Pub Date : 2024-08-01DOI: 10.3324/haematol.2022.282363
Emmanuelle Tchernonog, Aline Moignet, Amélie Anota, Sophie Bernard, Guy Bouguet, Fanny Colin, Catherine Rioufol, Loïc Ysebaert, Emmanuel Gyan
Chimeric antigen receptor (CAR) T-cell therapy has transformed the care of patients with relapsed/refractory B-cell-derived hematologic malignancies. To date, six CAR T-cell therapies, targeting either CD19 or B-cell maturation antigen, have received regulatory approval. Along with the promising survival benefit, CAR T-cell therapy is associated with potentially life-threatening adverse events, including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. While clinical trials evaluating CAR T-cell therapy consistently report the incidence of these adverse events, most trials do not collect health-related quality of life (HRQoL) data. As such, the impact of the CAR T-cell therapy process and related adverse events on the physical and psychological well-being of patients remains uncertain. HRQoL and other patient-reported outcome (PRO) assessments in patients with relapsed or refractory hematologic malignancies are of utmost importance, as individuals may have unmet needs and a high demand for tolerable therapy if a cure is not obtained. In addition, it is important to standardize methods of data collection to better assess the impact of CAR T-cell therapy on quality of life, optimize patients' care and costs, and enable comparisons between different studies. We conducted a literature search up to June 2023 to identify the HRQoL tools used in clinical trials and in real-world studies investigating CAR T-cell therapy in patients with lymphomas or leukemias. In the present comprehensive review, we summarize the most commonly used CAR T-cell specific and non-specific HRQoL tools and discuss how the use of HRQoL and other PRO tools may be optimized.
嵌合抗原受体(CAR)T 细胞疗法改变了对复发/难治性 B 细胞血液恶性肿瘤患者的治疗。迄今为止,已有六种以 CD19 或 B 细胞成熟抗原为靶点的 CAR T 细胞疗法获得了监管部门的批准。CAR T 细胞疗法在给患者带来生存益处的同时,也可能带来危及生命的不良反应(AE),包括细胞因子释放综合征和免疫效应细胞相关神经毒性综合征。尽管评估 CAR T 细胞疗法的临床试验一直在报告这些不良事件的发生率,但大多数试验并未收集与健康相关的生活质量(HRQoL)数据。因此,CAR T 细胞疗法过程和相关 AE 对患者身心健康的影响仍不确定。复发或难治性血液恶性肿瘤患者的 HRQoL 和其他患者报告结果 (PRO) 评估至关重要,因为如果不能治愈,患者可能有未满足的需求,并对可耐受的治疗有很高的要求。此外,为了更好地评估 CAR T 细胞疗法对生活质量的影响、优化患者护理和成本,并对不同研究进行比较,规范数据收集方法非常重要。我们对截至 2023 年 6 月的文献进行了检索,以确定在临床试验中以及在调查淋巴瘤或白血病患者 CAR T 细胞疗法的真实世界研究中使用的 HRQoL 工具。在本综述中,我们总结了最常用的 CAR T 细胞特异性和非特异性 HRQoL 工具,并讨论了如何优化 HRQoL 和其他 PRO 工具的使用。
{"title":"Health-related quality of life in patients with hematologic malignancies treated with chimeric antigen receptor T-cell therapy: review and current progress.","authors":"Emmanuelle Tchernonog, Aline Moignet, Amélie Anota, Sophie Bernard, Guy Bouguet, Fanny Colin, Catherine Rioufol, Loïc Ysebaert, Emmanuel Gyan","doi":"10.3324/haematol.2022.282363","DOIUrl":"10.3324/haematol.2022.282363","url":null,"abstract":"<p><p>Chimeric antigen receptor (CAR) T-cell therapy has transformed the care of patients with relapsed/refractory B-cell-derived hematologic malignancies. To date, six CAR T-cell therapies, targeting either CD19 or B-cell maturation antigen, have received regulatory approval. Along with the promising survival benefit, CAR T-cell therapy is associated with potentially life-threatening adverse events, including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. While clinical trials evaluating CAR T-cell therapy consistently report the incidence of these adverse events, most trials do not collect health-related quality of life (HRQoL) data. As such, the impact of the CAR T-cell therapy process and related adverse events on the physical and psychological well-being of patients remains uncertain. HRQoL and other patient-reported outcome (PRO) assessments in patients with relapsed or refractory hematologic malignancies are of utmost importance, as individuals may have unmet needs and