Acta Haematologica最新文献_第4页

Biologic and Clinical Characteristics of Isochromosome der(17)(q10)t(15;17) in Acute Promyelocytic Leukemia. 急性早幼粒细胞白血病中同源染色体 der(17)(q10)t(15;17) 的生物学和临床特征。

IF 1.7 4区医学

Acta Haematologica Pub Date : 2024-05-31 DOI: 10.1159/000539159

Yuchen Liu, Yi Ning, Gabriel Ghiaur, Ashkan Emadi

引用次数: 0

TES and SLC40A1 as Potential Biomarkers for Predicting Survival in T-Cell Acute Lymphoblastic Leukemia. TES和SLC40A1是预测T细胞急性淋巴细胞白血病存活率的潜在生物标记物。

IF 1.7 4区医学

Acta Haematologica Pub Date : 2024-05-28 DOI: 10.1159/000539435

Xiangyou Zeng, Kaifan Liu, Ruohao Xu, Lenghe Zhang, Peilong Lai, Xin Du, Jianyu Weng

{"title":"TES and SLC40A1 as Potential Biomarkers for Predicting Survival in T-Cell Acute Lymphoblastic Leukemia.","authors":"Xiangyou Zeng, Kaifan Liu, Ruohao Xu, Lenghe Zhang, Peilong Lai, Xin Du, Jianyu Weng","doi":"10.1159/000539435","DOIUrl":"10.1159/000539435","url":null,"abstract":"Introduction: Identifying patients with high-risk T-cell acute lymphoblastic leukemia (T-ALL) is crucial for personalized therapy; however, the lack of robust biomarkers hinders prognosis assessment. To address this issue, our study aimed to screen and validate genes whose expression may serve as predictive indicators of outcomes in T-ALL patients while also investigating the underlying molecular mechanisms.Methods: Differentially expressed genes (DEGs) between T-ALL patients and healthy controls were identified by integrating data from three independent public datasets. Functional annotation of these DEGs and protein-protein interactions were also conducted. Further, we enrolled a prospective cohort of T-ALL patients (n = 20) at our center, conducting RNA-seq analysis on their bone marrow samples. Survival-based univariate Cox analysis was employed to identify gene expressions related to survival, and an intersection algorithm was sequentially applied. Furthermore, we validated the identified genes using cases from the Therapeutically Applicable Research to Generate Effective Treatments database, plotting Kaplan-Meier curves for secondary validation.Results: Through the integration of survival-related genes with DEGs identified in T-ALL, our analysis revealed six T-ALL-specific genes, the expression levels of which were linked to prognostic value. Notably, the independent prognostic value of SLC40A1 and TES expression levels was confirmed in both an external cohort and a prospective cohort at our center.Conclusion: In summary, our preliminary study indicates that the expression levels of TES and SLC40A1 genes show promise as potential indicators for predicting survival outcomes in T-ALL patients.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-17"},"PeriodicalIF":1.7,"publicationDate":"2024-05-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141160096","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Eosinophilic Pleocytosis in the Cerebrospinal Fluid following CAR-T Cell Therapy for Central Nervous System Lymphoma: A Case for Warning? 中枢神经系统淋巴瘤 CAR-T 细胞疗法后脑脊液中出现嗜酸性粒细胞增多：需要警惕吗？

IF 1.7 4区医学

Acta Haematologica Pub Date : 2024-05-24 DOI: 10.1159/000539354

Mayasa Abu Ata, Israel Henig, Dana Yehudai-Ofir, Inna Tzoran, Shimrit Ringelstein-Harlev, Tsofia Inbar, Ilana Slouzkey, Michal Karmona Fintuch, Anat Stern, Olesya Stanevsky, Michal Weiler-Sagie, Yaniv Zohar, Ido Livneh, Goni Merhav, Ayelet Eran, Tsila Zuckerman, Ofrat Beyar Katz

