopag in Chinese children with primary immune thrombocytopenia: A real-world observational study 阿伐波帕治疗原发性免疫性血小板减少症儿童的长期疗效和安全性：一项真实世界的观察性研究。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-05 DOI: 10.1111/bjh.19973

Nan Wang, Zhifa Wang, Jingjing Liu, Yu Hu, Shuyue Dong, Hui Chen, Jinxi Meng, Jingyao Ma, Zhenping Chen, Xiaoling Cheng, Runhui Wu

{"title":"Long-term efficacy and safety of avatrombopag in Chinese children with primary immune thrombocytopenia: A real-world observational study","authors":"Nan Wang, Zhifa Wang, Jingjing Liu, Yu Hu, Shuyue Dong, Hui Chen, Jinxi Meng, Jingyao Ma, Zhenping Chen, Xiaoling Cheng, Runhui Wu","doi":"10.1111/bjh.19973","DOIUrl":"10.1111/bjh.19973","url":null,"abstract":"<div>\u0000 \u0000 <p>Avatrombopag is a newly approved thrombopoietin receptor agonist for second-line treatment of chronic immune thrombocytopenia (ITP) in adults. Our previous study showed its efficacy and safety in a small sample of paediatric ITP patients. However, large samples and long-term data are still lacking. Children diagnosed with ITP and treated with avatrombopag for at least 4 weeks were enrolled. In 94 ITP patients with a median age of 7.43 (interquartile range (IQR), 4.82, 10.80) years, the median effective dose was 10 (IQR, 10, 20) mg for children under 6 years old and 20 (IQR, 20, 40) mg for children under 18 years old. The overall response was achieved in 72.3% (68/94) and 73.4% (58/79) of patients within 4 weeks and 12 weeks. The sustained response at 24 weeks and 48 weeks were 62.3% (33/53) and 51.6% (16/31) respectively. The occurrence of bleeding events, rescue therapy and concomitant ITP medication decreased during the follow-up period. For safety, thrombocytosis (platelet count ≥400 × 10<sup>9</sup>/L) was the most frequent adverse event (AE) observed in 44 children 97 times. Long-term treatment with avatrombopag in ITP children showed a rapid and sustained platelet response and good bleeding control without significant or new AEs.</p>\u0000 </div>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 3","pages":"935-943"},"PeriodicalIF":5.1,"publicationDate":"2025-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142930097","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Avatrombopag in adults with immune thrombocytopenia: A multicentre real-life observational study in Madrid, Spain (AVAMAD study) avatromopag治疗成人免疫性血小板减少症：西班牙马德里的一项多中心现实观察研究（AVAMAD研究）。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-05 DOI: 10.1111/bjh.19975

Cristina Pascual-Izquierdo, Mª. Jose Llacer-Ferrandis, Almudena de-la-Iglesia, Silvia Monsalvo-Saornil, María Menor-Gómez, Juan Jose Gil-Fernández, Esther Chica-Gullon, María Teresa Álvarez-Román, Gloria Perez-Segura, Denis Zafra, Ariana Ortuzar-Pasalodos, Isabel Teresa González-Gascón-y-Marín, Gemma Moreno, Teresa Arquero-Portero, Marta Moreno-Carbonell, Nuria Revilla

{"title":"Avatrombopag in adults with immune thrombocytopenia: A multicentre real-life observational study in Madrid, Spain (AVAMAD study)","authors":"Cristina Pascual-Izquierdo, Mª. Jose Llacer-Ferrandis, Almudena de-la-Iglesia, Silvia Monsalvo-Saornil, María Menor-Gómez, Juan Jose Gil-Fernández, Esther Chica-Gullon, María Teresa Álvarez-Román, Gloria Perez-Segura, Denis Zafra, Ariana Ortuzar-Pasalodos, Isabel Teresa González-Gascón-y-Marín, Gemma Moreno, Teresa Arquero-Portero, Marta Moreno-Carbonell, Nuria Revilla","doi":"10.1111/bjh.19975","DOIUrl":"10.1111/bjh.19975","url":null,"abstract":"<p>Immune thrombocytopenia (ITP) is a rare acquired disorder where thrombopoietin-receptor agonists have become mainstays of ITP treatment. With the recent approval of avatrombopag (AVA), real-world studies are essential to evaluate its efficacy and safety. Our study of 66 adult ITP patients treated with AVA showed a high response rate. After starting AVA, 74.2% did not require rescue medications, with minimal adverse effects. Additionally, 56.0% of patients reduced other ITP medications, and all patients aged ≥65 years responded to AVA. Results should be confirmed in larger studies, but AVA appears to be an effective and safe treatment for ITP patients.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 2","pages":"652-656"},"PeriodicalIF":5.1,"publicationDate":"2025-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.19975","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142930094","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Should adolescents and young adults with Hodgkin lymphoma be treated as children or adults? 患有霍奇金淋巴瘤的青少年和年轻人应该作为儿童还是成人治疗？

