Azithromycin Treatment in Paediatric Primary Ciliary Dyskinesia

Table of Content

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.
Table 2: Clinical outcomes at follow-up of patients by treatment group

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

HRCT, High resolution computed tomography. *That is delineating physical activity patterns described by parents or child caretakers including shorter time of daily free play or short of breath after strenuous activities such as climbing 6 or more floors of stairs or frequent rest during running games compare to heathy children. This had been clarified in the method section.

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.