首页 > 最新文献

Haematologica最新文献

英文 中文
Response to DA-EPOCH-R is associated with activation of 'fitter' cytotoxic T cells in patients with newly diagnosed double and triple hit high-grade B-cell lymphoma. 对 DA-EPOCH-R 的反应与新诊断的双打和三打高级别 B 细胞淋巴瘤患者 "更合适的 "细胞毒性 T 细胞的激活有关。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-11-01 DOI: 10.3324/haematol.2024.285170
A Vera De Jonge, Wassilis S C Bruins, Carolien Duetz, Charlotte L B M Korst, Rosa Rentenaar, Meliha Cosovic, Merve Eken, Marie-Jose Kersten, Yorick Sandberg, Rozemarijn S Van Rijn, Rob Fijnheer, Pim Mutsaers, Vibeke K J Vergote, Djamila Issa, Aart Beeker, Yavuz M Bilgin, Otto Visser, Erik Van Werkhoven, Margaretha G M Roemer, Martine E D Chamuleau, Tuna Mutis
{"title":"Response to DA-EPOCH-R is associated with activation of 'fitter' cytotoxic T cells in patients with newly diagnosed double and triple hit high-grade B-cell lymphoma.","authors":"A Vera De Jonge, Wassilis S C Bruins, Carolien Duetz, Charlotte L B M Korst, Rosa Rentenaar, Meliha Cosovic, Merve Eken, Marie-Jose Kersten, Yorick Sandberg, Rozemarijn S Van Rijn, Rob Fijnheer, Pim Mutsaers, Vibeke K J Vergote, Djamila Issa, Aart Beeker, Yavuz M Bilgin, Otto Visser, Erik Van Werkhoven, Margaretha G M Roemer, Martine E D Chamuleau, Tuna Mutis","doi":"10.3324/haematol.2024.285170","DOIUrl":"10.3324/haematol.2024.285170","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"3760-3765"},"PeriodicalIF":8.2,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11532709/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141497899","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Extending duration of letermovir prophylaxis in haploidentical stem cell transplantation. 延长单倍体干细胞移植中的特莫病毒预防期。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-11-01 DOI: 10.3324/haematol.2024.285766
Pongthep Vittayawacharin, Benjamin J Lee, Shawn Griffin, Jean Doh, Julie Smith, Hannah Nam, Emily Blodget, Deepa Jeyakumar, Piyanuch Kongtim, Stefan O Ciurea
{"title":"Extending duration of letermovir prophylaxis in haploidentical stem cell transplantation.","authors":"Pongthep Vittayawacharin, Benjamin J Lee, Shawn Griffin, Jean Doh, Julie Smith, Hannah Nam, Emily Blodget, Deepa Jeyakumar, Piyanuch Kongtim, Stefan O Ciurea","doi":"10.3324/haematol.2024.285766","DOIUrl":"10.3324/haematol.2024.285766","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":"3806-3810"},"PeriodicalIF":8.2,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11532715/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141579525","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Bispecific antibodies in follicular lymphoma. 滤泡淋巴瘤中的双特异性抗体
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-10-31 DOI: 10.3324/haematol.2024.285245
Imran A Nizamuddin, Nancy L Bartlett

Bispecific antibodies, specifically anti-CD20 T-cell engaging constructs, are poised to alter the treatment paradigm of multiple B-cell malignancies, including follicular lymphoma. Two CD20xCD3 bispecific antibodies, mosunetuzumab and epcoritamab, are now approved in the United States for third-line or later treatment of follicular lymphoma. A third agent, odronextamab, remains under review by regulatory agencies. In pivotal phase II trials, these bispecific antibodies demonstrated overall response rates of approximately 80%, with complete response rates of 60-70%, the majority of which have been durable at two years. Important safety signals included risk of infections, neutropenia, and cytokine release syndrome, which occurred in approximately half of patients, but was rarely high grade. Despite similar efficacy and toxicity profiles, key differences exist among agents, primarily relating to treatment duration, route of administration, and prophylactic corticosteroid use. Several ongoing studies are exploring bispecific antibodies in earlier lines of treatment, either as single agents or in combination with other active therapies. This novel class of agents is likely to play a pivotal role in improving outcomes for patients with follicular lymphoma.

