Pub Date : 2025-03-06DOI: 10.1007/s44313-025-00062-w
John Jeongseok Yang, Sang-Hyun Hwang
Large Language Models (LLMs), such as ChatGPT (OpenAI, CA, US), have revolutionized scientific writing and research processes across academic disciplines, providing comprehensive support throughout the entire research lifecycle. Generative artificial intelligence (GAI) tools enhance every aspect of scientific writing, from hypothesis generation and methodology design to data analysis and manuscript preparation. This review examines the applications of LLMs in hematological research, with particular emphasis on advanced techniques, including prompt engineering and retrieval augmented generation (RAG) frameworks. Prompt engineering methods, including zero-shot and few-shot learning along with a chain-of-thought approach, enable researchers to generate more precise context-specific content, especially in scientific writing. Integrating RAG frameworks with the current medical literature and clinical guidelines significantly reduces the risk of misinformation while ensuring alignment with contemporary medical standards. Even though these GAI tools offer remarkable potential for streamlining research writing and enhancing documentation quality, the study also addresses the critical importance of maintaining scientific integrity, ethical considerations, and privacy concerns in hematological research.
{"title":"Transforming hematological research documentation with large language models: an approach to scientific writing and data analysis.","authors":"John Jeongseok Yang, Sang-Hyun Hwang","doi":"10.1007/s44313-025-00062-w","DOIUrl":"10.1007/s44313-025-00062-w","url":null,"abstract":"<p><p>Large Language Models (LLMs), such as ChatGPT (OpenAI, CA, US), have revolutionized scientific writing and research processes across academic disciplines, providing comprehensive support throughout the entire research lifecycle. Generative artificial intelligence (GAI) tools enhance every aspect of scientific writing, from hypothesis generation and methodology design to data analysis and manuscript preparation. This review examines the applications of LLMs in hematological research, with particular emphasis on advanced techniques, including prompt engineering and retrieval augmented generation (RAG) frameworks. Prompt engineering methods, including zero-shot and few-shot learning along with a chain-of-thought approach, enable researchers to generate more precise context-specific content, especially in scientific writing. Integrating RAG frameworks with the current medical literature and clinical guidelines significantly reduces the risk of misinformation while ensuring alignment with contemporary medical standards. Even though these GAI tools offer remarkable potential for streamlining research writing and enhancing documentation quality, the study also addresses the critical importance of maintaining scientific integrity, ethical considerations, and privacy concerns in hematological research.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"60 1","pages":"15"},"PeriodicalIF":2.3,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11885755/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143568530","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-10DOI: 10.1007/s44313-025-00060-y
Hyungsoon Kim, Haerim Chung, Hye Won Kook, Soo-Jeong Kim, Yu Ri Kim, Hyunsoo Cho, June-Won Cheong
Allogeneic stem cell transplantation (allo-SCT) is a salvage treatment option for patients with relapsed or refractory lymphoid malignancies. However, the clinical variables impacting outcomes in these patients remain unclear. We analyzed 58 patients who underwent allo-SCT for lymphoid malignancies, including B-cell lymphoma (BCL, n = 20), Hodgkin's disease (n = 3), multiple myeloma (n = 9), natural killer/T-cell lymphoma (NK/TCL, n = 4), and TCL (n = 22). The median progression-free survival (PFS) was 27.4 months, while the median overall survival (OS) was 30.6 months. In univariate analysis, human leukocyte antigen (HLA) matching and complete remission status post-transplantation were associated with improved PFS and OS. However, only post-transplant response remained significant for both survival outcomes in the multivariate analysis. Moreover, HLA matching was associated with a significantly improved PFS in patients with BCL and NK/TCL, but with better OS only in those with BCL. Complete remission after transplantation was associated with better PFS and OS in patients with BCL, NK/TCL, and TCL. Our results indicate that post-transplant response is an important prognostic indicator in allo-SCT for lymphoid malignancies and may guide clinical decisions and additional treatment.