{"title":"Eosinophilic Pleocytosis in the Cerebrospinal Fluid following CAR-T Cell Therapy for Central Nervous System Lymphoma: A Case for Warning?","authors":"Mayasa Abu Ata, Israel Henig, Dana Yehudai-Ofir, Inna Tzoran, Shimrit Ringelstein-Harlev, Tsofia Inbar, Ilana Slouzkey, Michal Karmona Fintuch, Anat Stern, Olesya Stanevsky, Michal Weiler-Sagie, Yaniv Zohar, Ido Livneh, Goni Merhav, Ayelet Eran, Tsila Zuckerman, Ofrat Beyar Katz","doi":"10.1159/000539354","DOIUrl":"10.1159/000539354","url":null,"abstract":"Introduction: Chimeric antigen receptor T (CAR-T) cell therapy, emerging as an efficient treatment option for patients with secondary central nervous system (CNS) lymphoma, is frequently complicated with immune effector cell-associated neurotoxicity syndrome (ICANS).Case presentation: We report a case of a 64-year-old woman with transformed follicular lymphoma, developing high-grade ICANS with eosinophilic pleocytosis following third-line therapy with CAR-T cells (tisagenlecleucel). During bridging therapy, she declined neurologically and was diagnosed with secondary CNS lymphoma. She received methotrexate-cytarabine-thiotepa-rituximab regimen with clinical and radiological improvement. Post-CAR-T cell infusion she developed cytokine release syndrome grade II and ICANS grade III. Given the lack of response to steroids, anakinra was initiated with complete ICANS resolution. Cerebrospinal fluid (CSF) analysis, performed only on day +10 due to thrombocytopenia, revealed eosinophils, while infections were excluded.Conclusion: This report emphasizes the importance of CSF analysis in individuals with CAR-T-related neurotoxicity for elucidating the role of specific immune cells in such complications.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-8"},"PeriodicalIF":1.7,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141086471","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Novel Compound Heterogeneous Mutations in CYB5R3 Gene Leading to Methemoglobinemia (Type I) in a Chinese Boy: Case Report and Relevant Comprehensive Analysis. 一名中国男孩的 CYB5R3 基因发生新型复合异质突变，导致高铁血红蛋白血症（I 型）。

IF 1.7 4区医学

Acta Haematologica Pub Date : 2024-05-24 DOI: 10.1159/000539448

Yeyi Yang, Yezhen Yang, Ye Meng, Lihua Huang, Zuocheng Yang

{"title":"Novel Compound Heterogeneous Mutations in CYB5R3 Gene Leading to Methemoglobinemia (Type I) in a Chinese Boy: Case Report and Relevant Comprehensive Analysis.","authors":"Yeyi Yang, Yezhen Yang, Ye Meng, Lihua Huang, Zuocheng Yang","doi":"10.1159/000539448","DOIUrl":"10.1159/000539448","url":null,"abstract":"Introduction: Recessive congenital methemoglobinemia (RCM) caused by CYB5R3 deficiency due to the mutations in the reduced nicotinamide adenine dinucleotide (NADH) cytochrome b5 reductase (CYB5R) gene is an autosomal recessive inherited disease. Clinically, it can be divided into two types, namely red blood cell affected type (RCM I) and systemically affected type (RCM II).Case presentation: A 5-year-old male patient was diagnosed with cyanosis for 5 years. Physical examination showed cyanosis in areas such as the lips, fingers, and toes. Laboratory examination revealed low pulse oxygen saturation (81%) and increased blood methemoglobin (23.6%). Gene testing revealed the compound heterozygous mutations in the CYB5R3 gene, c.149G>A (p.Arg50Gln) and c.331A>G (p.Lys111Glu), respectively originating from his parents. By constructing 3D models of CYB5R3 wild-type and mutant types using SWISS-MODEL software, it was found that the mutation caused significant structural abnormalities in the CYB5R protein. The relationship between CYB5R3 gene mutation sites, amino acid change, enzyme activity, and methemoglobinemia type I and II were listed and analyzed.Conclusion: A case of congenital RCM type I caused by compound heterozygous mutations in the CYB5R3 gene was reported, with c.331A>G (p.Lys111Glu) being the newly reported mutation. The homozygosity or heterozygosity of CYB5R3 gene mutations that lead to premature termination, loss of exons, and change in amino acid properties in FAD or NADH binding domains, is positively correlated with the severity (from type I to type II) of methemoglobinemia.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-7"},"PeriodicalIF":1.7,"publicationDate":"2024-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141086453","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Single-Center Experience of Patients with Plasma Cell Leukemia in the Era of New Therapeutics. 新疗法时代浆细胞白血病患者的单中心经验。

