Discussion
PWS, rhGH initiation, tonsillar hypertrophy, lymphoid hyperplasia, deterioration of motor function, ’ motor development quotients
SLEEP APNEA, AIRWAY OBSTRUCTION, ADENOIDAL HYPERTROPHY, TONSILLAR HYPERTROPHY, LYMPHOID HYPERPLASIA, REGRESSION
To our knowledge, this is the first study in China to describe the time effect of early rhGH treatment in infants and young children with PWS. After 52 weeks of treatment, besides significant improvement in anthropometric parameters, we found that PWS patients’ motor development quotients were negatively correlated with age when analyzed by linear regression models, and rhGH treatment slowed down the rate of deterioration in TMQ and GMQ. Mental development assessed by the GDS-C showed significant improvement both in GQ and AQ–EQ after treatment especially in subjects aged < 9 months.We found that TMQ, GMQ, and FMQ were negatively correlated with age in pediatric patients with PWS in a linear regression analysis. However, treatment alleviated the deterioration of TMQ and GMQ. Several studies reported improvement in motor development scores assessed using various motor scales with rhGH treatment among PWS patients [We also found that younger pediatric patients had a greater improvement in mental development using the GDS-C. When stratified according to age of rhGH initiation, those in the < 9-month group performed significantly better with rhGH treatment than baseline in GQ and the other sub-quotients. General and locomotor development were significantly improved in the < 9-month group compared with the ≥ 9-month group. Our results were in line with other studies that demonstrated the positive impact of GH treatment on mental functioning in children with PWS [Our results were consistent with other studies showing improvements in and normalization of anthropometric parameters [Children with PWS are sensitive to GH and have high levels of IGF-1 during rhGH treatment, increasing beyond + 2 SDS. This raises concerns about the safety issues related to high IGF-1 levels, as high levels of IGF-1 have been associated with lymphoid hyperplasia and this might increase the risk of sleep apnea [In general, daily rhGH was well tolerated in pediatric patients with PWS throughout the 52-week treatment. Safety concerns about the potential AEs of rhGH treatment in this study were mainly focused on tonsillar hypertrophy, adenoidal hypertrophy, and upper airway obstruction, which are well documented in children with PWS [The merit of our study was the simultaneous use of PDMS-2 and the GDS-C to comprehensively evaluate, monitor, and accurately capture the development of different motor skills and the overall development of pediatric patients with PWS, and this study enrolled younger patients with a median age of 7.0 months, in line with increasing evidence supporting the benefit of early intervention with rhGH [In conclusion, treatment with rhGH for 52 weeks in infants and young children with PWS improved growth (height and weight), BMI, and mental development. In addition, the results of this study support the premise that initiation of treatment at early infancy (before age nine months) yielded better mental outcomes than those who started treatment later. The impact on motor development remains inconclusive, although our linear regression analysis suggested a positive effect by alleviating the deterioration of motor function in infants and young children with PWS. The present findings of this study add to the growing evidence that rhGH administration in infants and young children with PWS is well tolerated and effective, providing benefits that extend beyond physical growth when initiated early. No rhGH was approved for the treatment of PWS in China at the time of study commencement. Therefore, the results of our study will provide more evidence to guide the clinical practice of rhGH therapy in Chinese PWS patients.