British Journal of Haematology最新文献_第10页

When is it time to bid farewell to anticoagulation? 什么时候是告别抗凝的时候？

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-22 DOI: 10.1111/bjh.70114

Julia Czuprynska, Roopen Arya

引用次数: 0

Refining the risk stratification in advanced-stage classical Hodgkin lymphoma: A critical analysis of clinical prediction models. 改进晚期经典霍奇金淋巴瘤的风险分层：临床预测模型的关键分析。

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-22 DOI: 10.1111/bjh.70115

Oguzhan Koca, Ahmet Emre Eskazan

{"title":"Refining the risk stratification in advanced-stage classical Hodgkin lymphoma: A critical analysis of clinical prediction models.","authors":"Oguzhan Koca, Ahmet Emre Eskazan","doi":"10.1111/bjh.70115","DOIUrl":"https://doi.org/10.1111/bjh.70115","url":null,"abstract":"Classical Hodgkin lymphoma (cHL) is a haematological malignancy with high curability; however, prognosis varies significantly based on clinical and biological factors. To enhance risk stratification, several clinical prediction models have been developed over time, particularly for advanced-stage cHL. The International Prognostic Score (IPS), introduced in 1998, was the first widely adopted model, later refined in 2012 (updated IPS) and further simplified in 2015 (IPS-3). Despite their prognostic utility, these models have demonstrated declining predictive performance due to advancements in cHL treatment. In response, the HoLISTIC consortium recently introduced the Advanced-Stage Hodgkin Lymphoma International Prognostic Index (A-HIPI) in 2023. Unlike previous models, A-HIPI incorporates continuous variables, aiming to provide a more precise risk assessment. However, its applicability to older patients remains uncertain, necessitating further validation studies. Additionally, none of the existing models incorporate dynamic treatment response markers such as interim positron emission tomography/computed tomography (PET/CT), which have shown strong prognostic value. This review comprehensively discusses the evolution, strengths and limitations of these prediction models, their clinical implications and the necessity for future refinements integrating dynamic biomarkers and treatment response indicators. The integration of machine learning and multi-omics approaches could further enhance risk stratification, improve treatment personalization and optimize patient outcomes in cHL.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-08-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144937014","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

aHSCT in multiple sclerosis aHSCT在多发性硬化中的应用。

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-19 DOI: 10.1111/bjh.70033

Guillermo José Ruiz-Argüelles

{"title":"aHSCT in multiple sclerosis","authors":"Guillermo José Ruiz-Argüelles","doi":"10.1111/bjh.70033","DOIUrl":"10.1111/bjh.70033","url":null,"abstract":"Kazmi, Muraro, Mehra et al. (1) report the United Kingdom experience with autologous haematopoietic stem cell transplantation (aHSCT) in multiple sclerosis (MS) in a group of 364 MS patients (pwMS) treated with aHSCT. Even though the response rate was superior in the relapsing-remitting forms of MS, the authors observed positive responses in all types of MS; this observation is in accord with other reports employing other conditioning regimens (2). We strongly believe that the world medical community will listen more closely to the advocacy by MS patients and their families who have attempted to divulge that HSCT is a very powerful tool in the treatment of persons with MS, and that the procedure is more effective, safer and more affordable than most of the novel drugs currently in use. The information presented in this paper strongly supports the encouraging role of aHSCT in the treatment of pwMS and is obviously most welcome.Commentary on: Kazmi et al. Autologous haematopoietic stem cell transplantation for multiple sclerosis in the UK: a twenty-year retrospective analysis of activity and haematological outcomes from the British Society of Blood and Marrow Transplantation and Cellular Therapy (BSBMTCT). Br J Haematol 2025 (Online ahead of print). doi: 10.1111/bjh.20199.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"207 3","pages":"1159-1160"},"PeriodicalIF":3.8,"publicationDate":"2025-08-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/bjh.70033","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144870543","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

Characteristics and prognosis of paediatric normal karyotype acute myeloid leukaemia: A NOPHO-DBH AML study. 儿童正常核型急性髓性白血病的特点和预后：一项nophoo - dbh AML研究。

