Outcomes of children and young adults with B-cell acute lymphoblastic leukemia given blinatumomab as last consolidation treatment before allogeneic hematopoietic stem cell transplantation
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
Affiliations
Mattia Algeri
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital; Department of Health Sciences, Magna Graecia University, Catanzaro
Michele Massa
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital; Department of Maternal and Child Health, Sapienza University of Rome
Daria Pagliara
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Valentina Bertaina
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Federica Galaverna
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Ilaria Pili
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Giuseppina Li Pira
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Roberto Carta
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Francesco Quagliarella
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Rita M. Pinto
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Chiara Rosignoli
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Barbarella Lucarelli
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Maria G. Cefalo
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Emilia Boccieri
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Francesca Benini
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital; Department of Maternal and Child Health, Sapienza University of Rome
Francesca Del Bufalo
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Marco Becilli
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Pietro Merli
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital
Gerhard Zugmaier
Amgen Research (Munich) GmbH, Munich
Franco Locatelli
Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children's Hospital; Catholic University of the Sacred Heart, Rome
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.