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-05 DOI: 10.1111/bjh.19985

Lucille Lew-Derivry, Florian Chevillon, Pauline Brice, Camille Bigenwald, Judith Landman-Parker, Thierry Leblanc, Nicolas Boissel, Aurélie Cabannes-Hamy

{"title":"Should adolescents and young adults with Hodgkin lymphoma be treated as children or adults?","authors":"Lucille Lew-Derivry, Florian Chevillon, Pauline Brice, Camille Bigenwald, Judith Landman-Parker, Thierry Leblanc, Nicolas Boissel, Aurélie Cabannes-Hamy","doi":"10.1111/bjh.19985","DOIUrl":"10.1111/bjh.19985","url":null,"abstract":"<div>\u0000 \u0000 <p>Hodgkin lymphoma (HL) is one of the most common cancers in adolescents and young adults (AYA). Paediatric and adult therapeutic strategies diverge while sharing the common objective: maintaining optimal efficacy with less long-term toxicity. However, few studies have compared the outcome of AYA treated according to one or the other approaches. Among the 148 patients aged 15–25 years, treated at Saint-Louis Hospital for newly diagnosed HL between 2012 and 2018, 71 were treated according to an adult protocol and 77 were treated according to a paediatric one. The 5-year overall survival (OS) and progression-free survival (PFS) were, respectively, 100% and 85%, with no significant difference between treatment groups (85% in paediatric vs. 86% in adult, <i>p</i> = 0.7). Overall, the 5-year PFS was 100% for early favourable stages and 78% for advanced stages. A higher risk of short-term steroid and vincristine-related toxicities was observed in paediatric regimen, whereas a higher risk of late toxicities was expected in adult regimen, due to higher anthracyclines, procarbazine, bleomycin and radiotherapy exposure. These results confirm the excellent outcome of AYA patients with HL, whatever the treatment strategies. They justify a tailor-made therapeutic decision and highlight the importance of managing AYA patients in dedicated units with trained professionals.</p>\u0000 </div>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 3","pages":"907-918"},"PeriodicalIF":5.1,"publicationDate":"2025-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142930099","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Iberdomide, ixazomib and dexamethasone in elderly patients with multiple myeloma at first relapse. 伊伯度胺、伊唑唑米和地塞米松在老年多发性骨髓瘤首次复发中的作用。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-05 DOI: 10.1111/bjh.19978

Cyrille Touzeau, Xavier Leleu, Mourad Tiab, Margaret Macro, Aurore Perrot, Julie Gay, Carine Chateleix, Stéphane Moreau, Lionel Karlin, Caroline Jacquet, Salomon Manier, Cyrille Hulin, Olivier Decaux, Valentine Richez, Thomas Chalopin, Mohamad Mohty, Frédérique Orsini-Piocelle, Denis Caillot, Cécile Sonntag, Marguerite Vignon, Arthur Bobin, Hervé Avet-Loiseau, Alexandra Jobert, Lucie Planche, Jill Corre, Philippe Moreau

{"title":"Iberdomide, ixazomib and dexamethasone in elderly patients with multiple myeloma at first relapse.","authors":"Cyrille Touzeau, Xavier Leleu, Mourad Tiab, Margaret Macro, Aurore Perrot, Julie Gay, Carine Chateleix, Stéphane Moreau, Lionel Karlin, Caroline Jacquet, Salomon Manier, Cyrille Hulin, Olivier Decaux, Valentine Richez, Thomas Chalopin, Mohamad Mohty, Frédérique Orsini-Piocelle, Denis Caillot, Cécile Sonntag, Marguerite Vignon, Arthur Bobin, Hervé Avet-Loiseau, Alexandra Jobert, Lucie Planche, Jill Corre, Philippe Moreau","doi":"10.1111/bjh.19978","DOIUrl":"https://doi.org/10.1111/bjh.19978","url":null,"abstract":"<p><p>Most transplant-ineligible patients present with multiple myeloma (MM) refractory to lenalidomide and/or anti-CD38 monoclonal antibody at first relapse and represent a difficult-to-treat population. The Intergroupe Francophone du Myélome phase 2 study iberdomide, ixazomib and dexamethasone (I2D) evaluated the oral triplet iberdomide, ixazomib and dexamethasone in MM patients aged ≥70 years at first relapse (NCT04998786). Seventy patients were enrolled to receive iberdomide (1.6 mg on day 1-21), ixazomib (3 mg on day 1, 8, 15) and dexamethasone (20 mg on day 1, 8, 15, 22 on cycle 1-2 and 10 mg on day 1, 8, 15, 22 on cycle 3-6) (28-day cycle) until disease progression. Median age was 76; 50% patients were frail according to the International Myeloma Working Group frailty score; 74% and 37% were refractory to lenalidomide and daratumumab respectively. With a median follow-up of 14 months, the overall response rate was 64%, including 36% very good partial response or better. The 12-month progression-free survival, duration of response and overall survival were 52%, 76% and 86% respectively. The most common (46%) grade 3-4 toxicity was neutropenia. Non-haematological adverse events were mostly grade 1 or 2. Overall, I2D demonstrated a favourable risk-benefit profile in elderly MM patients at first relapse, including in patients with lenalidomide and daratumumab refractory disease.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142930095","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

