ArticleMolecular genetics and metabolism reports2019
Transition of patients with mucopolysaccharidosis from paediatric to adult care.
Article in Molecular genetics and metabolism reports, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
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The trial behind it
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Who cites it
20 citing papers in PubMed, 34 citations in OpenAlex.
- Transition Care for Young People with Rare Bone and Mineral Conditions: A Scoping Review.Advances in therapy · 2026Article
- An international observational study on transition of care from paediatric to adult services for patients with mucopolysaccharidosis II.BMC health services research · 2026Observational
- Modelling the Transference of Paediatric Patients with Inborn Errors of Metabolism to Adult Hospitals: Clinical Experience.Journal of clinical medicine · 2025Article
- Transition in inherited metabolic diseases: the dietitians, pediatricians and adult physicians' point of view: the results of an Italian survey.Orphanet journal of rare diseases · 2025Article
- Transition of Care for Pediatric Neurologic Disorders - Are We There Yet?Annals of Indian Academy of Neurology · 2025Article
- Transition of patients with Gaucher disease type 1 from pediatric to adult care: results from two international surveys of patients and health care professionals.Frontiers in pediatrics · 2024Article
- Letting go/ moving on: A scoping review of relational effects on transition to adult care.Health care transitions · 2024Article
- Role of primary care for individuals with childhood-onset neurologic conditions.Health care transitions · 2024Article
- Understanding the challenges, unmet needs, and expectations of mucopolysaccharidoses I, II and VI patients and their caregivers in France: a survey study.Orphanet journal of rare diseases · 2022Article
- Healthcare Transition in Inherited Metabolic Disorders-Is a Collaborative Approach between US and European Centers Possible?Journal of clinical medicine · 2022Review
- Maple syrup urine disease decompensation misdiagnosed as a psychotic event.Molecular genetics and metabolism reports · 2022Article
- Consensus statement on enzyme replacement therapy for mucopolysaccharidosis IVA in Central and South-Eastern European countries.Orphanet journal of rare diseases · 2022Article
- The landscape of Mucopolysaccharidosis in Southern and Eastern European countries: a survey from 19 specialistic centers.Orphanet journal of rare diseases · 2022Article
- Transition from child to adult health care for patients with lysosomal storage diseases in France: current status and priorities-the TENALYS study, a patient perspective survey.Orphanet journal of rare diseases · 2022Article
- Unmet Cardiac Clinical Needs in Adult Mucopolysaccharidoses.Frontiers in cardiovascular medicine · 2022Review
- Non-cardiac Manifestations in Adult Patients With Mucopolysaccharidosis.Frontiers in cardiovascular medicine · 2022Review
- Social and medical needs of rare metabolic patients: results from a MetabERN survey.Orphanet journal of rare diseases · 2021Review
- The Impact of Excluding Nonrandomized Studies From Systematic Reviews in Rare Diseases: "The Example of Meta-Analyses Evaluating the Efficacy and Safety of Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis".Frontiers in molecular biosciences · 2021Review
- Challenges in Transition From Childhood to Adulthood Care in Rare Metabolic Diseases: Results From the First Multi-Center European Survey.Frontiers in medicine · 2021Article
- Critical clinical situations in adult patients with Mucopolysaccharidoses (MPS).Orphanet journal of rare diseases · 2020Article
Corrections and comments
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Authors and funding
9 authors at 5 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Mucopolysaccharidoses (MPS) are rare disorders associated with enzyme deficiencies, resulting in glycosaminoglycan (GAG) accumulation in multiple organ systems. As patients increasingly survive to adulthood, the need for a smooth transition into adult care is essential. Using case studies, we outline strategies and highlight the challenges of transition, illustrating practical solutions that may be used to optimise the transition process for patients with MPS disorders. Seven MPS case studies were provided by four European inherited metabolic disease centres; six of these patients transferred to an adult care setting and the final patient remained under paediatric care. Of the patients who transferred, age at the start of transition ranged between 14 and 18 years (age at transfer ranged from 16 to 19 years). While there were some shared features of transition strategies, they varied in duration, the healthcare professionals involved and the management of adult patients with MPS. Challenges included complex symptoms, patients' unwillingness to attend appointments with unfamiliar team members and attachment to paediatricians. Challenges were resolved by starting transition at an early age, educating patients and families, and providing regular communication with and reassurance to the patient and family. Sufficient time should be provided to allow patients to understand their responsibilities in the adult care setting while feeling assured of continued support from healthcare professionals. The involvement of a coordinated multidisciplinary team with expertise in MPS is also key. Overall, transition strategies must be comprehensive and individualised to patients' needs.
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Registered trials
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