2021 kolovoz: FDA je dala aksikabtagene ciloleucel (Yescarta, Kite Pharma, Inc.) ubrzano odobrenje za odrasle bolesnike s relapsom ili refraktornim folikularnim limfomom (FL) nakon dvije ili više linija sistemske terapije.
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.
Nepristrani odbor za reviziju definirao je glavne mjere učinkovitosti: stopu objektivnog odgovora (ORR) i trajanje odgovora (DOR). ORR je bio 91 posto (95 posto CI: 83, 96) među 81 pacijentom u primarnoj analizi učinkovitosti, sa stopom potpune remisije (CR) od 60 posto i srednjim vremenom do odgovora od mjesec dana. Medijan DOR nije postignut, a 76.2 posto bolesnika ostalo je u remisiji nakon jedne godine (95 posto CI: 63.9, 84.7). ORR je bio 89 posto (95 posto CI: 83, 94) za sve leukaferizirane pacijente u ovom ispitivanju (n=123), sa stopom CR od 62 posto.
A boxed warning for sindrom otpuštanja citokina (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.