双特异性抗体,特别是抗CD20 T细胞接合构建物,有望改变包括滤泡性淋巴瘤在内的多种B细胞恶性肿瘤的治疗模式。美国现已批准两种 CD20xCD3 双特异性抗体,即 mosunetuzumab 和 epcoritamab,用于滤泡性淋巴瘤的三线或更晚期治疗。第三种药物odronextamab仍在接受监管机构的审查。在关键的 II 期试验中,这些双特异性抗体的总体反应率约为 80%,完全反应率为 60-70%,其中大多数反应可持续两年。重要的安全性信号包括感染风险、中性粒细胞减少症和细胞因子释放综合征。尽管疗效和毒性特征相似,但不同药物之间存在主要差异,主要涉及治疗持续时间、给药途径和预防性皮质类固醇的使用。目前有几项研究正在探索将双特异性抗体作为单一药物或与其他活性疗法联合应用于早期治疗。这类新型药物很可能在改善滤泡性淋巴瘤患者的治疗效果方面发挥关键作用。
{"title":"Bispecific antibodies in follicular lymphoma.","authors":"Imran A Nizamuddin, Nancy L Bartlett","doi":"10.3324/haematol.2024.285245","DOIUrl":"https://doi.org/10.3324/haematol.2024.285245","url":null,"abstract":"<p><p>Bispecific antibodies, specifically anti-CD20 T-cell engaging constructs, are poised to alter the treatment paradigm of multiple B-cell malignancies, including follicular lymphoma. Two CD20xCD3 bispecific antibodies, mosunetuzumab and epcoritamab, are now approved in the United States for third-line or later treatment of follicular lymphoma. A third agent, odronextamab, remains under review by regulatory agencies. In pivotal phase II trials, these bispecific antibodies demonstrated overall response rates of approximately 80%, with complete response rates of 60-70%, the majority of which have been durable at two years. Important safety signals included risk of infections, neutropenia, and cytokine release syndrome, which occurred in approximately half of patients, but was rarely high grade. Despite similar efficacy and toxicity profiles, key differences exist among agents, primarily relating to treatment duration, route of administration, and prophylactic corticosteroid use. Several ongoing studies are exploring bispecific antibodies in earlier lines of treatment, either as single agents or in combination with other active therapies. This novel class of agents is likely to play a pivotal role in improving outcomes for patients with follicular lymphoma.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545068","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Comparative efficacy of lisocabtagene maraleucel in the PILOT study versus second-line chemotherapy regimens in the real world. PILOT 研究中的 lisocabtagene maraleucel 与现实世界中二线化疗方案的疗效比较。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-10-31 DOI: 10.3324/haematol.2024.285828
Nilanjan Ghosh, Alison Sehgal, Fei Fei Liu, Ana Kostic, Alessandro Crotta, Marc De Benedetti, Jillian Faccone, Lily Peng, Leo I Gordon

This study assessed the comparative efficacy of lisocabtagene maraleucel (liso-cel) in PILOT (NCT03483103), an open-label, phase II study, versus conventional second-line (2L) chemotherapy regimens in the real world administered to patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) who were not intended for hematopoietic stem cell transplantation (HSCT). The liso-cel-treated cohort (n=61) was based on patients who received liso-cel in the PILOT study. The conventional chemotherapy cohort included patients who met PILOT eligibility criteria and received conventional 2L chemotherapy in the real-world clinical setting (n=273). After using the trimmed stabilized inverse probability of treatment weighting method to balance cohorts according to baseline characteristics, there were statistically significant differences in all tested measures of efficacy. Compared with real-world conventional chemotherapy regimens, liso-cel demonstrated higher overall response rates (79.6% with liso-cel vs. 50.5% with conventional chemotherapy; relative risk [RR], 1.6; P.

本研究评估了开放标签II期研究PILOT(NCT03483103)中的lisocabtagene maraleucel(liso-cel)与传统二线(2L)化疗方案的疗效比较,后者用于复发或难治性(R/R)大B细胞淋巴瘤(LBCL)患者,且不打算进行造血干细胞移植(HSCT)。接受过liso-cel治疗的队列(n=61)是基于在PILOT研究中接受过liso-cel治疗的患者。常规化疗队列包括符合PILOT资格标准并在实际临床环境中接受常规2L化疗的患者(n=273)。根据基线特征使用修剪稳定化治疗逆概率加权法平衡队列后,所有测试的疗效指标均存在显著统计学差异。与真实世界中的传统化疗方案相比,liso-cel的总体应答率更高(liso-cel为79.6%,传统化疗为50.5%;相对风险[RR],1.6;P.
