TY  - JOUR
A1  - Gökbuget, Nicola
A1  - Kelsh, Michael
A1  - Chia, Victoria
A1  - Advani, Anjali
A1  - Bassan, Renato
A1  - Dombret, Hervè
A1  - Doubek, Michael
A1  - Fielding, Adele K.
A1  - Giebel, Sebastian
A1  - Haddad, Vincent
A1  - Hoelzer, Dieter
A1  - Holland, Chris
A1  - Ifrah, Norbert
A1  - Katz, Aaron
A1  - Maniar, Tapan
A1  - Martinelli, Giovanni
A1  - Morgades, Mireia
A1  - O'Brien, Susan
A1  - Ribera, Jose-Maria
A1  - Rowe, Jacob M.
A1  - Stein, Anthony S.
A1  - Topp, Max S.
A1  - Wadleigh, Martha
A1  - Kantarjian, Hagop
T1  - Blinatumomab vs historical standard therapy of adult relapsed/refractory acute lymphoblastic leukemia
T2  - Blood Cancer Journal
N2  - 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.
Y1  - 2016
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/41726
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-417262
N1  - This work is licensed under a Creative Commons Attribution 4.0 International License. The images or other third party material in this article are included in the article’s Creative Commons license, unless indicated otherwise in the credit line; if the material is not included under the Creative Commons license, users will need to obtain permission from the license holder to reproduce the material. To view a copy of this license, visit  http://creativecommons.org/licenses/by/4.0/
VL  - 6
IS  - e473
ER  -