Management of sleep-disordered breathing in achondroplasia: guiding principles of the European Achondroplasia Forum

Abstract Due to the craniofacial anatomy of people with achondroplasia, sleep-disordered breathing (SDB) occurs more frequently than in the average stature population. SDB, which comprises obstructive sleep apnoea (OSA), more rarely central sleep apnoea (CSA), and nocturnal alveolar hypoventilation...

Full description

Saved in:
Bibliographic Details
Main Authors: Brigitte Fauroux, Moeenaldeen AlSayed, Tawfeg Ben-Omran, Silvio Boero, Mieke Boon, Valérie Cormier-Daire, Svein Fredwall, Encarna Guillen-Navarro, Melita Irving, Philip Kunkel, Núria Madureira, Mohamad Maghnie, Josef Milerad, Klaus Mohnike, Geert Mortier, Lino Nobili, Zagorka Pejin, Marco Sessa, Sérgio B. Sousa
Format: Article
Language:English
Published: BMC 2025-05-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Online Access:https://doi.org/10.1186/s13023-025-03717-0
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1849309758296686592
author Brigitte Fauroux
Moeenaldeen AlSayed
Tawfeg Ben-Omran
Silvio Boero
Mieke Boon
Valérie Cormier-Daire
Svein Fredwall
Encarna Guillen-Navarro
Melita Irving
Philip Kunkel
Núria Madureira
Mohamad Maghnie
Josef Milerad
Klaus Mohnike
Geert Mortier
Lino Nobili
Zagorka Pejin
Marco Sessa
Sérgio B. Sousa
author_facet Brigitte Fauroux
Moeenaldeen AlSayed
Tawfeg Ben-Omran
Silvio Boero
Mieke Boon
Valérie Cormier-Daire
Svein Fredwall
Encarna Guillen-Navarro
Melita Irving
Philip Kunkel
Núria Madureira
Mohamad Maghnie
Josef Milerad
Klaus Mohnike
Geert Mortier
Lino Nobili
Zagorka Pejin
Marco Sessa
Sérgio B. Sousa
author_sort Brigitte Fauroux
collection DOAJ
description Abstract Due to the craniofacial anatomy of people with achondroplasia, sleep-disordered breathing (SDB) occurs more frequently than in the average stature population. SDB, which comprises obstructive sleep apnoea (OSA), more rarely central sleep apnoea (CSA), and nocturnal alveolar hypoventilation (NH), may present at any age in patients with achondroplasia. Untreated SDB is associated with neurocognitive dysfunction, cardiovascular, and metabolic complications in children and adults. There continues to be debate on the optimal assessment and management of SDB in achondroplasia. To help address this, the European Achondroplasia Forum (EAF), a network of clinicians and patient advocates representative of the achondroplasia clinical community, organised a virtual workshop in October 2023 to scrutinise, vote and agree upon five guiding principles for managing SDB in achondroplasia. This workshop was attended by 40 healthcare professionals, including clinical geneticists, general practitioners and consultants, orthodontic and orthopaedic surgeons, paediatricians, paediatric endocrinologists and pulmonologists, sleep researchers and specialists, and two patient advocacy group representatives. The five guiding principles focus on lifelong assessment and proactive management, incorporating individualised sleep studies, screening, and a stepwise approach to therapeutic management. The EAF was in favour of all guiding principles, with all achieving 100% consensus with high levels of agreement (range 8.9–9.7/10). In developing guiding principles for the management of SDB in achondroplasia, the EAF aims to facilitate optimal screening and management of SDB in infants, young children, and adults with achondroplasia.
format Article
id doaj-art-cbf99ce19ba5499b89b686711688feef
institution Kabale University
issn 1750-1172
language English
publishDate 2025-05-01
publisher BMC
record_format Article
series Orphanet Journal of Rare Diseases
spelling doaj-art-cbf99ce19ba5499b89b686711688feef2025-08-20T03:53:58ZengBMCOrphanet Journal of Rare Diseases1750-11722025-05-012011910.1186/s13023-025-03717-0Management of sleep-disordered breathing in achondroplasia: guiding principles of the European Achondroplasia ForumBrigitte Fauroux0Moeenaldeen AlSayed1Tawfeg Ben-Omran2Silvio Boero3Mieke Boon4Valérie Cormier-Daire5Svein Fredwall6Encarna Guillen-Navarro7Melita Irving8Philip Kunkel9Núria Madureira10Mohamad Maghnie11Josef Milerad12Klaus Mohnike13Geert Mortier14Lino Nobili15Zagorka Pejin16Marco Sessa17Sérgio B. Sousa18AP-HP Necker-Enfants Malades University Hospital and Paris Cité University, and EA 7330 VIFASOM, Paris UniversityKing Faisal Specialist Hospital and Research Centre in RiyadhDivision of Genetics and Genomic Medicine, Sidra Medicine & Hamad Medical CorporationIstituto Giannina GasliniDepartment of Pediatrics, University Hospital LeuvenAP-HP Necker-Enfants Malades University Hospital and Paris Cité University, and EA 7330 VIFASOM, Paris UniversityTRS National Resource Centre for Rare Disorders, Sunnaas Rehabilitation HospitalMedical Genetics Division & Pediatrics Department, Virgen de la Arrixaca University Hospital, IMIB-Pascual Parrilla, University of Murcia, CIBERER-ISCIIIGuy’s and St Thomas’ NHS Foundation TrustUniversity Medical Centre MannheimHospital Pediátrico de Coímbra, Unidade Local de Saúde de CoímbraIRCCS Istituto Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health, University of Genoa, and Pediatric Endocrinology Unit, IRCCS Giannina GasliniPaediatric Sleep Research Unit, Astrid Lindgren´S Children’s HospitalChildren’s