{"title":"Comparative efficacy of lisocabtagene maraleucel in the PILOT study <i>versus</i> second-line chemotherapy regimens in the real world.","authors":"Nilanjan Ghosh, Alison Sehgal, Fei Fei Liu, Ana Kostic, Alessandro Crotta, Marc De Benedetti, Jillian Faccone, Lily Peng, Leo I Gordon","doi":"10.3324/haematol.2024.285828","DOIUrl":"https://doi.org/10.3324/haematol.2024.285828","url":null,"abstract":"<p><p>This study assessed the comparative efficacy of lisocabtagene maraleucel (liso-cel) in PILOT (NCT03483103), an open-label, phase II study, versus conventional second-line (2L) chemotherapy regimens in the real world administered to patients with relapsed or refractory (R/R) large B-cell lymphoma (LBCL) who were not intended for hematopoietic stem cell transplantation (HSCT). The liso-cel-treated cohort (n=61) was based on patients who received liso-cel in the PILOT study. The conventional chemotherapy cohort included patients who met PILOT eligibility criteria and received conventional 2L chemotherapy in the real-world clinical setting (n=273). After using the trimmed stabilized inverse probability of treatment weighting method to balance cohorts according to baseline characteristics, there were statistically significant differences in all tested measures of efficacy. Compared with real-world conventional chemotherapy regimens, liso-cel demonstrated higher overall response rates (79.6% with liso-cel vs. 50.5% with conventional chemotherapy; relative risk [RR], 1.6; P.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545069","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Survival outcomes in diffuse large B-cell lymphoma patients with and without HIV in the United States from 2001 to 2016: a population-based analysis. 2001年至2016年美国感染和未感染艾滋病毒的弥漫大B细胞淋巴瘤患者的生存结果:基于人群的分析。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-10-31 DOI: 10.3324/haematol.2024.286343
Bryan Valcarcel, Sara J Schonfeld, Meredith S Shiels, Jorge J Castillo, Lindsay M Morton

Not available.

不详。
{"title":"Survival outcomes in diffuse large B-cell lymphoma patients with and without HIV in the United States from 2001 to 2016: a population-based analysis.","authors":"Bryan Valcarcel, Sara J Schonfeld, Meredith S Shiels, Jorge J Castillo, Lindsay M Morton","doi":"10.3324/haematol.2024.286343","DOIUrl":"https://doi.org/10.3324/haematol.2024.286343","url":null,"abstract":"<p><p>Not available.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545073","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Outcomes of children and young adults with B-cell acute lymphoblastic leukemia given blinatumomab as last consolidation treatment before allogeneic hematopoietic stem cell transplantation. B细胞急性淋巴细胞白血病儿童和青少年患者在接受同种异体造血干细胞移植前最后一次巩固治疗时使用blinatumomab的疗效。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-10-31 DOI: 10.3324/haematol.2024.286350
Mattia Algeri, Michele Massa, Daria Pagliara, Valentina Bertaina, Federica Galaverna, Ilaria Pili, Giuseppina Li Pira, Roberto Carta, Francesco Quagliarella, Rita M Pinto, Chiara Rosignoli, Barbarella Lucarelli, Maria G Cefalo, Emilia Boccieri, Francesca Benini, Francesca Del Bufalo, Marco Becilli, Pietro Merli, Gerhard Zugmaier, Franco Locatelli

Blinatumomab has remarkable efficacy in patients with relapsed/refractory (r/r) or measurable residual disease (MRD)-positive B-cell acute lymphoblastic leukemia (B-ALL). In many patients, blinatumomab treatment is followed by allogeneic hematopoietic stem cell transplantation (HSCT). However, the influence of blinatumomab on HSCT outcomes in children and young adults (YA) remains to be fully elucidated. We conducted a single-center, retrospective analysis on patients given blinatumomab as last treatment before HSCT. Seventy-eight pediatric and YA patients were evaluated. With a median follow-up of 23.23 months, the 2-year disease-free (DFS) and overall survival (OS) probability were 72.2% and 89.2%, respectively, with a 2-year cumulative incidence (CI) of non-relapse mortality (NRM) of 2.6%. A trend toward improved 2-year DFS, but not OS, was noted in patients transplanted in first complete remission (CR1) (92.9%) compared to those in second or greater remission (CR2/3) (68.5%, p=0.18) due to a lower CI of relapse (0% vs. 29.9%, p=0.05). Among CR2/3 patients, those receiving the sequential combination of inotuzumab and blinatumomab had a significantly lower CI of relapse as compared to those who did not receive inotuzumab (9.5% vs. 40.4%, p=0.023). Relapse after HSCT occurred in 16 patients, all exhibiting CD19-positive blasts; 10 of them received anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy and 2 inotuzumab as salvage therapy, leading to a 2-year post-relapse OS of 52.7%. Our results indicate that HSCT following blinatumomab in children and YA with B-ALL is highly effective, being associated with low NRM and not affecting the efficacy of subsequent salvage immunotherapies, including CAR-T cells.

