Christian Niederwieser, Anita Badbaran, Radwan Massoud, Nico Gagelmann, Ameya Kunte, Evgeny Klyuchnikov, Niloufar Seyedi, Silke Heidenreich, Ina Rudolph, Gaby Zeck, Catherina Lück, Dietlinde Janson, Christine Wolschke, Francis Ayuk, Nicolaus Kröger
{"title":"Different clearance of KITD816V mutation and tryptase levels after haematopoietic cell transplantation in patients with systemic mastocytosis with associated haematological neoplasm.","authors":"Christian Niederwieser, Anita Badbaran, Radwan Massoud, Nico Gagelmann, Ameya Kunte, Evgeny Klyuchnikov, Niloufar Seyedi, Silke Heidenreich, Ina Rudolph, Gaby Zeck, Catherina Lück, Dietlinde Janson, Christine Wolschke, Francis Ayuk, Nicolaus Kröger","doi":"10.1111/bjh.20211","DOIUrl":"https://doi.org/10.1111/bjh.20211","url":null,"abstract":"<p><p>The patterns of tryptase normalization, KITD816V clearance and establishment of donor cell chimerism were analysed in 13 patients with systemic mastocytosis and associated haematological neoplasm (AHN) after haematopoietic cell transplantation (HCT). The molecular marker of systemic mastocytosis (SM) (KITD816V) simultaneously disappeared (median + 36 days) with the establishment of donor chimerism (median: +31 days post-HCT; Pearson correlation: -0.99, p < 0.001). In contrast, tryptase normalized median of +228 days after HCT with significant delay to KITD816V clearance (p = 0.01) and full donor chimerism (p = 0.04). Faster normalization was observed after radiation-based conditioning and removal of an infiltrated spleen, while persistence was not associated with relapse of AHN.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144315567","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}
Kevin Chung, Lay She Ng, Claire I Yee, Radhika Bansal, Mohamed A Kharfan-Dabaja, Grzegorz S Nowakowski, Allison C Rosenthal, Javier Munoz, Januario Castro, Wern Lynn Ng, Hemant S Murthy, Madiha Iqbal, Yi Lin, Yucai Wang, Talal Hilal
{"title":"Impact of granulocyte colony-stimulating factor on safety of chimeric antigen receptor-T-cell therapy in non-Hodgkin lymphoma.","authors":"Kevin Chung, Lay She Ng, Claire I Yee, Radhika Bansal, Mohamed A Kharfan-Dabaja, Grzegorz S Nowakowski, Allison C Rosenthal, Javier Munoz, Januario Castro, Wern Lynn Ng, Hemant S Murthy, Madiha Iqbal, Yi Lin, Yucai Wang, Talal Hilal","doi":"10.1111/bjh.20207","DOIUrl":"https://doi.org/10.1111/bjh.20207","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144309337","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}
Michael R Savona, Olatoyosi Odenike, Gail J Roboz, Harshad Amin, Amy E DeZern, Elizabeth A Griffiths, Kim-Hien Dao, Amer M Zeidan, Bhavana Bhatnagar, Rena Buckstein, Brian Leber, Mary-Margaret Keating, Somedeb Ball, Aram Oganesian, Yuri Sano, Harold N Keer, Guillermo Garcia-Manero
{"title":"Efficacy and safety of oral decitabine/cedazuridine in the chronic myelomonocytic leukaemia subpopulations from phase 2 and 3 studies.","authors":"Michael R Savona, Olatoyosi Odenike, Gail J Roboz, Harshad Amin, Amy E DeZern, Elizabeth A Griffiths, Kim-Hien Dao, Amer M Zeidan, Bhavana Bhatnagar, Rena Buckstein, Brian Leber, Mary-Margaret Keating, Somedeb Ball, Aram Oganesian, Yuri Sano, Harold N Keer, Guillermo Garcia-Manero","doi":"10.1111/bjh.20203","DOIUrl":"https://doi.org/10.1111/bjh.20203","url":null,"abstract":"<p><p>DNA methyltransferase inhibitors (DNMTis) are commonly used in treating chronic myelomonocytic leukaemia (CMML); however, data from prospective studies of DNMTis in CMML are limited. The present analysis evaluated the efficacy, safety and pharmacodynamics of the oral DNMTi decitabine/cedazuridine in the subset of patients with CMML from the phase 2 and 3 trials, which led to the approval of this agent for myelodysplastic syndromes and CMML in the United States and Canada. Potential prognostic factors also were analysed. In all, 34 patients with CMML were screened and 33 were treated. Most patients (76% [n = 25]) had myelodysplastic type-CMML and 77% (n = 24/31 with DNA available for sequencing) had intermediate-2 