Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies
- Conditions
- Health Services AccessibilityBlood Coagulation Factor Deficiencies
- Registration Number
- NCT04775888
- Lead Sponsor
- RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vall茅e du Rh么ne
- Brief Summary
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-Rh么ne-Alpes region (France) in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.
- 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 Rh么ne Alpes region (France) and 2) a better understanding of their needs and their perceptions regarding access to on-demand and prophylactic treatments.
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- All
- Target Recruitment
- 500
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To assess the perception of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment 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).
To measure the accessibility of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment 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.
To compare the perception and the spatial accessibility of persons with haemophilia and other coagulation deficiencies regarding prophylactic and on-demand antihemorrhagic treatment 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).
- Secondary Outcome Measures
Name Time Method 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 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).
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 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).
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 Through study completion, on average 6 months. The satisfaction level of participants are stratified on the severity of the disease (minor, moderate, major).
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 Through study completion, on average 6 months. The satisfaction level of participants are stratified on the age of patient.
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 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).
Trial Locations
- Locations (34)
Centre Hospitalier Annecy Genevois
馃嚝馃嚪Annecy, France
Centre Hospitalier de Belley
馃嚝馃嚪Belley, France
Centre Hospitalier d'Ard猫che Nord
馃嚝馃嚪Annonay, France
Centre Hospitalier d'Ard猫che M茅ridionale
馃嚝馃嚪Aubenas, France
Centre Hospitalier Henri Mondor d'Aurillac
馃嚝馃嚪Aurillac, France
Centre hospitalier de Bourg St Maurice
馃嚝馃嚪Bourg St Maurice, France
Centre Hospitalier Fleyriat
馃嚝馃嚪Bourg-en-Bresse, France
Centre Hospitalier Pierre Oudot
馃嚝馃嚪Bourgoin-Jallieu, France
Centre Hospitalier de Chamb茅ry - H么pital Savoie
馃嚝馃嚪Chamb茅ry, France
Centre Hospitalier Universitaire de Clermont-Ferrand
馃嚝馃嚪Clermont-Ferrand, France
Centre Hospitalier Alpes L茅man
馃嚝馃嚪Contamine-sur-Arve, France
H么pital Nord Ouest de Villefranche-sur-Sa么ne
馃嚝馃嚪Gleiz茅, France
H么pital Edouard herriot, Hospices Civils de Lyon
馃嚝馃嚪Lyon, France
Centre Hospitalier du Forez
馃嚝馃嚪Montbrison, France
Centre Hospitalier de Moulins-Yzeure
馃嚝馃嚪Moulins, France
Centre Hospitalier Du Haut Bugey
馃嚝馃嚪Oyonnax, France
H么pital de Montlu莽on
馃嚝馃嚪Montlu莽on, France
Centre Hospitalier de Mont茅limar
馃嚝馃嚪Mont茅limar, France
Centre Hospitalier des Vals d'Ardeche
馃嚝馃嚪Privas, France
Centre Hospitalier de Roanne
馃嚝馃嚪Roanne, France
Pharmacie Centrale, Hospices Civils de Lyon
馃嚝馃嚪Saint-Genis-Laval, France
Centre Hospitalier Intercommunal Sud-L茅man Valserine
馃嚝馃嚪Saint-Julien-en-Genevois, France
H么pitaux Dr么me Nord
馃嚝馃嚪Romans-sur-Is猫re, France
Centre Hospitalier Uuniversitaire Nord, Saint-脡tienne
馃嚝馃嚪Saint-Priest-en-Jarez, France
H么pitaux du Pays du Mont-Blanc
馃嚝馃嚪Sallanches, France
Hopitaux du L茅man
馃嚝馃嚪Thonon-les-Bains, France
Centre Hospitalier de Valence
馃嚝馃嚪Valence, France
Centre Hospitalier de Vichy
馃嚝馃嚪Vichy, France
Centre Hospitalier Albertville-Mo没tiers
馃嚝馃嚪Albertville, France
Groupement Hospitalier Est, Hospices Civils de Lyon
馃嚝馃嚪Bron, France
Centre Hospitalier Universitaire Grenoble - Alpes
馃嚝馃嚪La Tronche, France
H么pital de la Croix-Rousse, Hospices Civils de Lyon
馃嚝馃嚪Lyon, France
H么pital Nord-Ouest Tarare-Grandris
馃嚝馃嚪Tarare, France
Centre Hospitalier Lucien Hussel
馃嚝馃嚪Vienne, France