Introduction
Current Primary ciliary dyskinesia (PCD) treatment mainly follows CF and non-CF bronchiectasis treatment recommendations, with azithromycin (AZM) maintenance therapy known to exert beneficial anti-inflammatory effects when use to treat CF and non-CF bronchiectasis
Aim
To assess AZM treatment effects on disease progression of pediatric PCD patients.
Patient Profile
Pediatric patients diagnosed with PCD
Methods
- Retrospective follow-up study
- PCD Pediatric Patients
- Changes of clinical outcomes, pulmonary function, and chest computed tomography findings were compared between untreated and AZM treated patients
Results
- Significant reduction in frequency of respiratory exacerbations was observed in the AZM-treated group as compared to the AZM-untreated group (mean, SD, 1.4 vs. 3.0, respectively, P = 0.001) and fewer AZM-treated group patients exhibited exercise intolerance
- Lung function indicator values increased after AZM treatment, but of no significance of difference with increases by 5.3% and 1.8%, respectively in AZM-treated and AZM-untreated groups, respectively.
|
Parameter |
AZM-treated (n = 34) |
AZM-untreated (n = 37) |
P-value |
|
Age (years) (mean, SD) |
10.8 |
11.5 |
0.412 |
|
Follow up duration (mean, SD, year) |
2.7 |
3.5 |
0.097 |
|
Changes of height (percentile) |
9.6 |
6.9 |
0.344 |
|
Increased (n, %) |
18 (75) |
14 (64) |
0.403 |
|
Decreased (n, %) |
6 (25) |
8(36) |
|
|
Changes of weight (percentile) |
5.1 |
10.2 |
0.800 |
|
Changes of BMI (percentile) |
1.5 |
1.7 |
0.085 |
|
Changes of BMI (Z score) (mean, SD) |
0.44 |
0.67 |
0.522 |
|
Changes of pulmonary function |
|
|
|
|
FVC% predicted |
6.7 |
1.6 |
0.328 |
|
FEV1% predicted |
5.3 |
1.8 |
0.477 |
|
FEF25- 75% predicted |
6.0) |
2.8 |
0.594 |
|
Increased (%) |
75% |
60% |
0.638 |
|
Decreased (%) |
25% |
40% |
|
|
Bronchiectasis on HRCT |
|
|
0.180 |
|
Aggravated or newly (n, %) |
1 (5) |
4 (25) |
|
|
Improved (n, %) |
7 (35) |
3 (19) |
|
|
Stable (n, %) |
12 (60) |
9 (56) |
|
|
Respiratory exacerbations (times/year) |
1.4(0.8) |
3.0 (2.1) |
0.001 |
|
Reduction of sputum volume |
93% (26/28) |
70% (21/30) |
0.043 |
|
Exercise intolerance* |
12% (3/26) |
24% (7/29) |
0.009 |
Conclusion
The study demonstrated that long-term AZM treatment can reduce respiratory infection frequency and may maintain pulmonary diseases stable in pediatric PCD patients with worse lung function
References
Front Pediatr. 2022 Jun 10; 10:905253.