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-18 DOI: 10.1111/bjh.70094

Morten Krogh Herlin, Eigil Kjeldsen, Jonas Abrahamsson, Nira Arad-Cohen, Daniel Cheuk, Barbara De Moerloose, Kirsi Jahnukainen, Ólafur Gísli Jónsson, Gertjan J L Kaspers, Zhanna Kovalova, Jose Maria Fernandez Navarro, Ulrika Noren-Nyström, Josefine Palle, Ramunė Pasaulienė, Kadri Saks, Bernward Zeller, Linda Holmfeldt, Henrik Hasle, Kristian Løvvik Juul-Dam

{"title":"Characteristics and prognosis of paediatric normal karyotype acute myeloid leukaemia: A NOPHO-DBH AML study.","authors":"Morten Krogh Herlin, Eigil Kjeldsen, Jonas Abrahamsson, Nira Arad-Cohen, Daniel Cheuk, Barbara De Moerloose, Kirsi Jahnukainen, Ólafur Gísli Jónsson, Gertjan J L Kaspers, Zhanna Kovalova, Jose Maria Fernandez Navarro, Ulrika Noren-Nyström, Josefine Palle, Ramunė Pasaulienė, Kadri Saks, Bernward Zeller, Linda Holmfeldt, Henrik Hasle, Kristian Løvvik Juul-Dam","doi":"10.1111/bjh.70094","DOIUrl":"10.1111/bjh.70094","url":null,"abstract":"Normal karyotype acute myeloid leukaemia (NK-AML) in children is a heterogeneous subgroup with scarce data on characteristics and prognosis. We investigated NK-AML in a large paediatric AML cohort from four trials of the Nordic Society for Paediatric Haematology and Oncology-Dutch Belgian Hongkong (NOPHO-DBH) group. Among 1476 AML patients, we identified 316 NK-AML patients (21%). NK-AML was characterized by high frequencies of FLT3 internal tandem duplications (ITD, 33%), mutated NPM1 (28%), WT1 (25%) and CEBPA (21%). Five-year event-free survival (EFS) and overall survival (OS) in NK-AML were 52% (95% confidence interval [CI]: 46-58) and 70% (CI: 65-75) respectively. Restricted to NPM1wt cases only (n = 959), NK-AML was associated with unfavourable outcome (relative risk [RR] of EFS = 0.80, p = 0.014; RR of OS = 0.87, p = 0.022). NK-AML with mutated NPM1 had excellent EFS (79%, CI: 66-88) and OS (97%, CI: 88-99), which was not influenced by concomitant FLT3-ITD. In multivariable analysis, mutated NPM1 in NK-AML was associated with favourable EFS (hazard ratio [HR]: 0.24, CI: 0.13-0.43, p < 0.001) and OS (HR: 0.10, CI: 0.03-0.35, p < 0.001). FLT3-ITD was associated with inferior EFS (HR: 1.56, CI: 1.03-2.35, p = 0.035) and OS (HR: 1.91, CI: 1.11-3.31, p = 0.02). We conclude that prognosis in paediatric NK-AML is independently affected by NPM1 and FLT3-ITD status. Further molecular characterization of NK-AML is needed, especially for NPM1wt NK-AML.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144870544","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

Patient survey in immune thrombocytopenia (ITP): Identifying unmet needs related to treatment and disease control in patients living in the United States 免疫性血小板减少症（ITP）患者调查：确定生活在美国的患者未满足的治疗和疾病控制需求。

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-17 DOI: 10.1111/bjh.20257

Nichola Cooper, Caroline Kruse, Sharon Deneen Morgan, Julie Laurent, Marleni Arvelo-Saillant, Jean-Pascal Roussy, Matias Cordoba, Imene Gouia, Lisa-Anne Schmitt, Erin Reineke, Terry Gernsheimer

