Evidence map›Paper›PMID 31270551›Full record

ReviewDer Nervenarzt2019

[Non-ambulatory patients with Duchenne muscular dystrophy : Recommendations for monitoring disease progression and course of treatment].

Marina Flotats-Bastardas, Daniel Ebrahimi-Fakhari, Günther Bernert, Andreas Ziegler, Kurt Schlachter, Martin Poryo, Andreas Hahn, Sascha Meyer

Abstract readReview
PubMed Publisher
In one paragraph

Review in Der Nervenarzt, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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

1 citing paper in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
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

8 authors.

Marina Flotats-BastardasKlinik für Allgemeine Pädiatrie und Neonatologie, Sektion Neuropädiatrie, Universitätsklinikum des Saarlandes, Geb. 9, 66421, Homburg, Deutschland.
Daniel Ebrahimi-FakhariKlinik für Allgemeine Pädiatrie und Neonatologie, Sektion Neuropädiatrie, Universitätsklinikum des Saarlandes, Geb. 9, 66421, Homburg, Deutschland.
Günther BernertSozialmedizinisches Zentrum Süd, Kaiser-Franz-Josef-Spital mit Gottfried von Preyer'schem Kinderspital, Wien, Österreich.
Andreas ZieglerSektion für Neuropädiatrie und Stoffwechselmedizin, Zentrum für Kinder- und Jugendmedizin, Universitätsklinikum Heidelberg, Heidelberg, Deutschland.
Kurt SchlachterKlinik für Kinder- und Jugendheilkunde, Landeskrankenhaus Bregenz, Bregenz, Österreich.
Martin PoryoKlinik für Pädiatrische Kardiologie, Universitätsklinikum des Saarlandes, Homburg, Deutschland.
Andreas HahnAbteilung Kinderneurologie, Sozialpädiatrie und Epileptologie, Universitätskinderklinik Gießen, Gießen, Deutschland.
Sascha MeyerKlinik für Allgemeine Pädiatrie und Neonatologie, Sektion Neuropädiatrie, Universitätsklinikum des Saarlandes, Geb. 9, 66421, Homburg, Deutschland. sascha.meyer@uks.eu.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundDuchenne muscular dystrophy (DMD) is a severe X‑linked recessive neuromuscular disorder. In children without corticosteroid therapy, progressive muscular weakness is associated with loss of ambulation on average by the age of 9.5 years. OBJECTIVE, MATERIAL AND

methodsOn the basis of current guidelines, a group of experts in this field defined a number of clinical parameters and examinations that should be performed on a regular basis to assess changes over time in non-ambulant patients. RESULTS AND

conclusionTo assess function of the upper extremities the Brooke upper extremity functional rating scale or the performance of upper limb test should be used. For assessment of pulmonary function measurement of forced vital capacity (FVC) is recommended. The extent of cardiac involvement can best be evaluated using cardiac magnetic resonance imaging (MRI), measurement of the ejection fraction (EF) and the left ventricular shortening fraction (LVSF) by echocardiography. The pediatric quality of life inventory should be used for assessment of quality of life. In addition, the body mass index (BMI), the number of infections and need for in-hospital treatment as well as early detection of orthopedic problems, most importantly the development of scoliosis should be monitored. After transition from pediatric to adult care DMD patients should be primarily cared for by adult neurologists and specialists in pulmonary and cardiac medicine.

Indexed as

Muscular Dystrophy, DuchenneDisease ProgressionHumansQuality of LifeDuchenne muscular dystrophyLeft ventricular dysfunctionLoss of ambulationPulmonary dysfunctionTransition

Identifiers

What OpenQuestion holds

Textmetadata
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.