对于复发/难治(r/r)或可测量残留病(MRD)阳性B细胞急性淋巴细胞白血病(B-ALL)患者,Blinatumomab具有显著疗效。许多患者在接受blinatumomab治疗后,会进行异基因造血干细胞移植(HSCT)。然而,blinatumomab对儿童和年轻成人(YA)造血干细胞移植结果的影响仍有待全面阐明。我们对造血干细胞移植前最后一次接受blinatumomab治疗的患者进行了单中心回顾性分析。共有 78 名儿童和青年患者接受了评估。中位随访时间为23.23个月,2年无病生存率(DFS)和总生存率(OS)分别为72.2%和89.2%,2年非复发死亡率(NRM)的累积发生率(CI)为2.6%。与第二次或更多次缓解(CR2/3)患者(68.5%,P=0.18)相比,首次完全缓解(CR1)移植患者(92.9%)的2年DFS有改善趋势,但OS没有改善,原因是复发的CI较低(0% vs. 29.9%,P=0.05)。在CR2/3患者中,与未接受伊妥珠单抗治疗的患者相比,接受伊妥珠单抗和blinatumomab序贯联合治疗的患者复发CI显著较低(9.5% vs. 40.4%,p=0.023)。造血干细胞移植后复发的患者有16例,均表现为CD19阳性囊泡,其中10例接受了抗CD19嵌合抗原受体T细胞(CAR-T)疗法,2例接受了伊妥珠单抗作为挽救疗法,复发后2年的OS为52.7%。我们的研究结果表明,B-ALL患儿和YA接受blinatumomab治疗后进行造血干细胞移植非常有效,NRM较低,而且不会影响包括CAR-T细胞在内的后续挽救性免疫疗法的疗效。
{"title":"Outcomes of children and young adults with B-cell acute lymphoblastic leukemia given blinatumomab as last consolidation treatment before allogeneic hematopoietic stem cell transplantation.","authors":"Mattia Algeri, Michele Massa, Daria Pagliara, Valentina Bertaina, Federica Galaverna, Ilaria Pili, Giuseppina Li Pira, Roberto Carta, Francesco Quagliarella, Rita M Pinto, Chiara Rosignoli, Barbarella Lucarelli, Maria G Cefalo, Emilia Boccieri, Francesca Benini, Francesca Del Bufalo, Marco Becilli, Pietro Merli, Gerhard Zugmaier, Franco Locatelli","doi":"10.3324/haematol.2024.286350","DOIUrl":"https://doi.org/10.3324/haematol.2024.286350","url":null,"abstract":"<p><p>Blinatumomab has remarkable efficacy in patients with relapsed/refractory (r/r) or measurable residual disease (MRD)-positive B-cell acute lymphoblastic leukemia (B-ALL). In many patients, blinatumomab treatment is followed by allogeneic hematopoietic stem cell transplantation (HSCT). However, the influence of blinatumomab on HSCT outcomes in children and young adults (YA) remains to be fully elucidated. We conducted a single-center, retrospective analysis on patients given blinatumomab as last treatment before HSCT. Seventy-eight pediatric and YA patients were evaluated. With a median follow-up of 23.23 months, the 2-year disease-free (DFS) and overall survival (OS) probability were 72.2% and 89.2%, respectively, with a 2-year cumulative incidence (CI) of non-relapse mortality (NRM) of 2.6%. A trend toward improved 2-year DFS, but not OS, was noted in patients transplanted in first complete remission (CR1) (92.9%) compared to those in second or greater remission (CR2/3) (68.5%, p=0.18) due to a lower CI of relapse (0% vs. 29.9%, p=0.05). Among CR2/3 patients, those receiving the sequential combination of inotuzumab and blinatumomab had a significantly lower CI of relapse as compared to those who did not receive inotuzumab (9.5% vs. 40.4%, p=0.023). Relapse after HSCT occurred in 16 patients, all exhibiting CD19-positive blasts; 10 of them received anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy and 2 inotuzumab as salvage therapy, leading to a 2-year post-relapse OS of 52.7%. Our results indicate that HSCT following blinatumomab in children and YA with B-ALL is highly effective, being associated with low NRM and not affecting the efficacy of subsequent salvage immunotherapies, including CAR-T cells.