Literature DB >> 34837063

International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia.

Ravi Savarirayan1, Penny Ireland2, Melita Irving3, Dominic Thompson4, Inês Alves5, Wagner A R Baratela6, James Betts7, Michael B Bober8, Silvio Boero9, Jenna Briddell8, Jeffrey Campbell8, Philippe M Campeau10, Patricia Carl-Innig11, Moira S Cheung3, Martyn Cobourne12, Valérie Cormier-Daire13, Muriel Deladure-Molla13, Mariana Del Pino14, Heather Elphick15, Virginia Fano14, Brigitte Fauroux13, Jonathan Gibbins3, Mari L Groves16, Lars Hagenäs17, Therese Hannon18, Julie Hoover-Fong16,19, Morrys Kaisermann20, Antonio Leiva-Gea21, Juan Llerena22, William Mackenzie8, Kenneth Martin23, Fabio Mazzoleni24, Sharon McDonnell18, Maria Costanza Meazzini25, Josef Milerad17, Klaus Mohnike26, Geert R Mortier27, Amaka Offiah15,28, Keiichi Ozono29, John A Phillips30, Steven Powell18, Yosha Prasad3, Cathleen Raggio31, Pablo Rosselli32, Judith Rossiter33, Angelo Selicorni34, Marco Sessa35, Mary Theroux8, Matthew Thomas18, Laura Trespedi36, David Tunkel16, Colin Wallis4, Michael Wright18, Natsuo Yasui37, Svein Otto Fredwall38,39.   

Abstract

Achondroplasia, the most common skeletal dysplasia, is characterized by a variety of medical, functional and psychosocial challenges across the lifespan. The condition is caused by a common, recurring, gain-of-function mutation in FGFR3, the gene that encodes fibroblast growth factor receptor 3. This mutation leads to impaired endochondral ossification of the human skeleton. The clinical and radiographic hallmarks of achondroplasia make accurate diagnosis possible in most patients. However, marked variability exists in the clinical care pathways and protocols practised by clinicians who manage children and adults with this condition. A group of 55 international experts from 16 countries and 5 continents have developed consensus statements and recommendations that aim to capture the key challenges and optimal management of achondroplasia across each major life stage and sub-specialty area, using a modified Delphi process. The primary purpose of this first International Consensus Statement is to facilitate the improvement and standardization of care for children and adults with achondroplasia worldwide in order to optimize their clinical outcomes and quality of life.
© 2021. Springer Nature Limited.

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Year:  2021        PMID: 34837063     DOI: 10.1038/s41574-021-00595-x

Source DB:  PubMed          Journal:  Nat Rev Endocrinol        ISSN: 1759-5029            Impact factor:   47.564


  126 in total

1.  Skeletal dysplasias.

Authors:  Ravi Savarirayan; David L Rimoin
Journal:  Adv Pediatr       Date:  2004

Review 2.  A review of imaging protocols for suspected skeletal dysplasia and a proposal for standardisation.

Authors:  Sarah G Watson; Alistair D Calder; Amaka C Offiah; Samantha Negus
Journal:  Pediatr Radiol       Date:  2015-06-17

Review 3.  A framework for the radiologic diagnosis of skeletal dysplasias and syndromes as revealed by molecular genetics.

Authors:  Jerry R Dwek
Journal:  Pediatr Radiol       Date:  2019-11-04

Review 4.  Clinical management of achondroplasia.

Authors:  M J Wright; M D Irving
Journal:  Arch Dis Child       Date:  2011-04-03       Impact factor: 3.791

Review 5.  Achondroplasia: a comprehensive clinical review.

Authors:  Richard M Pauli
Journal:  Orphanet J Rare Dis       Date:  2019-01-03       Impact factor: 4.123

Review 6.  Best practice guidelines regarding prenatal evaluation and delivery of patients with skeletal dysplasia.

Authors:  Ravi Savarirayan; Judith P Rossiter; Julie E Hoover-Fong; Melita Irving; Viviana Bompadre; Michael J Goldberg; Michael B Bober; Tae-Joon Cho; Shawn E Kamps; William G Mackenzie; Cathleen Raggio; Samantha S Spencer; Klane K White
Journal:  Am J Obstet Gynecol       Date:  2018-07-23       Impact factor: 8.661

Review 7.  Novel therapeutic approaches for the treatment of achondroplasia.

Authors:  Laurence Legeai-Mallet; Ravi Savarirayan
Journal:  Bone       Date:  2020-08-11       Impact factor: 4.398

Review 8.  Achondroplasia.

