The vast majority of Cystic Fibrosis (CF) patients worldwide are vitamin D insufficient. There is no evidence of benefit of vitamin D supplementation for CF patients yet. However, descriptive cross-sectional studies suggest that vitamin D might be beneficial with respect to bone health, as well as to the newly described "non-classical" functions of vitamin D such as the potential anti-diabetic and immunomodulatory effects. To prove causation, and to determine which serum vitamin D concentration is optimal for CF patients, vitamin D supplementation interventional studies are needed, such as our trial.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
16
Patients younger than 16 years of age are administered 35,000 IU ergo-/chole-calciferol per week divided into doses 5000 IU per day as a starting dose that is further adjusted by serum 25-hydroxy vitamin D concentration monitoring. The intervention time is 3 months, followed by 2-months wash-out period when patients do not take any more extra vitamin D but they are still monitored. Patients 16 or more years of age are administered 50,000 IU ergo-/chole-calciferol per week divided into doses 7150 IU per day as a starting dose that is further adjusted by serum 25-hydroxy vitamin D concentration monitoring.
Stockholm Cystic Fibrosis Center, Karolinska University Hospital Huddinge
Stockholm, Sweden
Serum 25-hydroxy vitamin D
This pilot study is primarily designed for establishing effective vitamin D dosing in our specific patient population, and only secondarily designed (and thus, not powered for) for the secondary outcome measures. The results of this study will make it possible for the first time to power the follow-up long-term study for some of the secondary outcome measures followed in this pilot study, some of which might therefore become primary outcome measures in the follow-up study.
Time frame: 3 months
Parathyroid hormone (PTH)
As a surrogate marker of bone health
Time frame: 3 months
Inflammatory parameters
Cytokine profiles, antimicrobial peptides, peripheral blood mononuclear cell profiles, immunoglobulines, acute phase markers, sedimentation rate and other
Time frame: 3 months
Infection parameters
Number of days on intravenous antibiotic treatment; number of infectious episodes; number of common cold episodes; relative number of sputum samples positive for pathological bacteria; and other
Time frame: 3 months
Lung function parameters
FEV1, FVC, PEF, FEF25, FEF50, FEF75 and other
Time frame: 3 months
Glucose tolerance parameters
Insulin, C-peptide, glucagon, fasting plasma glucose, HbA1c, 3-hour 75-g oral glucose tolerance test
Time frame: 3 months
Adherence with vitamin D treatment
Semi-quantitative assessment by a questionnaire (thus, a patient-reported outcome)
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Time frame: 3 months
Disease-specific quality of life
Assessment using "CFQ-R" questionnaire, specifically designed to measure the quality of life in CF patients
Time frame: 3 months
Plasma calcium
Proportion of patients with albumin-corrected serum calcium increasing to a concentration greater than 2,75 mmol/L in patients with no hypercalcaemia before vitamin D supplementation was started.
Time frame: 3 months
Relative number of patients reaching high abnormal 25(OH)D concentrations
Proportion of patients in the intervention arms reaching 25(OH)D \>250 nmol/L
Time frame: 3 months
Proportion of patients reaching toxic 25(OH)D concentrations
Proportion of patients in the intervention arms reaching 25(OH)D \>375 nmol/L
Time frame: 3 months
Proportion of patients with suspect hypercalcaemia symptoms
Proportion of patients with suspect hypercalcaemia symptoms in the intervention arms
Time frame: 3 months