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545072","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Impact of daratumumab/bortezomib/thalidomide and dexamethasone induction therapy on chemo-free stem cell mobilization in transplant eligible newly diagnosed multiple myeloma: a multicentre real-world experience. 达拉单抗/硼替佐米/他替佐米和地塞米松诱导疗法对符合移植条件的新诊断多发性骨髓瘤患者无化疗干细胞动员的影响:多中心真实世界经验。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-10-31 DOI: 10.3324/haematol.2024.286332
Marika Porrazzo, Tiziana Moscato, Giuseppe Sapienza, Fabrizio Accardi, Caterina Patti, Clotilde Cangialosi, Carmelo Costanza, Roberto Bono, Stefania Tringali, Cristina Rotolo, Emilia Gigliotta, Andrea Rizzuto, Manuela Giuseppa Ingrascè, Giulia Butera, Laura Di Noto, Alessandra Santoro, Anna Marfia, Cirino Botta, Sergio Siragusa, Massimo Martino, Luca Castagna

Not available.

不详。
{"title":"Impact of daratumumab/bortezomib/thalidomide and dexamethasone induction therapy on chemo-free stem cell mobilization in transplant eligible newly diagnosed multiple myeloma: a multicentre real-world experience.","authors":"Marika Porrazzo, Tiziana Moscato, Giuseppe Sapienza, Fabrizio Accardi, Caterina Patti, Clotilde Cangialosi, Carmelo Costanza, Roberto Bono, Stefania Tringali, Cristina Rotolo, Emilia Gigliotta, Andrea Rizzuto, Manuela Giuseppa Ingrascè, Giulia Butera, Laura Di Noto, Alessandra Santoro, Anna Marfia, Cirino Botta, Sergio Siragusa, Massimo Martino, Luca Castagna","doi":"10.3324/haematol.2024.286332","DOIUrl":"https://doi.org/10.3324/haematol.2024.286332","url":null,"abstract":"<p><p>Not available.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545071","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Early death and intracranial hemorrhage prediction in acute promyelocytic leukemia: validation of a risk score in a chemotherapy plus ATRA cohort from an international consortium. 急性早幼粒细胞白血病早期死亡和颅内出血预测:在一个国际联盟的化疗加ATRA队列中验证风险评分。
IF 8.2 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-10-31 DOI: 10.3324/haematol.2024.286338
Wellington F Silva, Haesook T Kim, Maria S Undurraga, Juan R Navarro-Cabrera, Victor Salinas, Pablo Muxi, Raul A M Melo, Ana Beatriz F Gloria, Katia B B Pagnano, Elenaide C Nunes, Rosane Isabel Bittencourt, Ninoska Rojas, Shirley M Q Truyenque, Ana Ilda Ayala-Lugo, Ana Carolina Oliver, Lorena L Figueiredo-Pontes, Fabiola Traina, Frederico Moreira, Evandro M Fagundes, Bruno K L Duarte, Analí Pamela Mora-Alferez, Percy Ortiz, Jose Luis Untama, Martin S Tallman, Raul C Ribeiro, Arnold Ganser, Richard James Dillon, Peter J M Valk, Miguel A Sanz, Bob Löwenberg, Nancy Berliner, Eduardo M Rego

Not available.

