ArticleJournal of multidisciplinary healthcare2026
Clinical Practice of Multidisciplinary Team-Guided Comprehensive Management for Pediatric Patients with Fabry Disease: A Single-Center Case Series.
Article in Journal of multidisciplinary healthcare, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Purpose: This study aimed to illustrate the practical application and preliminary outcomes of a multidisciplinary team (MDT) approach in managing pediatric Fabry disease (FD), and provide a reference for the clinical management of this rare disease. Methods: This single-center, prospective, observational case series was conducted at Beijing Children's Hospital. Between March 2021 and February 2024, five pediatric patients with FD who were managed by a dedicated MDT were enrolled. The MDT operated under a model of "identification and initiation in outpatient clinics, overall management by the core team, and specialist consultation as needed." The workflow covered a dual-path diagnostic pathway (MDT-initiated or external referral), pedigree screening, baseline assessment, individualized treatment, and long-term follow-up. Results: Five pediatric FD probands were enrolled, with four diagnosed through the MDT-initiated pathway and one via external referral. For the four newly diagnosed patients, the MDT achieved a definitive diagnosis within 15-30 days of its engagement, despite a prior diagnostic odyssey of 2.0-5.9 years. Pedigree screening identified an asymptomatic sibling, enabling pre-symptomatic diagnosis. All patients commenced enzyme replacement therapy (ERT, agalsidase α) and did not develop adverse events. Through MDT coordination, they currently maintain continuous ERT at medical institutions with a travel time of 20 minutes to 2 hours. MDT-guided pain treatment, primarily with oxcarbazepine, effectively controlled neuropathic pain in most cases and improved quality of life. Psychosocial support alleviated family burdens, achieving treatment cost reimbursement rates of 60%-85%. At one-year follow-up, symptomatic improvement and significant reductions in globotriaosylsphingosine (Lyso-GL-3) levels were observed. Conclusion: The structured MDT approach facilitated accelerated diagnosis, early intervention, and comprehensive care in this pediatric FD cohort, yielding positive short-term outcomes and providing a practical reference for rare disease management.
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