Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies (PHAREO)

May 13, 2021 updated by: RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône

Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies: a Comparison of Perceived and Observed Accessibility in the Auvergne-Rhône-Alpes Region (France)

The current treatment of people with haemophilia and other bleeding deficiencies is largely based on clotting factor replacement therapy. The injections can be repeated several times a week according to a personalized schedule. To date, medications are exclusively dispensed in hospital pharmacies to ensure traceability and safety. This retrocession imposes accessibility constraints on patients and on their caregivers, increasing the burden of the disease, particularly in the organization of personal and professional daily life. The PHAREO study aims to investigate patients' perception of accessibility to anti-haemophilia drugs in relation to an evaluation of spatial accessibility in the Auvergne-Rhône-Alpes region (France) in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.

Study Overview

Status

Unknown

Conditions

Detailed Description

Haemophilia is a rare constitutional hemorrhagic disease whose drug management is based on the use of chronic lifelong replacement therapy. For many years, the reference treatment, particularly in children, has been based on the use of anti-haemophilic drugs for prophylaxis requiring repeated injections several times a week according to a personalized schedule. In contrast, on-demand treatment is less and less used, particularly in patients with severe forms of haemophilia. Clinical and biological diagnosis, as well as the implementation and monitoring of treatments, are carried out within specialized hospital care structures affiliated with the French national reference center. Medications are dispensed as part of hospital retrocessions. This organization imposes constraints on patients and their caregivers due to their limited accessibility.

The burden related to this disease is probably due to the systematic use of specialized hospital teams such as doctors, nurses for self-injection training for example, and pharmacists. It now seems important to reflect on the evolution of patient pathways that were previously exclusively hospital-centred towards ambulatory care. To do this, various reflections need to be undertaken, including that relating to the accessibility of medication. Indeed, the monthly renewals of these chronic treatments force patients and their caregivers, as well as the parents of children, to go to the hospital, which frequently makes the organization of daily and professional life more cumbersome.

The PHAREO study aims to investigate patients' perception of accessibility to anti-haemophilia drugs in relation to an evaluation of spatial accessibility in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.

The expected benefits of the study are to have: 1) an exhaustive description of the spatial accessibility of the cohort of people living with haemophilia to anti-haemophilia medication within the Auvergne Rhône Alpes region (France) and 2) a better understanding of their needs and their perceptions regarding access to on-demand and prophylactic treatments.

Study Type

Observational

Enrollment (Anticipated)

