Efficience: Effectiveness of Medical Management of Fibrous Dysplasia of Bone.

Sponsor

Hospices Civils de Lyon (Other)

Overall Status

Completed

CT.gov ID

NCT05422833

Collaborator

(none)

528

Enrollment

Location

287

Actual Duration (Months)

1.8

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The objective of our study was to assess the effectiveness of our reference center since its constitution.

In a retrospective cohort study, we compared the activity of our center, including the time elapsed between diagnosis and access to the center and the diagnostic delay of patients with fibrous dysplasia between two periods, 1996-2006 (before certification of our center) and 2007-2019 (after certification of our center).

Condition or Disease	Intervention/Treatment	Phase
Fibrous Dysplasia of Bone McCune Albright Syndrome	Other: Evaluation and analysis of clinical data

Study Design

Study Type:

Observational

Actual Enrollment :

528 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

Effectiveness of Medical Management in the Field of Rare Diseases. The Example of Fibrous Dysplasia of Bone.

Actual Study Start Date :

Jan 1, 1996

Actual Primary Completion Date :

Dec 1, 2019

Actual Study Completion Date :

Dec 1, 2019

Arms and Interventions

Arm	Intervention/Treatment
Before Children and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients). and with the first visit in the center included between 1996 and 2006 - before certification of our center.	Other: Evaluation and analysis of clinical data Evaluation and analysis of clinical data (baseline demographic features (sex, age at diagnosis, age at first symptoms, age at first visit), initial presenting symptoms, affected bone sites (monostotic FD or polyostotic FD), renal phosphate wasting, MAS, Mazabraud syndrome, endocrine disease, fractures, bone specific treatment especially bisphosphonate and surgery, pain and disease's severity.
After Children and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients). and with the first visit in the center included between 2007 and 2019 - after certification of our center.	Other: Evaluation and analysis of clinical data Evaluation and analysis of clinical data (baseline demographic features (sex, age at diagnosis, age at first symptoms, age at first visit), initial presenting symptoms, affected bone sites (monostotic FD or polyostotic FD), renal phosphate wasting, MAS, Mazabraud syndrome, endocrine disease, fractures, bone specific treatment especially bisphosphonate and surgery, pain and disease's severity.

Arm

Intervention/Treatment

Before

Children and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients). and with the first visit in the center included between 1996 and 2006 - before certification of our center.

Other: Evaluation and analysis of clinical data

Evaluation and analysis of clinical data (baseline demographic features (sex, age at diagnosis, age at first symptoms, age at first visit), initial presenting symptoms, affected bone sites (monostotic FD or polyostotic FD), renal phosphate wasting, MAS, Mazabraud syndrome, endocrine disease, fractures, bone specific treatment especially bisphosphonate and surgery, pain and disease's severity.

After

Children and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients). and with the first visit in the center included between 2007 and 2019 - after certification of our center.

Other: Evaluation and analysis of clinical data

Outcome Measures

Primary Outcome Measures

to compare time elapsed between first symptomes or discovery of bone lesions and the diagnosis of FD [1996-2019]
The main objective was to compare time elapsed between first symptomes or discovery of bone lesions and the diagnosis of FD, over 2 periods of time: 1996-2006 (before certification) and 2007-2019 (after certification).

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

We have included children and adults with FD/MAS who visited at least once in our center (outpatient and hospitalized patients). The diagnosis was established by an expert of the center, based on clinical, biological, imaging and/or histological arguments