Pub Date : 2026-02-04DOI: 10.1007/s00277-026-06858-9
Devki D Sukhtankar, Pina M Cardarelli, Bita Jalilizeinali, Gayatri Setia, GaYeon Kim, Li-Wen Chang, Cheng-Yuan Tsai, Niña G Caculitan, Michael W Schuster
{"title":"Burixafor, a CXCR4 inhibitor with a differentiated kinetics profile: results of a phase 2 study for rapid cell mobilization in multiple myeloma and lymphoma patients undergoing transplant.","authors":"Devki D Sukhtankar, Pina M Cardarelli, Bita Jalilizeinali, Gayatri Setia, GaYeon Kim, Li-Wen Chang, Cheng-Yuan Tsai, Niña G Caculitan, Michael W Schuster","doi":"10.1007/s00277-026-06858-9","DOIUrl":"10.1007/s00277-026-06858-9","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 3","pages":"86"},"PeriodicalIF":2.4,"publicationDate":"2026-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12872619/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146117660","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-02-04DOI: 10.1007/s00277-026-06798-4
Yasmin H Karimi, Chan Y Cheah, Michael Roost Clausen, David Cunningham, Umar Farooq, Tatyana Feldman, Herve Ghesquieres, Wojciech Jurczak, Kim M Linton, Tycel Phillips, Julie M Vose, Won Seog Kim, Pegah Jafarinasabian, Barbara D'Angelo Månsson, David Soong, Andrew J Steele, Zhu Li, Christian W Eskelund, Martin Hutchings, Catherine Thieblemont
Epcoritamab, a CD3xCD20 bispecific antibody, resulted in deep, durable responses with a manageable safety profile in patients with relapsed/refractory large B-cell lymphoma (LBCL) in EPCORE® NHL-1 (NCT03625037). We report results from a 3-year follow-up. Adults with relapsed/refractory LBCL received epcoritamab until progressive disease or unacceptable toxicity. The primary endpoint was overall response rate (ORR). Median age was 64.0 years, 39% of patients received prior CAR T-cell treatment, and 75% were refractory to ≥ 2 consecutive lines of treatment. As of May 3, 2024 (median follow-up 37.1 months [range, 0.3-45.5]), ORR was 59% and complete response (CR) rate 41% by investigator assessment. Median duration of response was 20.8 months (95% confidence interval [CI], 13.0-32.0). Median duration of CR was 36.1 months (20.2-not reached [NR]); the longest ongoing CR was > 43 months. Median progression-free survival was 4.2 months (95% CI, 2.8-5.5) in all patients and 37.3 months (26.0-NR) in patients with CR. Median overall survival was 18.5 months (95% CI, 11.7-27.7) in all patients and NR in patients with CR. Of 119 patients evaluable for minimal residual disease (MRD) assessments, 54 (45%) were MRD-negative at any time during the study. Most common adverse events were cytokine release syndrome (51%), fatigue (25%), and pyrexia (25%), with no new safety signals. Grade 1, 2, and 3 infections occurred in 23%, 34%, and 24% of patients, respectively. The durability of responses and prolonged survival in complete responders suggest long-term disease-free survival with epcoritamab in these patients with relapsed/refractory LBCL.