500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Albertville, France, 73200
        • Recruiting
        • Centre Hospitalier Albertville-Moûtiers
        • Contact:
          • Thomas Gailland
      • Annecy, France, 74370
        • Recruiting
        • Centre Hospitalier Annecy Genevois
        • Contact:
          • Christelle Roch
      • Annonay, France, 07103
        • Recruiting
        • Centre Hospitalier d'Ardèche Nord
        • Contact:
          • Sophie Vernardet
      • Aubenas, France, 07200
        • Recruiting
        • Centre Hospitalier d'Ardèche Méridionale
        • Contact:
          • Alain Lenoir
      • Aurillac, France, 15000
        • Recruiting
        • Centre Hospitalier Henri Mondor d'Aurillac
        • Contact:
          • Valérie Millot
      • Belley, France, 01300
        • Recruiting
        • Centre Hospitalier de Belley
        • Contact:
          • Marie Geneste
      • Bourg St Maurice, France, 73700
        • Recruiting
        • Centre hospitalier de Bourg St Maurice
        • Contact:
          • Valérie Chaminant
      • Bourg-en-Bresse, France, 01012
        • Recruiting
        • Centre Hospitalier Fleyriat
        • Contact:
          • Stéphanie Claveranne
      • Bourgoin-Jallieu, France, 38300
        • Recruiting
        • Centre Hospitalier Pierre Oudot
        • Contact:
          • Clément Beyron
      • Bron, France, 69500
        • Recruiting
        • Groupement Hospitalier Est, Hospices Civils de Lyon
        • Contact:
          • Valérie Chamouard
      • Chambéry, France, 73000
        • Recruiting
        • Centre Hospitalier de Chambéry - Hôpital Savoie
        • Contact:
          • Elodie Bernard
      • Clermont-Ferrand, France, 63100
        • Recruiting
        • Centre Hospitalier Universitaire de Clermont-Ferrand
        • Contact:
          • Anne Boyer
      • Contamine-sur-Arve, France, 74130
        • Recruiting
        • Centre Hospitalier Alpes Leman
        • Contact:
          • Catherine Diakhate
      • Gleizé, France, 69400
        • Recruiting
        • Hôpital Nord Ouest de Villefranche-sur-Saône
        • Contact:
          • Magali Bourdelin
      • La Tronche, France, 38700
        • Recruiting
        • Centre Hospitalier Universitaire Grenoble - Alpes
        • Contact:
          • Sophie Cerena
      • Lyon, France, 69004
        • Recruiting
        • Hôpital de la Croix-Rousse, Hospices Civils de Lyon
        • Contact:
          • Anne-Claire Uhrès
      • Lyon, France, 69003
        • Recruiting
        • Hopital Edouard Herriot, Hospices Civils de Lyon
        • Contact:
          • Elodie Lanoi
      • Montbrison, France, 42600
        • Recruiting
        • Centre Hospitalier du Forez
        • Contact:
          • Hadyl Asfari
      • Montluçon, France, 03100
        • Recruiting
        • Hôpital de Montluçon
        • Contact:
          • Carole Rimpici
      • Montélimar, France, 26200
        • Recruiting
        • Centre Hospitalier de Montelimar
        • Contact:
          • Jérémy Rolain
      • Moulins, France, 03006
        • Recruiting
        • Centre Hospitalier de Moulins-Yzeure
        • Contact:
          • Emmanuel Deligeard
      • Oyonnax, France, 01100
        • Recruiting
        • Centre Hospitalier Du Haut Bugey
        • Contact:
          • Nathalie Dey
      • Privas, France, 07007
        • Recruiting
        • Centre Hospitalier des Vals d'Ardeche
        • Contact:
          • Dominique Quinard
      • Roanne, France, 42300
        • Recruiting
        • Centre hospitalier de Roanne
        • Contact:
          • Maud Rossignol
      • Romans-sur-Isère, France, 26100
        • Recruiting
        • Hopitaux Drome NOrd
        • Contact:
          • Xavier Cussoneau
      • Saint-Genis-Laval, France, 69230
        • Recruiting
        • Pharmacie Centrale, Hospices Civils de Lyon
        • Contact:
          • Isabelle Carpentier
      • Saint-Julien-en-Genevois, France, 74164
        • Recruiting
        • Centre Hospitalier Intercommunal Sud-Léman Valserine
        • Contact:
          • Alexandra Combes
      • Saint-Priest-en-Jarez, France, 42270
        • Recruiting
        • Centre Hospitalier Uuniversitaire Nord, Saint-Étienne
        • Contact:
          • Jihen Boussetta
      • Sallanches, France, 74700
        • Recruiting
        • Hôpitaux du Pays du Mont-Blanc
        • Contact:
          • Marion Filippi
      • Tarare, France, 69170
        • Recruiting
        • Hôpital Nord-Ouest Tarare-Grandris
        • Contact:
          • Christine Vray
      • Thonon-les-Bains, France, 74203
        • Recruiting
        • Hopitaux du Leman
        • Contact:
          • Denis Hardelin
      • Valence, France, 26000
        • Recruiting
        • Centre Hospitalier de Valence
        • Contact:
          • Stéphanie Bourget
      • Vichy, France, 03200
        • Recruiting
        • Centre Hospitalier de Vichy
        • Contact:
          • Jérôme Tavernier
      • Vienne, France, 38200
        • Recruiting
        • Centre Hospitalier Lucien Hussel
        • Contact:
          • Anne-Sophie Leromain

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • ADULT
  • OLDER_ADULT
  • CHILD

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

The included subjects comprise people with haemophilia or other coagulation deficiencies, with no limitation of age, treated by prophylaxis or on-demand treatment, in one of the authorized hospital pharmacies in the Auvergne-Rhone-Alpes region (France).

