We compared outcomes from a single-arm study of blinatumomab in adult patients with B-precursor Ph-negative relapsed/refractory acute lymphoblastic leukemia (R/R ALL) with a historical data set from Europe and the United States. Estimates of complete remission (CR) and overall survival (OS) were weighted by the frequency distribution of prognostic factors in the blinatumomab trial. Outcomes were also compared between the trial and historical data using propensity score methods. The historical cohort included 694 patients with CR data and 1112 patients with OS data compared with 189 patients with CR and survival data in the blinatumomab trial. The weighted analysis revealed a CR rate of 24% (95% CI: 20-27%) and a median OS of 3.3 months (95% CI: 2.8-3.6) in the historical cohort compared with a CR/CRh rate of 43% (95% CI: 36-50%) and a median OS of 6.1 months (95% CI: 4.2-7.5) in the blinatumomab trial. Propensity score analysis estimated increased odds of CR/CRh (OR=2.68, 95% CI: 1.67-4.31) and improved OS (HR=0.536, 95% CI: 0.394-0.730) with blinatumomab. The analysis demonstrates the application of different study designs and statistical methods to compare novel therapies for R/R ALL with historical data.

Blood Disease Department Hôpital Saint Louis Paris France

Blood Diseases Service Center Hospitalier Universitaire Angers France

Center for Observational Research Thousand Oaks CA Amgen USA

Clinical Hematology Department ICO Hospital Germans Trias 1 Pujol Jose Carreras Research Institute Barcelona Spain

Department of Biostatistics Amgen Inc Rockville MD USA

Department of Biostatistics Uxbridge Amgen Ltd UK

Department of Bone Marrow Transplantation and Onco Hematology Maria Sklodowska Curie Memorial Cancer Center and Institute of Oncology Gliwice Poland

Department of Clinical Development Amgen Inc Thousand Oaks CA USA

Department of Hematology and Bone Marrow Transplantation Rambam Medical Center Haifa Israel

Department of Hematology and Hematopoietic Cell Transplantation City of Hope Duarte CA USA

Department of Hematology Oncology Cleveland Clinic Cleveland OH USA

Department of Internal Medicine Hematology and Oncology University Hospital Brno Czech Republic

Department of Leukemia University of Texas MD Cancer Center Houston TX USA

Department of Medicine Dana Farber Cancer Institute Boston MA USA

Department of Medicine University Hospital Goethe University Frankfurt Germany

Institute of Hematology and Medical Oncology L and A Seragnoli S Orsola University Hospital Bologna Italy

Medizinische Klinik und Poliklinik 2 Universitätsklinikums Würzburg Würzburg Germany

Research Department of Haematology UCL Cancer Institute London UK

Unità Operativa Complessa di Ematologia Ospedale dell'Angelo Mestre Venezia Italy

Blood Cancer J. 2016 Dec 9;6(12):e509. doi: 10.1038/bcj.2016.119. PubMed

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Blinatumomab vs historic standard-of-care treatment for minimal residual disease in adults with B-cell precursor acute lymphoblastic leukaemia