Introduction Severe haemophilia is a rare disease characterised by spontaneous bleeding from early childhood, which may lead to various complications, especially in joints. It is nowadays possible to avoid these complications thanks to substitutive therapies for which the issue of adherence is major. The transition from adolescence to adulthood in young people with severe haemophilia is a critical period as it is associated with a high risk of lack of adherence to healthcare, which might have serious consequences on daily activities and on quality of life.
Methods and analysis We present the protocol for a cross-sectional, observational, multicentric study to assess the differences between adolescents and young adults with severe haemophilia in France through the transition process, especially on adherence to healthcare. This study is based on a mixed methods design, with two complementary and consecutive phases, comparing data from a group of adolescents (aged 14–17 years) with those from a group of young adults (aged 20–29 years). The quantitative phase focuses on the determinants (medical, organisational, sociodemographic and social and psychosocial and behavioural factors) of adherence to healthcare (considered as a marker of the success of transition). The qualitative phase explores participants’ views in more depth to explain and refine the results from the quantitative phase. Eligible patients are contacted by the various Haemophilia Treatment Centres participating in the French national registry FranceCoag.
Ethics and dissemination The study was approved by the French Ethics Committee and by the French National Agency for Medicines and Health Products Safety (number: 2016-A01034-47). Study findings will be disseminated to the scientific and medical community in peer-reviewed journals and presented at scientific meetings. Results will be popularised to be communicated via the French association for people with haemophilia to participants and to the general public.
Trial registration number NCT02866526; Pre-results.
- young adults
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Contributors NR, ABA, KB, TL, HC, PA contributed to the design of this study and wrote this article. The investigators (LA, SB, CB, M-AB, CB-A, AB-D, SC, PC, SCD, EDR, DD, CF, BF, VG, JG, YG, BG, AH, AH, YH, TL, AL, AL, MM, SM, FM, GM, CN, PN, PN, CO, BP-P, BP, AR, AR, DR, PS, AS, CS, BT, MT, J-BV, SV, FV, AV-E, BW) of the French Haemophilia Treatment Centres contribute to enroll participants, they revised the manuscript and approved the final version. Members of steering committee (NR-D, VM, TS) contributed to the design of this study, they revised the manuscript and approved the final version.
Funding The project was funded by a grant from the French Ministry of Social Affairs and Health (Ministère des Affaires sociales et de la Santé, grant number: PREPS-15-0597) and was supported by the ’Filière MHEMO' organisation.
Disclaimer The funders were neither involved in study design nor in data collection.
Competing interests None declared.
Patient consent Not required.
Ethics approval The study was approved by the French Ethics Committee (Comité de Protection des Personnes Sud Méditerranée V) on 8 November 2016 and by the French National Agency for Medicines and Health Products Safety on 22 September 2016 (reference number ID RCB: 2016-A01034-47). Data collection, recording and analysis process was approved by the French Data Protection Authority (Commission Nationale de l’Informatique et des Libertés, authorisation number 918045).
Provenance and peer review Not commissioned; externally peer reviewed.
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