不详。
{"title":"Early death and intracranial hemorrhage prediction in acute promyelocytic leukemia: validation of a risk score in a chemotherapy plus ATRA cohort from an international consortium.","authors":"Wellington F Silva, Haesook T Kim, Maria S Undurraga, Juan R Navarro-Cabrera, Victor Salinas, Pablo Muxi, Raul A M Melo, Ana Beatriz F Gloria, Katia B B Pagnano, Elenaide C Nunes, Rosane Isabel Bittencourt, Ninoska Rojas, Shirley M Q Truyenque, Ana Ilda Ayala-Lugo, Ana Carolina Oliver, Lorena L Figueiredo-Pontes, Fabiola Traina, Frederico Moreira, Evandro M Fagundes, Bruno K L Duarte, Analí Pamela Mora-Alferez, Percy Ortiz, Jose Luis Untama, Martin S Tallman, Raul C Ribeiro, Arnold Ganser, Richard James Dillon, Peter J M Valk, Miguel A Sanz, Bob Löwenberg, Nancy Berliner, Eduardo M Rego","doi":"10.3324/haematol.2024.286338","DOIUrl":"https://doi.org/10.3324/haematol.2024.286338","url":null,"abstract":"<p><p>Not available.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":" ","pages":""},"PeriodicalIF":8.2,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142545070","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Diagnosis of myelodysplastic syndromes: the classic and the novel. 骨髓增生异常综合征的诊断:经典与新颖。
IF 10.1 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-10-24 DOI: 10.3324/haematol.2023.284937
Howard S Oster,Arjan A Van de Loosdrecht,Moshe Mittelman
The Myelodysplastic syndromes (MDS) are a heterogenous group of clonal bone marrow (BM) stem cell myeloid neoplasms, characterized by bone marrow (BM) dysplasia, macrocytic anemia or cytopenia with a tendency for leukemic transformation. The suspicion of MDS is raised by a typical but not specific clinical picture and routine labs, but the gold standard for MDS diagnosis is still BM examination with the presence of uni-or multi-lineage dysplasia and blast percentage, together with exclusion of other reasons. Cytogenetics is also a part of the diagnostic process. Flow cytometry and genetics are helpful but are not always mandatory for MDS diagnosis. This review summarizes the current steps in the diagnostic approach for a patient suspected of having MDS. We also describe new concepts that use non-invasive diagnostic technologies, especially digital methods as well as peripheral blood genetics. The hope is that one day these will mature, be introduced into clinical practice, and perhaps in many cases even replace the invasive BM biopsy.
骨髓增生异常综合征(MDS)是一组异质性克隆性骨髓干细胞髓系肿瘤,其特征是骨髓(BM)发育不良、巨幼红细胞性贫血或全血细胞减少,并有白血病转化倾向。典型但非特异性的临床表现和常规实验室检查可引起对 MDS 的怀疑,但 MDS 诊断的金标准仍是骨髓检查,即出现单系或多系发育不良和胚泡百分比,并排除其他原因。细胞遗传学也是诊断过程的一部分。流式细胞术和遗传学对 MDS 诊断很有帮助,但并不总是必须的。本综述总结了目前对疑似 MDS 患者的诊断步骤。我们还介绍了使用无创诊断技术的新概念,尤其是数字方法和外周血遗传学。希望有一天这些技术能够成熟起来,并被引入临床实践,甚至在很多情况下取代有创的骨髓活检。
{"title":"Diagnosis of myelodysplastic syndromes: the classic and the novel.","authors":"Howard S Oster,Arjan A Van de Loosdrecht,Moshe Mittelman","doi":"10.3324/haematol.2023.284937","DOIUrl":"https://doi.org/10.3324/haematol.2023.284937","url":null,"abstract":"The Myelodysplastic syndromes (MDS) are a heterogenous group of clonal bone marrow (BM) stem cell myeloid neoplasms, characterized by bone marrow (BM) dysplasia, macrocytic anemia or cytopenia with a tendency for leukemic transformation. The suspicion of MDS is raised by a typical but not specific clinical picture and routine labs, but the gold standard for MDS diagnosis is still BM examination with the presence of uni-or multi-lineage dysplasia and blast percentage, together with exclusion of other reasons. Cytogenetics is also a part of the diagnostic process. Flow cytometry and genetics are helpful but are not always mandatory for MDS diagnosis. This review summarizes the current steps in the diagnostic approach for a patient suspected of having MDS. We also describe new concepts that use non-invasive diagnostic technologies, especially digital methods as well as peripheral blood genetics. The hope is that one day these will mature, be introduced into clinical practice, and perhaps in many cases even replace the invasive BM biopsy.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"4 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142489659","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
