Evidence map›Paper›PMID 41718944›Full record

Observational studyAdvances in therapy2026

Patient Burden in the Treatment of Wilson Disease in the United States: An Analysis of Real-World Health Insurance Claims Data from the Komodo database.

Peter Hedera, Megan Teynor, Carey Strader, Halley Costantino, Michael Schultze, Divine Akumo, Karl Heinz Weiss

Abstract readObservational Study
PubMed Publisher
In one paragraph

Observational study in Advances in therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–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. Article
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

7 authors.

Peter HederaDepartment of Neurology, University of Louisville, Louisville, KY, USA.ORCID http://orcid.org/0000-0003-2699-4085
Megan TeynorAlexion, AstraZeneca Rare Disease, Boston, USA.
Carey StraderOracle Life Sciences, Oncology and Specialty Therapeutics, 2800 Rock Creek Pkwy, North Kansas City, MO, 64117, USA. carey.strader@oracle.com.ORCID http://orcid.org/0009-0003-2522-7949
Halley CostantinoOracle Life Sciences, Oncology and Specialty Therapeutics, 2800 Rock Creek Pkwy, North Kansas City, MO, 64117, USA.ORCID http://orcid.org/0009-0000-6658-6057
Michael SchultzeZEG - Berlin Center for Epidemiology and Health Research GmbH, Berlin, Germany.ORCID http://orcid.org/0009-0002-1494-5446
Divine AkumoZEG - Berlin Center for Epidemiology and Health Research GmbH, Berlin, Germany.ORCID http://orcid.org/0009-0005-8366-9205
Karl Heinz WeissInternal Medicine, Krankenhaus Salem Der Evangelischen Stadtmission, Heidelberg, Germany.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionWilson disease (WD) is a rare inherited disorder that causes copper accumulation and can be fatal if untreated. This study used real-world data from the US Komodo Health claims database to describe healthcare resource utilization (HCRU) and evaluate direct economic costs among patients with WD.

methodsThis retrospective observational study identified patients with WD using ICD-9/10 codes (excluding Menkes disease) between 2016 and 2019, with data spanning 2012-2020. Sociodemographic characteristics, HCRU, and costs were analyzed using SPSS v23, SAS v9.4, and R v3.6.0. The study was approved by Pearl Pathways IRB (#20-KANT-224).

resultsA total of 2115 patients with prevalent WD, including 360 ever-treated (with reimbursable WD prescriptions), were identified. During the 2-year follow-up, about 25% were hospitalized, with a mean stay of 9 days, and most visited the ER three times annually. Hepatic patients with WD were more likely to undergo liver biopsy or transplant but had fewer home health visits and less use of assistive mobility devices. Annual liver transplant costs averaged $9094.72 ± 8110.23 per prevalent WD patient and $10,147.98 ± 7030.83 per ever-treated patient. Mean annual costs per prevalent versus ever-treated patients with WD were inpatient ($716.52 ± 2675.06 vs. $252.75 ± 333.39), pharmacy ($270.35 ± 1348.79 vs. $1284.51 ± 2994.85), and outpatient ($73.93 ± 156.89 vs. $60.82 ± 62.17), respectively. Pharmacy costs for adherent patients averaged $157,505.28 for any medication, $252,617.11 for D-penicillamine, $189,328.52 for trientine, and $1574.91 for zinc. Among ever-treated patients with WD, respective costs were lower at $85,117.60, $123,190.73, $100,017.20, and $820.35.

conclusionEstimated annual HCRU and treatment costs for patients with WD were lower than previously reported. These findings provide updated real-world insights into the economic burden of WD and highlight the cost implications of medication adherence in managing this rare disorder in the USA.

Indexed as

Cost of IllnessHealth Care CostsHepatolenticular DegenerationPatient Acceptance of Health CareAdolescentAdultChelating AgentsChildDatabases, FactualFemaleHospitalizationHumansMaleMiddle AgedRetrospective StudiesUnited StatesChelating AgentsHealthcare resource utilizationHealthcare services and costsPatient’s adherenceWilson disease

Identifiers

PMID41718944

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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.