同种异体干细胞移植是复发或难治性淋巴细胞恶性肿瘤患者的救助性治疗选择。然而,影响这些患者预后的临床变量仍不清楚。我们分析了58例接受同种异体细胞移植治疗淋巴细胞恶性肿瘤的患者,包括b细胞淋巴瘤(BCL, n = 20)、霍奇金病(n = 3)、多发性骨髓瘤(n = 9)、自然杀伤/ t细胞淋巴瘤(NK/TCL, n = 4)和TCL (n = 22)。中位无进展生存期(PFS)为27.4个月,中位总生存期(OS)为30.6个月。在单变量分析中,人类白细胞抗原(HLA)匹配和移植后完全缓解状态与PFS和OS的改善相关。然而,在多变量分析中,只有移植后反应对两种生存结果仍然显著。此外,HLA配型与BCL和NK/TCL患者的PFS显著改善相关,但只有BCL患者的OS更好。移植后完全缓解与BCL、NK/TCL和TCL患者更好的PFS和OS相关。我们的研究结果表明,移植后反应是淋巴细胞恶性肿瘤同种异体细胞移植的重要预后指标,可以指导临床决策和其他治疗。
{"title":"Prognostic factors and treatment outcomes of allogeneic stem cell transplantation in lymphoid malignancy.","authors":"Hyungsoon Kim, Haerim Chung, Hye Won Kook, Soo-Jeong Kim, Yu Ri Kim, Hyunsoo Cho, June-Won Cheong","doi":"10.1007/s44313-025-00060-y","DOIUrl":"10.1007/s44313-025-00060-y","url":null,"abstract":"<p><p>Allogeneic stem cell transplantation (allo-SCT) is a salvage treatment option for patients with relapsed or refractory lymphoid malignancies. However, the clinical variables impacting outcomes in these patients remain unclear. We analyzed 58 patients who underwent allo-SCT for lymphoid malignancies, including B-cell lymphoma (BCL, n = 20), Hodgkin's disease (n = 3), multiple myeloma (n = 9), natural killer/T-cell lymphoma (NK/TCL, n = 4), and TCL (n = 22). The median progression-free survival (PFS) was 27.4 months, while the median overall survival (OS) was 30.6 months. In univariate analysis, human leukocyte antigen (HLA) matching and complete remission status post-transplantation were associated with improved PFS and OS. However, only post-transplant response remained significant for both survival outcomes in the multivariate analysis. Moreover, HLA matching was associated with a significantly improved PFS in patients with BCL and NK/TCL, but with better OS only in those with BCL. Complete remission after transplantation was associated with better PFS and OS in patients with BCL, NK/TCL, and TCL. Our results indicate that post-transplant response is an important prognostic indicator in allo-SCT for lymphoid malignancies and may guide clinical decisions and additional treatment.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"60 1","pages":"12"},"PeriodicalIF":2.3,"publicationDate":"2025-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11811309/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143383592","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-06DOI: 10.1007/s44313-025-00056-8
Ye Li, Jingjing Deng, Yuan Jian, Zhiyao Zhang, Wenming Chen
Background: t(11;14) is considered a standard risk factor in multiple myeloma (MM). However, recent studies suggested that its impact in the context of novel agents remained controversial.
Methods: This retrospective analysis examined the clinical profiles of 375 newly diagnosed patients with MM and compared the outcomes between those with t(11;14) and those with normal cytogenetics.
Results: The median progression-free survival (PFS) of the 84 patients with t(11;14) was 36 months (95% confidence interval (CI), 23.5-48.5), which was significantly shorter than the median PFS of 65 months (95% CI, 23.0-107.0) for the 59 patients with normal cytogenetics (p = 0.011). Median overall survival (OS) was not reached in either group (p = 0.977). When combined with 1q21 + , t(11;14) showed a trend toward poorer PFS (median PFS: 36 vs. 65 months; p = 0.130). In the presence of high-risk cytogenetics (HRCAs), t(11;14) was associated with a worse PFS (median PFS: 9 vs. 38 months, p = 0.015) and a trend toward shorter OS (median OS: 33 vs. 49 months, p = 0.096). Multivariate analysis indicated that t(11;14) was a poor prognostic factor for PFS. 1q21 + was a detrimental prognostic factor, particularly in the t(11;14) group. Autologous stem cell transplantation (ASCT) may be a beneficial treatment option for patients with t(11;14).