a high demand for tolerable therapy if a cure is not obtained. In addition, it is important to standardize methods of data collection to better assess the impact of CAR T-cell therapy on quality of life, optimize patients' care and costs, and enable comparisons between different studies. We conducted a literature search up to June 2023 to identify the HRQoL tools used in clinical trials and in real-world studies investigating CAR T-cell therapy in patients with lymphomas or leukemias. In the present comprehensive review, we summarize the most commonly used CAR T-cell specific and non-specific HRQoL tools and discuss how the use of HRQoL and other PRO tools may be optimized.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":null,"pages":null},"PeriodicalIF":8.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11290540/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140049329","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}
Pub Date : 2024-08-01DOI: 10.3324/haematol.2023.284520
Laurent Garderet, Luuk Gras, Linda Koster, Liesbeth De Wreede, Rovira Montserrat, Laure Vincent, Roland Fenk, Kamaraj Karunanithi, Dries Deeren, Martin Kaufmann, Jürgen Kuball, Hakan Ozdogu, Maria Jesus Pascual Cascon, Jakob Passweg, Adam Rye, Urpu Salmenniemi, John Snowden, Charlotte Toftmann Hansen, Xavier Leleu, Lauris Gastaud, Joanna Drozd Sokolowska, Kavita Raj, Meral Beksac, Stefan Schönland, Patrick Hayden, Donal McLornan
There is little long-term outcome data on the efficacy of autologous hematopoietic stem cell transplantation (ASCT) in light chain deposition disease (LCDD). We identified 51 LCDD patients in the European Society for Blood and Bone Marrow transplantation registry who had undergone upfront ASCT between 1995 and 2021. The median serum creatinine was 280 μmol/L and 45% required renal replacement therapy (RRT) at time of transplant. The melphalan dose was 100 mg/m2 in 23%, 140 mg/m2 in 55% and 200 mg/m2 in 21%. The rate of very good partial response or better improved from 41% pretransplant to 66% at day +100 post- ASCT. In RRT-independent patients, there was a modest improvement in renal function within the first 3 months; the median estimated glomerular filtration rate increased from 44 to 51 mL/min/1.73 m2. There was no further change between 3 and 12 months post-ASCT. No patient who was RRT-independent at ASCT became RRT dependent by day + 100 post-ASCT. Median follow- up post-ASCT was 84 months (interquartile range [IQR]: 46-122). At 6-years post ASCT, overall survival was 88% (95% confidence interval [CI]: 78-98) and PFS was 44% (95% CI: 28-60). The 2-year cumulative incidence of relapse and non-relapse mortality was 17% (95% CI: 6-27) and 2% (95% CI: 0-6), respectively. The cumulative incidence of renal transplantation at 4 years after ASCT was 27% (95% CI: 13-41) with renal transplantation performed between 6.3 and 52.9 months post-ASCT (median 24.7 months). ASCT represents a feasible option for LCDD patients even if RRT dependent at time of transplant. Outcomes are favorable with low non-relapse mortality and good long-term overall survival.
{"title":"Long-term outcomes and renal responses following autologous hematopoietic stem cell transplantation for light chain deposition disease: a retrospective study on behalf of the Chronic Malignancies Working Party of the European Society for Blood and Marrow Transplantation.","authors":"Laurent Garderet, Luuk Gras, Linda Koster, Liesbeth De Wreede, Rovira Montserrat, Laure Vincent, Roland Fenk, Kamaraj Karunanithi, Dries Deeren, Martin Kaufmann, Jürgen Kuball, Hakan Ozdogu, Maria Jesus Pascual Cascon, Jakob Passweg, Adam Rye, Urpu Salmenniemi, John Snowden, Charlotte Toftmann Hansen, Xavier Leleu, Lauris Gastaud, Joanna Drozd Sokolowska, Kavita Raj, Meral Beksac, Stefan Schönland, Patrick Hayden, Donal