IF 2.4 4区医学

Acta Haematologica Pub Date : 2024-05-22 DOI: 10.1159/000539223

Maria Dampmann, Sarah Flossdorf, Julius Keyl, Hans Christian Reinhardt, Christine Hanoun

{"title":"Single-Center Experience of Patients with Plasma Cell Leukemia in the Era of New Therapeutics.","authors":"Maria Dampmann, Sarah Flossdorf, Julius Keyl, Hans Christian Reinhardt, Christine Hanoun","doi":"10.1159/000539223","DOIUrl":"10.1159/000539223","url":null,"abstract":"Introduction: Plasma cell leukemia (PCL) can occur de novo as primary PCL (pPCL), or in patients with prior diagnosis of multiple myeloma (MM) as secondary PCL (sPCL). In 2021, the diagnostic criteria have been revised, establishing a new cut-off of ≥5% plasma cells in the peripheral blood. Lacking specific clinical trials, PCL is treated similarly to MM; however, outcome for patients with PCL remains poor. Here, we report outcomes for patients with pPCL and sPCL in the era of novel agents.Methods: We performed a retrospective analysis and identified 19 patients (11 pPCL, 8 sPCL) who have been treated for PCL between 2010 and 2022 at University Hospital Essen.Results: Patients with pPCL had a median overall survival (OS) of 37.8 months (95% CI: [15.4; 52.3] months) from diagnosis, with a median time to next treatment (TTNT) of 18.4 (2.0; 22.9) months. All patients were treated with a proteasome-inhibitor (PI)-based induction therapy, and the majority was consolidated with an autologous stem cell transplantation (SCT). Five of these patients received a tandem transplantation. Patients with sPCL had a median OS of only 1.5 months after diagnosis of PCL. Only 1 patient achieved a remission with daratumumab and subsequent allogenic SCT.Conclusion: With our study, we add evidence for a PI-based induction therapy followed by a consolidating autologous SCT for patients with pPCL and give further evidence that a tandem transplant concept might be beneficial. The diagnosis of sPCL remains devastating and needs new therapeutic approaches.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-8"},"PeriodicalIF":2.4,"publicationDate":"2024-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141064633","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Racial and Ethnic Characteristics and Outcomes of Patients Diagnosed with CLL/SLL in the USA. 美国确诊的 CLL/SLL 患者的种族和民族特征及治疗效果。

IF 1.7 4区医学

Acta Haematologica Pub Date : 2024-05-20 DOI: 10.1159/000538836

Debora S Bruno, Manoj Khanal, Xiaohong I Li, Maricer P Escalon, Katherine B Winfree, Lisa M Hess

{"title":"Racial and Ethnic Characteristics and Outcomes of Patients Diagnosed with CLL/SLL in the USA.","authors":"Debora S Bruno, Manoj Khanal, Xiaohong I Li, Maricer P Escalon, Katherine B Winfree, Lisa M Hess","doi":"10.1159/000538836","DOIUrl":"10.1159/000538836","url":null,"abstract":"Introduction: This study was designed to compare outcomes among patients by race and ethnicity in the post-covalent Bruton tyrosine kinase inhibitor (cBTKi) treatment era.Methods: A nationwide electronic health record (EHR)-derived de-identified database was utilized that included patients diagnosed with CLL from 2013 to 2022 who received systemic therapy for their disease. Use of cBTKi therapy, time to next treatment or death (TTNT-D), and overall survival (OS) were compared by race in unadjusted (Kaplan-Meier method) and adjusted analyses (Cox proportional hazards regression).Results: This study included 4,572 White (71.8%) and 558 Black (8.8%) patients with CLL; 270 were Hispanic or Latino (4.2%). Patients who were Black were significantly younger, more were female, had later stage disease, were of lower socioeconomic status (SES), and were more likely to have unmutated immunoglobulin heavy chain gene (IGHV) and to have received cBTKi therapy than White patients (all p ≤ 0.002). SES was also significantly different by ethnicity. TTNT-D and OS were not different by race in either unadjusted or adjusted analyses (all p > 0.05).Conclusion: In unadjusted and adjusted analyses, TTNT-D and OS were not different by race. These data did not identify racial healthcare disparities in the era following the introduction of cBTKi therapy despite differences in baseline characteristics.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-15"},"PeriodicalIF":1.7,"publicationDate":"2024-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141070244","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