Conclusion: In this study, patients with MM with t(11;14) demonstrated poorer PFS than those with normal cytogenetics. Further investigations are required to evaluate the impact of t(11;14) in patients newly diagnosed with MM in the era of novel agents.
背景:t(11;14)被认为是多发性骨髓瘤(MM)的标准危险因素。然而,最近的研究表明,其在新药物背景下的影响仍然存在争议。方法:回顾性分析了375例新诊断的MM患者的临床资料,并比较了t(11;14)和正常细胞遗传学患者的结果。结果:84例t(11;14)患者的中位无进展生存期(PFS)为36个月(95%可信区间(CI) 23.5-48.5),显著短于59例细胞遗传学正常患者的中位无进展生存期(PFS) 65个月(95% CI, 23.0-107.0) (p = 0.011)。两组的中位总生存期均未达到(p = 0.977)。当与1q21 +联合使用时,t(11;14)显示出较差的PFS趋势(中位PFS: 36 vs 65个月;p = 0.130)。在存在高危细胞遗传学(HRCAs)的情况下,t(11;14)与更差的PFS(中位PFS: 9 vs. 38个月,p = 0.015)和更短的OS(中位OS: 33 vs. 49个月,p = 0.096)相关。多因素分析表明,t(11;14)是PFS的不良预后因素。1q21 +是一个不利的预后因素,特别是在t(11;14)组。自体干细胞移植(ASCT)可能是t患者有益的治疗选择(11;14)。结论:在本研究中,MM伴t(11;14)患者的PFS较细胞遗传学正常的患者差。在新药物时代,需要进一步的研究来评估t(11;14)对新诊断的MM患者的影响。
{"title":"Is t(11;14) in newly diagnosed multiple myeloma a favorable outcome in the novel agent era?","authors":"Ye Li, Jingjing Deng, Yuan Jian, Zhiyao Zhang, Wenming Chen","doi":"10.1007/s44313-025-00056-8","DOIUrl":"10.1007/s44313-025-00056-8","url":null,"abstract":"<p><strong>Background: </strong>t(11;14) is considered a standard risk factor in multiple myeloma (MM). However, recent studies suggested that its impact in the context of novel agents remained controversial.</p><p><strong>Methods: </strong>This retrospective analysis examined the clinical profiles of 375 newly diagnosed patients with MM and compared the outcomes between those with t(11;14) and those with normal cytogenetics.</p><p><strong>Results: </strong>The median progression-free survival (PFS) of the 84 patients with t(11;14) was 36 months (95% confidence interval (CI), 23.5-48.5), which was significantly shorter than the median PFS of 65 months (95% CI, 23.0-107.0) for the 59 patients with normal cytogenetics (p = 0.011). Median overall survival (OS) was not reached in either group (p = 0.977). When combined with 1q21 + , t(11;14) showed a trend toward poorer PFS (median PFS: 36 vs. 65 months; p = 0.130). In the presence of high-risk cytogenetics (HRCAs), t(11;14) was associated with a worse PFS (median PFS: 9 vs. 38 months, p = 0.015) and a trend toward shorter OS (median OS: 33 vs. 49 months, p = 0.096). Multivariate analysis indicated that t(11;14) was a poor prognostic factor for PFS. 1q21 + was a detrimental prognostic factor, particularly in the t(11;14) group. Autologous stem cell transplantation (ASCT) may be a beneficial treatment option for patients with t(11;14).</p><p><strong>Conclusion: </strong>In this study, patients with MM with t(11;14) demonstrated poorer PFS than those with normal cytogenetics. Further investigations are required to evaluate the impact of t(11;14) in patients newly diagnosed with MM in the era of novel agents.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"60 1","pages":"11"},"PeriodicalIF":2.3,"publicationDate":"2025-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11802943/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143257014","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
The efficacy and safety of polatuzumab vedotin combined with rituximab, cyclophosphamide, doxorubicin, and prednisolone (pola-R-CHP) in patients aged ≥ 80 years with untreated diffuse large B-cell lymphoma (DLBCL) remain largely unexplored. In this study, we administered a reduced-dose pola-R-CHP regimen to 38 patients with DLBCL aged > 80 years. Extending the findings of the POLARIX trial in this older individuals' cohort, we conducted a retrospective analysis to assess the efficacy and safety of the treatment in a real-world clinical setting. After 12 months, the overall and progression-free survival rates were 86.2% (95% confidence interval [CI]: 70.0-94.0) and 78.5% (95% CI: 59.2-89.5), respectively. Although the incidence of febrile neutropenia was relatively high (32%), an increased risk was observed in patients with an average relative dose intensity of < 70%, even with reduced treatment intensity. Notably, none of the patients required a dose reduction of polatuzumab vedotin owing to peripheral neuropathy. Therefore, our findings indicate that a reduced-dose pola-R-CHP regimen may be a viable and effective treatment option for older patients newly diagnosed with DLBCL.
{"title":"Real-world effectiveness and safety of rituximab and reduced-dose CHP with polatuzumab vedotin (pola-R-CHP) in patients aged > 80 years with diffuse large B-cell lymphoma: a retrospective analysis.","authors":"Shuku Sato, Shun Tsunoda, Wataru Kamata, Tomiteru Togano, Yotaro Tamai","doi":"10.1007/s44313-025-00059-5","DOIUrl":"10.1007/s44313-025-00059-5","url":null,"abstract":"<p><p>The efficacy and safety of polatuzumab vedotin combined with rituximab, cyclophosphamide, doxorubicin, and prednisolone (pola-R-CHP) in patients aged ≥ 80 years with untreated diffuse large B-cell lymphoma (DLBCL) remain largely unexplored. In this study, we administered a reduced-dose pola-R-CHP regimen to 38 patients with DLBCL aged > 80 years. Extending the findings of the POLARIX trial in this older individuals' cohort, we conducted a retrospective analysis to assess the efficacy and safety of the treatment in a real-world clinical setting. After 12 months, the overall and progression-free survival rates were 86.2% (95% confidence interval [CI]: 70.0-94.0) and 78.5% (95% CI: 59.2-89.5), respectively. Although the incidence of febrile neutropenia was relatively high (32%), an increased risk was observed in patients with an average relative dose intensity of < 70%, even with reduced treatment intensity. Notably, none of the patients required a dose reduction of polatuzumab vedotin owing to peripheral neuropathy. Therefore, our findings indicate that a reduced-dose pola-R-CHP regimen may be a viable and effective treatment option for older patients newly diagnosed with DLBCL.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"60 1","pages":"10"},"PeriodicalIF":2.3,"publicationDate":"2025-02-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11799502/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143190932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-02-04DOI: 10.1007/s44313-025-00055-9
Sung-Hoon Jung, Youngil Koh, Min Kyoung Kim, Jin Seok Kim, Joon Ho Moon, Chang-Ki Min, Dok Hyun Yoon, Sung-Soo Yoon, Je-Jung Lee, Chae Moon Hong, Ka-Won Kang, Jihyun Kwon, Kyoung Ha Kim, Dae Sik Kim, Sung Yong Kim, Sung-Hyun Kim, Yu Ri Kim, Young Rok Do, Yeung-Chul Mun, Sung-Soo Park, Young Hoon Park, Ho Jin Shin, Hyeon-Seok Eom, Sang Eun Yoon, Sang Mee Hwang, Won Sik Lee, Myung-Won Lee, Jun Ho Yi, Ji Yun Lee, Ji Hyun Lee, Ho Sup Lee, Sung-Nam Lim, Jihyang Lim, Ho-Young Yhim, Yoon Hwan Chang, Jae-Cheol Jo, Jinhyun Cho, Hyungwoo Cho, Yoon Seok Choi, Hee Jeong Cho, Ari Ahn, Jong Han Choi, Hyun Jung Kim, Kihyun Kim
Multiple myeloma (MM), a hematological malignancy, is characterized by malignant plasma cell proliferation in the bone marrow. Recent treatment advances have significantly improved patient outcomes associated with MM. In this study, we aimed to develop comprehensive, evidence-based guidelines for the diagnosis, prognosis, and treatment of MM. We identified 12 key clinical questions essential for MM management, guiding the extensive literature review and meta-analysis of the study. Our guidelines provide evidence-based recommendations by integrating patient preferences with survey data. These recommendations include current and emerging diagnostic tools, therapeutic agents, and treatment strategies. By prioritizing a patient-centered approach and rigorous data analysis, these guidelines were developed to enhance MM management, both in Korea and globally.