Description

Included:

  • Living person on the study start date and presenting with haemophilia or other coagulation deficiencies;
  • Treated with prophylaxis or on-demand anti-haemorrhagic treatments;
  • Location of medication dispensing within the hospital pharmacies of the HEsora network (Hémophilie Soins Rhône Alpes): 27 eligible hospital pharmacies in Rhône Alpes + <10 eligible hospital pharmacies in Auvergne

Excluded:

- Home delivery (not included in the study via hospital pharmacy)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To assess the perception of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment
Time Frame: Through study completion, on average 6 months.
The perception is defined by the satisfaction level of the survey participants, measured with on item in the questionnaire (Very satisfied / Rather satisfied / Rather not satisfied / Not satisfied).
Through study completion, on average 6 months.
To measure the accessibility of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment
Time Frame: Through study completion, on average 6 months.
The accessibility is measured by spatial analysis, representing the distance between the home and/or work zip codes and the hospital pharmacy zip code.
Through study completion, on average 6 months.
To compare the perception and the spatial accessibility of persons with haemophilia and other coagulation deficiencies regarding prophylactic and on-demand antihemorrhagic treatment
Time Frame: Through study completion, on average 6 months.
The mean distances between the 4-groups of satisfaction level are compared using an analysis of variance (ANOVA).
Through study completion, on average 6 months.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To determine if the severity of the disease would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time Frame: Through study completion, on average 6 months.
The satisfaction level of participants are stratified on the severity of the disease (minor, moderate, major).
Through study completion, on average 6 months.
To determine if the age of the patient would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time Frame: Through study completion, on average 6 months.
The satisfaction level of participants are stratified on the age of patient.
Through study completion, on average 6 months.
To determine if the condition of access to hospital would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time Frame: Through study completion, on average 6 months.
The satisfaction level of participants are stratified on the condition of access to hospital (relative to traffic conditions, the physical condition of the patient, the parking difficulties).
Through study completion, on average 6 months.
To determine if the level of autonomy of the participant would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time Frame: Through study completion, on average 6 months.
The satisfaction level of participants are stratified on the level of autonomy of the participant (relative to wheelchair, crutch/walking stick or other mobility aid).
Through study completion, on average 6 months.
To determine if the availability of the hospital pharmacy would be a factor for encouraging or limiting the perception of accessibility to prophylactic or on-demand anti-haemorrhagic treatments
Time Frame: Through study completion, on average 6 months.
The satisfaction level of participants are stratified on the availability of the hospital pharmacy (relative to the waiting times).
Through study completion, on average 6 months.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône

Collaborators

Hospices Civils de Lyon

Investigators

  • Study Director: Laurie Fraticelli, PhD, RESCUe-RESUVal
  • Study Director: Julie Freyssenge, PhD, RESCUe-RESUVal

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

September 21, 2020

Primary Completion (ACTUAL)

April 30, 2021

Study Completion (ANTICIPATED)

May 21, 2021

Study Registration Dates

First Submitted

February 11, 2021

First Submitted That Met QC Criteria

February 26, 2021

First Posted (ACTUAL)

March 1, 2021

Study Record Updates

Last Update Posted (ACTUAL)

May 14, 2021

Last Update Submitted That Met QC Criteria

May 13, 2021

Last Verified

February 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2020PHAREO

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Patient information is held under legal and ethical obligations of confidentiality. As the disease is rare, information should not be used or disclosed in a form that might identify a patient without his or her consent.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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