Determinants and Consequences of the Transition to Adulthood for Adolescents With Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project
TRANSHEMO2
2 other identifiers
observational
75
1 country
25
Brief Summary
Haemophilia is a rare genetic disorder which, in its severe form and in the absence of treatment, can be life-threatening. Since the 1960s and the introduction of coagulation factor concentrates, the life expectancy of people with haemophilia has increased rapidly. Today, for most affected individuals, the disease is experienced as a chronic condition. The transition process enabling adolescents and young adults (AYA) with a chronic disease to move into adult life can be complex, as they must face all the changes experienced by AYA in general, combined with issues related to their chronic condition and its management. A successful transition involves a transfer of responsibility from parents to AYA regarding the management of their health condition, as well as the acquisition by AYA of knowledge, skills and autonomy. A difficult transition may lead to decreased adherence to follow-up or treatment, deterioration of overall health status and/or quality of life, or difficulties in entering adult life. In this context, the national cross-sectional study TRANSHEMO was initiated in 2017. Its objective was to compare adherence to healthcare management between two groups of AYA with severe haemophilia (adolescents \[for whom the transition is ongoing\] versus young adults \[for whom the transition may have been completed\]), and to identify the determinants of this adherence. The results showed that young adults had a lower adherence rate than adolescents (82.2% vs. 61.2%, p\<0.001). Among the determinants studied, being a young adult, having repeated at least one school year, and presenting psychological and emotional difficulties were factors that had a negative effect on adherence to healthcare management. However, the cross-sectional nature of the study represents a limitation that restricts causal inference. Complementing this project with a longitudinal study would address this limitation. The results obtained from this new study (TRANSHEMO 2) may contribute to the literature by providing insights into both the determinants of sustained adherence to healthcare management among adolescents with severe haemophilia who become young adults, and the associations between these determinants. The longitudinal design of the project will allow the establishment of a higher level of causal inference between the maintenance of adherence during the transition to adult life and its determinants, which represents a major epidemiological strength. Moreover, results addressing the issue of transition in the context of chronic diseases and derived from longitudinal studies remain scarce, making the findings of this project particularly original. Finally, haemophilia, a relatively frequent condition among rare diseases, could represent an interesting model for understanding the impact of transition in this type of pathology. Study hypothesis The extent of the reduction in adherence to healthcare during the transition process, and/or the determinants of the maintenance of adherence identified in the longitudinal TRANSHEMO 2 project, may differ from those highlighted in the original cross-sectional TRANSHEMO project. Specific aims Main objective: To compare the rate of adherence to healthcare among adolescents who participated in the TRANSHEMO project, using data collected during the original TRANSHEMO study when they were adolescents (before transition) and data to be collected as part of the TRANSHEMO 2 study when they have become young adults (after transition). Secondary objective: To identify the determinants of the maintenance of this adherence and the associations between these determinants, within the framework of a longitudinal study.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Feb 2026
25 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 26, 2026
CompletedStudy Start
First participant enrolled
February 1, 2026
CompletedFirst Posted
Study publicly available on registry
February 18, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 1, 2027
February 18, 2026
February 1, 2026
10 months
January 26, 2026
February 11, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (8)
Number of follow-up visits over the last 2 years (FranceCoag registry)
Quantitative variable. Dichotomized in agreement and not in agreement with the recommended number
1 day
Rate of Physician-reported adherence to clinical follow-up
Ordinal variable in 5 modalities (Very high/High/Average/Low/Very low). Dichotomized in very high and high to very low.
1 day
Rate of Patient-reported adherence to clinical follow-up
Ordinal variable in 2 modalities : visiting Haemophilia Treatment Centre (HTC) at least once a year and Not visiting HTC every year.
1 day
If under prophylaxis, number of prophylactic injections over the last 3 months (FranceCoag registry)
Quantitative variable. Dichotomized in agreement and not in agreement with the recommended number.
1 day
If under prophylaxis, rate of physician-reported adherence to prophylaxis
Ordinal variable in 5 modalities (Very high/High/Average/Low/Very low). Dichotomized in very high and high to very low.
1 day
Rate of Patient-reported adherence to treatment
Nominal variable. If under prophylaxis: \- Dichotomized in having no difficulties in adherence to prophylaxis or having difficulties but missing treatment less than once a week and having difficulties and missing treatment once a week or more If under on-demand treatment: \- Dichotomized in having no difficulties in recognizing early signs of haemorrhage and having difficulties inrecognizing early signs of haemorrhage
1 day
Physician-reported number of haemorrhagic events over the last two years
Quantitative variable. Dichotomized in none and at least one
1 day
Rate of composite endpoint
A composite quantitative endpoint was created by adding the criteria described above (seven for people under prophylaxis, five for people under ondemandtreatment). Adherent peoplewere defined as those having 4 to 7 points if under prophylaxis, and as those having 3 to 5 points if underon-demand treatment. Dichotomized in adherent and non adherent
1 day
Secondary Outcomes (9)
Quality of life analysis
1 day
Coping strategies analysis
1 day
Autonomy analysis
1 day
Time perspective analysis
1 day
Family functioning analysis
1 day
- +4 more secondary outcomes
Study Arms (1)
adolescents with severe hemophilia who participated in the TRANSHEMO project
Interventions
Participants will have questionnaires
Eligibility Criteria
Young adults with severe haemophilia (haemophilia A or B)
You may qualify if:
- Young adults who participated in the TRANSHEMO project during adolescence and who are currently aged 20-29 years;
- Young adults with severe haemophilia (haemophilia A or B);
- Young adults registered in the FranceCoag registry;
- Young adults who have received the participant information sheet for the TRANSHEMO 2 project;
- Young adults who did not object to participation in the present study.
You may not qualify if:
- patients with comprehension difficulties;
- patients who are unable to read and/or write;
- patients who express opposition to participation in this study.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (25)
CHU La Reunion
Saint-Denis, La Réunion, France
CH Annecy - St Julien
Annecy, France
Chu de Bordeaux
Bordeaux, France
Chu de Caen
Caen, France
Centre hospitalier Métropole Savoie
Chambéry, France
CHU Clermont-Ferrand
Clermont-Ferrand, France
Chu de Dijon
Dijon, France
Chu de Grenoble
Grenoble, France
CHRU de Lille
Lille, France
CHU Limoges
Limoges, France
Hospices Civils de Lyon
Lyon, France
Assistance publique - Hôpitaux de Marseille
Marseille, France
CH Montmorency
Montmorency, France
CHU Montpellier
Montpellier, France
Chu de Nancy
Nancy, France
CHU Nantes
Nantes, France
AP-HP (Hôpital Kremlin Bicetre)
Paris, France
AP-HP (Hôpital NECKER)
Paris, France
CHU Reims
Reims, France
CHU de Rennes
Rennes, France
CHU de Rouen
Rouen, France
CHU St Etienne
Saint-Etienne, France
CHU Strasbourg
Strasbourg, France
CHU Toulouse
Toulouse, France
CH Versailles
Versailles, France
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
François Cremieux
AP-HM
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- CROSS SECTIONAL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 26, 2026
First Posted
February 18, 2026
Study Start
February 1, 2026
Primary Completion (Estimated)
December 1, 2026
Study Completion (Estimated)
June 1, 2027
Last Updated
February 18, 2026
Record last verified: 2026-02