Evidence map›Paper›PMID 28606101›Full record

ArticleOrphanet journal of rare diseases2017

International physician survey on management of FOP: a modified Delphi study.

Maja Di Rocco, Genevieve Baujat, Marta Bertamino, Matthew Brown, Carmen L De Cunto, Patricia L R Delai, Elisabeth M W Eekhoff, Nobuhiko Haga, Edward Hsiao, Richard Keen and 3 more

Open access · goldAbstract read
In one paragraph

Article in Orphanet journal of rare diseases, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed
0.9field-weighted citation impact, top 25% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

13 citing papers in PubMed, 21 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

13 authors at 12 institutions in 10 countries.

Maja Di RoccoDepartment of Pediatrics, Unit of Rare Diseases, Giannina Gaslini Institute, Largo Gaslini 5, 16147, Genoa, Italy. majadirocco@gaslini.org.
Genevieve BaujatService of Medical Genetics CHU Paris - Hôpital Necker-Enfants Malades, 149 rue de Sèvres, 75743, Paris, France.
Marta BertaminoDepartment of Pediatrics, Unit of Rare Diseases, Giannina Gaslini Institute, Largo Gaslini 5, 16147, Genoa, Italy.
Matthew BrownInstitute of Health and Biomedical Innovation, Queensland University of Technology, Translational Research Institute, Princess Alexandra Hospital, Woolloongabba, QLD, 4069, Australia.
Carmen L De CuntoDepartment of Pediatrics, Pediatric Rheumatology Section, Hospital Italiano de Buenos Aires, Gascón 450, 1181, Ciudad Autónoma de Buenos Aires, Argentina.
Patricia L R DelaiOrthopaedic Department of Santa Casa de Misericórdia de São Paulo, School of Medicine Faculdade de Ciências Médicas da Santa Casa de São Paulo, Rua Pedro de Toledo 129 cj 121, Vila Clementino, 04039-001, São Paulo, Brazil.
Elisabeth M W EekhoffDepartment of Internal Medicine/Section Endocrinology, VU Medical Center Amsterdam, De Boelelaan 1117, 1081 HV, Amsterdam, The Netherlands.
Nobuhiko HagaDepartment of Rehabilitation Medicine Graduate School of Medicine, The University of Tokyo, 7-3-1 Hongo, Bunkyo-ku, Tokyo, 113-8655, Japan.
Edward HsiaoDepartment of Endocrinology, Faculty Practice University of California-San Francisco, 400 Parnassus Ave., San Francisco, CA, 94143-1222, USA.
Richard KeenUniversity College London Hospitals, London, NW1 2PQ, UK.
Rolf MorhartDepartment of Pediatrics Klinikum Garmisch-Partenkirchen GmbH, Auenstraße 6, 82467, Garmisch-Partenkirchen, Germany.
Robert J PignoloDepartment of Medicine, Division of Geriatric Medicine & Gerontology, Mayo Clinic College of Medicine, 200 First Street SW, Rochester, MN, USA.
Frederick S KaplanDepartment of Orthopaedic Surgery, Center for Research in FOP & Related Disorders, The Perelman School of Medicine, The University of Pennsylvania, 3737 Market Street, Philadelphia, PA, 19104, USA.
Istituto Giannina Gaslini · ITAmsterdam UMC Location Vrije Universiteit Amsterdam · NLFaculdade de Ciências Médicas da Santa Casa de São Paulo · BRGarmisch-Partenkirchen Medical Center · DEHôpital Necker-Enfants Malades · FRHospital Italiano de Buenos Aires · ARMayo Clinic · USThe University of Tokyo · JPTranslational Research Institute · AUUniversity College London · GBUniversity of California, San Francisco · USUniversity of Pennsylvania · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Fibrodysplasia ossificans progressiva (FOP), a disabling disorder of progressive heterotopic ossification (HEO), is caused by heterozygous gain-of- function mutations in Activin receptor A, type I (ACVR1, also known as ALK2), a bone morphogenetic protein (BMP) type I receptor. Presently, symptomatic management is possible, but no definitive treatments are available. Although extensive guidelines for symptomatic management are widely used, regional preferences exist. In order to understand if there was worldwide consensus among clinicians treating FOP patients, an expert panel of physicians directly involved in FOP patient care was convened. Using a modified Delphi method, broad international consensus was reached on four main topics: diagnosis, prevention of flare-ups, patient and family-centered care and general clinical management issues. This study of physician preferences provides a basis for standardization of clinical management for FOP.

Indexed as

Delphi TechniquePhysiciansActivin Receptors, Type IData CollectionGlobal HealthHumansMyositis OssificansActivin Receptors, Type IACVR1 protein, humanACVR1Fibrodysplasia ossificans progressiva

Identifiers

PMID28606101
PMCPMC5468985
OpenAlexW2627047415

What OpenQuestion holds

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LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.