Response to treatment in a prospective national XXXX .

@entity1 @entity4545 @entity410 @entity5305 @entity739 @entity2473 @entity302 @entity288
OBJECTIVE: Infantile @entity2473 are @entity410 associated with a severe @entity302 presenting in the first 2 years of life, and optimal treatment continues to be debated. This study evaluates early and sustained response to initial treatments and addresses both clinical remission and electrographic resolution of @entity739 . Secondarily, it assesses whether response to treatment differs by etiology or developmental status. METHODS: The National @entity739 Consortium established a multicenter, prospective database enrolling @entity1 with new diagnosis of @entity739 . @entity1 were considered responders if there was clinical remission and resolution of @entity739 that was sustained at 3 months after first treatment initiation. Standard treatments of @entity4545 ( @entity4545 ), oral @entity288 , and @entity5305 were considered individually, and all other nonstandard therapies were analyzed collectively. Developmental status and etiology were assessed. We compared response rates by treatment group using chi-square tests and multivariate logistic regression models. RESULTS: Two hundred thirty @entity1 were enrolled from 22 centers. Overall, 46% of @entity1 receiving standard therapy responded, compared to only 9% who responded to nonstandard therapy (p   <   0.001). Fifty-five percent of @entity1 receiving @entity4545 as initial treatment responded, compared to 39% for oral @entity288 , 36% for @entity5305 , and 9% for other (p   <   0.001). Neither etiology nor development significantly modified the response pattern by treatment group. INTERPRETATION: Response rate varies by treatment choice. Standard therapies should be considered as initial treatment for @entity739 , including those with impaired development or known structural or genetic/metabolic etiology. @entity4545 appeared to be more effective than other standard therapies. Ann Neurol 2016.