{"title":"Evidence-based Korean guidelines for the clinical management of multiple myeloma: addressing 12 key clinical questions.","authors":"Sung-Hoon Jung, Youngil Koh, Min Kyoung Kim, Jin Seok Kim, Joon Ho Moon, Chang-Ki Min, Dok Hyun Yoon, Sung-Soo Yoon, Je-Jung Lee, Chae Moon Hong, Ka-Won Kang, Jihyun Kwon, Kyoung Ha Kim, Dae Sik Kim, Sung Yong Kim, Sung-Hyun Kim, Yu Ri Kim, Young Rok Do, Yeung-Chul Mun, Sung-Soo Park, Young Hoon Park, Ho Jin Shin, Hyeon-Seok Eom, Sang Eun Yoon, Sang Mee Hwang, Won Sik Lee, Myung-Won Lee, Jun Ho Yi, Ji Yun Lee, Ji Hyun Lee, Ho Sup Lee, Sung-Nam Lim, Jihyang Lim, Ho-Young Yhim, Yoon Hwan Chang, Jae-Cheol Jo, Jinhyun Cho, Hyungwoo Cho, Yoon Seok Choi, Hee Jeong Cho, Ari Ahn, Jong Han Choi, Hyun Jung Kim, Kihyun Kim","doi":"10.1007/s44313-025-00055-9","DOIUrl":"10.1007/s44313-025-00055-9","url":null,"abstract":"<p><p>Multiple myeloma (MM), a hematological malignancy, is characterized by malignant plasma cell proliferation in the bone marrow. Recent treatment advances have significantly improved patient outcomes associated with MM. In this study, we aimed to develop comprehensive, evidence-based guidelines for the diagnosis, prognosis, and treatment of MM. We identified 12 key clinical questions essential for MM management, guiding the extensive literature review and meta-analysis of the study. Our guidelines provide evidence-based recommendations by integrating patient preferences with survey data. These recommendations include current and emerging diagnostic tools, therapeutic agents, and treatment strategies. By prioritizing a patient-centered approach and rigorous data analysis, these guidelines were developed to enhance MM management, both in Korea and globally.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"60 1","pages":"9"},"PeriodicalIF":2.3,"publicationDate":"2025-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11794900/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143190931","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-30DOI: 10.1007/s44313-024-00053-3
Angelo Nigro
This case report describes the clinical course of a 78-year-old patient diagnosed with polycythemia vera (PV), who presented with pronounced acrocyanosis of the hands in 2021. The patient was treated with hydroxyurea (oncocarbide), and nailfold capillaroscopy revealed an "abnormal pattern" characterized by pronounced architectural disarray and capillary tortuosity, which is uncommon in patients with myeloproliferative neoplasms (MPNs). In 2023, owing to suboptimal symptom management and hematological side effects, the treatment was switched to ruxolitinib, which led to significant clinical improvements by 2024, including near-complete resolution of acrocyanosis and substantial improvement in capillaroscopic abnormalities, with only residual capillary tortuosity noted. This case emphasizes the need for individualized therapeutic interventions for PV, and underscores the potential role of ruxolitinib in ameliorating microvascular dysfunction.