McLornan","doi":"10.3324/haematol.2023.284520","DOIUrl":"10.3324/haematol.2023.284520","url":null,"abstract":"<p><p>There is little long-term outcome data on the efficacy of autologous hematopoietic stem cell transplantation (ASCT) in light chain deposition disease (LCDD). We identified 51 LCDD patients in the European Society for Blood and Bone Marrow transplantation registry who had undergone upfront ASCT between 1995 and 2021. The median serum creatinine was 280 μmol/L and 45% required renal replacement therapy (RRT) at time of transplant. The melphalan dose was 100 mg/m2 in 23%, 140 mg/m2 in 55% and 200 mg/m2 in 21%. The rate of very good partial response or better improved from 41% pretransplant to 66% at day +100 post- ASCT. In RRT-independent patients, there was a modest improvement in renal function within the first 3 months; the median estimated glomerular filtration rate increased from 44 to 51 mL/min/1.73 m2. There was no further change between 3 and 12 months post-ASCT. No patient who was RRT-independent at ASCT became RRT dependent by day + 100 post-ASCT. Median follow- up post-ASCT was 84 months (interquartile range [IQR]: 46-122). At 6-years post ASCT, overall survival was 88% (95% confidence interval [CI]: 78-98) and PFS was 44% (95% CI: 28-60). The 2-year cumulative incidence of relapse and non-relapse mortality was 17% (95% CI: 6-27) and 2% (95% CI: 0-6), respectively. The cumulative incidence of renal transplantation at 4 years after ASCT was 27% (95% CI: 13-41) with renal transplantation performed between 6.3 and 52.9 months post-ASCT (median 24.7 months). ASCT represents a feasible option for LCDD patients even if RRT dependent at time of transplant. Outcomes are favorable with low non-relapse mortality and good long-term overall survival.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":null,"pages":null},"PeriodicalIF":8.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11290545/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140305426","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}
Pub Date : 2024-08-01DOI: 10.3324/haematol.2023.284121
Wenqian Xu, Feng Tian, Xiaolu Tai, Gaoxian Song, Yuanfang Liu, Liquan Fan, Xiangqin Weng, Eunjeong Yang, Meng Wang, Martin Bornhäuser, Chao Zhang, Richard B Lock, Jason W H Wong, Jin Wang, Duohui Jing, Jian-Qing Mi
ETV6::ACSL6 represents a rare genetic aberration in hematopoietic neoplasms and is often associated with severe eosinophilia, which confers an unfavorable prognosis requiring additional anti-inflammatory treatment. However, since the translocation is unlikely to produce a fusion protein, the mechanism of ETV6::ACSL6 action remains unclear. Here, we performed multi-omics analyses of primary leukemia cells and patient-derived xenografts from an acute lymphoblastic leukemia (ALL) patient with ETV6::ACSL6 translocation. We identified a super-enhancer located within the ETV6 gene locus, and revealed translocation and activation of the super-enhancer associated with the ETV6::ACSL6 fusion. The translocated super-enhancer exhibited intense interactions with genomic regions adjacent to and distal from the breakpoint at chromosomes 5 and 12, including genes coding inflammatory factors such as IL-3. This led to modulations in DNA methylation, histone modifications, and chromatin structures, triggering transcription of inflammatory factors leading to eosinophilia. Furthermore, the bromodomain and extraterminal domain (BET) inhibitor synergized with standard-of-care drugs for ALL, effectively reducing IL-3 expression and inhibiting ETV6::ACSL6 ALL growth in vitro and in vivo. Overall, our study revealed for the first time a cis-regulatory mechanism of super-enhancer translocation in ETV6::ACSL6ALL, leading to an ALL-accompanying clinical syndrome. These findings may stimulate novel treatment approaches for this challenging ALL subtype.
