ABSTRACT
Objectives: Primary ciliary dyskinesia (PCD) is a rare genetic
disease mainly involved in lung dysfunction. PCD patient outcomes after
azithromycin (AZM) treatment have rarely been reported. This study was
aimed to assess AZM treatment effects on disease progression of
pediatric PCD patients.
Study design: This retrospective follow-up study involved PCD
patients diagnosed from December 2009 to December 2020. Changes of
clinical outcomes, pulmonary function, and chest computed tomography
findings were compared between untreated and AZM-treated patients.
Results: Of 63 enrolled patients (median follow-up duration of
3.1 years), 30 received AZM (AZM-treated group) and 33 received no AZM
(AZM-untreated group). At diagnosis, no significant intergroup
differences in age, sex, height, weight, respiratory infection
frequency, and FEV1% and FVC% predicted values were found, although
FEF25-75% predicted was lower in AZM group. Between
treatment initiation and follow-up, patients in AZM-treated group had
less respiratory infection frequency than that of controls (1.4 ± 0.8VS 3.0 ± 2.1 times/year, respectively, P = 0.001) and
fewer AZM-treated group patients exhibited exercise intolerance.
Increases above baseline of AZM-treated group
FEV1% and FVC% predicted values
exceeded corresponding control increases, but intergroup differences
were insignificant (FEV1% predicted: 5.3 (-13.4, 9.4) VS 1.8
(-12.1, 9.5), P = 0.477; FVC% predicted: 6.7 (-7.6, 18.8)VS 1.6 (-5.6, 7.6), P = 0·328).
Conclusions: Long-term AZM treatment can reduce respiratory
infection frequency and may
maintain pulmonary diseases stable in pediatric PCD patients.