{"title":"Reversibility of acrocyanosis and improvement of capillaroscopic pattern in a patient with polycythemia vera treated with ruxolitinib: a case report.","authors":"Angelo Nigro","doi":"10.1007/s44313-024-00053-3","DOIUrl":"10.1007/s44313-024-00053-3","url":null,"abstract":"<p><p>This case report describes the clinical course of a 78-year-old patient diagnosed with polycythemia vera (PV), who presented with pronounced acrocyanosis of the hands in 2021. The patient was treated with hydroxyurea (oncocarbide), and nailfold capillaroscopy revealed an \"abnormal pattern\" characterized by pronounced architectural disarray and capillary tortuosity, which is uncommon in patients with myeloproliferative neoplasms (MPNs). In 2023, owing to suboptimal symptom management and hematological side effects, the treatment was switched to ruxolitinib, which led to significant clinical improvements by 2024, including near-complete resolution of acrocyanosis and substantial improvement in capillaroscopic abnormalities, with only residual capillary tortuosity noted. This case emphasizes the need for individualized therapeutic interventions for PV, and underscores the potential role of ruxolitinib in ameliorating microvascular dysfunction.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"60 1","pages":"8"},"PeriodicalIF":2.3,"publicationDate":"2025-01-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11782742/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143068265","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-23DOI: 10.1007/s44313-025-00057-7
Kyung Taek Hong, Bo Kyung Kim, Hong Yul An, Jung Yoon Choi, Sang Hoon Song, Kyung-Sang Yu, In-Jin Jang, Hyoung Jin Kang
Purpose: This study compared the outcomes of haploidentical-related donor (HRD) and umbilical cord blood (UCB) hematopoietic stem cell transplantation (HSCT) in pediatric patients with hematologic malignancies.
Methods: Data on patients who underwent HRD HSCT with post-transplant cyclophosphamide (n = 41) and UCB HSCT (n = 24) after targeted busulfan-based myeloablative conditioning with intensive pharmacokinetic monitoring between 2009 and 2018 were retrospectively analyzed.
Results: The median follow-up durations in the HRD and UCB groups were 7.0 and 10.9 years, respectively. The cumulative incidence of acute graft-versus-host disease (GVHD) grades II-IV and moderate-to-severe chronic GVHD did not differ significantly between the groups. However, the HRD group demonstrated significantly lower rates of acute GVHD grades III-IV (4.9% vs. 29.2%, p = 0.009) and non-relapse mortality (2.6% vs. 34.2%, p < 0.001) but a higher relapse incidence (32.1% vs. 8.8%, p = 0.004) than the UCB group. The 5-year event-free and overall survival rates were 65.8% and 54.2% (p = 0.204) and 78.0% and 65.7% (p = 0.142) for the HRD and UCB groups, respectively. Multivariate analysis identified disease status as a significant risk factor for overall survival (hazard ratio, 3.24; p = 0.016). Additionally, UCB HSCT exhibited a trend toward worse event-free survival compared to HRD HSCT (hazard ratio, 2.63; p = 0.05).
Conclusions: These findings indicate that HRD HSCT with post-transplant cyclophosphamide provides promising outcomes compared to UCB HSCT in pediatric patients, with a trend toward improved survival over a long-term follow-up period exceeding a median of 7 years. Thus, HRD HSCT may be a valuable option for pediatric patients without human leukocyte antigen-matched donors.