HLA-DRB5 Overexpression Promotes Platelet Reduction in Immune Thrombocytopenia Mice Model by Facilitating MHC-II-Mediated Antigen Presentation. 通过促进 MHC-II 介导的抗原呈递，HLA-DRB5 的过表达可促进免疫性血小板减少症小鼠模型中血小板的减少。

IF 2.4 4区医学

Acta Haematologica Pub Date : 2024-05-15 DOI: 10.1159/000538749

Yujuan Ren, Qianqian Ying, Ying Chen, Cong Liao, Anrong Li, Qidong Ye

{"title":"HLA-DRB5 Overexpression Promotes Platelet Reduction in Immune Thrombocytopenia Mice Model by Facilitating MHC-II-Mediated Antigen Presentation.","authors":"Yujuan Ren, Qianqian Ying, Ying Chen, Cong Liao, Anrong Li, Qidong Ye","doi":"10.1159/000538749","DOIUrl":"10.1159/000538749","url":null,"abstract":"Introduction: Major histocompatibility complex II (MHC-II)-mediated antigen presentation contributes to the pathogenesis of immune thrombocytopenia (ITP). Human leukocyte antigen (HLA)-DRB5 is an MHC-II molecule and this study aims to investigate its role and mechanisms in ITP development.Methods: Guinea pig anti-mouse platelet (PLT) serum-induced ITP mice received tail vein injection of HLA-DRB5 overexpressing adenoviral vector/immune receptor expressed on myeloid cells-1 (IREM-1) monoclonal antibody (mAb). PLT count changes in mice blood were assessed by a hematology analyzer. MHC-II/CD80/CD86 expression in mice blood was measured by quantitative real-time-PCR and immunofluorescence assay. CD8+ T-cell proportion in mice blood was detected by flow cytometry.Results: HLA-DRB5 overexpression exacerbated PLT reduction since the 5th day of the establishment of ITP mice model and enhanced MHC-II/CD80/CD86 expression upregulation as well as CD8+ T-cell ratio elevation in the blood of ITP mice, while its effects were reversed by IREM-1 mAb.Conclusion: HLA-DRB5 overexpression upregulates MHC-II-mediated antigen presentation to CD8+ T cells, thus lowering PLT count in the ITP mice model.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-9"},"PeriodicalIF":2.4,"publicationDate":"2024-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140920761","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Real-World Risk of Severe Cytopenias in Multiple Myeloma Patients Sequentially Treated with Immunomodulatory Drugs. 多发性骨髓瘤患者接连接受免疫调节药物治疗后出现严重细胞减少症的现实风险。

IF 1.7 4区医学

Acta Haematologica Pub Date : 2024-05-10 DOI: 10.1159/000539127

Julie Barberio, Timothy L Lash, Ajay K Nooka, Ashley I Naimi, Rachel E Patzer, Christopher Kim

{"title":"Real-World Risk of Severe Cytopenias in Multiple Myeloma Patients Sequentially Treated with Immunomodulatory Drugs.","authors":"Julie Barberio, Timothy L Lash, Ajay K Nooka, Ashley I Naimi, Rachel E Patzer, Christopher Kim","doi":"10.1159/000539127","DOIUrl":"10.1159/000539127","url":null,"abstract":"Introduction: Most multiple myeloma (MM) patients experience cytopenias, likely driven by both disease and treatment-related factors. Immunomodulatory agents (IMiDs), which form the backbone of most anti-myeloma regimens, are known to cause higher grade cytopenias. In this context, the impact of sequential IMiD treatments on cytopenia risk is unknown.Methods: We evaluated the cumulative risks of severe cytopenias following second line of therapy (LOT) initiation in 5,573 MM patients in the Flatiron Health database. Patients for whom both LOTs 1 and 2 contained IMiDs were considered \"sequentially exposed\"; those for whom neither contained IMiDs were \"never exposed.\"Results: For the neutropenia outcome, compared to the never exposed, the sequentially exposed had the highest 1-year risk (risk difference [RD] 12%), followed by those only recently exposed during LOT 2 (RD 8%), then by those with only past exposure during LOT 1 (RD 5%). A similar pattern was observed for leukopenia, but no meaningful differences were observed for anemia or thrombocytopenia. The associations between sequential exposure, versus never, with neutropenia and leukopenia were even stronger among those with a recent cytopenia history.Conclusion: Results suggest that sequential exposure to IMiDs is a risk factor for higher grade cytopenias. These findings have profound clinical implications in choosing newer LOTs with potential risks of cytopenia.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1"},"PeriodicalIF":1.7,"publicationDate":"2024-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140911212","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Toxicity of CAR T-Cell Therapy for Multiple Myeloma. CAR T 细胞疗法治疗多发性骨髓瘤的毒性。

