PHILEOS (haemoPHILia and ostEoporOSis) Study: protocol of a multicentre prospective case-control study.
BMJ Open
; 11(1): e042283, 2021 01 13.
Article
en En
| MEDLINE
| ID: mdl-33441362
ABSTRACT
INTRODUCTION:
Two meta-analyses showed lower bone mineral density (BMD) in patients with haemophilia (haemophilia type and severity were often not specified) compared with healthy controls. This finding could be related to reduced mobility and sedentary lifestyle, and/or hepatitis C or HIV infection. The aim of this study is to determine osteoporosis prevalence in patients with haemophilia classified in function of the disease type (A or B) and severity, and to evaluate the potential role of regular prophylactic factor replacement (early vs delayed initiation) in preserving or restoring BMD. METHODS ANDANALYSIS:
The haemoPHILia and ostEoporOSis Study is a prospective, controlled, multicentre study that will include patients in France (13 haemophilia treatment centres), Belgium (1 centre) and Romania (1 centre). In total, 240 patients with haemophilia and 240 matched healthy controls will be recruited (11). The primary objective is to determine osteoporosis prevalence in patients with severe haemophilia A and B (HA and HB) without prophylaxis, compared with healthy controls. Secondary outcomes include prevalence of osteoporosis and osteopenia in patients with mild, moderate and severe HA or HB with prophylaxis (grouped in function of their age at prophylaxis initiation), compared with healthy subjects; BMD in patients with HA and HB of comparable severity; correlation between BMD and basal factor VIII/IX levels and thrombin potential; and quantification of plasmatic markers of bone remodelling (formation and resorption) in patients with haemophilia. ETHICS AND DISSEMINATION The protocol was approved by the French Ethics Committee and by the French National Agency for Medicines and Health Products Safety (number 2019-A03358-49). The results of this study will be actively disseminated through scientific publications and conference presentations. TRIAL REGISTRATION NUMBER NCT04384341.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Contexto en salud:
1_ASSA2030
/
2_ODS3
Problema de salud:
1_doencas_nao_transmissiveis
/
2_enfermedades_transmissibles
Asunto principal:
Osteoporosis
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Infecciones por VIH
/
Hemofilia A
Tipo de estudio:
Clinical_trials
/
Guideline
/
Observational_studies
/
Risk_factors_studies
Aspecto:
Ethics
Límite:
Humans
País/Región como asunto:
Europa
Idioma:
En
Revista:
BMJ Open
Año:
2021
Tipo del documento:
Article
País de afiliación:
Francia