Platelets and monocytes: A dynamic duo aiding in the prediction of the clinical response to thrombopoietin receptor agonists in immune thrombocytopenia? 血小板和单核细胞：在预测免疫血小板减少患者对血小板生成素受体激动剂的临床反应中的一个动态组合？

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-03 DOI: 10.1111/bjh.19991

Rick Kapur

{"title":"Platelets and monocytes: A dynamic duo aiding in the prediction of the clinical response to thrombopoietin receptor agonists in immune thrombocytopenia?","authors":"Rick Kapur","doi":"10.1111/bjh.19991","DOIUrl":"https://doi.org/10.1111/bjh.19991","url":null,"abstract":"<p><p>Patients with immune thrombocytopenia (ITP) suffer from an autoimmune bleeding disorder with an isolated low number of platelets. Platelets and megakaryocytes are targeted by the immune system, due to a loss of immune tolerance, via the action of platelet-autoantibodies and/or cytotoxic T cells. ITP is a highly variable disorder regarding the underlying biological mechanisms, disease trajectories and treatment responses. Predictive indicators are therefore strongly warranted. To analyze the predictive response to the thrombopoietin receptor agonist eltrombopag, in this issue of the British Journal of Haematology, Osuna-Gomez and co-workers research platelet-leukocyte complexes in 38 ITP patients upon treatment with eltrombopag. Elevated platelet counts upon treatment with eltrombopag were associated with increased complexes of platelets bound to leucocytes. A higher percentage of monocytes bound to platelets, with an increased IL-10 production in monocytes, was found to be associated to a favorable clinical response to treatment with eltrombopag. Further validation is required but these findings reveal the potential of analyzing platelet-monocyte complexes as a predictive indicator for the clinical response to eltrombopag in patients with ITP. Commentary on: Osuna-Gómez et al. Interplay of leucocyte-platelet complexes and clinical response to eltrombopag in immune thrombocytopenia patients. Br J Haematol 2025 (Online ahead of print). doi: 10.1111/bjh.19779.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142918719","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

UK consensus statement on the use of plerixafor in healthy stem cell donors 英国关于在健康干细胞供体中使用普利沙福的共识声明。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-02 DOI: 10.1111/bjh.19963

Tania Dexter, Sara Lozano, Angela Leather, Mary Slatter, Kalinga Perera, James Griffin, Khaled El-Ghariani, Fiona Dignan, Adrian Bloor, Chloe Anthias, the UK Unrelated Donor Registries and BSBMTCT

引用次数: 0

Update on B-cell maturation antigen-directed therapies in AL amyloidosis b细胞成熟抗原导向治疗AL淀粉样变性的最新进展。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-02 DOI: 10.1111/bjh.19960

