osteoporosis
Conditions
Interventions
Observational Studies — Intervention (follow-up/observation methodology) will be conducted with a convenience sample from the HBDF/IGESDF reference center, composed of approximately 200 participants (
Cohort 2, with approximately 50 individuals with cystic fibrosis not eligible under label criteria for modulators/potentiators
and Control Group 3, with about 100 participants with chronic pancreatic diseases without relevant CFTR mutations. The follow-up period will be 10 years, with annual evaluations by ultrasound with hep
V03.175.500
Sponsors
Ricardo Jacarandá de Faria
Instituto Hospital de Base do Distrito Federal - Ihbdf
Eligibility
Age
16 Years to No maximum
Inclusion criteria
Inclusion criteria: Individuals followed at the cystic fibrosis outpatient clinic and at the pancreas outpatient clinic of HBDF will be invited for inclusion in the cohort,;with sequencing for CFTR mutations;aged over 16 years.
Exclusion criteria
Exclusion criteria: Individuals who have undergone pancreatectomy ;and those with alcohol consumption greater than 20 g per day.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the progression of fat deposition and pancreatic fibrosis in adult patients with cystic fibrosis using CFTR modulators/potentiators, verified through ultrasound with Shear Wave Elastography (SWE) for pancreatic and hepatic stiffness, complemented by the analysis of abdominal CT scans performed in the last 5 years for pancreatic fat quantification expressed in Hounsfield Units (HU), with variations in elastography values (kPa) and pancreatic/hepatic density in HU being observed throughout the follow-up period of up to 10 years, comparing baseline and follow-up.;To evaluate the progression of cystic fibrosis–related bone disease (CFOD) in adult patients, using annual bone densitometry by DEXA (Dual-Energy X-ray Absorptiometry) with measurement of bone mineral density in the lumbar spine, hip, and whole body, considering the variation of Z-scores and T-scores obtained by DEXA throughout the follow-up, identifying significant reductions or stabilization of bone mass. | — |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate the progression of glycemia and the risk of cystic fibrosis–related diabetes (CFRD) through serial laboratory tests performed quarterly or semiannually, including fasting plasma glucose, serum insulin, and calculation of the HOMA-IR index (Homeostasis Model Assessment of Insulin Resistance), with the variation of plasma glucose levels (mg/dL), insulin (µU/mL), and HOMA-IR values being observed in relation to baseline and throughout the follow-up.;To evaluate the progression of liver function in patients with cystic fibrosis using or not using CFTR modulators, through laboratory tests including ALT (alanine aminotransferase), AST (aspartate aminotransferase), GGT (gamma-glutamyltransferase), alkaline phosphatase, and bilirubins, in addition to annual assessment with FibroScan® (transient elastography for non-invasive measurement of liver stiffness), with changes in serum levels of liver enzymes (IU/L) and in liver stiffness (kPa) being observed throughout the follow-up.;To evaluate the progression of pulmonary function in patients with cystic fibrosis using or not using CFTR modulators through serial spirometry performed according to routine protocols, with the variation in FEV1 (Forced Expiratory Volume in the first second, expressed as % predicted) and FVC (Forced Vital Capacity, expressed as % predicted) being observed throughout the follow-up.;To evaluate the overall clinical progression and quality of life of patients with cystic fibrosis through periodic clinical assessments and the application of standardized quality-of-life questionnaires specific for cystic fibrosis, with observation of changes in the obtained scores as well as in the records of relevant clinical events, including exacerbations, hospital admissions, and antibiotic use. | — |
Countries
Brazil
Contacts
Public ContactRicardo de Faria
Instituto Hospital de Base do Distrito Federal - Ihbdf
Outcome results
None listed