Presidential Symposium: Friday, June 10
Blinatumomab improved OS in patients with relapsed or refractory Philadelphia-negative B-cell precursor ALL in a randomized, open-label phase 3 study (TOWER)
In this large phase 3 trial, blinatumomab, a bispecific CD19-directed CD3 T-cell engager (BiTE®) antibody construct, is compared with protocol-defined standard-of-care chemotherapy regimens in adults with relapsed or refractory Philadelphia-negative B-cell precursor acute lymphoblastic leukemia (ALL). TOWER is the first randomized study of an immunotherapy to demonstrate an overall survival (OS) benefit in this difficult-to-treat patient population. The magnitude of this benefit will be unveiled tomorrow.
Stopping tyrosine kinase inhibitors (TKIs) in a very large cohort of European CML patients: results of the EURO-SKI trial
TKIs have dramatically improved survival in chronic myeloid leukemia (CML). Most patients achieve deep molecular responses, such that TKIs can be safely and successfully stopped. The initial univariate analysis of the European Stop TKI (EURO-SKI) study verifies that high molecular relapse-free remission rates are achievable, and that duration of TKI treatment and duration of deep molecular response affect relapse-free survival. Results of a multivariate analysis from this trial will be presented in tomorrow’s session.
Persistence of driver mutations during complete remission associates with shorter survival and contributes to the inferior outcomes of elderly patients with AML
Patients with acute myeloid leukemia (AML) who are in morphologic complete remission (CR) can harbor persistent preleukemic clones that influence disease relapse. In this evaluation of more than 100 patients with AML who achieved CR or incomplete CR after initial therapy, gene sequencing shows persistent leukemia-associated driver mutations. These mutations appear more often in older patients with AML and are correlated with shorter relapse-free survival and OS.
ETV6-related thrombocytopenia (ETV6-RT): clinical and pathogenetic characterization of an inherited thrombocytopenia (IT) predisposing to childhood ALL
Using gene sequencing, researchers find that monoallelic ETV6 mutations cause one of the most frequent forms of IT. This study confirms that affected subjects have a low bleeding tendency and a high propensity to hematologic malignancies, including childhood ALL. Because ETV6-RT is an autosomal-dominant form of IT without platelet macrocytosis, screening for ETV6 mutations is recommended in patients with these characteristics.
Dissecting the contribution of unregulated macrophage iron recycling and dietary iron uptake in generating systemic iron overload in hemochromatosis
This translational study shows that increased duodenal iron export is a major contributor to systemic iron overload in patients with hemochromatosis, a disorder in which the body absorbs too much iron from food. This finding will advance pharmacologic interventions for patients with primary and secondary iron-overload diseases.
An open-label, randomised phase 3 study of daratumumab, lenalidomide, and dexamethasone (DRd) versus lenalidomide and dexamethasone (Rd) in relapsed or refractory MM (rrMM): POLLUX
The phase 3 POLLUX study shows significantly improved progression-free survival after the preplanned interim analysis. For patients with rrMM receiving DRd, risk of disease progression is lowered by more than 60% compared with those who did not receive daratumumab.