A parent language intervention based on story-telling eases the severity of speech and social impairments in children with fragile X syndrome, a study shows.
The study also found that adding lovastatin, a medication widely prescribed for lowering cholesterol, leads to no additional benefits.
The study “Controlled trial of lovastatin combined with an open-label treatment of a parent-implemented language intervention in youth with fragile X syndrome” was published in the Journal of Neurodevelopmental Disorders.
Research in preclinical models of fragile X has suggested that treatment with certain medicines to lower cholesterol — such as lovastatin, sold under the brand names Altocor, Altoprev, and Mevacor — corrected memory deficits.
Previous research showed that a parent-implemented language intervention, or PILI, that used story-telling improved language skills in children and adolescents with fragile X.
A team of researchers at University of California Davis Health conducted a Phase 4 trial (NCT02642653) testing whether the combination of lovastatin with PILI, delivered via video teleconferencing, would provide greater benefits than PILI alone. A total of 30 youngsters (28 boys and two girls), ages 10 to 17, participated in the study.
Fourteen participants were assigned randomly to lovastatin and 16 to a placebo, in addition to PILI. Both lovastatin and placebo were given orally at a starting dose of 10 mg once daily in the evening, and increasing up to a maximum dose of 40 mg daily. Treatment was maintained for 20 weeks.
PILI sessions lasted for 12 weeks, with four activities per week, delivered to the parent’s computers by an American speech-language certified specialist, in collaboration with a behavior analyst.
Parents were taught to use language facilitation strategies while interacting with their children over a shared storytelling activity. After digital wordless picture books, parents used example scripts that served as a guide. After the activities, parents met with the researchers to discuss challenging behaviors during the shared storytelling, which enabled the establishment of individualized plans.
The study’s main aims were to assess the effectiveness of lovastatin plus PILI in improving child language outcomes relative to PILI alone — focusing on the diversity of vocabulary used — how parent use of PILI impacted child language outcomes, and to identify potential biomarkers of lovastatin benefits.
Results showed that, after adjusting to the child’s performance at the start of the trial, children in both lovastatin and placebo groups experienced a significant improvement in the total number of story-related utterances, i.e., the ability to maintain spoken or written language.
Likewise, a significant improvement was seen in both groups for the number of different word roots, or types — an indicator of a child’s speech diversity — and in the total number of story-related utterances in parents. No differences were found between lovastatin and placebo.
Also, no differences were observed in the Clinical Global Impressions Scale-improvement scores of disease severity, assessed at a post-treatment visit. Parents in both groups reported lessened spoken language and social impairments in their children.
Improvements across the treatment period in the Aberrant Behavior Checklist-Community (ABC-C) also were similar between lovastatin and the placebo. Notably, the ABC-C is a 58-item caregiver-rated scale used to examine treatment effects on challenging behaviors in fragile X, such as irritability, hyperactivity, inappropriate speech, and social avoidance.
The analysis also revealed that parents are able to learn PILI-targeted intervention, but such learning is correlated only with speech benefits in the placebo group. No differences were found in blood markers associated with lovastatin’s effects, or in the level of FMR1 gene silencing — the root cause of fragile X.
Overall, “participants in both groups demonstrated significant changes in the primary outcome measures,” the researchers wrote.
“The magnitude of change observed across the two groups was comparable, providing additional support for the efficacy of the use of PILI in youth with FXS [fragile x syndrome],” they concluded.
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