Evidence map›Paper›PMID 37368924›Full record

SynthesisPloS one2023

Duchenne muscular dystrophy in Italy: A systematic review of epidemiology, quality of life, treatment adherence, and economic impact.

Massimiliano Orso, Antonio Migliore, Barbara Polistena, Eleonora Russo, Francesca Gatto, Mauro Monterubbianesi, Daniela d'Angela, Federico Spandonaro, Marika Pane

Open access · goldAbstract readSystematic Review
In one paragraph

Synthesis in PloS one, 2023. 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
2.9field-weighted citation impact, top 9% 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, 19 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

9 authors at 4 institutions in 1 country.

Massimiliano OrsoC.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy.ORCID 0000-0003-0200-1202
Antonio MiglioreC.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy.
Barbara PolistenaC.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy.
Eleonora RussoMedical Department, Pfizer Italia, Rome, Italy.
Francesca GattoMedical Department, Pfizer Italia, Rome, Italy.
Mauro MonterubbianesiMedical Department, Pfizer Italia, Rome, Italy.
Daniela d'AngelaC.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy.
Federico SpandonaroC.R.E.A. Sanità (Centre for Applied Economic Research in Healthcare), Rome, Italy.
Marika PaneCentro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.
Pfizer (Italy) · ITUniversity of Rome Tor Vergata · ITSan Raffaele University of Rome · ITUniversità Cattolica del Sacro Cuore · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

objectiveThis systematic review aims to update the evidence on Duchenne muscular dystrophy (DMD) in Italy, describing the epidemiology, quality of life (QoL) of patients and caregivers, treatment adherence, and economic impact of DMD.

methodsSystematic searches were conducted in PubMed, Embase and Web of Science up to January 2023. Literature selection process, data extraction and quality assessment were performed by two independent reviewers. Study protocol was registered in PROSPERO (CRD42021245196).

resultsThirteen studies were included. The prevalence of DMD in the general population is 1.7-3.4 cases per 100,000, while the birth prevalence is 21.7-28.2 per 100,000 live male births. The QoL of DMD patients and caregivers is lower than that of healthy subjects, and the burden for caregivers of DMD children is higher than that of caregivers of children with other neuromuscular disorders. The compliance of real-world DMD care to clinical guidelines recommendations in Italy is lower than in other European countries. The annual cost of illness for DMD in Italy is € 35,000-46,000 per capita while, adding intangible costs, the total cost amounts to € 70,000.

conclusionAlthough it is a rare disease, DMD represents a significant burden in terms of quality of life of patients and their caregivers, and economic impact.

Indexed as

Muscular Dystrophy, DuchenneQuality of LifeChildEuropeHumansItalyMaleTreatment Adherence and Compliance

Identifiers

PMID37368924
PMCPMC10298760
OpenAlexW4382318617

What OpenQuestion holds

Textmetadata
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.