ETV6::ACSL6是造血肿瘤中一种罕见的基因畸变,常伴有严重的嗜酸性粒细胞增多,预后不良,需要额外的抗炎治疗。然而,由于易位不太可能产生融合蛋白,ETV6::ACSL6 的作用机制仍不清楚。在这里,我们对一名患有ETV6::ACSL6易位的急性淋巴细胞白血病(ALL)患者的原代白血病细胞和患者衍生异种移植物进行了多组学分析。我们确定了位于 ETV6 基因座内的超级增强子,并发现了与 ETV6::ACSL6 融合相关的超级增强子的易位和激活。易位的超级增强子与 5 号和 12 号染色体断点邻近和远端的基因组区域(包括编码 IL-3 等炎症因子的基因)发生了强烈的相互作用。这导致了 DNA 甲基化、组蛋白修饰和染色质结构的改变,引发了炎症因子的转录,导致嗜酸性粒细胞增多。此外,溴化多聚酶域和髓外结构域(BET)抑制剂与治疗ALL的标准药物协同作用,有效降低了IL-3的表达,抑制了ETV6::ACSL6 ALL在体外和体内的生长。总之,我们的研究首次揭示了ETV6::ACSL6 ALL中超级增强子易位的顺式调控机制,从而导致ALL伴随临床综合征。这些发现可能会激发针对这种具有挑战性的 ALL 亚型的新型治疗方法。
{"title":"<i>ETV6::ACSL6</i> translocation-driven super-enhancer activation leads to eosinophilia in acute lymphoblastic leukemia through IL-3 overexpression.","authors":"Wenqian Xu, Feng Tian, Xiaolu Tai, Gaoxian Song, Yuanfang Liu, Liquan Fan, Xiangqin Weng, Eunjeong Yang, Meng Wang, Martin Bornhäuser, Chao Zhang, Richard B Lock, Jason W H Wong, Jin Wang, Duohui Jing, Jian-Qing Mi","doi":"10.3324/haematol.2023.284121","DOIUrl":"10.3324/haematol.2023.284121","url":null,"abstract":"<p><p>ETV6::ACSL6 represents a rare genetic aberration in hematopoietic neoplasms and is often associated with severe eosinophilia, which confers an unfavorable prognosis requiring additional anti-inflammatory treatment. However, since the translocation is unlikely to produce a fusion protein, the mechanism of ETV6::ACSL6 action remains unclear. Here, we performed multi-omics analyses of primary leukemia cells and patient-derived xenografts from an acute lymphoblastic leukemia (ALL) patient with ETV6::ACSL6 translocation. We identified a super-enhancer located within the ETV6 gene locus, and revealed translocation and activation of the super-enhancer associated with the ETV6::ACSL6 fusion. The translocated super-enhancer exhibited intense interactions with genomic regions adjacent to and distal from the breakpoint at chromosomes 5 and 12, including genes coding inflammatory factors such as IL-3. This led to modulations in DNA methylation, histone modifications, and chromatin structures, triggering transcription of inflammatory factors leading to eosinophilia. Furthermore, the bromodomain and extraterminal domain (BET) inhibitor synergized with standard-of-care drugs for ALL, effectively reducing IL-3 expression and inhibiting ETV6::ACSL6 ALL growth in vitro and in vivo. Overall, our study revealed for the first time a cis-regulatory mechanism of super-enhancer translocation in ETV6::ACSL6ALL, leading to an ALL-accompanying clinical syndrome. These findings may stimulate novel treatment approaches for this challenging ALL subtype.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":null,"pages":null},"PeriodicalIF":8.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11290521/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139735037","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}
Pub Date : 2024-08-01DOI: 10.3324/haematol.2023.283597
Kathrin Hammon, Kathrin Renner, Michael Althammer, Florian Voll, Nathalie Babl, Sonja-Maria Decking, Peter J Siska, Carina Matos, Zugey Elizabeth Cárdenas Conejo, Karina Mendes, Friederike Einwag, Heiko Siegmund, Sabine Iberl, Raffaela S Berger, Katja Dettmer, Rebecca Schoenmehl, Christoph Brochhausen, Wolfgang Herr, Peter J Oefner, Michael Rehli, Simone Thomas, Marina Kreutz
D-2-hydroxyglutarate (D-2-HG) accumulates in patients with acute myeloid leukemia (AML) with mutated isocitrate dehydrogenase (IDH) and in other malignancies. D-2-HG suppresses antitumor T-cell immunity but little is known about potential effects on non-malignant myeloid cells. Here we show that D-2-HG impairs human but not murine dendritic cell differentiation, resulting in a tolerogenic phenotype with low major histocompatibility class II expression. In line with this, IDH-mutated AML blasts exhibited lower expression of HLA-DP and were less susceptible to lysis by HLA-DP-specific T cells. Interestingly, besides its expected impact on DNA demethylation, D-2-HG reprogrammed metabolism towards increased lactate production in dendritic cells and AML. Vitamin C accelerated DNA demethylation, but only the combination of vitamin C and glycolytic inhibition lowered lactate levels and supported major histocompatibility complex class II expression. Our results indicate an unexpected link between the immunosuppressive metabolites 2-HG and lactic acid and suggest a potentially novel therapeutic strategy with combinations of anti-glycolytic drugs and epigenetic modulators (hypomethylating agents) or other therapeutics for the treatment of AML.
