Augusta 2021: FDA dal axicabtagene ciloleucel (Yescarta, Kite Pharma, Inc.) urýchlené schválenie pre dospelých pacientov s relapsujúcim alebo refraktérnym folikulárnym lymfómom (FL) po dvoch alebo viacerých líniách systémovej terapie.
A single-arm, open-label, multicenter trial (ZUMA-5; NCT03105336) evaluated axicabtagene ciloleucel, a CD19-directed chimeric antigen receptor (CAR) T cell therapy, in adult patients with relapsed or refractory FL after two or more lines of systemic therapy, including the combination of an anti-CD20 monoclonal antibody and an alkylating agent, in adult patients with relapsed A single intravenous infusion of axicabtagene ciloleucel was given after lymphodepleting chemotherapy.
Nestranný hodnotiaci výbor definoval hlavné opatrenia účinnosti: mieru objektívnej odpovede (ORR) a trvanie odpovede (DOR). ORR bola 91 % (95 percent IS: 83, 96) medzi 81 pacientmi v primárnej analýze účinnosti s mierou úplnej remisie (CR) 60 percent a mediánom času do odpovede jeden mesiac. Medián DOR nebol dosiahnutý a 76.2 percenta pacientov zostalo v remisii po jednom roku (95 percent CI: 63.9, 84.7). ORR bola 89 percent (95 percent CI: 83, 94) pre všetkých pacientov s leukaferou v tejto štúdii (n = 123), s mierou CR 62 percent.
A boxed warning for syndróm uvoľnenia cytokínov (CRS) and neurologic toxicities is included in the prescribing material for axicabtagene ciloleucel. CRS occurred in 88 percent (Grade 3, 10%) of patients with non-lymphoma Hodgkin’s (NHL) in investigations using axicabtagene ciloleucel, while neurologic toxicities occurred in 81 percent (Grade 3, 26 percent). CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, febrile neutropenia, nausea, infections with pathogen unspecified, decreased appetite, chills, diarrhoea, tremor, musculoskeletal pain, cough, hypoxia, constipation, vomiting, arrhythmias, and dizziness are the most common non-laboratory adverse reactions (incidence 20%) in patients with NHL.