or high-risk disease noted by CMML-specific prognostic scoring systems. The overall response rate was 76%, with 21% (n = 7) of patients achieving a complete response. Nearly half of the 11 patients who were red blood cell-transfusion dependent at baseline (46%) attained transfusion independence for ≥12 weeks, which was associated with survival. Median overall and transformation-free survival were 35.7 and 28.3 months, respectively, and the safety profile was similar to that previously reported for decitabine. This analysis described the use of decitabine/cedazuridine in CMML from consecutive, prospective, randomised trials and illustrated a median survival of nearly 3 years.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144309336","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}
{"title":"Prognostic factors for allogeneic haematopoietic cell transplantation outcomes in primary refractory acute myeloid leukaemia (2013-2022): A retrospective study by the adult acute myeloid leukaemia working group of the Japanese Society for Transplantation and Cellular Therapy.","authors":"Takaaki Konuma, Yoshimitsu Shimomura, Shohei Mizuno, Satoshi Yamasaki, Shunsuke Yui, Naoyuki Uchida, Noriko Doki, Masatsugu Tanaka, Satoshi Yoshihara, Tetsuya Eto, Yuta Katayama, Yuna Katsuoka, Masahito Tokunaga, Shuichi Ota, Mamiko Sakata-Yanagimoto, Kazuya Ishiwata, Yoshinobu Kanda, Toshiro Kawakita, Makoto Onizuka, Junya Kanda, Takahiro Fukuda, Yoshiko Atsuta, Masamitsu Yanada","doi":"10.1111/bjh.20208","DOIUrl":"https://doi.org/10.1111/bjh.20208","url":null,"abstract":"<p><p>Primary refractory acute myeloid leukaemia (AML) remains a major clinical challenge, with poor outcomes despite salvage chemotherapy. Allogeneic haematopoietic cell transplantation (HCT) continues to be a potentially curative option for these patients. However, recent data on outcomes and prognostic factors specific to primary refractory AML remain limited. We conducted a retrospective analysis of 2600 adult patients with primary refractory AML who underwent their first allogeneic HCT between 2013 and 2022, using data from the Japanese national registry. The 3-year overall survival (OS) and leukaemia-free survival (LFS) rates were 28.5% and 24.4% respectively. The multivariate analysis identified older age (≥50 years), poor performance status (≥2), adverse cytogenetics, extramedullary disease at diagnosis and higher peripheral blood blast count at HCT (≥10%) as significant risk factors for worse OS and LFS. The cumulative incidences of relapse and non-relapse mortality at 3 years were 49.8% and 25.8% respectively. Based on the five significant risk factors, we developed a scoring system that effectively stratified patients into distinct prognostic groups for OS and LFS. This nationwide analysis demonstrated that allogeneic HCT offers the potential for long-term survival in adult patients with primary refractory AML. The proposed risk-scoring system may support clinical decision-making and patient counselling.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144300723","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}
Umut Yılmaz, Esra Terzi Demirsoy, Fatma Keklik Karadağ, Uğur Hatipoğlu, Meral Uluköylü Mengüç, Ebru Pekgüç, Taner Tan, Eda Nuhoğlu Kantarcı, Metban Mastanzade, Ali Durdu, Merve Apaydın Kayer, Murat Özbalak, Tuğrul Elverdi, Asu Fergün Yılmaz, Deniz Gören, Tayfur Toptaş, Turgay Ulaş, Özgür Mehtap, Mehmet Sinan Dal, Olga Meltem Akay, Muhlis Cem Ar, Güray Saydam, Fevzi Altuntaş, Elif Birtaş Ateşoğlu, Burhan Ferhanoğlu