{"title":"Patient survey in immune thrombocytopenia (ITP): Identifying unmet needs related to treatment and disease control in patients living in the United States","authors":"Nichola Cooper, Caroline Kruse, Sharon Deneen Morgan, Julie Laurent, Marleni Arvelo-Saillant, Jean-Pascal Roussy, Matias Cordoba, Imene Gouia, Lisa-Anne Schmitt, Erin Reineke, Terry Gernsheimer","doi":"10.1111/bjh.20257","DOIUrl":"10.1111/bjh.20257","url":null,"abstract":"Immune thrombocytopenia (ITP) is a chronic disease with primary therapeutic goals of platelet count recovery to safe levels to minimize active/future bleeding, alongside easing additional symptoms negatively impacting overall patient well-being with consequent improvement in physical fatigue/energy levels, daily/work-related activities and social/emotional health. Documentation of this rare disease is important for evaluating real-world experiences in treatment satisfaction, expectations and unmet needs in disease management. This cross-sectional, real-world evidence survey was conducted from 9 February 2023 to 4 April 2023, by the Platelet Disorder Support Association and Sanofi in US adults diagnosed with ITP for ≥1 year. Results showed that although most patients receive and adhere to treatment, lack of sustained efficacy fuels a need for more effective therapy. Patients desired long-term ITP control and were most concerned with increasing/stabilizing platelet counts and decreasing fatigue and bleeding severity. Better treatment was needed to ease burdensome health-related quality of life symptoms, especially physical fatigue and anxiety, and decrease effects on daily life and activities. Additionally, involvement in shared decision-making and engagement in educational, digital healthcare and psychological support were preferred. Overall, ITP patients desired long-term disease management options that improved platelet counts, minimized bleeding and reduced physical fatigue and anxiety, while also engaging in patient support options.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"207 3","pages":"1038-1046"},"PeriodicalIF":3.8,"publicationDate":"2025-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12436224/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144870545","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

Poor prognosis of newly diagnosed multiple myeloma patients with 1p32.3 deletion in single monoallelic deletion and/or in main clone 单个单等位基因缺失和/或主克隆缺失1p32.3的新诊断多发性骨髓瘤患者预后差

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-17 DOI: 10.1111/bjh.70015

Hongying You, Weiqin Yao, Lingzhi Yan, Yingying Zhai, Zhi Yan, Jingjing Shang, Shuang Yan, Xiaolan Shi, Song Jin, Xinxin Ge, Hongjie Shen, Jinlan Pan, Depei Wu, Chengcheng Fu

{"title":"Poor prognosis of newly diagnosed multiple myeloma patients with 1p32.3 deletion in single monoallelic deletion and/or in main clone","authors":"Hongying You, Weiqin Yao, Lingzhi Yan, Yingying Zhai, Zhi Yan, Jingjing Shang, Shuang Yan, Xiaolan Shi, Song Jin, Xinxin Ge, Hongjie Shen, Jinlan Pan, Depei Wu, Chengcheng Fu","doi":"10.1111/bjh.70015","DOIUrl":"10.1111/bjh.70015","url":null,"abstract":"Del(1p32.3) by FISH detection in multiple myeloma (MM) has not been routinely carried out in China. Its clinical significance was not clearly demonstrated. This study analysed clinical characteristics, treatment response and prognostic significance of del(1p32.3). We analysed 345 newly diagnosed multiple myeloma (NDMM) samples, and cytogenetic analysis was performed using Cytoscan and FISH results, enabling comprehensive disease-specific cohort analysis and prognostic model development. The total proportion of chromosomal 1 abnormality was 64.1%, including 189 cases of 1q21 gain/amplification and 88 cases of 1p deletion, 40 patients had 1p32.3/CDKN2Cdeletion. Del(1p32.3) patients were strongly correlated with 1q21 gain/amplification and 17p deletion. Del(1p32.3) patients were more likely accompanied with extra-medullary multiple myeloma (EMM) and complex karyotype. Del(1p32.3) had a worse effect on progression-free survival (PFS) and overall survival (OS), alongside other high-risk cytogenetic abnormalities that further worsened prognosis, especially 1q gain/amplification. Patients with 1p32.3 in the main clone or with a single monoallelic deletion had significantly poorer survival outcomes. Autologous stem cell transplantation (ASCT) cannot completely overcome its adverse effects on prognosis. In multivariate analysis, 1p32.3 was an important independent adverse PFS factor. Patients harbouring single monoallelic del(1p32.3) and/or main clone deletions demonstrated inferior outcomes despite lenalidomide, bortezomib and dexamethasone (VRD) induction and transplantation. Del(1p32.3) had synergistic effects frequently co-occurring with 1q21 gain/amplification, thus, we strongly advocate for routine del(1p32.3) testing in clinical practice.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"207 3","pages":"869-880"},"PeriodicalIF":3.8,"publicationDate":"2025-08-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12436220/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144870546","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

