Long-term GH treatment appears safe for children with Prader-Willi syndrome
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With proper follow-up evaluations, long-term growth hormone treatment in children with Prader-Willi syndrome is safe, according to researchers from Italy. Previous research in the literature has linked short-term growth hormone therapy to adenotonsillar tissue hypertrophy and obstructive sleep apnea in this patient population.
“The overall results of this 48-month follow-up study confirm that GH treatment, on average, does not impair respiratory function during sleep,” Jenny Berini, MD, of the pediatric unit at Ospedale Del Ponte, Insubria University, in Varese, Italy, and colleagues wrote.
According to data, 50 patients with Prader-Willi syndrome were examined before GH treatment (dosage range: 0.01 mg/kg daily to 0.03 mg/kg daily), after 6 weeks, at 12 months and annually for up to 4 years. Researchers wrote that all of the patients underwent biochemical and anthropometric evaluations, polysomnography and otolaryngology examinations to evaluate the effects of long-term GH treatment.
The percentage of patients with an obstructive apnea-hypopnea index of more than 1 increased from 3% to 22% after 6 weeks, to 36% at 2 years and 38% at 4 years (P<.05), according to data. Conversely, reductions in the respiratory disturbance index (1.4 before treatment to 0.8 after 12 months of treatment; P<.05) and central apnea index were observed (1.2 before treatment to 0.1 after 2 years of treatment; P<.0001).
Due to severe obstructive sleep apnea, the researchers wrote that three patients were temporarily removed from GH treatment at 6 weeks and years 2 and 3.
The percentage of patients with severe adenotonsillar hypertrophy was significantly greater at years 2 and 3 compared with those examined before treatment, according to data.
“We found a direct relationship between the [obstructive apnea-hypopnea index] and the age-adjusted adenoid size, showing a probable major causative role of adenoid hypertrophy in the pathogenesis of [obstructive sleep apnea],” researchers wrote.
These data indicate that children with Prader-Willi syndrome can be safely administered long-term GH treatments with caution, they wrote.
Disclosure: The researchers report no relevant financial disclosures.