Evaluation of miglustat treatment in patients with type III mucopolysaccharidosis: a randomized, double-blind, placebo-controlled study.

OBJECTIVE: To evaluate the efficacy and safety of oral miglustat treatment in patients with mucopolysaccharidosis type III. The primary outcome was efficacy with improvement or stabilization in at least two domains of Vineland Adaptative Behavior Scales at 6 months. The secondary outcome measured the evolution of other cognitive tests at 12 months. The safety and tolerability were assessed throughout the study. STUDY DESIGN: This was a randomized, double-blind, placebo-controlled, monocenter, institutional, phase IIb to III study. In case of efficacy at 6 months, the study would go on for another 6 months on an open design with all patients receiving miglustat. In the absence of efficacy at 6 months, the trial had to be continued for 6 more months with the initial design. RESULTS: After 6 months, efficacy was not superior in patients with miglustat. The independent review board confirmed continuing the study until 12 months. CONCLUSION: Miglustat treatment was not associated with any improvement/stabilization in behavior problems in patients with mucopolysaccharidosis type III. Miglustat has an acceptable safety profile. However, the study has confirmed that miglustat is able to pass through the blood-brain barrier without significantly decreasing ganglioside levels.

Growth and bone mineralization in preterm infants fed preterm formula or standard term formula after discharge.

OBJECTIVE: To evaluate growth and bone mineralization in very low birth weight (VLBW) infants fed preterm formula (PF) or term formula (TF). STUDY DESIGN: In a double-blind prospective study, 49 preterm infants of gestational age 33 weeks or less were randomly fed PF or TF for 2 months after discharge, then all of the infants were fed TF for the next 2 months. Anthropometric and dual-energy x-ray absorptiometry data were collected at discharge and at 2 months and 4 months after discharge. Anthropometric data also were collected at 12 months postterm. RESULTS: Four months after discharge, both body weight (6139 +/- 1254 g vs 5540 +/- 863 g; P = .03) and bone mineral content (104.4 +/- 29.2 g vs 87.5 +/- 17.1 g; P = .01) were significantly higher in the PF group compared with the TF group. At 12 months postterm, mean body weight, length, and head circumference remained higher in the PF group than in the TF group, and body mass index was similar and within the normal range in the 2 groups. CONCLUSIONS: At 4 months after discharge, growth and mineralization were better in the VLBW infants who were fed PF during the first 2 months after discharge compared with those who were fed TF, suggesting that PF may be particularly valuable at this early stage of development.