{"title":"Efficacy and safety of epcoritamab in relapsed or refractory large B-cell lymphoma: 3-year update from the EPCORE NHL-1 trial.","authors":"Yasmin H Karimi, Chan Y Cheah, Michael Roost Clausen, David Cunningham, Umar Farooq, Tatyana Feldman, Herve Ghesquieres, Wojciech Jurczak, Kim M Linton, Tycel Phillips, Julie M Vose, Won Seog Kim, Pegah Jafarinasabian, Barbara D'Angelo Månsson, David Soong, Andrew J Steele, Zhu Li, Christian W Eskelund, Martin Hutchings, Catherine Thieblemont","doi":"10.1007/s00277-026-06798-4","DOIUrl":"10.1007/s00277-026-06798-4","url":null,"abstract":"<p><p>Epcoritamab, a CD3xCD20 bispecific antibody, resulted in deep, durable responses with a manageable safety profile in patients with relapsed/refractory large B-cell lymphoma (LBCL) in EPCORE<sup>®</sup> NHL-1 (NCT03625037). We report results from a 3-year follow-up. Adults with relapsed/refractory LBCL received epcoritamab until progressive disease or unacceptable toxicity. The primary endpoint was overall response rate (ORR). Median age was 64.0 years, 39% of patients received prior CAR T-cell treatment, and 75% were refractory to ≥ 2 consecutive lines of treatment. As of May 3, 2024 (median follow-up 37.1 months [range, 0.3-45.5]), ORR was 59% and complete response (CR) rate 41% by investigator assessment. Median duration of response was 20.8 months (95% confidence interval [CI], 13.0-32.0). Median duration of CR was 36.1 months (20.2-not reached [NR]); the longest ongoing CR was > 43 months. Median progression-free survival was 4.2 months (95% CI, 2.8-5.5) in all patients and 37.3 months (26.0-NR) in patients with CR. Median overall survival was 18.5 months (95% CI, 11.7-27.7) in all patients and NR in patients with CR. Of 119 patients evaluable for minimal residual disease (MRD) assessments, 54 (45%) were MRD-negative at any time during the study. Most common adverse events were cytokine release syndrome (51%), fatigue (25%), and pyrexia (25%), with no new safety signals. Grade 1, 2, and 3 infections occurred in 23%, 34%, and 24% of patients, respectively. The durability of responses and prolonged survival in complete responders suggest long-term disease-free survival with epcoritamab in these patients with relapsed/refractory LBCL.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 3","pages":"79"},"PeriodicalIF":2.4,"publicationDate":"2026-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12868022/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146112015","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-02-04DOI: 10.1007/s00277-026-06760-4
Garret M K Leung, Joycelyn P Y Sim, Thomas S Y Chan, Carol Y M Cheung, Eric Tse, Albert K W Lie, Harinder Gill, Yok-Lam Kwong
{"title":"Efficacy and safety of belumosudil for refractory chronic graft-versus-host disease in routine practice.","authors":"Garret M K Leung, Joycelyn P Y Sim, Thomas S Y Chan, Carol Y M Cheung, Eric Tse, Albert K W Lie, Harinder Gill, Yok-Lam Kwong","doi":"10.1007/s00277-026-06760-4","DOIUrl":"10.1007/s00277-026-06760-4","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 3","pages":"84"},"PeriodicalIF":2.4,"publicationDate":"2026-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12872659/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146117726","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative treatment for aplastic anemia (AA). For patients lacking a matched sibling donor (MSD) or matched unrelated donor (MUD), haploidentical HSCT (haplo-HSCT) has become an alternative with comparable efficacy. However, donor-specific anti-HLA antibodies (DSAs) remain the principal barrier. In this study, we compared transplant outcomes in DSA-positive recipients after desensitization with plasma exchange (PE) combined with anti-B-cell therapy. Between January 2019 and December 2023, we enrolled 30 DSA-positive AA patients who underwent haplo-HSCT at our center. All received a desensitization regimen combining PE with anti-B-cell agents. By propensity-score matching (1:2 ratio, caliper 0.02), we additionally enrolled 60 DSA-negative controls matched for key clinical variables to compare therapeutic efficacy and long-term outcomes between the two groups. This study enrolled 90 AA patients who underwent haplo-HSCT: 30 were DSA-positive and 60 were DSA-negative. Baseline characteristics were comparable between the two groups. After desensitization with PE combined with anti-B-cell agents, the DSA-positive group achieved outcomes equivalent to those of the DSA-negative group. Specifically, EBV reactivation (46.7% vs. 68.3%; P = 0.002) was significantly better in the DSA-positive group. Engraftment, aGVHD, Effacacy, OS, and GRFS rates were similar between two groups. However, the risk of cGVHD remains higher in the DSA-positive group (40% vs. 12%; P = 0.009). For DSA-positive AA patients who are candidates for haplo-HSCT and for whom no DSA-negative donor can be identified, desensitization with PE combined with anti-B-cell agents represents an established strategy to improve transplant outcomes; nevertheless, close surveillance for cGVHD is warranted after transplantation.
