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
Authors: Resseguier Noémie , Rosso-Delsemme Natacha , Beltran Anzola Any , Baumstarck Karine , Milien Vanessa , Ardillon Laurent , Bayart Sophie , Berger Claire , Bertrand Marie-Anne , Biron-Andreani Christine , Borel-Derlon Annie , Castet Sabine , Chamouni Pierre , Claeyssens Donadel Ségolène , De Raucourt Emmanuelle , Desprez Dominique , Falaise Céline , Frotscher Birgit , Gay Valérie , Goudemand Jenny , Gruel Yves , Guillet Benoît , Harroche Annie , Hassoun Abel , Huguenin Yoann , Lambert Thierry , Lebreton Aurélien , Lienhart Anne , Martin Michèle , Meunier Sandrine , Monpoux Fabrice , Mourey Guillaume , Negrier Claude , Nguyen Philippe , Nyombe Placide , Oudot Caroline , Pan-Petesch Brigitte , Polack Benoît , Rafowicz Anne , Rauch Antoine , Rivaud Delphine , Schneider Pascale , Spiegel Alexandra , Stoven Cecile , Tardy Brigitte , Trossaërt Marc , Valentin Jean-Baptiste , Vanderbecken Stéphane , Volot Fabienne , Voyer-Ebrard Annelise , Wibaut Bénédicte , Leroy Tanguy , Sannie Thomas , Chambost Hervé , Auquier Pascal 
Published: July 2018

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.