LentiGlobin reduces transfusion dependence in young thalassemia patients
REPORTING FROM 2019 ASPHO CONFERENCE
Safety in both trials
“Very importantly, there were no deaths, there were no engraftment failures, there was no evidence of vector-mediated replication-competent lentivirus, and integration site analysis revealed no evidence of clonal dominance,” Dr. Olson said.
He added that most of the grade 3 or greater adverse events seen in both trials were directly attributable to busulfan-based myeloablative conditioning, including four episodes of veno-occlusive disease.
Nonhematologic grade 3 or higher adverse events in HGB-204 included stomatitis (n = 8), febrile neutropenia (n = 6), irregular menstruation (n = 3), pharyngeal inflammation (n = 2), and veno-occlusive liver disease (n = 1).
Nonhematologic grade 3 or higher adverse events in HGB-207 included stomatitis (n = 9), febrile neutropenia (n = 4), pharyngeal inflammation (n = 2), epistaxis (n = 3), pyrexia (n = 3), veno-occlusive liver disease (n = 3), ALT increase (n = 2), bilirubin increase (n = 2), and hypoxia (n = 2).
One patient in HGB-207 had grade 3 thrombocytopenia considered possibly related to LentiGlobin.
Dr. Olson reported advisory board engagement with bluebird bio, which sponsored both trials.
SOURCE: Olson TS et al. ASPHO 2019. Abstract 2002.
