Clinical trial NCT05004701: Impact of the Lockdown Due to the COVID Pandemic in French Wilson's Disease Patients
Official title: Impact of the Lockdown Due to the COVID Pandemic in French Wilson's Disease Patients
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Wilson's Disease and COVID-19
Lead sponsor: Fondation Ophtalmologique Adolphe de Rothschild
Sponsor class: NETWORK
First posted: 2021-08-13
Enrollment: 120
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patient with Wilson's disease followed by doctors from the Wilson national reference centers between the first confinement due to COVID and the end of the last lockdown

Brief summary: Wilson's disease is a rare genetic disorder that causes copper to build up in the body. This overload is initially localized in the liver and the brain, but can spread throughout the body and cause systemic damage if copper chelation or zinc salt therapy is not implemented quickly. Treatment should be taken daily and continued all the lifelong.

Patients usually have a follow-up (clinical examination, ultrasound of the liver, blood and urine samples) every six months in the maintenance phase of the disease and more frequently in the event of destabilization of the disease which requires adaptation of the doses of treatment or when initiating treatment. Some patients also benefit from regular psychological follow-up and patients with a disabling neurological form may have physiotherapy, and speech therapy. The Covid 19 pandemic has imposed the lockdown of the entire population, including patients with Wilson's disease. The non-urgent care of these patients was therefore suspended. Medical consultations and paramedical care (physiotherapy, speech therapy, psychologist, etc.) have been postponed. Only very urgent hospitalizations in the event of imbalance of their illness with life-threatening risk were maintained.

Wilson's disease patients could in this situation be particularly anxious and present disturbances of their quality of life. The psychiatric consequences could not be limited to the current period but also concern long-term patients, in particular if there is a worsening of the disease. The consequences of inactivity and the end of specific treatments (physiotherapy and speech therapy) could also be sources of aggravation. The behavioral and cognitive characteristics of the disease and the major difficulties in adherence to treatment already observed in this chronic disease, may suggest a repercussion of the pandemic in this population.

The consequences of the COVID pandemic in these fragile patients with a rare disease must be assessed. It will be important to look at the consequences of the lockdown on the adherence to treatment and on the course of the disease.

Detailed description: During the lockdown period, the first part of a detailed questionnaire is proposed to Wilson's disease patients. The second part of the questionnaire will be carried out away from the lockdown This is an observational, French multicenter study, of an uncontrolled cohort of Wilsonian patients followed by doctors from the Wilson national reference centers

Intervention 1:

Other: Questionnaires The questionnaires are WHOQOL-BREF (quality of life) Compliance questionnaire with a Visual Analog Scale (VAS) from 0 to 100 Hospital Anxiety and Depression scale (HAD)

Eligibility criteria: Inclusion Criteria:

* Wilson's disease patient
* Patient over 12 years of age
* Patient followed by doctors from the Wilson national reference centers (Paris, Lyon, Toulouse, Besançon, Bordeaux, Rennes, Marseilles, Tours, Lille )
* Patients hospitalized or seen in outpatient consultation (or teleconsultation) during the first confinement and at the end of the last one
* Non-opposition of participation in the study and for minor patient non-opposition of one of the two holders of the exercise of parental authority

Exclusion criteria :

* Patient subject to a legal protection (tutorship)
* Patient or patient representative not wishing to answer questions

Primary outcomes:
measure: Quality of life was measured using the World Health Organization Quality of Life-BREF (WHOQOL-BREF) questionnaire
timeFrame: 6 months
Secondary outcomes:
Other outcomes:
Locations:
city: Paris
country: France
facility: Fondation A De Rothschild
Study officials:
affiliation: Fondation A. de Rothschild
name: Aurélia Poujois, MD
role: PRINCIPAL_INVESTIGATOR
References: