Efficacy and safety of avapritinib in advanced systemic mastocytosis: 4-year follow-up of the PATHFINDER study

Gotlib J, Reiter A, Radia DH, Álvarez-Twose I, Deininger MW, George TI, Panse JP, Mital A, Pettit KM, Vannucchi AM, Platzbecker U, Hermine O, Elshoury A, Livideanu C, Mesa RA, Ustun C, Triggiani M, Dybedal I, Jurcic JG, Zanotti R, Oh ST, Yacoub A, Hexner EO, Bose P, Lee SG, Sperr WR, Griffiths EA, Butler M, Lübke J, Bidollari I, Lin HM, Rylova SN, Dimitrijević S, Muñoz-González JI, DeAngelo DJ

Advanced systemic mastocytosis (AdvSM) is a rare blood disease that can damage organs and shorten life expectancy. In this study, treatment with avapritinib produced long-lasting benefits, with nearly three-quarters of patients responding to therapy and many maintaining their response for several years.

After more than four years of follow-up, avapritinib continued to reduce signs of disease and showed a manageable safety profile. These results suggest that avapritinib can provide durable disease control and meaningful long-term benefits for people with AdvSM.