Has early mortality following single-unit unrelated cord blood transplantation improved in recent years? A nationwide registry data analysis from 2003 to 2022 in Japan. 近年来，单单位非亲属脐带血移植后的早期死亡率有所改善吗？日本2003年至2022年全国登记数据分析。

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-16 DOI: 10.1111/bjh.70099

Takaaki Konuma, Junya Kanda, Naoyuki Uchida, Masatsugu Tanaka, Fumihiko Kimura, Hideki Nakasone, Yasufumi Uehara, Masahito Tokunaga, Masako Toyosaki, Shigesaburo Miyakoshi, Noriko Doki, Kazuya Ishiwata, Hikaru Kobayashi, Yasushi Onishi, Yasuji Kozai, Koji Kato, Fumihiko Ishimaru, Takahiro Fukuda, Yoshiko Atsuta, Satoshi Takahashi

{"title":"Has early mortality following single-unit unrelated cord blood transplantation improved in recent years? A nationwide registry data analysis from 2003 to 2022 in Japan.","authors":"Takaaki Konuma, Junya Kanda, Naoyuki Uchida, Masatsugu Tanaka, Fumihiko Kimura, Hideki Nakasone, Yasufumi Uehara, Masahito Tokunaga, Masako Toyosaki, Shigesaburo Miyakoshi, Noriko Doki, Kazuya Ishiwata, Hikaru Kobayashi, Yasushi Onishi, Yasuji Kozai, Koji Kato, Fumihiko Ishimaru, Takahiro Fukuda, Yoshiko Atsuta, Satoshi Takahashi","doi":"10.1111/bjh.70099","DOIUrl":"https://doi.org/10.1111/bjh.70099","url":null,"abstract":"Unrelated single-unit cord blood transplantation (CBT) is a valuable alternative donor source for patients without matched related or unrelated donors. Although initial concerns included limited cell dose, delayed haematopoietic recovery and higher early mortality, advancements in transplant practices may have led to improved outcomes. However, it remains uncertain whether these improvements extend to the most recent years. We conducted a nationwide, registry-based retrospective study of 15 816 patients who received unrelated single-unit CBT in Japan and analysed across four time periods: 2003-2007, 2008-2012, 2013-2017 and 2018-2022. Overall survival (OS) improved significantly across time periods, with 3-year OS increasing from 38.8% (2003-2007) to 54.4% (2018-2022; p < 0.001). Similarly, 100-day non-relapse mortality declined from 19.6% to 9.5% (p < 0.001). Neutrophil and platelet engraftment rates also rose steadily, reaching 89.6% and 78.0%, respectively, in the most recent period. The most notable improvement in 100-day OS occurred among patients aged ≥60 years. Early mortality within 100 days due to bacterial and fungal infections, graft failure, haemorrhage and graft-versus-host disease (GVHD) significantly decreased over time. This study shows clear and consistent improvements in transplant outcomes, especially in the most recent 5-year period.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144858667","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

Graft-versus-host disease is not associated with reduced incidence of relapse following haploidentical stem cell transplantation with post-transplant cyclophosphamide for acute lymphoblastic leukaemia: A study on behalf of the Acute Leukaemia Working Party of European Society for Blood and Marrow Transplantation. 移植物抗宿主病与单倍体干细胞移植后环磷酰胺治疗急性淋巴细胞白血病的复发率降低无关：代表欧洲血液和骨髓移植学会急性白血病工作组的一项研究。