Authors:  William A Horton; Judith G Hall; Jacqueline T Hecht
Journal:  Lancet       Date:  2007-07-14       Impact factor: 79.321

Review 9.  The radiologic diagnosis of skeletal dysplasias: past, present and future.

Authors:  Amaka C Offiah; Christine M Hall
Journal:  Pediatr Radiol       Date:  2020-11-02

10.  Birth prevalence of achondroplasia: A systematic literature review and meta-analysis.

Authors:  Pamela K Foreman; Femke van Kessel; Rosa van Hoorn; Judith van den Bosch; Renée Shediac; Sarah Landis
Journal:  Am J Med Genet A       Date:  2020-08-17       Impact factor: 2.802

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  7 in total

1.  Literature review and expert opinion on the impact of achondroplasia on medical complications and health-related quality of life and expectations for long-term impact of vosoritide: a modified Delphi study.

Authors:  Ravi Savarirayan; Wagner Baratela; Thomas Butt; Valérie Cormier-Daire; Melita Irving; Bradley S Miller; Klaus Mohnike; Keiichi Ozono; Ron Rosenfeld; Angelo Selicorni; Dominic Thompson; Klane K White; Michael Wright; Svein O Fredwall
Journal:  Orphanet J Rare Dis       Date:  2022-06-13       Impact factor: 4.303

2.  Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies.

Authors:  Ravi Savarirayan; Josep Maria De Bergua; Paul Arundel; Helen McDevitt; Valerie Cormier-Daire; Vrinda Saraff; Mars Skae; Borja Delgado; Antonio Leiva-Gea; Fernando Santos-Simarro; Jean Pierre Salles; Marc Nicolino; Massimiliano Rossi; Peter Kannu; Michael B Bober; John Phillips; Howard Saal; Paul Harmatz; Christine Burren; Garrett Gotway; Terry Cho; Elena Muslimova; Richard Weng; Daniela Rogoff; Julie Hoover-Fong; Melita Irving
Journal:  Ther Adv Musculoskelet Dis       Date:  2022-03-21       Impact factor: 5.346

3.  Experiences of children and adolescents living with achondroplasia and their caregivers.

Authors:  Renée Shediac; Olga Moshkovich; Heather Gerould; Rachel Ballinger; Agnes Williams; M Alex Bellenger; Jennifer Quinn; Julie Hoover-Fong; Klaus Mohnike; Ravi Savarirayan; Dominique Kelly
Journal:  Mol Genet Genomic Med       Date:  2022-02-09       Impact factor: 2.183

4.  Optimising the diagnosis and referral of achondroplasia in Europe: European Achondroplasia Forum best practice recommendations.

Authors:  Valerie Cormier-Daire; Moeenaldeen AlSayed; Inês Alves; Joana Bengoa; Tawfeg Ben-Omran; Silvio Boero; Svein Fredwall; Catherine Garel; Encarna Guillen-Navarro; Melita Irving; Christian Lampe; Mohamad Maghnie; Geert Mortier; Sérgio B Sousa; Klaus Mohnike
Journal:  Orphanet J Rare Dis       Date:  2022-07-27       Impact factor: 4.303

5.  Optimising care and follow-up of adults with achondroplasia.

Authors:  Svein Fredwall; Yana Allum; Moeenaldeen AlSayed; Inês Alves; Tawfeg Ben-Omran; Silvio Boero; Valerie Cormier-Daire; Encarna Guillen-Navarro; Melita Irving; Christian Lampe; Mohamad Maghnie; Klaus Mohnike; Geert Mortier; Sérgio B Sousa; Michael Wright
Journal:  Orphanet J Rare Dis       Date:  2022-08-20       Impact factor: 4.303

6.  Achondroplasia in Latin America: practical recommendations for the multidisciplinary care of pediatric patients.

Authors:  Juan Llerena; Chong Ae Kim; Virginia Fano; Pablo Rosselli; Paulo Ferrez Collett-Solberg; Paula Frassinetti Vasconcelos de Medeiros; Mariana Del Pino; Débora Bertola; Charles Marques Lourenço; Denise Pontes Cavalcanti; Têmis Maria Félix; Antonio Rosa-Bellas; Norma Teresa Rossi; Fanny Cortes; Flávia Abreu; Nicolette Cavalcanti; Maria Cecilia Hervias Ruz; Wagner Baratela
Journal:  BMC Pediatr       Date:  2022-08-19       Impact factor: 2.567

7.  Foramen magnum stenosis in a 5-month-old boy with achondroplasia.

Authors:  Armaan K Malhotra; Whitney Parker; Peter B Dirks
Journal:  CMAJ       Date:  2022-09-06       Impact factor: 16.859

  7 in total

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