Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the Joint Outcome Continuation Study.

The Joint Outcome Study (JOS), a randomized controlled trial, demonstrated that children with severe hemophilia A (HA) initiating prophylactic factor VIII (FVIII) prior to age 2.5 years had reduced joint damage at age 6 years compared with those treated with episodic FVIII for bleeding. The Joint Outcome Continuation Study (JOS-C) evaluated early vs delayed prophylaxis effects on long-term joint health, following JOS participants to age 18 years in an observational, partially retrospective study. Index joint magnetic resonance imaging (MRI) scores of osteochondral (OC) damage (primary outcome), joint physical examination scores, and annualized rates of joint/other bleeding episodes (secondary outcomes) were collected. Thirty-seven of 65 JOS participants enrolled in JOS-C, including 15 randomized to prophylaxis at mean age 1.3 years ("early prophylaxis"); 18 initially randomized to episodic treatment, starting "delayed prophylaxis" at mean age 7.5 years; and 4 with high-titer inhibitors. At JOS-C exit, MRI OC damage was found in 77% of those on delayed and 35% of those on early prophylaxis for an odds ratio of OC damage, in the delayed vs early prophylaxis group, of 6.3 (95% confidence interval, 1.3, 29.9; P = .02). Annualized bleeding rates were higher with delayed prophylaxis (mean plus or minus standard deviation, 10.6 ± 6.6 vs 3.5 ± 2.1; P < .001), including when only comparing time periods on prophylaxis (6.2 ± 5.3 vs 3.3 ± 1.9; P < .05). In severe HA, early initiation of prophylaxis provided continued protection against joint damage throughout childhood compared with delayed initiation, but early prophylaxis was not sufficient to fully prevent damage. This trial was registered at www.clinicaltrials.gov as #NCT01000844.

A randomized comparative effectiveness trial of using cable television to deliver diabetes prevention programming.

OBJECTIVE: To evaluate the use and effectiveness of two "in-home" strategies for delivering diabetes prevention programming using cable television. METHODS: An individually randomized, two-arm intervention trial including adults with diabetes risk factors living in two US cities. Interventions involved a 16-session lifestyle intervention delivered via "video-on-demand" cable television, offered alone versus in combination with web-based lifestyle support tools. Repeated measures longitudinal linear regression with imputation of missing observations was used to compare changes in body weight. RESULTS: A total of 306 individuals were randomized and offered the interventions. After 5 months, 265 (87%) participants viewed at least 1, and 110 (36%) viewed ≥9 of the video episodes. A total of 262 (86%) participants completed a 5-month weight measurement. In intention-to-treat analysis with imputation of missing observations, mean weight loss at 5 months for both treatment groups combined was 3.3% (95% CI 0.7-5.0%), regardless of intervention participation (with no differences between randomized groups (P = 0.19)), and was 4.9% (95% CI 2.1-6.5%) for participants who viewed ≥9 episodes. CONCLUSIONS: In-home delivery of evidence-based diabetes prevention programming in a reality television format, offered with or without online behavioral support tools, can achieve modest weight losses consistent with past implementation studies of face-to-face programs using similar content.