British Journal of Haematology最新文献_第10页

Olutasidenib demonstrates significant clinical activity in mutated IDH1 acute myeloid leukaemia arising from a prior myeloproliferative neoplasm. Olutasidenib在先前骨髓增殖性肿瘤引起的突变IDH1急性髓性白血病中显示出显着的临床活性。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-19 DOI: 10.1111/bjh.19944

Stéphane De Botton, Christian Récher, Jorge Cortes, Antonio Curti, Pierre Fenaux, Pierre Peterlin, Arnaud Pigneux, Karen Yee, Andrew Wei, Alice Mims, Gary Schiller, Mwe Mwe Chao, Hua Tian, Justin M Watts

{"title":"Olutasidenib demonstrates significant clinical activity in mutated IDH1 acute myeloid leukaemia arising from a prior myeloproliferative neoplasm.","authors":"Stéphane De Botton, Christian Récher, Jorge Cortes, Antonio Curti, Pierre Fenaux, Pierre Peterlin, Arnaud Pigneux, Karen Yee, Andrew Wei, Alice Mims, Gary Schiller, Mwe Mwe Chao, Hua Tian, Justin M Watts","doi":"10.1111/bjh.19944","DOIUrl":"https://doi.org/10.1111/bjh.19944","url":null,"abstract":"Acute myeloid leukaemia (AML) arising from a myeloproliferative neoplasm (MPN) is more aggressive and less responsive to therapies compared to de novo AML. Olutasidenib, an oral small-molecule inhibitor of mutated IDH1 (mIDH1), showed encouraging and durable responses in a phase 1/2 study of adults with post-MPN mIDH1 AML. Patients received olutasidenib 150 mg BID monotherapy or in combination with azacitidine. Primary end-points: safety and best response defined as complete remission (CR), CR with partial haematological recovery or morphological leukaemia-free state (MLFS). Analysis included 15 patients with post-MPN mIDH1 AML; 10 had relapsed or refractory AML and five had newly diagnosed AML. Six were treated with olutasidenib monotherapy and nine in combination with azacitidine. Treatment emergent adverse events occurred in 15 patients, three of whom discontinued therapy. CR: 40% (n = 6/15); median duration of response: 15.6 months (range: 1.7-44.3); CR with incomplete haematological recovery: 13% (n = 2/15); MLFS: 7% (n = 1/15); composite complete remission (CRc): 53% (n = 8/15); and overall response rate (ORR): 60% (9/18). Median duration of CRc and ORR: 13.15 (range: 2.4-48.7) and 14.3 months (range: 2.4-48.7), respectively, and median overall survival: 13.8 months (95% confidence interval: 3.70-23.7). Olutasidenib demonstrated encouraging response rates with a manageable safety profile for patients with post-MPN mIDH1 AML.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142862636","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

Landscape of somatic mutations and clonal evolution in NUP98-rearranged adult acute myeloid leukaemia. nup98重排成人急性髓性白血病的体细胞突变和克隆进化景观。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-19 DOI: 10.1111/bjh.19962

Candace Frerich, Peng Li, Philipp W Raess, Jennifer Dunlap, Curtis Lachowiez, Jose Solis-Ruiz, Richard Press, Nicola Long, Ronan T Swords, Joanna Wiszniewska, Guang Fan, Wei Xie

引用次数: 0

Treating asymptomatic follicular lymphoma: What is the score? 治疗无症状滤泡性淋巴瘤：评分是多少？

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-18 DOI: 10.1111/bjh.19949

Ghassan Zammar, Chan Y Cheah

引用次数: 0

Corticosteroids plus metformin versus corticosteroids as front-line treatment for patients with newly diagnosed ITP and pre-existing type 2 diabetes mellitus: A multicentre propensity score-matched study. 糖皮质激素加二甲双胍与糖皮质激素作为新诊断ITP和既往2型糖尿病患者的一线治疗：一项多中心倾向评分匹配研究

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-18 DOI: 10.1111/bjh.19940

Xi-Ran Hou, Zhen-Yu Yan, Shuang Liu, Na Gao, Jian Chen, Ya-Wen Wang, Liang Wang, Zhao Li, Xin-Ru Wang, Qiao-Feng Dong, Qiu-Yan Wang, Lin Sun, Yan-Ming Wang, Ji Ma, Ya-Jing Zhao, Zhi-Long Xu, Cong-Cong Cao, Jun Peng, Ming Hou, Xin-Guang Liu

