Rationale: Patients with Primary Antibody Deficiencies (PADs) frequently encounter chronic lung disease, caused by recurrent airway disease and/or interstitial lung disease. Chronic lung disease leads to absence from work and school and significant health costs. Patients with PAD are treated with immunoglobulin replacement therapy. Optimal dosing to prevent lung disease is unclear and different dosing regimens, all within prescription label, are used nationwide. We and others recently showed that higher immunoglobulin (Ig) trough levels were related to less airway infections and slower progression of airway disease. These findings need confirmation in a prospective randomized setting.
Objective: To show the protective value and to measure cost effectiveness of higher Ig dosing on progression of lung disease in PAD.
Study design: Multicenter, prospective randomized controlled trial, performed by 3 academic centers in The Netherlands.
Study population: Total of N=100 adult and pediatric PAD patients
Intervention (if applicable): Two Ig dosing regimens (both in normal range) are compared: Control group: Ig dose 0.4-0.6 g/kg/L, vs intervention group: dose increase of 33% (relative to pre-study dose) will be administered for 2 years. CT scanning and pulmonary function tests will be performed at t=0 and 24 months.
Main study parameters/endpoints: 1. Progression of lung disease, determined by computed tomography (CT) scanning and pulmonary function test 2. Number of respiratory infections 3. Days missed from school/work. 4. Cost savings 5. Quality of life.
TIME SCHEDULE: inclusion and follow up 2017-2020, data analysis and publications 2020-2021.