Background:Currently,no worldwide approved therapies exist for adults with neurofibromatosis type 1(NF1)and symptomatic,inoperable plexiform neurofibromas.The KOMET study aimed to evaluate selumetinib(ARRY-142886,AZD6...Background:Currently,no worldwide approved therapies exist for adults with neurofibromatosis type 1(NF1)and symptomatic,inoperable plexiform neurofibromas.The KOMET study aimed to evaluate selumetinib(ARRY-142886,AZD6244)efficacy and safety in this population.Methods:This ongoing multicentre,international,randomised,placebo-controlled,phase 3,parallel,double-blind trial randomly assigned adults with NF1-plexiform neurofibroma 1:1 to 28-day cycles of oral selumetinib 25 mg/m2 twice daily,or placebo with crossover to selumetinib at confirmed radiological progression or the end of cycle 12.展开更多
文摘Background:Currently,no worldwide approved therapies exist for adults with neurofibromatosis type 1(NF1)and symptomatic,inoperable plexiform neurofibromas.The KOMET study aimed to evaluate selumetinib(ARRY-142886,AZD6244)efficacy and safety in this population.Methods:This ongoing multicentre,international,randomised,placebo-controlled,phase 3,parallel,double-blind trial randomly assigned adults with NF1-plexiform neurofibroma 1:1 to 28-day cycles of oral selumetinib 25 mg/m2 twice daily,or placebo with crossover to selumetinib at confirmed radiological progression or the end of cycle 12.