Genome sequencing in the management of myelodysplastic syndromes and related disorders. 骨髓增生异常综合征及相关疾病治疗中的基因组测序。
IF 10.1 1区 医学 Q1 HEMATOLOGY Pub Date : 2024-10-24 DOI: 10.3324/haematol.2023.284947
Mario Cazzola,Luca Malcovati
Myeloid neoplasms originate from the clonal proliferation of hematopoietic stem cells, which is driven by the acquisition of somatic genetic mutations. Within these disorders, myelodysplastic syndromes (MDS) are specifically characterized by morphologic abnormalities (dysplasia) and impaired maturation of myeloid precursors (ineffective hematopoiesis), resulting in peripheral blood cytopenia. Several studies have advanced the field of MDS, with a few landmark papers leading to a paradigm shift, opening new avenues of research and enabling a molecular revolution. These seminal papers include the first description of the 5q- syndrome, the identification of somatic mutations of TET2 in myeloid neoplasms, the detection of common pathway mutations in the splicing machinery, and the discovery of clonal hematopoiesis. The somatic genomic landscape of MDS is now well-defined. Genes that are recurrently mutated include epigenetic regulators, as well as genes of RNA splicing machinery, transcription regulation, DNA repair control, cohesin complex, and signal transduction. Furthermore, several disorders with a germline genetic predisposition to MDS have been identified, collectively accounting for up to 15% of all MDS cases. Genomic profiling can significantly improve the diagnostic approach to MDS, allowing the identification of distinct nosologic entities such as SF3B1-mutant or TP53-mutant MDS. The Molecular International Prognostic Scoring System for MDS (IPSS-M) has already proven to be a valuable tool for individualized risk assessment and treatment decisions. In addition, the recently developed molecular taxonomy of MDS will likely facilitate the implementation of precision medicine approaches for these disorders. This will necessitate the establishment of specialized infrastructures within public health systems, involving close collaboration between healthcare institutions, academia, and the life sciences industry.
髓系肿瘤起源于造血干细胞的克隆性增殖,其驱动力来自体细胞基因突变。在这些疾病中,骨髓增生异常综合征(MDS)的具体特征是形态异常(发育不良)和骨髓前体成熟受损(无效造血),导致外周血全血细胞减少。有几项研究推动了 MDS 领域的发展,其中几篇具有里程碑意义的论文导致了范式的转变,开辟了新的研究途径,促成了一场分子革命。这些开创性论文包括首次描述 5q- 综合征、确定骨髓肿瘤中 TET2 的体细胞突变、检测剪接机制中的常见通路突变以及发现克隆造血。目前,MDS 的体细胞基因组图谱已经明确。反复发生突变的基因包括表观遗传调控因子、RNA剪接机制基因、转录调控基因、DNA修复调控基因、粘合素复合物基因和信号转导基因。此外,还发现了几种具有MDS种系遗传易感性的疾病,它们合起来占所有MDS病例的15%。基因组图谱分析可大大改进 MDS 的诊断方法,从而确定不同的病理实体,如 SF3B1 突变或 TP53 突变 MDS。事实证明,MDS 分子国际预后评分系统(IPSS-M)是个体化风险评估和治疗决策的重要工具。此外,最近开发的 MDS 分子分类法可能会促进针对这些疾病的精准医疗方法的实施。这将需要在公共卫生系统内建立专门的基础设施,涉及医疗机构、学术界和生命科学行业之间的密切合作。