同种异体造血干细胞移植(allo-HSCT)仍然是治疗再生障碍性贫血的唯一方法。对于缺乏匹配兄弟姐妹供体(MSD)或匹配非亲属供体(MUD)的患者,单倍同型HSCT(单倍-HSCT)已成为具有相当疗效的替代方法。然而,供体特异性抗hla抗体(dsa)仍然是主要的障碍。在这项研究中,我们比较了dsa阳性受体在血浆置换(PE)联合抗b细胞治疗脱敏后的移植结果。在2019年1月至2023年12月期间,我们招募了30名dsa阳性AA患者,他们在我们的中心接受了单倍造血干细胞移植。所有患者均接受PE联合抗b细胞药物的脱敏治疗。通过倾向评分匹配(1:2比,卡尺0.02),我们额外招募了60名dsa阴性对照,匹配关键临床变量,比较两组的治疗效果和长期结局。本研究招募了90例接受单倍造血干细胞移植的AA患者:30例dsa阳性,60例dsa阴性。两组患者的基线特征具有可比性。经PE联合抗b细胞药物脱敏后,dsa阳性组的结果与dsa阴性组相当。具体而言,dsa阳性组EBV再激活(46.7% vs. 68.3%; P = 0.002)明显更好。两组间植入率、aGVHD、Effacacy、OS和GRFS相似。然而,dsa阳性组cGVHD的风险仍然较高(40% vs. 12%; P = 0.009)。对于dsa阳性的AA患者,作为单倍hsct的候选者,并且无法确定dsa阴性供体,PE脱敏联合抗b细胞药物代表了改善移植结果的既定策略;然而,移植后密切监测cGVHD是必要的。
{"title":"Plasma exchange combined with anti-B-cell therapy enables comparable outcomes between DSA-positive and DSA-negative aplastic anemia after haplo-HSCT: a propensity-score-matched cohort study.","authors":"Zhengwei Tan, Jinyu Hu, Yuechao Zhao, Huijin Hu, Qinghong Yu, Yu Zhang, Liqiang Wu, Tonglin Hu, Dijiong Wu, Baodong Ye, Wenbin Liu","doi":"10.1007/s00277-026-06815-6","DOIUrl":"10.1007/s00277-026-06815-6","url":null,"abstract":"<p><p>Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only curative treatment for aplastic anemia (AA). For patients lacking a matched sibling donor (MSD) or matched unrelated donor (MUD), haploidentical HSCT (haplo-HSCT) has become an alternative with comparable efficacy. However, donor-specific anti-HLA antibodies (DSAs) remain the principal barrier. In this study, we compared transplant outcomes in DSA-positive recipients after desensitization with plasma exchange (PE) combined with anti-B-cell therapy. Between January 2019 and December 2023, we enrolled 30 DSA-positive AA patients who underwent haplo-HSCT at our center. All received a desensitization regimen combining PE with anti-B-cell agents. By propensity-score matching (1:2 ratio, caliper 0.02), we additionally enrolled 60 DSA-negative controls matched for key clinical variables to compare therapeutic efficacy and long-term outcomes between the two groups. This study enrolled 90 AA patients who underwent haplo-HSCT: 30 were DSA-positive and 60 were DSA-negative. Baseline characteristics were comparable between the two groups. After desensitization with PE combined with anti-B-cell agents, the DSA-positive group achieved outcomes equivalent to those of the DSA-negative group. Specifically, EBV reactivation (46.7% vs. 68.3%; P = 0.002) was significantly better in the DSA-positive group. Engraftment, aGVHD, Effacacy, OS, and GRFS rates were similar between two groups. However, the risk of cGVHD remains higher in the DSA-positive group (40% vs. 12%; P = 0.009). For DSA-positive AA patients who are candidates for haplo-HSCT and for whom no DSA-negative donor can be identified, desensitization with PE combined with anti-B-cell agents represents an established strategy to improve transplant outcomes; nevertheless, close surveillance for cGVHD is warranted after transplantation.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 3","pages":"73"},"PeriodicalIF":2.4,"publicationDate":"2026-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12868018/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146111984","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Neurofibromatosis type I -related severe aplastic anemia: an unusual association with a complicated bone marrow transplantation course.","authors":"Farah Malaeb, Nahla Mobarak, Dayel Alshahrani, Fahad Almanjomi","doi":"10.1007/s00277-026-06752-4","DOIUrl":"10.1007/s00277-026-06752-4","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 3","pages":"83"},"PeriodicalIF":2.4,"publicationDate":"2026-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12872736/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146117651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-02-04DOI: 10.1007/s00277-026-06744-4
Linli Lu, Qi Liang, YuHan Yan, Yishu Tang, Erhua Wang, Ying Li, Jian Zhang, Han Xiao, Qian Cheng, Xin Li
