Prader-Willi syndrome: guidance for children and transition into adulthood.
Mohamad Guftar ShaikhTimothy G BarrettNicola BridgesRobin ChungEvelien F GeversAnthony Peter GoldstoneAnthony HollandShankar KanumakalaRuth KroneAndreas KyriakouE Anne LiveseyAngela K Lucas-HeraldChristina MeadeSusan PassmoreEdna RocheChris SmithSarita SoniPublished in: Endocrine connections (2024)
Prader-Willi syndrome (PWS) is a rare orphan disease and complex genetic neurodevelopmental disorder, with a birth incidence of approximately 1 in 10,000-30,000. Management of people with PWS requires a multi-disciplinary approach, ideally through a multi-disciplinary team (MDT) clinic with community support. Hypotonia, poor feeding and faltering growth are characteristic features in the neonatal period, followed by hyperphagia and risk of rapid weight gain later in childhood. Children and adolescents (CA) with PWS usually display developmental delay and mild learning disability and can develop endocrinopathies, scoliosis, respiratory difficulties (both central and obstructive sleep apnoea), challenging behaviours, skin picking, and mental health issues, especially into adulthood. This consensus statement is intended to be a reference document for clinicians managing children and adolescents (up to 18 years of age) with PWS. It considers the bio-psycho-social domains of diagnosis, clinical assessment, and management in the paediatric setting as well as during and after transition to adult services. The guidance has been developed from information gathered from peer-reviewed scientific reports and from the expertise of a range of experienced clinicians in the United Kingdom and Ireland involved in the care of patients with PWS.
Keyphrases
- mental health
- weight gain
- growth hormone
- palliative care
- healthcare
- early life
- body mass index
- birth weight
- depressive symptoms
- primary care
- intensive care unit
- mental illness
- young adults
- quality improvement
- case report
- emergency department
- multiple sclerosis
- gestational age
- pregnant women
- sleep quality
- pain management
- health information
- cross sectional
- affordable care act
- respiratory tract
- copy number
- adverse drug
- loop mediated isothermal amplification
- dna methylation
- sensitive detection
- positive airway pressure