Ana Alfonso-Pierola, David Martinez-Cuadrón, Rebeca Rodríguez-Veiga, Cristina Gil, Pilar Martínez-Sánchez, Teresa Bernal, Celina Benavente, Mónica A. Romero-Riquelme, Josefina Serrano-Lopez, Juan M. Bergua, Raimundo García-Boyero, Mar Tormo, Pilar Herrera, Claudia L. Sossa-Melo, José A. Pérez-Simon, Carlos Rodríguez-Medina, María F. Bass-Maturana, José L. López-Lorenzo, Lorenzo Algarra-Algarra, Belén Vidriales-Vicente, Manuel Pérez-Encinas, Manuel Barrios-García, Susana Vives, María J. Sayas-Lloris, Marisa Capurro, Sebastián Hidalgo, Mayte Olave, Diana Cuervo-Lozada, Esperanza Lavilla-Rubira, Felipe Casado, Armando Mena-Durán, Marta Valero-Nuñez, Soledad Casado-Calderón, Amaia Balerdi, Vivianne Torres, Rosa Fernández, Víctor Noriega, Mariana Stevenazzi, Jorge Labrador, Pilar León-Maldonado, Beatriz de Rueda-Ciller, Olga Arce-Fernández, María L. Amigo, José Ángel Raposo-Puglia, María Solé, Blanca Boluda, Rosa Ayala, Eva Barragán, Pau Montesinos
{"title":"自体干细胞移植与强化化疗巩固对急性髓性白血病患者的长期益处:来自PETHEMA AML登记的倾向评分匹配分析","authors":"Ana Alfonso-Pierola, David Martinez-Cuadrón, Rebeca Rodríguez-Veiga, Cristina Gil, Pilar Martínez-Sánchez, Teresa Bernal, Celina Benavente, Mónica A. Romero-Riquelme, Josefina Serrano-Lopez, Juan M. Bergua, Raimundo García-Boyero, Mar Tormo, Pilar Herrera, Claudia L. Sossa-Melo, José A. Pérez-Simon, Carlos Rodríguez-Medina, María F. Bass-Maturana, José L. López-Lorenzo, Lorenzo Algarra-Algarra, Belén Vidriales-Vicente, Manuel Pérez-Encinas, Manuel Barrios-García, Susana Vives, María J. Sayas-Lloris, Marisa Capurro, Sebastián Hidalgo, Mayte Olave, Diana Cuervo-Lozada, Esperanza Lavilla-Rubira, Felipe Casado, Armando Mena-Durán, Marta Valero-Nuñez, Soledad Casado-Calderón, Amaia Balerdi, Vivianne Torres, Rosa Fernández, Víctor Noriega, Mariana Stevenazzi, Jorge Labrador, Pilar León-Maldonado, Beatriz de Rueda-Ciller, Olga Arce-Fernández, María L. Amigo, José Ángel Raposo-Puglia, María Solé, Blanca Boluda, Rosa Ayala, Eva Barragán, Pau Montesinos","doi":"10.1038/s41375-025-02744-x","DOIUrl":null,"url":null,"abstract":"<p>While allogeneic stem cell transplantation (allo-SCT) is the preferred consolidation for high and most intermediate-risk acute myeloid leukemia (AML) patients in first remission, the role of autologous SCT (auto-SCT) vs. chemotherapy (CT) when allo-SCT is not feasible or indicated, remains controversial. We conducted a real-world, retrospective cohort study using the PETHEMA AML registry to compare auto-SCT and CT. Multivariate Cox regression and propensity score matching (PS-matching) were used to adjust for confounding factors. A total of 1272 patients in first remission and who received 2 consolidation courses were included (615 receiving additional CT cycles and 657 undergoing auto-SCT). Overall, 78.08% of auto-SCT patients were diagnosed before 2017, compared to 38.11% in the CT cohort (<i>p</i> < 0.001). In the overall cohort, auto-SCT was associated with significantly prolonged overall survival (OS) (HR: 0.73, <i>p</i> < 0.001) and relapse-free survival (RFS) (HR: 0.73, <i>p</i> < 0.001). This benefit was particularly evident in patients ≤65 years, those with normal karyotype, and FLT3-ITD negativity. In the PS-matched cohort, the RFS advantage persisted (HR: 0.80, <i>p</i> = 0.092), but OS differences were not statistically significant (HR: 0.91, <i>p</i> = 0.563). The role of auto-SCT in the genomic and targeted agent era should not be discarded.</p>","PeriodicalId":18109,"journal":{"name":"Leukemia","volume":"60 1","pages":""},"PeriodicalIF":13.4000,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Long-term benefits of autologous stem cell transplantation versus intensive chemotherapy consolidation for acute myeloid leukemia patients: A propensity score matching analysis from the PETHEMA AML registry\",\"authors\":\"Ana Alfonso-Pierola, David Martinez-Cuadrón, Rebeca Rodríguez-Veiga, Cristina Gil, Pilar Martínez-Sánchez, Teresa Bernal, Celina Benavente, Mónica A. Romero-Riquelme, Josefina Serrano-Lopez, Juan M. Bergua, Raimundo García-Boyero, Mar Tormo, Pilar Herrera, Claudia L. Sossa-Melo, José A. Pérez-Simon, Carlos Rodríguez-Medina, María F. Bass-Maturana, José L. 