Acute leukemia (AL) is an aggressive hematologic malignancy that often causes serious complications and requires intensive care unit (ICU) treatment. Identifying risk factors for mortality in AL patients during and post-ICU admission can improve prognosis. This retrospective study enrolled AL patients first admitted to the ICU of the Third Xiangya Hospital, Central South University, from November 2008 to October 2023. It analyzed risk factors associated with ICU mortality, 1-month mortality and 6-month mortality, and conducted a subgroup analysis of the prognosis of ICU survivors. A total of 126 patients were included in this study, with a median age of 42 years. Acute respiratory failure (46%) and sepsis (23%) were the main reasons for ICU admission. Overall, the mortality rates were 57.9% in the ICU, 67.5% at 1 month, and 69% at 6 months. Notably, among the ICU survivors, 26.4% (14/53) died within 6 months, with 85.7% (12/14) occurring within the first month. The time from hospital admission to ICU transfer >10 days and invasive mechanical ventilation were independent risk factors for ICU mortality, 1-month mortality and 6-month mortality. Relapsed or refractory leukemia (OR = 20.715) and low albumin levels (OR = 34.428) were independent risk factors for death within one month among ICU survivors. The risk factors for mortality in AL patients admitted to the ICU exhibit dynamic changes across different stages. For ICU survivors, optimizing nutritional support and initiating anti-tumor treatment as early as possible after discharge are crucial for improving prognosis.
{"title":"Dynamic changes in mortality risk factors for acute leukemia patients in the ICU and Post-ICU periods.","authors":"Linli Lu, Qi Liang, YuHan Yan, Yishu Tang, Erhua Wang, Ying Li, Jian Zhang, Han Xiao, Qian Cheng, Xin Li","doi":"10.1007/s00277-026-06744-4","DOIUrl":"10.1007/s00277-026-06744-4","url":null,"abstract":"<p><p>Acute leukemia (AL) is an aggressive hematologic malignancy that often causes serious complications and requires intensive care unit (ICU) treatment. Identifying risk factors for mortality in AL patients during and post-ICU admission can improve prognosis. This retrospective study enrolled AL patients first admitted to the ICU of the Third Xiangya Hospital, Central South University, from November 2008 to October 2023. It analyzed risk factors associated with ICU mortality, 1-month mortality and 6-month mortality, and conducted a subgroup analysis of the prognosis of ICU survivors. A total of 126 patients were included in this study, with a median age of 42 years. Acute respiratory failure (46%) and sepsis (23%) were the main reasons for ICU admission. Overall, the mortality rates were 57.9% in the ICU, 67.5% at 1 month, and 69% at 6 months. Notably, among the ICU survivors, 26.4% (14/53) died within 6 months, with 85.7% (12/14) occurring within the first month. The time from hospital admission to ICU transfer >10 days and invasive mechanical ventilation were independent risk factors for ICU mortality, 1-month mortality and 6-month mortality. Relapsed or refractory leukemia (OR = 20.715) and low albumin levels (OR = 34.428) were independent risk factors for death within one month among ICU survivors. The risk factors for mortality in AL patients admitted to the ICU exhibit dynamic changes across different stages. For ICU survivors, optimizing nutritional support and initiating anti-tumor treatment as early as possible after discharge are crucial for improving prognosis.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 3","pages":"76"},"PeriodicalIF":2.4,"publicationDate":"2026-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12868027/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146112023","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-02-03DOI: 10.1007/s00277-026-06834-3
Chaoshuai Fan, Jiabang Yang, Yanni Peng, Juan Hu, Mingyue Cao, Ruobing Li, Dongmei Jiang, Tao Wang, Wen Luo, Li Chen, Yanrong Luo, Guihua Lu
{"title":"Addition of rituximab to CEAC conditioning for autologous hematopoietic stem cell transplantation provides no survival benefit in diffuse large B-Cell lymphoma: A propensity score-matched cohort study.","authors":"Chaoshuai Fan, Jiabang Yang, Yanni Peng, Juan Hu, Mingyue Cao, Ruobing Li, Dongmei Jiang, Tao Wang, Wen Luo, Li Chen, Yanrong Luo, Guihua Lu","doi":"10.1007/s00277-026-06834-3","DOIUrl":"10.1007/s00277-026-06834-3","url":null,"abstract":"","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 2","pages":"71"},"PeriodicalIF":2.4,"publicationDate":"2026-02-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12868091/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146111976","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2026-02-02DOI: 10.1007/s00277-026-06731-9
