2021 avgust: FDA je dala aksikabtagene ciloleucel (Yescarta, Kite Pharma, Inc.) hitra odobritev za odrasle bolnike z recidivom ali neodzivnim folikularnim limfomom (FL) po dveh ali več linijah sistemskega zdravljenja.
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.
Nepristranska revizijska komisija je opredelila glavne ukrepe učinkovitosti: objektivno stopnjo odziva (ORR) in trajanje odziva (DOR). ORR je bil med 91 bolniki v primarni analizi učinkovitosti 95 odstotkov (83 odstotkov IZ: 96, 81), s popolno remisijo (CR) 60 odstotkov in povprečnim časom do odziva en mesec. Mediana DOR ni bila dosežena in 76.2 odstotka bolnikov je po enem letu ostalo v remisiji (95 odstotkov IZ: 63.9, 84.7). ORR je bil 89 % (95 % IZ: 83, 94) za vse bolnike z levkoferezo v tem preskušanju (n = 123), s stopnjo CR 62 %.
A boxed warning for sindrom sproščanja citokinov (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.