Hospital, Otto-Von-Guericke-UniversityCenter for Human Genetics, University Hospitals Leuven and KU LeuvenIRCCS Istituto G. Gaslini, DiNOGMI, University of GenoaAP-HP Necker-Enfants Malades University Hospital and Paris Cité University, and EA 7330 VIFASOM, Paris UniversityItalian Association on AchondroplasiaHospital Pediátrico de Coímbra, Unidade Local de Saúde de CoímbraAbstract Due to the craniofacial anatomy of people with achondroplasia, sleep-disordered breathing (SDB) occurs more frequently than in the average stature population. SDB, which comprises obstructive sleep apnoea (OSA), more rarely central sleep apnoea (CSA), and nocturnal alveolar hypoventilation (NH), may present at any age in patients with achondroplasia. Untreated SDB is associated with neurocognitive dysfunction, cardiovascular, and metabolic complications in children and adults. There continues to be debate on the optimal assessment and management of SDB in achondroplasia. To help address this, the European Achondroplasia Forum (EAF), a network of clinicians and patient advocates representative of the achondroplasia clinical community, organised a virtual workshop in October 2023 to scrutinise, vote and agree upon five guiding principles for managing SDB in achondroplasia. This workshop was attended by 40 healthcare professionals, including clinical geneticists, general practitioners and consultants, orthodontic and orthopaedic surgeons, paediatricians, paediatric endocrinologists and pulmonologists, sleep researchers and specialists, and two patient advocacy group representatives. The five guiding principles focus on lifelong assessment and proactive management, incorporating individualised sleep studies, screening, and a stepwise approach to therapeutic management. The EAF was in favour of all guiding principles, with all achieving 100% consensus with high levels of agreement (range 8.9–9.7/10). In developing guiding principles for the management of SDB in achondroplasia, the EAF aims to facilitate optimal screening and management of SDB in infants, young children, and adults with achondroplasia.https://doi.org/10.1186/s13023-025-03717-0AchondroplasiaEuropean Achondroplasia ForumObstructive sleep apnoeaCentral sleep apnoeaAlveolar hypoventilationConsensus
spellingShingle Brigitte Fauroux
Moeenaldeen AlSayed
Tawfeg Ben-Omran
Silvio Boero
Mieke Boon
Valérie Cormier-Daire
Svein Fredwall
Encarna Guillen-Navarro
Melita Irving
Philip Kunkel
Núria Madureira
Mohamad Maghnie
Josef Milerad
Klaus Mohnike
Geert Mortier
Lino Nobili
Zagorka Pejin
Marco Sessa
Sérgio B. Sousa
Management of sleep-disordered breathing in achondroplasia: guiding principles of the European Achondroplasia Forum
Orphanet Journal of Rare Diseases
Achondroplasia
European Achondroplasia Forum
Obstructive sleep apnoea
Central sleep apnoea
Alveolar hypoventilation
Consensus
title Management of sleep-disordered breathing in achondroplasia: guiding principles of the European Achondroplasia Forum
title_full Management of sleep-disordered breathing in achondroplasia: guiding principles of the European Achondroplasia Forum
title_fullStr Management of sleep-disordered breathing in achondroplasia: guiding principles of the European Achondroplasia Forum
title_full_unstemmed Management of sleep-disordered breathing in achondroplasia: guiding principles of the European Achondroplasia Forum
title_short Management of sleep-disordered breathing in achondroplasia: guiding principles of the European Achondroplasia Forum
title_sort management of sleep disordered breathing in achondroplasia guiding principles of the european achondroplasia forum
topic Achondroplasia
European Achondroplasia Forum
Obstructive sleep apnoea
Central sleep apnoea
Alveolar hypoventilation
Consensus
url https://doi.org/10.1186/s13023-025-03717-0
work_keys_str_mv AT brigittefauroux managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT moeenaldeenalsayed managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT tawfegbenomran managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT silvioboero managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT miekeboon managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT valeriecormierdaire managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT sveinfredwall managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT encarnaguillennavarro managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT melitairving managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT philipkunkel managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT nuriamadureira managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT mohamadmaghnie managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT josefmilerad managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT klausmohnike managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT geertmortier managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT linonobili managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT zagorkapejin managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT marcosessa managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum
AT sergiobsousa managementofsleepdisorderedbreathinginachondroplasiaguidingprinciplesoftheeuropeanachondroplasiaforum