2022 júna: Po dvoch alebo viacerých líniách systémovej terapie FDA udelila tisagenlecleucel (Kymriah, Novartis Pharmaceuticals Corporation) urýchlené schválenie pre dospelých pacientov s recidivujúcim alebo refraktérnym folikulárnym lymfómom (FL).
The approval was based on the results of the ELARA trial (NCT03568461), a multicenter, single-arm, open-label trial evaluating tisagenlecleucel, a CD19-directed chimeric antigen receptor (CAR) T cell therapy, in adult patients who were refractory or relapsed within 6 months after completing two or more lines of systemic therapy (including an anti-CD20 antibody and an alkylating agent) or Tisagenlecleucel was given as a single intravenous infusion after lymphodepleting chemotherapy, with a target dosage of 0.6 to 6.0 x 108 CAR-positive viable T cells.
Hlavnými mierami účinnosti boli celková miera odpovede (ORR) a trvanie odpovede (DOR), ako ich stanovila nezávislá hodnotiaca komisia. ORR bola 86 percent (95 percent CI: 77, 92) medzi 90 pacientmi v primárnej analýze účinnosti, s mierou CR 68 percent (95 percent CI: 57, 77). Medián DOR nebol splnený, pričom 75 % respondentov (95 percent CI: 63, 84) odpovedalo aj po 9 mesiacoch. ORR bola 86 percent (95 percent CI: 77, 92) u všetkých pacientov, ktorí mali leukaferézu (n = 98), s mierou CR 67 percent (95 percent CI: 57, 76).
Syndróm uvoľňovania cytokínov, infection, weariness, musculoskeletal pain, headache, and diarrhoea were the most prevalent adverse effects in patients (>20 percent). 0.6 to 6.0 x 108 CAR-positive viable T cells is the suggested tisagenlecleucel dose.
