Mucopolysaccharidoses Patient's Transition To Adulthood

Dr. Lampe begins by defining “transition” as a purposeful, planned process that addresses the medical, psychosocial, and educational needs of adolescents as they move from child-centered to adult-oriented healthcare systems. She highlights the unique challenges adolescents with MPS face—already a turbulent phase of life, made more complex by chronic, multisystem diseases that often require lifelong medical support.

The session presents key contrasts between pediatric and adult care models. Pediatric care tends to be family-centered, holistic, and development-focused, whereas adult care emphasizes independence, patient responsibility, and compartmentalized specialist input. Dr. Lampe notes that many adult care providers lack training in adolescent medicine, creating gaps that must be bridged through deliberate planning and communication.

She shares findings from European surveys that reveal major deficits in structured transition protocols, financial support, and adult metabolic physician availability. For example, only 11% of respondents were adult metabolic specialists, and nearly 20% of patients never transitioned, continuing lifelong care with pediatricians. Dr. Lampe stresses the urgent need for transition coordinators and flexible, individualized programs supported by standardized documentation and cross-disciplinary collaboration.

The webinar reviews several successful international transition models, including France’s “Jump” program and a U.S. six-step system, both starting around age 12 and gradually introducing adult services over time. In the MPS context, she stresses the need to address two patient groups: those with and without cognitive involvement. While cognitively intact patients may navigate independence and adult planning, those with severe cognitive deficits rely heavily on their families and require sensitive, ongoing support.

She emphasizes that successful transition hinges on education, early planning, psychological support, and family involvement. Pediatricians play a central role in preparing both patients and adult care teams, and adult physicians must be engaged well in advance to foster trust and familiarity. Above all, Dr. Lampe advocates for flexible, patient-centered approaches that respect the individual needs, developmental status, and preferences of each MPS patient.