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Child's Nervous System

, Volume 34, Issue 9, pp 1759–1765 | Cite as

Spinal cord issues in adult patients with MPS: transition of care survey

  • Kemel A. Ghotme
  • Fernando Alvarado-Gomez
  • Christina Lampe
  • Klane K. White
  • Martha Solano-Villareal
  • Roberto Giugliani
  • Paul R. Harmatz
Original Paper
  • 46 Downloads

Abstract

Purpose

This study aims to raise awareness of the need for research and appropriate guidelines for managing spinal cord issues in adult patients with mucopolysaccharidosis (MPS) and transition of these patients from pediatric to adult care.

Methods

Pediatric/adult neurosurgeons, orthopedic spine surgeons, and treating physicians with expertise in metabolic disorders and spinal cord issues were invited to complete a survey to assess their experience with spinal cord problems in MPS and their opinion on transitioning routes from pediatric to adult care.

Results

Twenty specialists completed the survey; 16 had treated spinal cord issues in patients with MPS. Foramen magnum and cervical stenosis (87%), atlanto-axial instability (67%), and lumbar spine instability (33%) were the main spinal cord issues encountered; 28% had treated adult patients for one or more spinal cord issues. In 40% of cases, this concerned an intervention or procedures performed during childhood. The main specialist responsible for the care of adult patients with MPS differed considerably between institutions and included both pediatric and adult specialists (30% pediatric neurosurgeons, 10% pediatric spine orthopedic surgeons, 30% adult spine neurosurgeons, 20% general adult surgeons). The preferred option (> 50%) for the transition of care was an interdisciplinary team of pediatric and adult specialists.

Conclusions

Further work needs to be done to address problems of managing spinal cord issues in adult patients with MPS. Currently, the responsibility for the care of patients with MPS with spinal cord issues is inconsistent. The best strategy for transitioning these patients from pediatric to adult care is likely an interdisciplinary approach.

Keywords

Mucopolysaccharidoses Spinal cord Transition to adult care Survey 

Notes

Acknowledgements

The authors are grateful to Ismar Healthcare NV for their assistance in the writing of this manuscript, which was funded by BioMarin Pharmaceutical Inc.

Compliance with ethical standards

Conflict of interest

The writing of this manuscript was funded by BioMarin Pharmaceutical Inc.

Dr. Ghotme has received speaking fees, honoraria, and travel support to attend scientific meetings from BioMarin Pharmaceutical. The survey was conducted independently and did not have external financial support.

Dr. Alvarado reports no conflicts of interest concerning this manuscript.

Dr. Lampe has received travel support, speaker fees, and honoraria from Alexion, Actelion, BioMarin, Genzyme Sanofi, and Shire.

Dr. White has received travel support, research support, and honoraria from BioMarin, travel support from Medicrea, and royalties from UptoDate.com.

Dr. Solano Villareal has received speaking fees, honoraria, and travel support to attend scientific meetings from BioMarin Pharmaceutical.

Prof. Giugliani declares board membership for Amicus, BioMarin, Lysogene, Sanofi-Genzyme, and Shire; consultancy for BioMarin, Sanofi-Genzyme, and Shire; grants from Actelion, BioMarin, Sanofi-Genzyme, and Shire; payments for lectures from Actelion, BioMarin, Sanofi-Genzyme, and Shire; and payments for travel/accommodation/meeting expenses from Actelion, Amicus, BioMarin, Sanofi-Genzyme, and Shire.

Dr. Harmatz declares board membership for BioMarin and PTC; consultancy for BioMarin, Shire, Genzyme, PTC, Chiesi, Armagen, Inventiva, REGENXBIO, Sangamo, and Alexion; grants from BioMarin; payments for lectures from BioMarin, Shire, Genzyme, Alexion, and PTC; and payments for travel/accommodation/meeting expenses from BioMarin, Genzyme, Chiesi, and Armagen.

Ethical approval

This article does not contain any studies with human participants or animals performed by any of the authors.

Supplementary material

381_2018_3834_MOESM1_ESM.docx (17 kb)
Online Resource 1 Survey questions. (DOCX 16 kb)
381_2018_3834_MOESM2_ESM.docx (39 kb)
Online Resource 2 Main professional activity of survey participants. (DOCX 38 kb)

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Copyright information

© Springer-Verlag GmbH Germany, part of Springer Nature 2018

Authors and Affiliations

  1. 1.Translational Neuroscience Research Lab, Faculty of MedicineUniversidad de La SabanaChiaColombia
  2. 2.Neurosurgery UnitFundación Santafé de BogotáBogotaColombia
  3. 3.Department of Orthopedics, Spine Surgery SectionFundación Santafé de BogotáBogotaColombia
  4. 4.Spine Surgery SectionInstituto de Ortopedia Infantil RooseveltBogotaColombia
  5. 5.Center for Rare Diseases, Clinic for Children and AdolescentsHelios Dr. Horst Schmidt KlinikenWiesbadenGermany
  6. 6.Department of Orthopedics and Sport MedicineUniversity of Washington School of Medicine and Seattle Children’s HospitalSeattleUSA
  7. 7.Fundacion CardioinfantilBogotaColombia
  8. 8.Med Genet Serv HCPA, Dep Genet UFRGS & INAGEMPPorto AlegreBrazil
  9. 9.UCSF Benioff Children’s Hospital OaklandOaklandUSA

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