目的:本研究比较了单倍体相关供体(HRD)和脐带血(UCB)造血干细胞移植(HSCT)治疗儿童血液病恶性患者的结果。方法:回顾性分析2009年至2018年接受移植后环磷酰胺(n = 41)和UCB HSCT (n = 24)的HRD HSCT患者的数据,这些患者在接受靶向布磺胺为基础的清髓调节后进行了强化药代动力学监测。结果:HRD组和UCB组的中位随访时间分别为7.0年和10.9年。急性移植物抗宿主病(GVHD) II-IV级和中度至重度慢性GVHD的累积发病率在两组之间无显著差异。然而,HRD组显示出明显较低的急性GVHD III-IV级发生率(4.9% vs. 29.2%, p = 0.009)和非复发死亡率(2.6% vs. 34.2%, p)。结论:这些发现表明,与UCB HSCT相比,HRD移植后环磷酰胺HSCT在儿科患者中提供了有希望的结果,在超过中位7年的长期随访期间有改善生存率的趋势。因此,对于没有人类白细胞抗原匹配供体的儿科患者,HRD HSCT可能是一个有价值的选择。
{"title":"Comparing haploidentical transplantation with post-transplantation cyclophosphamide and umbilical cord blood transplantation using targeted busulfan in children and adolescents with hematologic malignancies.","authors":"Kyung Taek Hong, Bo Kyung Kim, Hong Yul An, Jung Yoon Choi, Sang Hoon Song, Kyung-Sang Yu, In-Jin Jang, Hyoung Jin Kang","doi":"10.1007/s44313-025-00057-7","DOIUrl":"10.1007/s44313-025-00057-7","url":null,"abstract":"<p><strong>Purpose: </strong>This study compared the outcomes of haploidentical-related donor (HRD) and umbilical cord blood (UCB) hematopoietic stem cell transplantation (HSCT) in pediatric patients with hematologic malignancies.</p><p><strong>Methods: </strong>Data on patients who underwent HRD HSCT with post-transplant cyclophosphamide (n = 41) and UCB HSCT (n = 24) after targeted busulfan-based myeloablative conditioning with intensive pharmacokinetic monitoring between 2009 and 2018 were retrospectively analyzed.</p><p><strong>Results: </strong>The median follow-up durations in the HRD and UCB groups were 7.0 and 10.9 years, respectively. The cumulative incidence of acute graft-versus-host disease (GVHD) grades II-IV and moderate-to-severe chronic GVHD did not differ significantly between the groups. However, the HRD group demonstrated significantly lower rates of acute GVHD grades III-IV (4.9% vs. 29.2%, p = 0.009) and non-relapse mortality (2.6% vs. 34.2%, p < 0.001) but a higher relapse incidence (32.1% vs. 8.8%, p = 0.004) than the UCB group. The 5-year event-free and overall survival rates were 65.8% and 54.2% (p = 0.204) and 78.0% and 65.7% (p = 0.142) for the HRD and UCB groups, respectively. Multivariate analysis identified disease status as a significant risk factor for overall survival (hazard ratio, 3.24; p = 0.016). Additionally, UCB HSCT exhibited a trend toward worse event-free survival compared to HRD HSCT (hazard ratio, 2.63; p = 0.05).</p><p><strong>Conclusions: </strong>These findings indicate that HRD HSCT with post-transplant cyclophosphamide provides promising outcomes compared to UCB HSCT in pediatric patients, with a trend toward improved survival over a long-term follow-up period exceeding a median of 7 years. Thus, HRD HSCT may be a valuable option for pediatric patients without human leukocyte antigen-matched donors.</p>","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"60 1","pages":"7"},"PeriodicalIF":2.3,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11757842/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143025222","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-01-21DOI: 10.1007/s44313-025-00058-6
Jeong-Moo Lee
{"title":"Correction: Strategies for integrating ChatGPT and generative AI into clinical studies.","authors":"Jeong-Moo Lee","doi":"10.1007/s44313-025-00058-6","DOIUrl":"10.1007/s44313-025-00058-6","url":null,"abstract":"","PeriodicalId":46224,"journal":{"name":"Blood Research","volume":"60 1","pages":"6"},"PeriodicalIF":2.3,"publicationDate":"2025-01-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751195/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143013340","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}