Determinants of adherence and consequences of the transition from adolescence to adulthood among young people with severe haemophilia (TRANSHEMO): study protocol for a multicentric French national observational cross-sectional study

Abstract

IntroductionSevere 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 analysisWe 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 disseminationThe 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 numberNCT02866526; Pre-results.

Health Care Transition of Adolescents and Young Adults with Haemophilia: the Situation in Germany and the Munich experience

“…Valentino et al 20 ), treatment adherence (e.g. Schrijvers et al 21 ), caregiver impact, 22 and possible studies 23 and outcome indicators of HCT programmes. 24 However, these studies together with the above-reviewed experience in HCT from other disease areas support the need for education and transition programmes, joint paediatric/ adult clinics, and special youth and transition clinics.…”

“…The study aims for results by December 2019 and will hopefully add evidence to the sparse knowledge on HCT in patients with haemophilia. 23 For AYAs with malignant and haematological diseases, the 'Working Group on Adolescents, Young Adults, and Transition (Arbeitsgemeinschaft Adoleszenten, junge Erwachsene, Transition, AjET)' has been established within the Society for Paediatric Oncology and Haematology (GPOH). This group intends to build a national, multidisciplinary network to increase awareness of the topic and to define and evaluate standards of care.…”

Cost‐effectiveness of emicizumab vs bypassing agents in the prevention of bleeding episodes in haemophilia A patients with anti‐FVIII inhibitors in France

“…Nevertheless, clinical and subclinical bleeding events may still occur even with prophylactic FVIII replacement therapy 12 . However, many PwHA avoid prophylaxis because of the short half‐life of current products and the treatment burden imposed by the need for frequent intravenous infusions 12‐14 . Such patients rely on episodic treatment of bleeding events, increasing their risk to develop long‐term complications 3 .…”

Evaluation of the U.S. Adherence Questionnaires VERITAS-PRO and VERITAS-PRN for Use in Patients with Hemophilia in the German Healthcare System

“…In 2014 alone, there were almost 20 publications on this topic according to a counting of Khair 5 ; hemophilia treaters now attach greater importance to this topic. [6][7][8] In hemophilia patients (PWH [people with hemophilia]), adherence has a major impact on their health state and thus also on the financial burden or relief of the respective healthcare systems in which PWH are cared for. 9 In a 2013 study with 80 U.S. adolescents and young adults (AYA) with moderate or severe hemophilia, it could be shown that the higher their adherence was, the less chronic pain the AYA-PWH reported.…”

“…11 Nonadherence in hemophilia treatment limits the effectiveness of the factor concentrates and contributes to suboptimal avoidance of bleeding and its long-term consequences, especially related to orthopaedic sequelae. 7 In addition, nonadherence negatively influences the prescribing behavior of hemophilia treaters and can therefore lead to a vicious circle in the relationship between hemophilia treaters and PWH. 12 Miesbach and Kalnins had identified potential risk factors for nonadherence in 397 hemophilia patients (age range: 0-80 years) from the German patient organization; they found mainly condition-related, therapy-related, and healthcare system/ healthcare team-related factors.…”