Krzysztof Jamroziak, Klaudia Zielonka, Jahanzaib Khwaja, Ashutosh D. Wechalekar

{"title":"Update on B-cell maturation antigen-directed therapies in AL amyloidosis","authors":"Krzysztof Jamroziak, Klaudia Zielonka, Jahanzaib Khwaja, Ashutosh D. Wechalekar","doi":"10.1111/bjh.19960","DOIUrl":"10.1111/bjh.19960","url":null,"abstract":"<div>\u0000 \u0000 <p>Systemic light chain (AL) amyloidosis is a rare clonal plasma cell disorder characterized by the production of amyloidogenic immunoglobulin light chains, which causes the formation and deposition of amyloid fibrils, leading to multi-organ dysfunction. Current treatment is directed at the underlying plasma cell clone to achieve a profound reduction in the monoclonal free light chain production. The standard-of-care first-line therapy is a combination of daratumumab, cyclophosphamide, bortezomib and dexamethasone (D-VCd regimen), resulting in high rates of haematological and organ responses. However, AL amyloidosis remains incurable, and all patients inevitably relapse. Hence, novel treatment options are needed for patients with an inadequate response or relapsed/refractory disease. B-cell maturation antigen (BCMA) is a tumour necrosis factor (TNF receptor superfamily receptor overexpressed on plasma cells in multiple myeloma (MM) and AL amyloidosis. Recently, several novel anti-BCMA immunotherapies have been approved for the treatment of relapsed/refractory MM, including antibody–drug conjugate belantamab mafodotin, bispecific antibodies teclistamab and elranatamab and chimeric antigen receptor T-cell therapies idecabtagene vicleucel and ciltacabtagene autoleucel. Despite lower expression than in MM, BCMA is also a promising target in AL amyloidosis. This review aims to provide up-to-date information on the efficacy and toxicity of anti-BCMA therapy in AL amyloidosis.</p>\u0000 </div>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 3","pages":"817-831"},"PeriodicalIF":5.1,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.19960","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142918728","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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

EGLN1 mutations in Cis can induce congenital erythrocytosis with thromboses by increasing protein instability EGLN1突变可通过增加蛋白不稳定性诱导先天性红细胞增多伴血栓形成。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-02 DOI: 10.1111/bjh.19932

Serge Carillo, Marine Delamare, Laurent Henry, Nada Maaziz, Hana Safraou, Betty Gardie, Thierry Lavabre-Bertrand

引用次数: 0

Systemic lupus erythematosus-associated autoantibodies in sickle cell disease: Spontaneous emergence in a patient and in transgenic sickle mice. 镰状细胞病中的系统性红斑狼疮相关自身抗体：在患者和转基因镰状细胞小鼠中自发出现。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-02 DOI: 10.1111/bjh.19972

Urshita Sinha, Suman Setty, Céleste Pilon, Julia J Brown, Maria Armila Ruiz, Guohui Ren, Joyce Rauch, Santosh L Saraf, Jerrold S Levine

引用次数: 0

Plasma inflammatory and angiogenic protein profiling of patients with sickle cell disease 镰状细胞病患者血浆炎症和血管生成蛋白谱分析

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2025-01-01 DOI: 10.1111/bjh.19970

L. A. de Ligt, A. E. Gaartman, K. Konté, S. Thakoerdin, K. Fijnvandraat, T. W. Kuijpers, R. van Bruggen, B. J. Biemond, E. Nur

{"title":"Plasma inflammatory and angiogenic protein profiling of patients with sickle cell disease","authors":"L. A. de Ligt, A. E. Gaartman, K. Konté, S. Thakoerdin, K. Fijnvandraat, T. W. Kuijpers, R. van Bruggen, B. J. Biemond, E. Nur","doi":"10.1111/bjh.19970","DOIUrl":"10.1111/bjh.19970","url":null,"abstract":"<p>In this study, we aimed to explore the inflammatory and angiogenic pathways in sickle cell disease (SCD). We used proximity extension assay technology (Olink) to measure 92 plasma proteins involved in inflammation and angiogenesis. Plasma samples were collected from 57 SCD patients (sickle cell anaemia/HbS-β<sup>0</sup> thalassaemia-thalassaemia) in steady-state and 13 healthy ethnicity-matched healthy controls (HCs). From 15 patients, paired samples were collected during both steady-state and vaso-occlusive episodes (VOEs) and from 23 SCD patients longitudinal samples were collected before and after treatment with either voxelotor (<i>n</i> = 10), hydroxyurea (<i>n</i> = 8) or allogeneic haematopoietic stem-cell transplantation (<i>n</i> = 5). Fifty plasma proteins were differentially expressed in steady-state SCD patients as compared to HC. These included proteins involved in angiogenesis (i.e. ANGPT1, ANGPT2 and VEGFA), the IL-18 signalling pathway (i.e. IL-6, IL-10, IL-18), T-cell activation (i.e. LAG3, PDCD1) and natural killer (NK)-cell activation (CD244, NCR1, GZMB). While proteins involved in angiogenesis and the IL-18 signalling pathway were further upregulated during VOE, levels of several proteins involved in the IL-18 pathway, T-cell and NK-cell activation and angiogenesis, restored towards levels detected in HCs after curative or disease-modifying treatment. These findings might contribute to a better understanding of SCD pathophysiology and identifying potential new targets for therapeutic interventions.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"206 3","pages":"954-964"},"PeriodicalIF":5.1,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.19970","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142913164","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"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