{"title":"Genome sequencing in the management of myelodysplastic syndromes and related disorders.","authors":"Mario Cazzola,Luca Malcovati","doi":"10.3324/haematol.2023.284947","DOIUrl":"https://doi.org/10.3324/haematol.2023.284947","url":null,"abstract":"Myeloid neoplasms originate from the clonal proliferation of hematopoietic stem cells, which is driven by the acquisition of somatic genetic mutations. Within these disorders, myelodysplastic syndromes (MDS) are specifically characterized by morphologic abnormalities (dysplasia) and impaired maturation of myeloid precursors (ineffective hematopoiesis), resulting in peripheral blood cytopenia. Several studies have advanced the field of MDS, with a few landmark papers leading to a paradigm shift, opening new avenues of research and enabling a molecular revolution. These seminal papers include the first description of the 5q- syndrome, the identification of somatic mutations of TET2 in myeloid neoplasms, the detection of common pathway mutations in the splicing machinery, and the discovery of clonal hematopoiesis. The somatic genomic landscape of MDS is now well-defined. Genes that are recurrently mutated include epigenetic regulators, as well as genes of RNA splicing machinery, transcription regulation, DNA repair control, cohesin complex, and signal transduction. Furthermore, several disorders with a germline genetic predisposition to MDS have been identified, collectively accounting for up to 15% of all MDS cases. Genomic profiling can significantly improve the diagnostic approach to MDS, allowing the identification of distinct nosologic entities such as SF3B1-mutant or TP53-mutant MDS. The Molecular International Prognostic Scoring System for MDS (IPSS-M) has already proven to be a valuable tool for individualized risk assessment and treatment decisions. In addition, the recently developed molecular taxonomy of MDS will likely facilitate the implementation of precision medicine approaches for these disorders. This will necessitate the establishment of specialized infrastructures within public health systems, involving close collaboration between healthcare institutions, academia, and the life sciences industry.","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":"5 1","pages":""},"PeriodicalIF":10.1,"publicationDate":"2024-10-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142489796","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"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
期刊
Haematologica
全部 Acc. Chem. Res. ACS Applied Bio Materials ACS Appl. Electron. Mater. ACS Appl. Energy Mater. ACS Appl. Mater. Interfaces ACS Appl. Nano Mater. ACS Appl. Polym. Mater. ACS BIOMATER-SCI ENG ACS Catal. ACS Cent. Sci. ACS Chem. Biol. ACS Chemical Health & Safety ACS Chem. Neurosci. ACS Comb. Sci. ACS Earth Space Chem. ACS Energy Lett. ACS Infect. Dis. ACS Macro Lett. ACS Mater. Lett. ACS Med. Chem. Lett. ACS Nano ACS Omega ACS Photonics ACS Sens. ACS Sustainable Chem. Eng. ACS Synth. Biol. Anal. Chem. BIOCHEMISTRY-US Bioconjugate Chem. BIOMACROMOLECULES Chem. Res. Toxicol. Chem. Rev. Chem. Mater. CRYST GROWTH DES ENERG FUEL Environ. Sci. Technol. Environ. Sci. Technol. Lett. Eur. J. Inorg. Chem. IND ENG CHEM RES Inorg. Chem. J. Agric. Food. Chem. J. Chem. Eng. Data J. Chem. Educ. J. Chem. Inf. Model. J. Chem. Theory Comput. J. Med. Chem. J. Nat. Prod. J PROTEOME RES J. Am. Chem. Soc. LANGMUIR MACROMOLECULES Mol. Pharmaceutics Nano Lett. Org. Lett. ORG PROCESS RES DEV ORGANOMETALLICS J. Org. Chem. J. Phys. Chem. J. Phys. Chem. A J. Phys. Chem. B J. Phys. Chem. C J. Phys. Chem. Lett. Analyst Anal. Methods Biomater. Sci. Catal. Sci. Technol. Chem. Commun. Chem. Soc. Rev. CHEM EDUC RES PRACT CRYSTENGCOMM Dalton Trans. Energy Environ. Sci. ENVIRON SCI-NANO ENVIRON SCI-PROC IMP ENVIRON SCI-WAT RES Faraday Discuss. Food Funct. Green Chem. Inorg. Chem. Front. Integr. Biol. J. Anal. At. Spectrom. J. Mater. Chem. A J. Mater. Chem. B J. Mater. Chem. C Lab Chip Mater. Chem. Front. Mater. Horiz. MEDCHEMCOMM Metallomics Mol. Biosyst. Mol. Syst. Des. Eng. Nanoscale Nanoscale Horiz. Nat. Prod. Rep. New J. Chem. Org. Biomol. Chem. Org. Chem. Front. PHOTOCH PHOTOBIO SCI PCCP Polym. Chem.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1