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We conducted a real-world, retrospective cohort study using the PETHEMA AML registry to compare auto-SCT and CT. Multivariate Cox regression and propensity score matching (PS-matching) were used to adjust for confounding factors. A total of 1272 patients in first remission and who received 2 consolidation courses were included (615 receiving additional CT cycles and 657 undergoing auto-SCT). Overall, 78.08% of auto-SCT patients were diagnosed before 2017, compared to 38.11% in the CT cohort (<i>p</i> < 0.001). In the overall cohort, auto-SCT was associated with significantly prolonged overall survival (OS) (HR: 0.73, <i>p</i> < 0.001) and relapse-free survival (RFS) (HR: 0.73, <i>p</i> < 0.001). This benefit was particularly evident in patients ≤65 years, those with normal karyotype, and FLT3-ITD negativity. In the PS-matched cohort, the RFS advantage persisted (HR: 0.80, <i>p</i> = 0.092), but OS differences were not statistically significant (HR: 0.91, <i>p</i> = 0.563). The role of auto-SCT in the genomic and targeted agent era should not be discarded.</p>\",\"PeriodicalId\":18109,\"journal\":{\"name\":\"Leukemia\",\"volume\":\"60 1\",\"pages\":\"\"},\"PeriodicalIF\":13.4000,\"publicationDate\":\"2025-09-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Leukemia\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1038/s41375-025-02744-x\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"HEMATOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Leukemia","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1038/s41375-025-02744-x","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}
引用次数: 0
摘要
虽然同种异体干细胞移植(allogeneic stem cell transplantation, alloc -SCT)是高风险和大多数中危急性髓系白血病(AML)患者首次缓解的首选巩固治疗,但当同种异体干细胞移植不可行或适应症时,自体干细胞移植(auto-SCT)与化疗(chemotherapy, CT)的作用仍然存在争议。我们使用PETHEMA AML注册表进行了一项真实世界的回顾性队列研究,以比较auto-SCT和CT。采用多变量Cox回归和倾向评分匹配(PS-matching)对混杂因素进行校正。共纳入1272例首次缓解并接受2个巩固疗程的患者(615例接受额外CT周期,657例接受自动sct)。总体而言,78.08%的auto-SCT患者在2017年之前被诊断,而CT队列中的这一比例为38.11% (p < 0.001)。在整个队列中,auto-SCT与显著延长总生存期(OS) (HR: 0.73, p < 0.001)和无复发生存期(RFS) (HR: 0.73, p < 0.001)相关。这种益处在≤65岁、核型正常、FLT3-ITD阴性的患者中尤为明显。在ps匹配的队列中,RFS优势持续存在(HR: 0.80, p = 0.092),但OS差异无统计学意义(HR: 0.91, p = 0.563)。auto-SCT在基因组和靶向药物时代的作用不应被抛弃。
Long-term benefits of autologous stem cell transplantation versus intensive chemotherapy consolidation for acute myeloid leukemia patients: A propensity score matching analysis from the PETHEMA AML registry
While allogeneic stem cell transplantation (allo-SCT) is the preferred consolidation for high and most intermediate-risk acute myeloid leukemia (AML) patients in first remission, the role of autologous SCT (auto-SCT) vs. chemotherapy (CT) when allo-SCT is not feasible or indicated, remains controversial. We conducted a real-world, retrospective cohort study using the PETHEMA AML registry to compare auto-SCT and CT. Multivariate Cox regression and propensity score matching (PS-matching) were used to adjust for confounding factors. A total of 1272 patients in first remission and who received 2 consolidation courses were included (615 receiving additional CT cycles and 657 undergoing auto-SCT). Overall, 78.08% of auto-SCT patients were diagnosed before 2017, compared to 38.11% in the CT cohort (p < 0.001). In the overall cohort, auto-SCT was associated with significantly prolonged overall survival (OS) (HR: 0.73, p < 0.001) and relapse-free survival (RFS) (HR: 0.73, p < 0.001). This benefit was particularly evident in patients ≤65 years, those with normal karyotype, and FLT3-ITD negativity. In the PS-matched cohort, the RFS advantage persisted (HR: 0.80, p = 0.092), but OS differences were not statistically significant (HR: 0.91, p = 0.563). The role of auto-SCT in the genomic and targeted agent era should not be discarded.
期刊介绍:
Title: Leukemia
Journal Overview:
Publishes high-quality, peer-reviewed research
Covers all aspects of research and treatment of leukemia and allied diseases
Includes studies of normal hemopoiesis due to comparative relevance
Topics of Interest:
Oncogenes
Growth factors
Stem cells
Leukemia genomics
Cell cycle
Signal transduction
Molecular targets for therapy
And more
Content Types:
Original research articles
Reviews
Letters
Correspondence
Comments elaborating on significant advances and covering topical issues