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

Health Services Accessibility Clinical Trial

— PHAREO  

Official title:

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04775888
• Other study ID #: 2020PHAREO
• Status: Recruiting
• Start date: September 21, 2020
• Est. completion date: May 21, 2021

Study information

• Verified date: February 2021
• Source: RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône
• Contact: Valérie Chamouard, PharmD
• Phone: +33(0)472118829
• Email: valerie.chamouard[@]chu-lyon.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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-Rhône-Alpes region (France) in order to consider, if necessary, ways of improving the pathway for patients and their caregivers.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: May 21, 2021
• Est. primary completion date: April 30, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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)

Related Conditions & MeSH terms

• Blood Coagulation Factor Deficiencies
• Coagulation Protein Disorders
• Health Services Accessibility
• Hemophilia A

Locations

Country	Name	City	State
France	Centre Hospitalier Albertville-Moûtiers	Albertville
France	Centre Hospitalier Annecy Genevois	Annecy
France	Centre Hospitalier d'Ardèche Nord	Annonay
France	Centre Hospitalier d'Ardèche Méridionale	Aubenas
France	Centre Hospitalier Henri Mondor d'Aurillac	Aurillac
France	Centre Hospitalier de Belley	Belley
France	Centre hospitalier de Bourg St Maurice	Bourg St Maurice
France	Centre Hospitalier Fleyriat	Bourg-en-Bresse
France	Centre Hospitalier Pierre Oudot	Bourgoin-Jallieu
France	Groupement Hospitalier Est, Hospices Civils de Lyon	Bron
France	Centre Hospitalier de Chambéry - Hôpital Savoie	Chambéry
France	Centre Hospitalier Universitaire de Clermont-Ferrand	Clermont-Ferrand
France	Centre Hospitalier Alpes Léman	Contamine-sur-Arve
France	Hôpital Nord Ouest de Villefranche-sur-Saône	Gleizé
France	Centre Hospitalier Universitaire Grenoble - Alpes	La Tronche
France	Hôpital de la Croix-Rousse, Hospices Civils de Lyon	Lyon
France	Hôpital Edouard herriot, Hospices Civils de Lyon	Lyon
France	Centre Hospitalier du Forez	Montbrison
France	Centre Hospitalier de Montélimar	Montélimar
France	Hôpital de Montluçon	Montluçon
France	Centre Hospitalier de Moulins-Yzeure	Moulins
France	Centre Hospitalier Du Haut Bugey	Oyonnax
France	Centre Hospitalier des Vals d'Ardeche	Privas
France	Centre Hospitalier de Roanne	Roanne
France	Hôpitaux Drôme Nord	Romans-sur-Isère
France	Pharmacie Centrale, Hospices Civils de Lyon	Saint-Genis-Laval
France	Centre Hospitalier Intercommunal Sud-Léman Valserine	Saint-Julien-en-Genevois
France	Centre Hospitalier Uuniversitaire Nord, Saint-Étienne	Saint-Priest-en-Jarez
France	Hôpitaux du Pays du Mont-Blanc	Sallanches
France	Hôpital Nord-Ouest Tarare-Grandris	Tarare
France	Hopitaux du Léman	Thonon-les-Bains
France	Centre Hospitalier de Valence	Valence
France	Centre Hospitalier de Vichy	Vichy
France	Centre Hospitalier Lucien Hussel	Vienne

Sponsors (2)

Lead Sponsor	Collaborator
RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône	Hospices Civils de Lyon

Country where clinical trial is conducted

France, 

References & Publications (2)

Leroy V, Freyssenge J, Renard F, Tazarourte K, Négrier C, Chamouard V. Access to treatment among persons with hemophilia: A spatial analysis assessment in the Rhone-Alpes region, France. J Am Pharm Assoc (2003). 2019 Nov - Dec;59(6):797-803. doi: 10.1016/j.japh.2019.07.006. Epub 2019 Aug 9. — View Citation

Susen S, Gruel Y, Godier A, Harroche A, Chambost H, Lasne D, Rauch A, Roullet S, Fontana P, Goudemand J, de Maistre E, Chamouard V, Wibaut B, Albaladejo P, Négrier C. Management of bleeding and invasive procedures in haemophilia A patients with inhibitor treated with emicizumab (Hemlibra(®) ): Proposals from the French network on inherited bleeding disorders (MHEMO), the French Reference Centre on Haemophilia, in collaboration with the French Working Group on Perioperative Haemostasis (GIHP). Haemophilia. 2019 Sep;25(5):731-737. doi: 10.1111/hae.13817. Epub 2019 Jul 11. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To assess the perception of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment	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.
Primary	To measure the accessibility of persons with haemophilia and other coagulation deficiencies regarding the accessibility to prophylactic and on-demand antihemorrhagic treatment	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.
Primary	To compare the perception and the spatial accessibility of persons with haemophilia and other coagulation deficiencies regarding prophylactic and on-demand antihemorrhagic treatment	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	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	The satisfaction level of participants are stratified on the severity of the disease (minor, moderate, major).	Through study completion, on average 6 months.
Secondary	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	The satisfaction level of participants are stratified on the age of patient.	Through study completion, on average 6 months.
Secondary	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	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.
Secondary	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	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.
Secondary	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	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.