Evidence map›Paper›PMID 31718567›Full record

Observational studyBMC neurology2019

Persistence with treatment for Wilson disease: a retrospective study.

Wojciech Masełbas, Anna Członkowska, Tomasz Litwin, Maciej Niewada

Open access · goldAbstract readObservational Study
PubMed Full text at PMC Publisher
In one paragraph

Observational study in BMC neurology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
31citing papers in PubMed, 1 pooled it
4.4field-weighted citation impact, top 5% 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

31 citing papers in PubMed, 1 synthesis or guideline pooled it, 58 citations in OpenAlex.

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  20. The Role of Zinc in the Treatment of Wilson's Disease.International journal of molecular sciences · 2022
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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

4 authors at 2 institutions in 1 country.

Wojciech MasełbasDepartment of Experimental and Clinical Pharmacology, Medical University of Warsaw, Warsaw, Poland.
Anna CzłonkowskaDepartment of Experimental and Clinical Pharmacology, Medical University of Warsaw, Warsaw, Poland. czlonkow@ipin.edu.pl.ORCID http://orcid.org/0000-0002-1956-1866
Tomasz Litwin2nd Department of Neurology, Institute of Psychiatry and Neurology, Warsaw, Poland.
Maciej NiewadaDepartment of Experimental and Clinical Pharmacology, Medical University of Warsaw, Warsaw, Poland.
Medical University of Warsaw · PLInstitute of Psychiatry and Neurology · PL

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundWilson disease (WD) is genetically induced failure of copper metabolism which can be successfully treated with pharmacological agents. The prognosis for survival in most WD patients is favorable if diagnosis and anti-copper treatment are provided early. Many observations imply that persistence with drug treatment is generally low in patients with chronic diseases, which impact the treatment effectiveness, but such results are very limited in WD. The aim of our study was to assess persistence with treatment among WD patients, to analyze its effect on patient outcome and to identify factors that might be related to persistence.

methods170 newly diagnosed, symptomatic patients with WD who started treatment between 1995 and 2005 were analyzed retrospectively to assess treatment non-persistence, which was defined as at least one reported break of more than 3 months or minimum two breaks lasting longer than 2 months. Results were further analyzed according to selected clinical variables.

resultsOnly 74.1% of patients were persistent with treatment during the mean 11.7 years of follow up. Treatment persistence closely impacted positive clinical outcomes. In patients classified as persistent, improvement and lack of WD progression were observed more often compared to those classified as non-persistent (29.4 and 68.3% vs. 2.3 and 45.5%; p < 0.001, respectively). In contrast, non-persistent patients presented more often with worsening WD than persistent patients (52.3% vs. 2.4%). Type of WD treatment, gender, phenotypic presentation, adverse events and duration of treatment were not related to treatment persistence. Higher or upper/post-secondary education and a supportive family attitude towards treatment were the most important factors related to persistence.

conclusionsOne quarter of WD patients were not taking anti-copper treatment regularly and this had an important negative effect on clinical outcome. Family support played an important role in treatment persistence.

Indexed as

Drug MonitoringAdultChelating AgentsFemaleHepatolenticular DegenerationHumansMalePenicillaminePolandRetrospective StudiesZinc SulfateChelating AgentsPenicillamineZinc SulfatePersistenceTreatment outcomeWilson disease

Identifiers

PMID31718567
PMCPMC6849180
OpenAlexW2986960958

What OpenQuestion holds

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Registered trials

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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.