Table 6.
Areas Regarding rhGH Use for PWS Requiring Prioritized Attention in Future Studiesa
| Top 10 areas for further research |
| i. Effects of rhGH therapy in adults with PWS on quality of life |
| ii. Long-term post-treatment effect of rhGH on mortality and morbidity using registries |
| iii. The optimal timing and dosage of rhGH treatment initiation in early life |
| iv. The effect of rhGH interruption at completion of growth |
| v. Effects of rhGH on behavior and cognitive function across the age range |
| vi. Impact of rhGH treatment on activities of daily living and well-being as defined by WHO |
| vii. Influence of IGF-I titration on clinical effects |
| viii. Effect of rhGH on glucose metabolism/diabetes risk, mainly long-term effect |
| ix. Effects of rhGH therapy on sleep and sleep-disordered breathing in PWS adults |
| x. RCTs investigating combination approaches to treatment |
| Additional areas for future research |
| xi. Effects of GH/IGF-I on nasopharyngeal tissue and mainly whether adenotonsillectomy changes the course or may avoid potential side effects of rhGH on sleep disorders and obstructive sleep apnea |
| xii. Dose-response relationships investigating efficacy of physiological (rather than pharmacological) dosing |
| xiii. Effects of rhGH treatment in children and adults on visceral adiposity and ectopic fat, eg, muscle, liver, and pancreas |
| xiv. Effects of rhGH on timing of development or severity of hyperphagia |
| xv. Effects of rhGH on bone maturation and premature pubarche |
| xvi. Effects on structural brain development |
| xvii. Scoliosis and slipped capital femoral epiphysis in children |
| xviii. Is there hypersensitivity to rhGH in PWS? |
| xix. Thyroid function before and after rhGH |
| xx. Effects on cardiac function |
| xxi. Effects of rhGH on lipid metabolism |
| xxii. Effects of rhGH on water retention |
| xxiii. Intracranial hypertension (difficult to assess in young children) |
a
All participants were asked to discuss areas for future investigation within breakout groups. All participants were then asked to order the areas, by priority, using a secret ballot.