{"title":"Simplified Molecular International Prognostic Index as an eligibility criterion for clinical trials: Analysis of the Turkish Lymphoma Study Group's large B-cell lymphoma cohort.","authors":"Umut Yılmaz, Esra Terzi Demirsoy, Fatma Keklik Karadağ, Uğur Hatipoğlu, Meral Uluköylü Mengüç, Ebru Pekgüç, Taner Tan, Eda Nuhoğlu Kantarcı, Metban Mastanzade, Ali Durdu, Merve Apaydın Kayer, Murat Özbalak, Tuğrul Elverdi, Asu Fergün Yılmaz, Deniz Gören, Tayfur Toptaş, Turgay Ulaş, Özgür Mehtap, Mehmet Sinan Dal, Olga Meltem Akay, Muhlis Cem Ar, Güray Saydam, Fevzi Altuntaş, Elif Birtaş Ateşoğlu, Burhan Ferhanoğlu","doi":"10.1111/bjh.20111","DOIUrl":"https://doi.org/10.1111/bjh.20111","url":null,"abstract":"<p><p>Comparative first-line trials in large B-cell lymphoma (LBCL) have mostly failed over the last decade. Failures were commonly attributed to overestimation of the progression risk for the control arms, bringing the precision of the international prognostic index (IPI) into scrutiny. Simplified molecular IPI (smIPI), introduced at American Society of Hematology 2023, was developed to address the shortcomings of IPI. This study investigated smIPI and its potential implications as trial eligibility criteria among a multicentre LBCL cohort of 1439 patients. Patients diagnosed between 2012 and 2024 were included. Data were collected from institutional archives. The primary end-point was risk stratification for progression-free survival (PFS). Kaplan-Meier and Cox regression methods were used for survival analyses. The smIPI reclassified 38.2% and 5.3% of patients to higher and lower risk groups from IPI, respectively. Patients reclassified to higher risk groups by smIPI had an increased risk of progression than the rest of the group (hazard ratio = 1.34, p = 0.003). High-risk groups classified by IPI and smIPI had similar outcomes (3-year PFS 54.9% vs. 56.3%); however, the high-risk group size was expanded by 35.2% when defined by smIPI. The smIPI is easily applicable and more sensitive than IPI in identifying patients under high risk of progression who are ideal candidates to participate in clinical research.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144309338","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}
Rahul Lakhotia, Christopher Melani, Stefania Pittaluga, Max J Gordon, James D Phelan, Jagan R Muppidi, Atekelt Tadese, Sarah Evans, Elaine S Jaffe, Louis M Staudt, Wyndham H Wilson, Mark Roschewski
{"title":"The anti-CD47 antibody magrolimab with obinutuzumab and venetoclax in relapsed or refractory indolent B-cell lymphomas.","authors":"Rahul Lakhotia, Christopher Melani, Stefania Pittaluga, Max J Gordon, James D Phelan, Jagan R Muppidi, Atekelt Tadese, Sarah Evans, Elaine S Jaffe, Louis M Staudt, Wyndham H Wilson, Mark Roschewski","doi":"10.1111/bjh.20219","DOIUrl":"https://doi.org/10.1111/bjh.20219","url":null,"abstract":"<p><p>Follicular lymphoma (FL), marginal zone lymphoma (MZL), chronic lymphocytic leukaemia (CLL) and mantle cell lymphoma (MCL) are characterized by a continuous incidence of relapse and increasing resistance to therapy. Novel immunotherapy approaches are needed. Magrolimab, a CD47-blocking antibody, disrupts CD47:SIRPα-mediated antiphagocytic signalling. When combined with a prophagocytic signal from an anti-CD20 antibody rituximab, it has shown activity in relapsed or refractory FL and MZL. In this phase 1 study, adding the BCL2-inhibitor venetoclax to magrolimab and the anti-CD20 antibody obinutuzumab resulted in complete responses in 6 of 10 (60%) evaluable patients with FL, MZL or CLL. Notably, we did not observe increased risk of infections previously reported from studies of magrolimab in acute myeloid leukaemia and higher risk myelodysplastic syndromes.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144309339","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}
Liting Niu, Minli Hu, Yi Xia, Meng Lv, Daoxing Deng, Leqing Cao, Jun Kong, Yanmin Zhao, Xiaodong Mo