{"title":"Corticosteroids plus metformin versus corticosteroids as front-line treatment for patients with newly diagnosed ITP and pre-existing type 2 diabetes mellitus: A multicentre propensity score-matched study.","authors":"Xi-Ran Hou, Zhen-Yu Yan, Shuang Liu, Na Gao, Jian Chen, Ya-Wen Wang, Liang Wang, Zhao Li, Xin-Ru Wang, Qiao-Feng Dong, Qiu-Yan Wang, Lin Sun, Yan-Ming Wang, Ji Ma, Ya-Jing Zhao, Zhi-Long Xu, Cong-Cong Cao, Jun Peng, Ming Hou, Xin-Guang Liu","doi":"10.1111/bjh.19940","DOIUrl":"https://doi.org/10.1111/bjh.19940","url":null,"abstract":"Corticosteroids are the standard first-line treatment for primary immune thrombocytopenia (ITP), with a high initial response but unsatisfactory sustained response (SR). Additionally, corticosteroids usually lead to hyperglycaemia especially in patients with pre-existing type 2 diabetes mellitus (T2DM). Besides reducing the blood glucose levels, metformin was found to have immunomodulatory effects. We hereby conducted a multicentre propensity score matching analysis of corticosteroids plus metformin versus corticosteroids for newly diagnosed ITP patients with pre-existing T2DM. After matching at a ratio of 1:1, there were 57 patients in each group. Baseline characteristics, comorbidities and other medications including concurrent hypoglycaemic medications were balanced between the two groups. No statistical difference was observed in the initial response rate at day 14. It was notable that patients in the metformin group had a significantly higher SR rate and longer duration of response compared to the non-metformin group. Metformin inclusion was associated with a higher incidence of stomach upset, which were generally tolerable. Our study provided evidence that the addition of metformin to corticosteroids might be a promising front-line treatment for newly diagnosed ITP patients with pre-existing T2DM.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142851710","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

Chronic lymphocytic leukaemia with trisomy 12. 伴有 12 三体综合征的慢性淋巴细胞白血病。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-17 DOI: 10.1111/bjh.19946

Priscilla Quach, Keenan O Hogan

引用次数: 0

Reframing thalassaemia syndrome as a benign haematopoietic stem cell disorder. 将地中海贫血综合征重新定义为良性造血干细胞疾病。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-15 DOI: 10.1111/bjh.19919

Aurelio Maggio, Mariasanta Napolitano, Ali T Taher, Rayan Bou-Fakhredin, Mariano Anibal Ostuni

{"title":"Reframing thalassaemia syndrome as a benign haematopoietic stem cell disorder.","authors":"Aurelio Maggio, Mariasanta Napolitano, Ali T Taher, Rayan Bou-Fakhredin, Mariano Anibal Ostuni","doi":"10.1111/bjh.19919","DOIUrl":"https://doi.org/10.1111/bjh.19919","url":null,"abstract":"Thalassaemia, caused by over 250 mutations in the beta globin gene, changes the haematopoietic stem cell (HSC) differentiation, leading to ineffective erythropoiesis. This Wider Perspective article overlooks its underlying nature as a benign HSC disorder with a significant impact on the erythroid cell lineage. The simplicity of managing symptoms through transfusions and iron chelation therapy has shifted the focus away from the development of cell-based treatments. The identification of the beta039 mutation by Chang and Kan in 1979 marked a turning point, suggesting as main approach the molecular level by correcting the beta globin chain imbalances through gene insertion and editing. However, challenges of technology have delayed the implementation of these strategies for over four decades. In contrast, the past two decades have witnessed significant advances in the treatment of HSC disorders of the myeloid clone which are driven by a 'target cell strategy'. Many current and innovative treatments for thalassaemia are now adopting this approach, highlighting the importance of identifying suitable candidates through risk stratification. This manuscript explores the evolving understanding of thalassaemia syndromes as congenital HSC disorders of the erythroid clone and examines the implications of this perspective for the development of future treatments.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142826893","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

Increasing the dose of recombinant human thrombopoietin can enhance platelet engraftment after allogeneic haematopoietic stem cell transplantation: A NICHE cohort study. 增加重组人血小板生成素的剂量可促进异体造血干细胞移植后的血小板移植：NICHE队列研究。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-15 DOI: 10.1111/bjh.19954

Dan Feng, Qingzhen Liu, Hongye Gao, Yigeng Cao, Xin Chen, Rongli Zhang, Weihua Zhai, Donglin Yang, Jialin Wei, Yi He, Aiming Pang, Sizhou Feng, Mingzhe Han, Qiaoling Ma, Erlie Jiang

引用次数: 0

Exhaled breath analysis during vaso-occlusive events in sickle cell disease. 镰状细胞病血管闭塞事件中的呼气分析。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-15 DOI: 10.1111/bjh.19942

C Vuong, P Brinkman, H Heijboer, E Nur, C L Eckhardt, S W J Terheggen-Lagro, B J Biemond, A H Maitland-van der Zee, K Fijnvandraat

引用次数: 0

Acalabrutinib in combination with rituximab and lenalidomide in patients with relapsed or refractory follicular lymphoma: Results of the phase 1b open-label study (ACE-LY-003). 阿卡拉布替尼联合利妥昔单抗和来那度胺治疗复发或难治性滤泡性淋巴瘤：1b期开放标签研究（ACE-LY-003）的结果