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-14 DOI: 10.1111/bjh.70101

Avichai Shimoni, Christophe Peczynski, Myriam Labopin, Alida Dominietto, Mariya Koc, Mutlu Arat, Johanna Tischer, Simona Sica, Zafer Gülbas, Gerard Socié, Didier Blaise, Pietro Pioltelli, Hakan Ozdogu, Jan Vydra, Fabio Ciceri, Arnon Nagler, Mohamad Mohty

{"title":"Graft-versus-host disease is not associated with reduced incidence of relapse following haploidentical stem cell transplantation with post-transplant cyclophosphamide for acute lymphoblastic leukaemia: A study on behalf of the Acute Leukaemia Working Party of European Society for Blood and Marrow Transplantation.","authors":"Avichai Shimoni, Christophe Peczynski, Myriam Labopin, Alida Dominietto, Mariya Koc, Mutlu Arat, Johanna Tischer, Simona Sica, Zafer Gülbas, Gerard Socié, Didier Blaise, Pietro Pioltelli, Hakan Ozdogu, Jan Vydra, Fabio Ciceri, Arnon Nagler, Mohamad Mohty","doi":"10.1111/bjh.70101","DOIUrl":"https://doi.org/10.1111/bjh.70101","url":null,"abstract":"The graft-versus-leukaemia effect (GVL) is closely associated with graft-versus-host disease (GVHD) after human leucocyte antigen (HLA)-matched allogeneic stem-cell transplantation (SCT) in acute lymphoblastic leukaemia (ALL). However, there are no data on this association following haploidentical SCT (haploSCT) with post-transplant cyclophosphamide (PTCy). We assessed the impact of acute and chronic GVHD on haploSCT outcomes in 516 adult ALL patients. The cumulative incidence of acute GVHD grade II-IV and III-IV, chronic GVHD and extensive chronic GVHD was 33.3%, 11.7%, 35.3% and 11.8% respectively. The 2-year relapse incidence (RI), non-relapse mortality (NRM) and overall survival (OS) were 27.1%, 17.3% and 64.4% respectively. The time-dependent hazard ratios (HRs) of acute GVHD grade II, grade III-IV, limited and extensive chronic GVHD associated with RI were 0.92 (95% confidence interval [CI] 0.58-1.48, p = 0.74), 0.57 (95% CI, 0.27-1.22, p = 0.15), 1.06 (95% CI, 0.62-1.82, p = 0.83) and 0.95 (95% CI, 0.42-2.17, p = 0.91) respectively. Acute GVHD grade III-IV and extensive chronic GVHD were associated with higher NRM (hazard ratio [HR] 1.95 [95% CI, 1.09-3.48, p = 0.002] and 3.3 [95% CI, 1.41-7.73, p = 0.006]) and reduced OS (HR 1.91 [95% CI, 1.07-3.39, p = 0.03] and 3.27 [95% CI, 1.4-7.66, p = 0.006]) respectively. In conclusion, acute and chronic GVHD are not statistically associated with reduced RI after haploSCT with PTCy in ALL. Higher GVHD grades are associated with higher NRM and lower OS.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144854100","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

White blood cell count as a powerful prognostic marker and treatment guide in paediatric acute myeloid leukaemia with NUP98 rearrangement. 白细胞计数作为NUP98重排儿童急性髓性白血病的有力预后标志物和治疗指南

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-14 DOI: 10.1111/bjh.70096

Hui Shi, Wenjing Shu, Yu Tao, Xueming Jia, Yali Shen, Hua Yang, Yang Xun, Daniel Tomas Baptista-Hon, Hua You