Songyuan He, Hao Song, Weiyi Zuo, Shu Liu, Xiaojie Fang, Xudong Li
Although marital status has been associated with survival in several cancers, evidence regarding its prognostic relevance in follicular lymphoma (FL), an indolent lymphoma characterized by prolonged survival, remains limited. In this population-based study using the SEER database, we identified 23,434 adult patients diagnosed with FL between 2013 and 2022. Propensity score matching was performed to balance baseline characteristics, and survival outcomes were assessed using Kaplan-Meier analysis and Cox proportional hazards models. In the overall cohort, unmarried patients were more likely to be female, older, and present with B symptoms (all P < 0.001), and demonstrated significantly lower 5-year overall survival (OS: 72.8% vs. 81.0%, P < 0.001) and cause-specific survival (CSS: 84.9% vs. 90.6%, P < 0.001) compared with married patients. After 1:1 propensity score matching on age, sex, race/ethnicity, Ann Arbor stage, B symptoms, treatment status, prior cancer history, and diagnosis year (matched cohort, n = 16,384), this survival disparity persisted (5-year OS: 72.5% vs. 81.3%; CSS: 84.7% vs. 90.7%; both P < 0.001). Multivariable Cox regression confirmed that unmarried status was independently associated with increased risks of OS (HR, 1.62; 95% CI, 1.53-1.71, P < 0.001) and CSS (HR, 1.77; 95% CI, 1.63-1.92, P < 0.001). These findings suggest that unmarried status is an independent adverse prognostic factor in patients with FL and highlight additional attention may be warranted for unmarried individuals during long-term disease management.
{"title":"Impact of marital status on survival in patients with follicular lymphoma.","authors":"Songyuan He, Hao Song, Weiyi Zuo, Shu Liu, Xiaojie Fang, Xudong Li","doi":"10.1007/s00277-026-06731-9","DOIUrl":"10.1007/s00277-026-06731-9","url":null,"abstract":"<p><p>Although marital status has been associated with survival in several cancers, evidence regarding its prognostic relevance in follicular lymphoma (FL), an indolent lymphoma characterized by prolonged survival, remains limited. In this population-based study using the SEER database, we identified 23,434 adult patients diagnosed with FL between 2013 and 2022. Propensity score matching was performed to balance baseline characteristics, and survival outcomes were assessed using Kaplan-Meier analysis and Cox proportional hazards models. In the overall cohort, unmarried patients were more likely to be female, older, and present with B symptoms (all P < 0.001), and demonstrated significantly lower 5-year overall survival (OS: 72.8% vs. 81.0%, P < 0.001) and cause-specific survival (CSS: 84.9% vs. 90.6%, P < 0.001) compared with married patients. After 1:1 propensity score matching on age, sex, race/ethnicity, Ann Arbor stage, B symptoms, treatment status, prior cancer history, and diagnosis year (matched cohort, n = 16,384), this survival disparity persisted (5-year OS: 72.5% vs. 81.3%; CSS: 84.7% vs. 90.7%; both P < 0.001). Multivariable Cox regression confirmed that unmarried status was independently associated with increased risks of OS (HR, 1.62; 95% CI, 1.53-1.71, P < 0.001) and CSS (HR, 1.77; 95% CI, 1.63-1.92, P < 0.001). These findings suggest that unmarried status is an independent adverse prognostic factor in patients with FL and highlight additional attention may be warranted for unmarried individuals during long-term disease management.</p>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 2","pages":"70"},"PeriodicalIF":2.4,"publicationDate":"2026-02-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12862019/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146099506","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
