Determinants and Consequences of the Transition to Adulthood for Adolescents With Severe Haemophilia: TRANSHEMO 2, an Ancillary Study to the TRANSHEMO Project (TRANSHEMO2)

February 11, 2026 updated by: Assistance Publique Hopitaux De Marseille

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

75

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Young adults with severe haemophilia (haemophilia A or B)

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • patients with comprehension difficulties;
  • patients who are unable to read and/or write;
  • patients who express opposition to participation in this study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
adolescents with severe hemophilia who participated in the TRANSHEMO project
Other: questionnaires
Participants will have questionnaires

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of follow-up visits over the last 2 years (FranceCoag registry)
Time Frame: 1 day
Quantitative variable. Dichotomized in agreement and not in agreement with the recommended number
1 day
Rate of Physician-reported adherence to clinical follow-up
Time Frame: 1 day
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
Time Frame: 1 day
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)
Time Frame: 1 day
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
Time Frame: 1 day
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
Time Frame: 1 day

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
Time Frame: 1 day
Quantitative variable. Dichotomized in none and at least one
1 day
Rate of composite endpoint
Time Frame: 1 day
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Quality of life analysis
Time Frame: 1 day
Generic quality of life (QoL) measured by Short form 12 Health Survey (SF-12). Two subscores: Physical component score (PCS) for physical health-related quality of life (HRQoL), mental component score (MCS) for mental HRQoL. Scores range from 0 to 100 with higher scores indicating better HRQoL.
1 day
Coping strategies analysis
Time Frame: 1 day
Coping strategies measured by Brief Coping Orientation to Problems Experienced Scale (Brief COPE). 28-item questionnaire evaluating 14 coping strategies, which are grouped into four main strategies: Social support, problem solving, avoidance, and positive thinking. Scores ranged from 0 to 100. Higher scores indicate a more frequent use of the assessed coping strategy
1 day
Autonomy analysis
Time Frame: 1 day
Autonomy measured by autonomy questionnaire Noom. 15-item questionnaire classified into three dimensions: Attitudinal autonomy, Functional autonomy, Emotional autonomy. Scores range from 5 to 25 with higher scores indicating higher level of autonomy
1 day
Time perspective analysis
Time Frame: 1 day
Time perspective measured by Zimbardo Time Perspective Inventory (ZTPI). Two subscales "Negative past" (nine items) and "Future" (12 items) were retained from the original 56-item questionnaire, measuring tendency to focus on different aspects of the past and future. Score range from 1 to 5. Higher scores indicate stronger projection in the assessed time perspective .
1 day
Family functioning analysis
Time Frame: 1 day
Family functioning measured by Family Assessment Device (FAD). 6-item questionnaire. Scores range from 1 to 4 with lower score indicating better general family functioning.
1 day
Rate of academic difficulty
Time Frame: 1 day
Nominal variable with two modalities : at least one grade repetition at school and none
1 day
Rate of type of healthcare transition in the HTC
Time Frame: 1 day
Nominal variable with two modalities : same hospital and service for adolescents and adults and same hospital, two different services (paediatric/adult)
1 day
Rate of paediatrician in the medical team of the HTC
Time Frame: 1 day
Nominal variable with two modalities : yes and no
1 day
Rate of setup of therapeutic education activities for transition in the HTC
Time Frame: 1 day
Nominal variable with two modalities : yes and no
1 day

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique Hopitaux De Marseille

Investigators

  • Study Director: François CREMIEUX, AP-HM

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

February 1, 2026

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

June 1, 2027

Study Registration Dates

First Submitted

January 26, 2026

First Submitted That Met QC Criteria

February 11, 2026

First Posted (Actual)

February 18, 2026

Study Record Updates

Last Update Posted (Actual)

February 18, 2026

Last Update Submitted That Met QC Criteria

February 11, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RCAPHM25_0354
  • 2025-A01711-48 (Other Identifier: AP-HM)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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