Influencing Progression of Airway Disease in Primary Antibody Deficiency
- Conditions
- immunodeficiencyPAD1002146010019815
- Registration Number
- NL-OMON51878
- Lead Sponsor
- niversitair Medisch Centrum Utrecht
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 100
In order to be eligible to participate in this study, a subject must meet all
of the following criteria:
1. Age 8-60 years
2. Diagnosis of Primary Antibody Deficiency / Common Variable Immunodeficiency
Disorder (see Appendix 1).
3. Indication for immunoglobulin replacement therapy and/or treated with
immunoglobulin replacement therapy
4. Current IgG dosing 0.25 - 0.6 gr / kg / 3-4 weeks
5. Receiving treatment and follow-up for PAD by a physician in one of the
participating centers
6. Written informed consent
A potential subject who meets any of the following criteria will be excluded
from participation in this study:
1. Diagnosis of Combined Immunodeficiency (CID) disease at onset of study (see
Appendix 1). Explanation: Combined Immunodeficiency is featured by the
occurrence of more viral infections and reactivations and thus less comparable
to PAD.
2. Severe pulmonary disease, determined by an independent radiologist:
a. Baseline AD score > 7 and/or ILD score > 5, in combination with:
i. Saccular bronchiectasis on CT scan, or;
ii. Clinical diagnosis of severe respiratory insufficiency ( (defined as:
saturations in room air <92%, and/ or oxygen dependency).
b. Baseline pulmonary function (FEV1 and FVC) <70% expected for age and body
weight / length)
3. Active smoker
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Difference in mean AD and ILD scores (as measured with CT scanning) between t=0<br /><br>and t=2 years in patients with standard vs higher Ig replacement therapy<br /><br>dosing. </p><br>
- Secondary Outcome Measures
Name Time Method <p>1. The incidence of symptomatic lower pulmonary infections in PAD patients with<br /><br>high Ig replacement therapy dosing versus standard Ig replacement therapy<br /><br>dosing.<br /><br>2. Number of physician diagnosed lower respiratory tract infections in patients<br /><br>with high vs standard Ig replacement therapy dosing.<br /><br>3. Number of hospital admissions and duration of hospital admissions for<br /><br>pulmonary complications (ao exacerbations of bronchiectasis)<br /><br>4. Outcomes of pulmonary function tests (specifically: Total Lung Capacity<br /><br>(TLC), Forced Expiratory Volume after 1 second (FEV1), CO diffusion) on t=0 and<br /><br>t=2 years in all patients<br /><br>5. Days missed from work / school in patients with high vs standard Ig<br /><br>replacement therapy dosing.<br /><br>6. Total therapeutic and preventive costs </p><br>