Ottobre 2021: Brexucabtagene autoleucel (Tecartus, Kite Pharma, Inc.) has been approved by the Food and Drug Administration for adult patients with relapsed or refractory B-cell precursor leucemia linfoblastica acuta (LLA).
In ZUMA-3 (NCT02614066), a single-arm multicenter trial in individuals with relapsed or refractory B-cell precursor TUTTO, the efficacy of brexucabtagene autoleucel, a CD19-directed chimeric antigen receptor (CAR) T-cell treatment, was assessed. Following lymphodepleting chemotherapy, patients received a single infusion of brexucabtagene autoleucel.
La risposta completa (CR) entro 3 mesi dall'infusione e la durata della CR erano i criteri di efficacia utilizzati per supportare l'approvazione. Entro tre mesi, 28 (52 percento; 95 percento CI: 38, 66) dei 54 pazienti valutabili per l'efficacia hanno raggiunto la CR. La durata mediana della CR non è stata raggiunta con un follow-up mediano di 7.1 mesi per i responder; si prevedeva che la durata della CR superasse i 12 mesi per più della metà dei pazienti.
Un avvertimento in scatola per sindrome da rilascio di citochine (CRS) and neurologic toxicities is included in the prescribing material for brexucabtagene autoleucel. In 92 percent of cases (Grade 3, 26 percent), CRS developed, and in 87 percent of cases (Grade 3, 35 percent), neurologic toxicities occurred. Fever, CRS, hypotension, encephalopathy, tachycardias, nausea, chills, headache, fatigue, febrile neutropenia, diarrhoea, musculoskeletal pain, hypoxia, rash, edoema, tremor, infection with an unspecified pathogen, constipation, decreased appetite, and vomiting were the most common non-laboratory adverse reactions (incidence 20%).
A single intravenous infusion of 1 x 106 CAR-positive viable T cells per kg body weight (maximum 1 x 108 CAR-positive viable T cells) is advised for brexucabtagene autoleucel treatment, followed by fludarabine and cyclophosphamide for lymphodepleting chemotherapy.
