Developmental Outcomes in Duarte Galactosemia.

: media-1vid110.1542/5849572227001PEDS-VA_2018-2516Video Abstract OBJECTIVES: For decades, infants with Duarte galactosemia (DG) have been identified by newborn screening (NBS), but whether they should be treated with dietary restrictions of galactose has remained unknown. To clarify, we conducted a study of dietary and developmental outcomes in 206 children with DG (case patients) and 144 controls, all of whom were 6 to 12 years old. METHODS: We recruited case patients from states where they were identified by NBS; unaffected siblings served as controls. Diet in infancy was ascertained by retrospective parent surveys; developmental outcomes were assessed in 5 domains, yielding 73 outcome measures for each child. We divided subjects randomly into independent discovery (n = 87) and validation (n = 263) sets. We tested the discovery set to order the 73 outcome measures by ascending P values and tested the 10 outcomes with the lowest P values for possible association with DG in the validation set. We also tested these same 10 outcomes for possible association with milk exposure in infancy among case patients in the validation set. RESULTS: None of the 73 outcomes tested in the discovery set revealed significant association with DG, and none of the 10 outcomes tested in the validation set revealed either significant association with DG or significant association with milk exposure among children with DG. CONCLUSIONS: Through our results, we demonstrated that there were no significant differences in outcomes tested between case patients and controls or among case patients as a function of milk exposure in infancy. In this study, we provide a long-needed foundation of knowledge for health care providers, families, and NBS professionals seeking to make evidence-based decisions about DG.

Virtual reality exposure therapy for social anxiety disorder: a randomized controlled trial.

OBJECTIVE: This is the first randomized trial comparing virtual reality exposure therapy to in vivo exposure for social anxiety disorder. METHOD: Participants with a principal diagnosis of social anxiety disorder who identified public speaking as their primary fear (N = 97) were recruited from the community, resulting in an ethnically diverse sample (M age = 39 years) of mostly women (62%). Participants were randomly assigned to and completed 8 sessions of manualized virtual reality exposure therapy, exposure group therapy, or wait list. Standardized self-report measures were collected at pretreatment, posttreatment, and 12-month follow-up, and process measures were collected during treatment. A standardized speech task was delivered at pre- and posttreatment, and diagnostic status was reassessed at 3-month follow-up. RESULTS: Analysis of covariance showed that, relative to wait list, people completing either active treatment significantly improved on all but one measure (length of speech for exposure group therapy and self-reported fear of negative evaluation for virtual reality exposure therapy). At 12-month follow-up, people showed significant improvement from pretreatment on all measures. There were no differences between the active treatments on any process or outcome measure at any time, nor differences on achieving partial or full remission. CONCLUSION: Virtual reality exposure therapy is effective for treating social fears, and improvement is maintained for 1 year. Virtual reality exposure therapy is equally effective as exposure group therapy; further research with a larger sample is needed, however, to better control and statistically test differences between the treatments.