在异柠檬酸脱氢酶(IDH)突变的原发性急性髓性白血病(AML)患者和其他恶性肿瘤患者体内,D-2-羟基戊二酸(D-2-HG)会累积。D-2-HG 可抑制抗肿瘤 T 细胞免疫,但对非恶性髓系细胞的潜在影响却知之甚少。我们在这里发现,D-2-HG 会影响人类树突状细胞(DC)的分化,但不会影响鼠类树突状细胞的分化,从而导致主要组织相容性(MHC)II 类低表达的耐受表型。同样,IDH突变的急性髓细胞白细胞表现出较低的HLA-DP表达,并且不易被HLA-DP特异性T细胞溶解。有趣的是,D-2-HG 除了对 DNA 去甲基化产生预期的影响外,还对 DCs 和 AML 的新陈代谢进行了重编程,使乳酸生成增加。维生素 C 加速了 DNA 的去甲基化,但只有维生素 C 和糖酵解抑制的结合才能降低乳酸水平并支持 MHC II 类表达。我们的研究结果表明,免疫抑制代谢物2-HG与乳酸之间存在着意想不到的联系,并提出了一种潜在的新型治疗策略,即结合使用抗糖酵解药物和表观遗传调节剂(低甲基化药物)或其他治疗药物来治疗急性髓细胞白血病。
{"title":"D-2-hydroxyglutarate supports a tolerogenic phenotype with lowered major histocompatibility class II expression in non-malignant dendritic cells and acute myeloid leukemia cells.","authors":"Kathrin Hammon, Kathrin Renner, Michael Althammer, Florian Voll, Nathalie Babl, Sonja-Maria Decking, Peter J Siska, Carina Matos, Zugey Elizabeth Cárdenas Conejo, Karina Mendes, Friederike Einwag, Heiko Siegmund, Sabine Iberl, Raffaela S Berger, Katja Dettmer, Rebecca Schoenmehl, Christoph Brochhausen, Wolfgang Herr, Peter J Oefner, Michael Rehli, Simone Thomas, Marina Kreutz","doi":"10.3324/haematol.2023.283597","DOIUrl":"10.3324/haematol.2023.283597","url":null,"abstract":"<p><p>D-2-hydroxyglutarate (D-2-HG) accumulates in patients with acute myeloid leukemia (AML) with mutated isocitrate dehydrogenase (IDH) and in other malignancies. D-2-HG suppresses antitumor T-cell immunity but little is known about potential effects on non-malignant myeloid cells. Here we show that D-2-HG impairs human but not murine dendritic cell differentiation, resulting in a tolerogenic phenotype with low major histocompatibility class II expression. In line with this, IDH-mutated AML blasts exhibited lower expression of HLA-DP and were less susceptible to lysis by HLA-DP-specific T cells. Interestingly, besides its expected impact on DNA demethylation, D-2-HG reprogrammed metabolism towards increased lactate production in dendritic cells and AML. Vitamin C accelerated DNA demethylation, but only the combination of vitamin C and glycolytic inhibition lowered lactate levels and supported major histocompatibility complex class II expression. Our results indicate an unexpected link between the immunosuppressive metabolites 2-HG and lactic acid and suggest a potentially novel therapeutic strategy with combinations of anti-glycolytic drugs and epigenetic modulators (hypomethylating agents) or other therapeutics for the treatment of AML.</p>","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":null,"pages":null},"PeriodicalIF":8.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11290548/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139485431","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}
Pub Date : 2024-08-01DOI: 10.3324/haematol.2023.284658
Léa Boulnois, Margot Robles, Nada Maaziz, Bernard Aral, Martin Gauthier, Francis Duchene, Marie-Amélie Goujart, Betty Gardie, François Girodon
{"title":"Benefit of phlebotomy and low-dose aspirin in the prevention of vascular events in patients with <i>EPOR</i> primary familial polycythemia on the island of New Caledonia.","authors":"Léa Boulnois, Margot Robles, Nada Maaziz, Bernard Aral, Martin Gauthier, Francis Duchene, Marie-Amélie Goujart, Betty Gardie, François Girodon","doi":"10.3324/haematol.2023.284658","DOIUrl":"10.3324/haematol.2023.284658","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":null,"pages":null},"PeriodicalIF":8.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11290533/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140305419","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}
Pub Date : 2024-08-01DOI: 10.3324/haematol.2024.284962
Nickolas Steinauer, Kristen McCullough, Aref Al-Kali, Hassan B Alkhateeb, Kebede H Begna, Abhishek A Mangaonkar, Antoine N Saliba, Mehrdad Torghabeh, Mark R Litzow, William J Hogan, Mithun Shah, Mrinal M Patnaik, Animesh Pardanani, Talha Badar, Hemant Murthy, James Foran, Cecilia Arana Yi, Ayalew Tefferi, Naseema Gangat