{"title":"White blood cell count as a powerful prognostic marker and treatment guide in paediatric acute myeloid leukaemia with NUP98 rearrangement.","authors":"Hui Shi, Wenjing Shu, Yu Tao, Xueming Jia, Yali Shen, Hua Yang, Yang Xun, Daniel Tomas Baptista-Hon, Hua You","doi":"10.1111/bjh.70096","DOIUrl":"https://doi.org/10.1111/bjh.70096","url":null,"abstract":"NUP98-rearranged paediatric acute myeloid leukaemia (NUP98-r pAML) has an extremely poor prognosis, and the impact of clinical parameters and therapeutic schemes on its outcomes remains unclear. We conducted a retrospective study of the largest pAML cohort (1779 patients) and found that NUP98-r pAML has the worst prognosis among all subtypes. Furthermore, we identified white blood cell (WBC) count as the sole predictor of overall survival (OS) in NUP98-r pAML patients and validated its adverse prognostic impact in both external paediatric and adult cohorts. NUP98-r pAML patients were categorized into low-risk (WBC count ≤150 × 109/L) and high-risk (WBC count >150 × 109/L) groups based on WBC levels. Haematopoietic stem cell transplantation (HSCT) significantly improved OS and reduced the cumulative incidence of relapse (CIR) in the high-risk group but not in the low-risk group. Bortezomib significantly increased OS in NUP98::NSD1 patients within the low-risk group, and the combination of bortezomib and HSCT significantly enhanced OS in the entire NUP98-r pAML cohort. CD33 antibody (Gemtuzumab ozogamicin, GO) is not recommended for the entire NUP98-r pAML patients. In summary, WBC count is a pivotal marker for risk stratification and treatment decision-making in NUP98-r pAML patients.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144854101","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

Treatment-free remission in chronic myeloid leukaemia patients with accelerated phase or tyrosine kinase inhibitor therapy failure. 加速期或酪氨酸激酶抑制剂治疗失败的慢性髓性白血病患者的无治疗缓解。

IF 3.8 2区医学

British Journal of Haematology Pub Date : 2025-08-13 DOI: 10.1111/bjh.70080

Mengyao Yuan, Yanli Zhang, Bingcheng Liu, Li Zhou, Yu Zhu, Zhenling Li, Shaolei Zang, Zhenfang Liu, Weiming Li, Qian Jiang

{"title":"Treatment-free remission in chronic myeloid leukaemia patients with accelerated phase or tyrosine kinase inhibitor therapy failure.","authors":"Mengyao Yuan, Yanli Zhang, Bingcheng Liu, Li Zhou, Yu Zhu, Zhenling Li, Shaolei Zang, Zhenfang Liu, Weiming Li, Qian Jiang","doi":"10.1111/bjh.70080","DOIUrl":"https://doi.org/10.1111/bjh.70080","url":null,"abstract":"We studied 60 chronic myeloid leukaemia (CML) patients with a prior history of accelerated phase (AP) including de novo AP (n = 19) and transformation to AP (n = 4), or tyrosine kinase inhibitor (TKI) therapy failure in the chronic phase (CP, n = 37), who discontinued TKI therapy. Median interval from diagnosis with AP or TKI therapy failure to achieving a deep molecular response (DMR) was 19 months (interquartile range [IQR], 9-30 months). Median TKI treatment and DMR duration were 108 months (IQR, 72-137 months) and 59 months (IQR, 39-87 months) respectively. At a median follow-up of 21 months (IQR, 11-36 months) after TKI discontinuation, 19 (31%) patients lost the major molecular response (MMR). The 3-year probability of a sustained MMR was 59% (95% confidence interval [CI], [45%, 78%]). In the multivariable analyses, age at discontinuation <32 years (hazard ratio [HR] = 4.1 [1.3, 12.7], p = 0.014) and BCR::ABL1 >0.1% at 12 months on TKI therapy (reference, ≤0.1%; HR = 3.9 [1.4, 11.5], p = 0.011) were significantly associated with a higher probability of MMR loss after TKI discontinuation. CML patients with a history of AP or TKI therapy failure may achieve successful treatment-free remission after an adequate TKI therapy duration and a sustained DMR.","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":3.8,"publicationDate":"2025-08-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144833598","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