Real-world use of blinatumomab in adult patients with B-cell acute lymphoblastic leukemia in clinical practice: results from the NEUF study

Abstract

This retrospective observational study (NEUF) included adult patients with B-cell acute lymphoblastic leukemia (B-cell ALL) who had received blinatumomab for the treatment of minimal residual disease-positive (MRD+) or relapsed/refractory (R/R) B-cell ALL via an expanded access program (EAP). Patients were eligible if blinatumomab was initiated via the EAP between January 2014 and June 2017. Patients were followed from blinatumomab initiation until death, entry into a clinical trial, the end of follow-up, or the end of the study period (December 31, 2017), whichever occurred first. Of the 249 adult patients included, 109 were MRD+ (83 Philadelphia chromosome-negative [Ph−] and 26 Philadelphia chromosome-positive [Ph+]) and 140 had a diagnosis of R/R B-cell ALL (106 Ph− and 34 Ph+). In the MRD+ group, within the first cycle of blinatumomab treatment, 93% (n = 49/53) of Ph− and 64% (n = 7/11) of Ph+ patients with evaluable MRD achieved an MRD response (MRD <0.01%). Median overall survival (OS) was not reached over a median follow-up time of 18.5 months (Ph−, 18.8 [range: 5.1–34.8] months; Ph+, 16.5 [range: 1.8–31.6] months). In the R/R group, within two cycles of blinatumomab, 51% of Ph− and 41% of Ph+ patients achieved complete hematologic remission (CR/CRh/CRi), and 83% of Ph− and 67% of Ph+ MRD-evaluable patients in CR/CRh/CRi achieved an MRD response. Median (95% confidence interval) OS was 12.2 (7.3–24.2) months in the R/R Ph− subgroup and 16.3 (5.3–not estimated) months in the R/R Ph+ subgroup. This large, real-world data set of adults with B-cell ALL treated with blinatumomab confirms efficacy outcomes from published studies.

NG2 molecule expression in acute lymphoblastic leukemia B cells: a flow-cytometric marker for the rapid identification of KMT2A gene rearrangements

“…Therefore, as the presence of KMT2Ar increases CD38 MFI and does not impact the intensity of CD19 antigen expression, these patients can still benefit from the specific therapy currently available (monoclonal antibodies). 28 – 29 On the other hand, no differences in any of the antigens analysed were observed comparing KMT2Ar+ vs KMT2Ar– B-common ALLs, probably due to the low number of KMT2Ar + cases characterized by this EGIL immunologic subtype. However, no differences were observed in any of the analysed antigens when comparing KMT2Ar+ and KMT2Ar-B-common ALLs.…”

Impact of minimal residual disease response and of status of disease on survival after Blinatumomab in B-Cell Acute Lymphoblastic Leukemia: results from a Real-Life Study. Running title: MRD-response and Disease Status correlate with survival after Blinatumomab in ALL-B patients

“…The largest real-life blinatumomab studies up to date, are a US study by Badar and colleagues [19], the European multicenter study by Boissel and colleagues in adults (NEUF study) [20] and the study by Locatelli and colleagues in a pediatric population (RIALTO study) [21]. In addition other retrospective [17, 24 -26] studies addressed the outcome of blinatumomab both for MRD and for relapsed/refractory B-ALL.…”

“…The NEUF study [20] and the RIALTO study [21] enroll 249 adult patients and 110 pediatric patients, respectively. In both the patients enrolled were those treated under an Expanded Access program (EAP) prior to the introduction of the drug on the European market.…”

“…In addition other retrospective [17, 24 -26] studies addressed the outcome of blinatumomab both for MRD and for relapsed/refractory B-ALL. Overall they report a median relapse free survival (RFS) ranging from 8.5 moths to 32 months, with the better results for patients treated for MRD [17,[19][20][21][24][25][26]. The table 6 summarizes the results of such studies.…”

Therapeutic tactics for targeting B lymphocytes in autoimmunity and cancer

“…Blinatumomab dually targets CD3 and CD19, is used to treat B‐cell acute lymphocytic leukemia, and has been observed to grant complete remission in some patients [117]. A wealth of clinical information has emerged since the approval of Blinatumomab in 2014 and a retrospective observational study published in January 2023 confirms efficacy outcomes in real‐world use [118].…”