Clinical Outcomes
Growth Changes
Average percentage-based increases in height of children were 9.6% and 6.9% for AZM-treated and AZM-untreated groups, respectively, between the time of initial diagnosis to the final follow-up visit, with percentage change in height increasing in 75% of AZM-treated group patients as compared to 64% of AZM-untreated group patients. Meanwhile, BMI percentile increases did not significantly differ between AZM-treated and AZM-untreated groups [(1.5, 1.0-2.0) VS (1.7, -6.4-17.9), respectively], while the average AZM-treated group weight increased by only 5.1% as compared to 10.2% for the AZM-untreated group (Table 3) also with no significance of difference.
PCD Manifestations
The mean frequency of infections was significantly reduced in the AZM-treated group as compared to the AZM-untreated group (1.4 ± 0.8VS 3.0 ± 2.1, respectively, P =0.001). In addition, significantly decreased sputum volumes were observed in AZM-treated group patients as compared to those of AZM-untreated group (93%VS 70%, respectively, P =0.043). Moreover, the percentage number of AZM-treated group patients with exercise intolerance was lower than that of the AZM-untreated group (12% VS 24%, respectively,P =0.009).
Changes in Chest CT Findings and Pulmonary Function Test (PFT) Results
Only 28 patients (16 in AZM-treated group and 12 in the AZM-untreated group respectively) received repeated chest CT scans. Scans of 1 patient in AZM-treated group revealing aggravation of lung damage with pathological involvement of new bronchiectasis, while, results obtained for 5 patients showed signs of lung improvement and results for 10 patients showed no obvious change in CT findings, number of which in the AZM-untreated group were 3, 2 and 7 (Table 3) respectively.
Results of cross-sectional analysis of lung function-based measurements collected during the follow-up period are shown in Table 3. The first measured FEV1% predicted of patients at baseline varied widely between normal and subnormal values (i.e., <80% of predicted), with a substantial number (n=10; 33%) demonstrating subnormal values (59%–79% of predicted). Importantly, lung function indicator values seemed 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. Meanwhile, although the FEF25-75% predicted value in the AZM-treated group was significantly lower than that of the AZM-untreated group at baseline, intergroup FEF25-75% predicted change differences were of no statistically significance [6.0 (-7.6-18.8)VS -2.8 (-20.3, 11.2), respectively, P =0.594].