{"title":"Upadacitinib, a selective JAK1 inhibitor, for the treatment of steroid refractory chronic graft-versus-host disease: A multicentre real-world study","authors":"Liting Niu, Minli Hu, Yi Xia, Meng Lv, Daoxing Deng, Leqing Cao, Jun Kong, Yanmin Zhao, Xiaodong Mo","doi":"10.1111/bjh.20183","DOIUrl":"10.1111/bjh.20183","url":null,"abstract":"<div>\u0000 \u0000 <p>Graft-versus-host disease (GVHD) remains an important cause of morbidity and mortality after allo-HSCT. Upadacitinib is a selective JAK1 inhibitor. This study investigated the clinical outcomes of upadacitinib in patients with steroid refractory (SR) chronic graft-versus-host disease (cGVHD). Thirty-two patients with steroid refractory cGVHD (SR-cGVHD) who received upadacitinib treatment were enrolled from three hospitals in China. The overall response rates (ORRs) at 24 weeks and at any time were 81.3% (complete response [CR] rate: 21.9%; partial response [PR] rate: 59.4%) and 84.4% (CR rate: 21.9%; PR rate: 62.5%) respectively. In particular, the cutaneous-specific ORRs at week 24 and at any time were 84.4% (CR rate: 40.6%, PR rate: 43.8%) and 87.6% (CR rate: 43.8%; PR rate: 43.8%) respectively. The rates of infection and haematological toxicity were 18.75% and 6.25%, and two patients (6.25%) experienced grade III adverse events after upadacitinib treatment. One patient died from relapse, and no patient died from non-relapse mortality. The 1-year probabilities of overall survival, failure-free survival and disease-free survival after upadacitinib were 96.6%, 64.9% and 83.1% respectively. In summary, upadacitinib may be an effective and safe treatment for SR-cGVHD.</p>\u0000 </div>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":"207 1","pages":"171-179"},"PeriodicalIF":5.1,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144300724","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}
Moshe Mittelman, Ariel Israel, Howard S Oster, Michael Leshchinsky, Yatir Ben-Shlomo, Eldad Kepten, Osnat Jarchowsky Dolberg, Ran Balicer, Galit Shaham
{"title":"Can we identify individuals at risk to develop multiple myeloma? A machine learning-based predictive model.","authors":"Moshe Mittelman, Ariel Israel, Howard S Oster, Michael Leshchinsky, Yatir Ben-Shlomo, Eldad Kepten, Osnat Jarchowsky Dolberg, Ran Balicer, Galit Shaham","doi":"10.1111/bjh.20136","DOIUrl":"https://doi.org/10.1111/bjh.20136","url":null,"abstract":"<p><p>Multiple myeloma evolves unnoticed over years, and when diagnosed, organ damage is common. Electronic health records (EHR) can help in developing predictive models identifying 'healthy' people at risk. MM patients from Clalit Health Services (2002-2019) were matched with healthy controls. Stage I: EHR from 5 years prior to MM diagnosis were reviewed and >200 parameters were compared (patients vs. controls). Stage II: Establishing xgboost model predicting 5 year risk for MM, with validation. Stage III: A simplified logistic regression model for community, requiring 20 variables (Age; Hb; RBC; MCV; RDW; WBC; neutrophils; lymphocytes; monocytes; basophils; glucose; creatinine; total protein; albumin; calcium; uric acid; bilirubin; HDL-C; LDL-C; triglycerides). EHR from the pre-MM period of 4256 patients were compared to controls. Future MM patients had higher ESR, lower Hb, ANC, neutrophil/lymphocyte ratio, higher globulins and ferritin, more immune deficiencies, MDS and FMF. They took fewer tranquilizers, anti-diabetics and statins. Using labs from future MM (n = 19 129) and controls (n = 382 580, 20:1), a predictive model was developed (ROC AUC = 0.836). The simple LR model provided individual risk prediction for MM within 5 years (AUC = 0.72). Two models with machine learning predict the risk of myeloma in 'healthy' individuals within 5 years. The models can be used in practice.</p>","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-06-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144309334","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}
Laura Distelmaier, Wolfgang G Kunz, Sebastian Theurich
{"title":"Extensive extramedullary haematopoiesis in a patient with non-transfusion-dependent beta-thalassaemia.","authors":"Laura Distelmaier, Wolfgang G Kunz, Sebastian Theurich","doi":"10.1111/bjh.20169","DOIUrl":"https://doi.org/10.1111/bjh.20169","url":null,"abstract":"","PeriodicalId":135,"journal":{"name":"British Journal of Haematology","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2025-06-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144300722","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}
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