Effects of Childhood Multidisciplinary Care and Growth Hormone Treatment on Health Problems in Adults with Prader-Willi Syndrome

Abstract

Prader-Willi syndrome (PWS) is a complex hypothalamic disorder. Features of PWS include hyperphagia, hypotonia, intellectual disability, and pituitary hormone deficiencies. The combination of growth hormone treatment and multidisciplinary care (GHMDc) has greatly improved the health of children with PWS. Little is known about the effects of childhood GHMDc on health outcomes in adulthood. We retrospectively collected clinical data of 109 adults with PWS. Thirty-nine had received GHMDc during childhood and adolescence (GHMDc+ group) and sixty-three had never received growth hormone treatment (GHt) nor multidisciplinary care (GHMDc− group). Our systematic screening revealed fewer undetected health problems in the GHMDc+ group (10%) than in the GHMDc− group (84%). All health problems revealed in the GHMDc+ group had developed between the last visit to the paediatric and the first visit to the adult clinic and/or did not require treatment. Mean BMI and the prevalence of diabetes mellitus type 2 were significantly lower in the GHMDc+ group compared to the GHMDc− group. As all patients who received GHt were treated in a multidisciplinary setting, it is unknown which effects are the result of GHt and which are the result of multidisciplinary care. However, our data clearly show that the combination of both has beneficial effects. Therefore, we recommend continuing GHMDc after patients with PWS have reached adult age.

Health Problems in Adults with Prader–Willi Syndrome of Different Genetic Subtypes: Cohort Study, Meta-Analysis and Review of the Literature

“…Patients who had experienced at least one episode of psychotic behavior (diagnosed by a psychologist/psychiatrist) were considered as having a (history of) psychosis. Part of the clinical data of this patient group has been previously published in another context [ 22 , 23 , 24 , 25 , 26 , 27 ].…”

Facilitating the transition from paediatric to adult care in endocrinology: a focus on growth disorders

“…AYAs with PWS who have had appropriate transition of care have been reported to have lower BMI, improved metabolic parameters and fewer antidepressant treatments compared with those who did not have coordinated transitional care [68]. Multidisciplinary models of care, including joint visits with paediatric and adult endocrinologists, dieticians and therapists, have also been shown to improve outcomes in adulthood [69]. In a recent retrospective study, fewer undetected health problems during the transition period and lower mean BMI and prevalence of T2D occurred in adults with PWS who received GH therapy and multidisciplinary care during the transition period, compared with those who did not receive this care [69].…”

“…Multidisciplinary models of care, including joint visits with paediatric and adult endocrinologists, dieticians and therapists, have also been shown to improve outcomes in adulthood [69]. In a recent retrospective study, fewer undetected health problems during the transition period and lower mean BMI and prevalence of T2D occurred in adults with PWS who received GH therapy and multidisciplinary care during the transition period, compared with those who did not receive this care [69]. The benefits of care coordinators have been studied in AYAs with PWS.…”

A questionnaire-based survey of medical conditions in adults with Prader-Willi syndrome in Japan: implications for transitional care

“…The effectiveness of a multidisciplinary team approach including pediatric endocrinologists, dieticians, nurse practitioners, geneticists, and psychologists for the management of the body composition and obesity-related complications in children with PWS has been demonstrated [12][13][14]. Although the majority of children with PWS are treated with GH, which allows for the provision of frequent and sustained interventions by the multidisciplinary team, the multidisciplinary team approach is generally difficult to implement during adulthood, which is partly due to the lack of a structured transitional care program for PWS [12,15].…”

“…The effectiveness of a multidisciplinary team approach including pediatric endocrinologists, dieticians, nurse practitioners, geneticists, and psychologists for the management of the body composition and obesity-related complications in children with PWS has been demonstrated [12][13][14]. Although the majority of children with PWS are treated with GH, which allows for the provision of frequent and sustained interventions by the multidisciplinary team, the multidisciplinary team approach is generally difficult to implement during adulthood, which is partly due to the lack of a structured transitional care program for PWS [12,15]. Transitional care is a process during which dedicated health care programs are provided when care is transferred from pediatric to adult caregivers [16]; however, transitional care in PWS has been difficult and challenging because of the multisystem nature of this disorder, which includes cognitive and psychiatric symptoms [15,17].…”