Growth hormone for adults with PWS

The clinical experience of GH in adults with PWS is not very well studied, but the first reports are encouraging. Two previous studies have suggested there are benefits to GH therapy in adults with respect body composition and physical performance [Hoybye 2003], as well as increased mental speed and flexibility [Hoybye 2005].

A new study from a group in Italy adds to this positive trend in GH outcomes. This study included 13 adults (20-33 years old) with PWS and growth hormone deficiency (GHD) and followed them over a period of 2 years, assessing quality of life and sense of well-being. [Quality of life and psychological well-being in GH-treated, adult PWS patients: a longitudinal study. L. Bertella et al, 2007]. Based on laboratory testing, 5 had severe GHD, 5 were GHD, and 3 had partial GHD. These adults were treated with 0.04 mg/kg/week for a month, then moved to 0.08 mg/kg/week for a year, with subsequent dosing modified according to serum IGF-1 levels.

After 24 months, data was collected on the 9 adults still enrolled (the remainder had moved or had work-related reasons for not completing the study). GH therapy was well tolerated. In a self-evaluation, these adults with PWS reported a significant increase in “Psychological General Well-Being Index”, a questionnaire looking at anxiety, depression, positive-well being, self-control, general health and vitality. They reported improvements on all measures, with the mean score rising from 75 at the start of the therapy to 101 at its completion, on a scale from 0-110. Another questionnaire (short form health survey) showed similar improvements in all areas, including general physical and mental health.

Interestingly, parents were also given the questionnaires (all of these adults lived with their parents), and also reported improvements in all areas, but in general these were more modest those reported by the individuals. Parents did report a significant increase in self-control in the PWS individuals, and ‘role limitation because of physical and emotional problems’ also showed strong improvement. Importantly, nobody (parents or adults with PWS) reported any declines in well-being over the growth hormone therapy period. These families had been seen at the clinic for years prior to this study, so the improvements cannot be attributed to a newly found clinical support team. Unfortunately, body mass index did not improve for these adults.

Thus, overall, GH therapy again seems to have beneficial effects in several areas for those with PWS of all ages.