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
{"title":"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。","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":null,"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":8.2000,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"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\":null,\"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%. 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引用次数: 0
摘要
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。
Outcomes of children and young adults with B-cell acute lymphoblastic leukemia given blinatumomab as last consolidation treatment before allogeneic hematopoietic stem cell transplantation.
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.
期刊介绍:
Haematologica is a journal that publishes articles within the broad field of hematology. It reports on novel findings in basic, clinical, and translational research.
Scope:
The scope of the journal includes reporting novel research results that:
Have a significant impact on understanding normal hematology or the development of hematological diseases.
Are likely to bring important changes to the diagnosis or treatment of hematological diseases.