{"title":"Cladribine plus cytarabine plus venetoclax in acute myeloid leukemia relapsed or refractory to venetoclax plus hypomethylating agent.","authors":"Nickolas Steinauer, Kristen McCullough, Aref Al-Kali, Hassan B Alkhateeb, Kebede H Begna, Abhishek A Mangaonkar, Antoine N Saliba, Mehrdad Torghabeh, Mark R Litzow, William J Hogan, Mithun Shah, Mrinal M Patnaik, Animesh Pardanani, Talha Badar, Hemant Murthy, James Foran, Cecilia Arana Yi, Ayalew Tefferi, Naseema Gangat","doi":"10.3324/haematol.2024.284962","DOIUrl":"10.3324/haematol.2024.284962","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":null,"pages":null},"PeriodicalIF":8.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11290516/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140305420","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}
Pub Date : 2024-08-01DOI: 10.3324/haematol.2024.285083
Armin P Piehler, Marietta Truger, Jan-Hendrik Kozik, Sandra Weissmann, Martin Schwonzen, Manja Meggendorfer, Wolfgang Kern, Torsten Haferlach, Gregor Hoermann, Claudia Haferlach
{"title":"Classical meets malignant hematology: a case of acquired εγδβ-thalassemia in clonal hematopoiesis.","authors":"Armin P Piehler, Marietta Truger, Jan-Hendrik Kozik, Sandra Weissmann, Martin Schwonzen, Manja Meggendorfer, Wolfgang Kern, Torsten Haferlach, Gregor Hoermann, Claudia Haferlach","doi":"10.3324/haematol.2024.285083","DOIUrl":"10.3324/haematol.2024.285083","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":null,"pages":null},"PeriodicalIF":8.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11290575/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140143266","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}
Pub Date : 2024-08-01DOI: 10.3324/haematol.2024.285845
Zachary D Epstein-Peterson, Esther Drill, Umut Aypar, Connie Lee Batlevi, Philip Caron, Ahmet Dogan, Pamela Drullinsky, John Gerecitano, Paul A Hamlin, Caleb Ho, Allison Jacob, Ashlee Joseph, Leana Laraque, Matthew J Matasar, Alison J Moskowitz, Craig H Moskowitz, Chelsea Mullins, Colette Owens, Gilles Salles, Heiko Schöder, David J Straus, Anas Younes, Andrew D Zelenetz, Anita Kumar
{"title":"<i>Erratum</i> to: Immunochemotherapy plus lenalidomide for high-risk mantle cell lymphoma with measurable residual disease evaluation.","authors":"Zachary D Epstein-Peterson, Esther Drill, Umut Aypar, Connie Lee Batlevi, Philip Caron, Ahmet Dogan, Pamela Drullinsky, John Gerecitano, Paul A Hamlin, Caleb Ho, Allison Jacob, Ashlee Joseph, Leana Laraque, Matthew J Matasar, Alison J Moskowitz, Craig H Moskowitz, Chelsea Mullins, Colette Owens, Gilles Salles, Heiko Schöder, David J Straus, Anas Younes, Andrew D Zelenetz, Anita Kumar","doi":"10.3324/haematol.2024.285845","DOIUrl":"10.3324/haematol.2024.285845","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":null,"pages":null},"PeriodicalIF":8.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11290576/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141859565","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}
Pub Date : 2024-08-01DOI: 10.3324/haematol.2024.284976
Do Hwan Kim, Sa A Wang, Wei Wang, Guilin Tang, Shaoying Li, C Cameron Yin, Pei Lin, Marina Konopleva, M James You, Roberto N Miranda, Xiaoqiong Wang, Qing Wei, L Jeffrey Medeiros, Jie Xu
{"title":"Acute myeloid leukemia with mast cell differentiation is characterized by interstitial mast cells, complex karyotype, <i>TP53</i> alterations and poor prognosis.","authors":"Do Hwan Kim, Sa A Wang, Wei Wang, Guilin Tang, Shaoying Li, C Cameron Yin, Pei Lin, Marina Konopleva, M James You, Roberto N Miranda, Xiaoqiong Wang, Qing Wei, L Jeffrey Medeiros, Jie Xu","doi":"10.3324/haematol.2024.284976","DOIUrl":"10.3324/haematol.2024.284976","url":null,"abstract":"","PeriodicalId":12964,"journal":{"name":"Haematologica","volume":null,"pages":null},"PeriodicalIF":8.2,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11290508/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140305417","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}