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-12 DOI: 10.1111/bjh.19951

Paolo Strati, Richy Agajanian, Izidore S Lossos, Morton Coleman, Robert Kridel, Andrew Wood, Robin Lesley, Chuan-Chuan Wun, Deborah M Stephens

{"title":"Acalabrutinib in combination with rituximab and lenalidomide in patients with relapsed or refractory follicular lymphoma: Results of the phase 1b open-label study (ACE-LY-003).","authors":"Paolo Strati, Richy Agajanian, Izidore S Lossos, Morton Coleman, Robert Kridel, Andrew Wood, Robin Lesley, Chuan-Chuan Wun, Deborah M Stephens","doi":"10.1111/bjh.19951","DOIUrl":"https://doi.org/10.1111/bjh.19951","url":null,"abstract":"Patients with relapsed/refractory (R/R) follicular lymphoma (FL) have limited effective treatment options. Bruton tyrosine kinase inhibitors (BTKis) increase the anti-tumoural phenotype of tumour-associated macrophages, providing rationale to combine them with rituximab and lenalidomide (R2). Acalabrutinib, a second-generation BTKi, has potential to improve R2 efficacy without increasing T-cell-mediated toxicity due to its lack of interleukin-2-inducible T-cell kinase inhibition. Here, we report safety and efficacy from a phase 1b dose-finding study (NCT02180711) evaluating acalabrutinib plus R2 in patients with R/R FL. Overall, 29 patients received acalabrutinib plus R2 (lenalidomide 15 mg, n = 8; lenalidomide 20 mg, n = 21). At a median acalabrutinib exposure of 21 months, the most common grade ≥3 treatment-emergent adverse event (TEAE) was neutropenia (37.9%). The incidence of grade ≥3 serious TEAEs was 37.5% and 52.4% in the lenalidomide 15-mg and 20-mg cohorts, respectively; overall, the most common were COVID-19 pneumonia, COVID-19 infection and pneumonia. Earlier treatment withholdings/reductions were observed in the 20-mg cohort. With a median follow-up of 34.1 months, the overall response rate was 75.9%. The complete response rate was 25.0% and 42.9% in the lenalidomide 15- and 20-mg cohorts, respectively. Due to acceptable toxicity and preliminary efficacy, the lenalidomide 20-mg dose was selected for further investigation.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142816746","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

Development and validation of a deep learning model for morphological assessment of myeloproliferative neoplasms using clinical data and digital pathology. 利用临床数据和数字病理学对骨髓增生性肿瘤进行形态学评估的深度学习模型的开发和验证。

IF 5.1 2区医学

British Journal of Haematology Pub Date : 2024-12-10 DOI: 10.1111/bjh.19938

Rong Wang, Zhongxun Shi, Yuan Zhang, Liangmin Wei, Minghui Duan, Min Xiao, Jin Wang, Suning Chen, Qian Wang, Jianyao Huang, Xiaomei Hu, Jinhong Mei, Jieyu He, Feng Chen, Lei Fan, Guanyu Yang, Wenyi Shen, Yongyue Wei, Jianyong Li

{"title":"Development and validation of a deep learning model for morphological assessment of myeloproliferative neoplasms using clinical data and digital pathology.","authors":"Rong Wang, Zhongxun Shi, Yuan Zhang, Liangmin Wei, Minghui Duan, Min Xiao, Jin Wang, Suning Chen, Qian Wang, Jianyao Huang, Xiaomei Hu, Jinhong Mei, Jieyu He, Feng Chen, Lei Fan, Guanyu Yang, Wenyi Shen, Yongyue Wei, Jianyong Li","doi":"10.1111/bjh.19938","DOIUrl":"https://doi.org/10.1111/bjh.19938","url":null,"abstract":"The subjectivity of morphological assessment and the overlapping pathological features of different subtypes of myeloproliferative neoplasms (MPNs) make accurate diagnosis challenging. To improve the pathological assessment of MPNs, we developed a diagnosis model (fusion model) based on the combination of bone marrow whole-slide images (deep learning [DL] model) and clinical parameters (clinical model). Thousand and fifty-one MPN and non-MPN patients were divided into the training, internal testing and one internal and two external validation cohorts (the combined validation cohort). In the combined validation cohort, fusion model achieved higher areas under curve (AUCs) than clinical or DL model or both for MPNs and subtype identification. Compared with haematopathologists with different experience, clinical model achieved AUC which was comparable to seniors and higher than juniors (p = 0.0208) for polycythaemia vera. The AUCs of fusion model were comparable to seniors and higher than juniors for essential thrombocytosis (p = 0.0141), prefibrotic primary myelofibrosis (p = 0.0085) and overt primary myelofibrosis (p = 0.0330) identification. In conclusion, the performances of our proposed models are equivalent to senior haematopathologists and better than juniors, providing a new perspective on the utilization of DL algorithms in MPN morphological assessment.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2024-12-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142805575","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