The outcomes of children with aplastic anemia receiving cyclosporine monotherapy vary significantly in terms of mortality risk; therefore, a prognostic model for predicting mortality risk was constructed to optimize risk-stratified treatment strategies. This retrospective cohort study included children with acquired AA receiving cyclosporine-based immunosuppression, stratified by disease severity (vSAA/SAA/NSAA) and randomly split into training (70%) and validation (30%) cohorts. Ten machine learning models were developed; hyperparameters were optimized via grid search with 10-fold cross-validation exclusively within the training cohort to prevent data leakage. Model performance was evaluated using area under the ROC curve (AUC), accuracy, recall, specificity, precision, F1 score, and Brier score. Decision curve analysis (DCA) quantified clinical net benefit. The calibration curve was used to evaluate the reliability of the predicted probabilities. The SHapley Additive exPlanations (SHAP) framework was used to interpret feature contributions and ensure model transparency. Least absolute shrinkage and selection operator (LASSO) regression on the training cohort identified 5 predictors: reticulocyte count (RC), platelet count (PLT), disease subtype (vSAA/SAA/NSAA), total bilirubin (TB), and bone marrow myeloid proportion. The CatBoost model achieved the highest performance: AUC 0.834 (95% CI: 0.774–0.895) in training and 0.826 (95% CI: 0.743–0.910) in validation, with acceptable calibration (Brier score: 0.206 in training cohort, 0.207 in validation cohort). SHAP analysis confirmed RC as the top contributor, with lower RC values associated with higher predicted mortality risk. The CatBoost model demonstrates robust performance and transparency for predicting mortality risk in children with AA after cyclosporine treatment. Adherence to TRIPOD + AI guidelines ensures methodological rigor, supporting its potential as a clinical decision tool to stratify patients into distinct mortality risk groups and optimize individualized treatment strategies.
{"title":"Machine learning mortality prediction model for cyclosporine therapy in pediatric aplastic anemia","authors":"Xianhao Wen, Li Xiao, Danni Li, Meiling Liao, Yuye Liu, Qihui Liu, Xianmin Guan, Ying Dou, Ziyu Hua","doi":"10.1007/s00277-026-06842-3","DOIUrl":"10.1007/s00277-026-06842-3","url":null,"abstract":"<div><p>The outcomes of children with aplastic anemia receiving cyclosporine monotherapy vary significantly in terms of mortality risk; therefore, a prognostic model for predicting mortality risk was constructed to optimize risk-stratified treatment strategies. This retrospective cohort study included children with acquired AA receiving cyclosporine-based immunosuppression, stratified by disease severity (vSAA/SAA/NSAA) and randomly split into training (70%) and validation (30%) cohorts. Ten machine learning models were developed; hyperparameters were optimized via grid search with 10-fold cross-validation exclusively within the training cohort to prevent data leakage. Model performance was evaluated using area under the ROC curve (AUC), accuracy, recall, specificity, precision, F1 score, and Brier score. Decision curve analysis (DCA) quantified clinical net benefit. The calibration curve was used to evaluate the reliability of the predicted probabilities. The SHapley Additive exPlanations (SHAP) framework was used to interpret feature contributions and ensure model transparency. Least absolute shrinkage and selection operator (LASSO) regression on the training cohort identified 5 predictors: reticulocyte count (RC), platelet count (PLT), disease subtype (vSAA/SAA/NSAA), total bilirubin (TB), and bone marrow myeloid proportion. The CatBoost model achieved the highest performance: AUC 0.834 (95% CI: 0.774–0.895) in training and 0.826 (95% CI: 0.743–0.910) in validation, with acceptable calibration (Brier score: 0.206 in training cohort, 0.207 in validation cohort). SHAP analysis confirmed RC as the top contributor, with lower RC values associated with higher predicted mortality risk. The CatBoost model demonstrates robust performance and transparency for predicting mortality risk in children with AA after cyclosporine treatment. Adherence to TRIPOD + AI guidelines ensures methodological rigor, supporting its potential as a clinical decision tool to stratify patients into distinct mortality risk groups and optimize individualized treatment strategies.</p></div>","PeriodicalId":8068,"journal":{"name":"Annals of Hematology","volume":"105 2","pages":""},"PeriodicalIF":2.4,"publicationDate":"2026-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://link.springer.com/content/pdf/10.1007/s00277-026-06842-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146083010","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}