Parkinson's disease in Gaucher disease patients: what's changing in the counseling and management of patients and their relatives?


Journal

Orphanet journal of rare diseases
ISSN: 1750-1172
Titre abrégé: Orphanet J Rare Dis
Pays: England
ID NLM: 101266602

Informations de publication

Date de publication:
23 09 2020
Historique:
received: 12 05 2020
accepted: 07 09 2020
entrez: 24 9 2020
pubmed: 25 9 2020
medline: 19 5 2021
Statut: epublish

Résumé

How to address the counseling of lifetime risk of developing Parkinson's disease in patients with Gaucher disease and their family members carrying a single variant of the GBA1 gene is not yet clearly defined. In addition, there is no set way of managing Gaucher disease patients, taking into account the possibility that they may show features of Parkinson's disease. Starting from an overview on what has recently changed in our knowledge on this issue and grouping the experiences of healthcare providers of Gaucher disease patients, we outline a path of counseling and management of Parkinson's disease risk in Gaucher disease patients and their relatives. The approach proposed here will help healthcare providers to communicate Parkinson's disease risk to their patients and will reduce the possibility of patients receiving inaccurate information from inadequate sources. Furthermore, this resource will help to empower healthcare providers to identify early signs and/or symptoms of Parkinson's disease and decide when to refer these patients to the neurologist for appropriate specific therapy and follow-up.

Sections du résumé

BACKGROUND
How to address the counseling of lifetime risk of developing Parkinson's disease in patients with Gaucher disease and their family members carrying a single variant of the GBA1 gene is not yet clearly defined. In addition, there is no set way of managing Gaucher disease patients, taking into account the possibility that they may show features of Parkinson's disease.
METHODS
Starting from an overview on what has recently changed in our knowledge on this issue and grouping the experiences of healthcare providers of Gaucher disease patients, we outline a path of counseling and management of Parkinson's disease risk in Gaucher disease patients and their relatives.
CONCLUSION
The approach proposed here will help healthcare providers to communicate Parkinson's disease risk to their patients and will reduce the possibility of patients receiving inaccurate information from inadequate sources. Furthermore, this resource will help to empower healthcare providers to identify early signs and/or symptoms of Parkinson's disease and decide when to refer these patients to the neurologist for appropriate specific therapy and follow-up.

Identifiants

pubmed: 32967694
doi: 10.1186/s13023-020-01529-y
pii: 10.1186/s13023-020-01529-y
pmc: PMC7510137
doi:

Substances chimiques

Glucosylceramidase EC 3.2.1.45

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

262

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Auteurs

Maja Di Rocco (M)

Unit of Rare Diseases, Department of Pediatrics, IRCCS Istituto Giannina Gaslini, Via Gerolamo Gaslini 3, 16147, Genoa, Italy. Majadirocco@gaslini.org.

Alessio Di Fonzo (A)

Neuroscience Section, Department of Pathophysiology and Transplantation, Dino Ferrari Center, IRCCS Foundation Ca' Granda Ospedale Maggiore Policlinico, University of Milan Neurology Unit, Milan, Italy. alessio.difonzo@policlinico.mi.it.

Antonio Barbato (A)

Department of Clinical Medicine and Surgery, "Federico II" University Hospital, Naples, Italy.

Maria Domenica Cappellini (MD)

Department of Medical Science and Community, University of Milan, Milan, Italy.

Francesca Carubbi (F)

Regional Referral Centre for Lysosomal Storage Diseases, Division of Internal Medicine and Metabolism, Civil Hospital, AOU of Modena, University of Modena and Reggio Emilia, Modena, Italy.

Fiorina Giona (F)

Department of Translational and Precision Medicine, Sapienza University, Rome, Italy.

Gaetano Giuffrida (G)

Regional Reference Center for Rare Diseases, Clinical Division of Haematology and Transplantation, PO Ferrarotto Hospital, Azienda Ospedaliera-Universitaria Policlinico-Vittorio Emanuele, Catania, Italy.

Silvia Linari (S)

Center for Bleeding Disorders and Coagulation, Careggi University Hospital, Florence, Italy.

Andrea Pession (A)

Pediatric Unit, Department of Medical and Surgical Sciences, S. Orsola Hospital, University of Bologna, Bologna, Italy.

Antonella Quarta (A)

Center for Microcythemia, Iron Metabolism disorders, Gaucher disease-Hematology and Transplantation Unit, "A. Perrino" Hospital, Brindisi, Italy.

Maurizio Scarpa (M)

Regional Coordinating Center for Rare Disease, University Hospital of Udine, Udine, Italy.

Marco Spada (M)

Department of Pediatrics, AOU Città della Salute e della Scienza di Torino, University of Torino, Torino, Italy.

Pietro Strisciuglio (P)

Department of Translational Medical Sciences, Section of Pediatrics, Federico II University, 80131, Naples, Italy.

Generoso Andria (G)

Professor Emeritus "Federico II" University Hospital, Naples, Italy.

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Classifications MeSH