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A 30-minute webinar with Dr Christina Lampe (Director, Rare Disease Centre, Gießen) on the transition of mucopolysaccharidosis (MPS) patients from paediatric to adult care — the structural, emotional and clinical gaps, and international models for bridging them. For paediatricians and metabolic teams.
As treatment lets more children with MPS reach adulthood, transition — a purposeful, planned process addressing the medical, psychosocial and educational needs of adolescents moving from child-centred to adult-oriented care — has become essential. Dr Christina Lampe contrasts the two care models: paediatric care is family-centred, holistic, protective and development-focused, while adult care emphasises independence, patient responsibility and compartmentalised specialist input, and many adult providers lack training in adolescent medicine — leaving a gap precisely where adolescents (a wide span of developmental stages) need continuity most. Drawing on European MetabERN surveys, she documents the deficits: only about 11% of respondents were adult metabolic physicians, nearly 20% of patients never transition (continuing lifelong with paediatricians), and there is a widespread lack of transition coordinators, standardised documentation and financial support. She reviews successful international models — France's 'Jump' programme and a US six-step system, both starting around age 12 and introducing adult services gradually over years — and stresses that transition is a long, planned process, not an event at 18. In MPS specifically, two groups need different approaches: cognitively intact patients, who can pursue independence, work, relationships and family planning ('I'm different, but I want a normal life'), and cognitively involved patients, who depend on their families, are sensitive to changes of environment, and need sustained, sensitive support including end-of-life planning — while both need continued monitoring of progressive somatic complications and, as adults, care for ordinary adult conditions. Her recommendations: plan early, educate and engage both patients and adult teams, involve adult physicians before transfer, give young people time to be seen without their parents, appoint a coordinator (not necessarily a physician), document the process in a standardised way, involve families, and keep every plan individualised and flexible.
After viewing this webinar, participants will be able to:
Transition is a purposeful, planned process that addresses the medical, psychosocial and educational needs of adolescents as they move from child-centred to adult-oriented healthcare. It matters because treatment now lets many more children with MPS reach adulthood, so these 'childhood' diseases increasingly need adult physicians — and adolescence, already a turbulent stage, is made harder by a chronic, multisystem disease that requires lifelong care. Done poorly, patients fall into the gap between two very different care systems.
Paediatric care is family-centred, holistic, protective and focused on development and growth; adult care emphasises independence, patient responsibility, and compartmentalised specialist input. Many providers in both — but especially in adult services — have limited training in adolescent medicine, so the adolescent group in between is poorly covered. Practical gaps include the loss of multidisciplinary, holistic coordination, less time for psychosocial needs, and adult settings less used to involving parents, whose expertise remains important.
MetabERN surveys found major deficits: only about 11% of respondents were adult metabolic physicians, most centres are followed by paediatricians (often lifelong), and nearly 20% of patients never transition at all. There is a widespread lack of transition coordinators, no standardised written transition protocol in most places, a need for training in adolescent metabolic care, and a lack of financial support in around 90% of cases — with lack of time and of adult metabolic physicians the most cited reasons for not running a programme.
As a gradual, planned, individualised process — not an event at 18 — ideally starting around age 12 and introducing adult services over several years, as in France's 'Jump' programme or the US six-step model. Two MPS groups need different approaches: cognitively intact patients working towards independence, work, relationships and family planning; and cognitively involved patients who depend on family, are sensitive to new environments, and need sustained support and end-of-life planning. Give young people time to be seen without their parents, appoint a coordinator, document the process, involve adult physicians before transfer and involve families throughout — keeping every plan flexible.
This content is intended for healthcare professionals only. The views expressed are those of the presenters and do not necessarily reflect those of Excellence in Pediatrics; their inclusion does not imply endorsement. The content is provided for educational purposes only and does not constitute medical advice or replace independent clinical judgement.