IF 2.4 4区医学

Acta Haematologica Pub Date : 2024-05-08 DOI: 10.1159/000539134

Aimaz Afrough, Pearl Rajan Abraham, Laura Turer, Gurbakhash Kaur, Aishwarya Sannareddy, Doris K Hansen, Larry D Anderson

{"title":"Toxicity of CAR T-Cell Therapy for Multiple Myeloma.","authors":"Aimaz Afrough, Pearl Rajan Abraham, Laura Turer, Gurbakhash Kaur, Aishwarya Sannareddy, Doris K Hansen, Larry D Anderson","doi":"10.1159/000539134","DOIUrl":"10.1159/000539134","url":null,"abstract":"Background: Idecabtagene vicleucel (ide-cel) and ciltacabtagene autoleucel (cilta-cel) are novel chimeric antigen receptor (CAR)-T cell therapies targeting B-cell maturation antigen (BCMA), and both have recently gained approval by the US Food Drug Administration (FDA) for the treatment of relapsed and refractory multiple myeloma (RRMM).Summary: These therapies offer unprecedented responses in RRMM but present new challenges including cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), non-ICANS neurotoxicity, cytopenias, infections, and hypogammaglobulinemia.Key messages: In the evolving CAR-T landscape, a primary objective is to develop innovative strategies for managing associated toxicities. Through meticulous exploration of underlying mechanisms and tailored interventions, we aim to enhance safety and enable broader outpatient utilization. Refinement of protocols, biomarker identification, and robust monitoring are imperative for sustained efficacy. This comprehensive approach guarantees the continuous advancement and optimization of CAR-T therapy.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-15"},"PeriodicalIF":2.4,"publicationDate":"2024-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140891042","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Historical Perspective of High-Dose Therapy Followed by Autologous Stem Cell Transplantation in Multiple Myeloma. 多发性骨髓瘤自体干细胞移植后大剂量治疗的历史展望

IF 2.4 4区医学

Acta Haematologica Pub Date : 2024-05-06 DOI: 10.1159/000539225

Inbar Cohen, Iuliana Vaxman, Morie A Gertz

{"title":"Historical Perspective of High-Dose Therapy Followed by Autologous Stem Cell Transplantation in Multiple Myeloma.","authors":"Inbar Cohen, Iuliana Vaxman, Morie A Gertz","doi":"10.1159/000539225","DOIUrl":"10.1159/000539225","url":null,"abstract":"Background: High-dose therapy (HDT) followed by autologous stem cell transplantation (ASCT) has become part of standard of care (SOC) in newly diagnosed multiple myeloma. In this review, we provide a historical perspective on ASCT since its introduction in the 1990s.Summary: Overall survival (OS) benefit for HDT followed by ASCT was demonstrated in studies comparing HDT with ASCT to standard-dose therapy (SDT) before the era of novel agents. Conditioning is done with melphalan 200 mg/m2. Lower doses (MEL140, MEL150) for older patients with comorbidities are safe and have comparable results. The addition of busulfan to melphalan improves progression-free survival (PFS) but not OS. HDT with ASCT after induction with novel agents prolongs PFS but not OS compared to SDT alone. The benefit is more evident in patients with high-risk cytogenetics. Mobilization can be achieved with granulocyte colony-stimulating factor alone, but is improved with the addition of chemotherapy. Plerixafor reduces mobilization failure and enables sufficient stem cell collection after induction with novel agents. ASCT is safe with a low rate of mortality (1%), and selected patients can be managed as outpatients.Key messages: HDT followed by ASCT remains part of SOC due to its PFS benefit and relatively low toxicity.","PeriodicalId":6981,"journal":{"name":"Acta Haematologica","volume":" ","pages":"1-10"},"PeriodicalIF":2.4,"publicationDate":"2024-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140846838","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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