Background
Methods
Demographic, clinical, and laboratory markers
Spirometry
SKS analysis
Sputum collection
Statistical analysis
Results
Demographic and clinical markers | Category | DI (1990–2000) | DII (2000–2010) | p |
---|---|---|---|---|
Number of patients | 104 | 181 | ||
Age | Mean: 10 years 9 months ± 6.33 months | Mean :16 years 7 months ± 13.16 months |
<0.001
| |
Median: 9 years | Median: 12 years 10 months | |||
Range: 11 months to 31 years 2 months | Range: 6 months to 73 years 10 months | |||
Sex | Male | 53.8% | 49.7% | 0.539 |
Female | 46.2% | 50.3% | ||
Ethnicity | Caucasian | 93.3% | 92.0% | 0.818 |
NonCaucasian | 6.7% | 8.0% | ||
Consanguineous parents | 6.2% | 1.1% | 0.054 | |
Manifestation | Respiratory | 89.4% | 91.7% | 0.528 |
Digestive | 59.6% | 83.3% |
<0.001
| |
Onset of symptoms | Mean: 16 months | Mean: 91.75 months |
<0.001
| |
Median: 3 months | Median: 3 months | |||
Range: 0–20 years | Range: 0–60 years | |||
Age at diagnosis | Mean: 4 years 2 months | Mean: 2 years 10 months |
<0.001
| |
Median: 2 years 4 months | Median: 2 years | |||
Range: 0 to 29 years 11 months | Range: 0–60 years | |||
Meconium ileus | 5.8% | 15.0% |
0.021
| |
Diabetes mellitus | 4.8% | 18.5% |
0.001
| |
Nutritional status | Weight below 10th percentile | 69.9% | 35.71% |
<0.001
|
Height below 10th percentile | 56.6% | 40.82% |
0.025
| |
SpO2 | >95% | 59.5% | 55.5% | 0.713 |
91%–95% | 32.9% | 34.7% | ||
<91% | 7.6% | 9.8% | ||
Sweat test | <60 mEq/L* | 10.6% | – | |
60–100 mEq/L | 28.8% | 40.51% | ||
>100 mEq/L | 60.6% | 59.49% | ||
Bacteria |
Staphylococcus aureus
| 80.2% | 78.5% | 0.880 |
Pseudomonas aeruginosa
| 76.0% | 55.8% |
0.001
| |
Mucoid P. aeruginosa | 53.1% | 42.0% | 0.085 | |
Burkholderia cepacia
| 5.2% | 14.4% |
0.016
| |
Mucoid and nonmucoid P. aeruginosa | 51.0% | 21.85% |
<0.001
| |
Spirometry | Normal | 27.3% | 34.4% |
<0.001
|
Restrictive ventilatory disorder | 18.2% | 48.9% | ||
Obstructive lung disorder | 25.4% | 14.5% | ||
Mixed respiratory disorder | 29.1% | 2.3% | ||
CFTR mutation | F508del homozygotes | 18.75% | 26.5% |
<0.001
|
F508del heterozygotes | 62.5% | 22.7% | ||
G542X | 4.17% | 6.45% | ||
N1303K | 2.08% | 1.1% | ||
G551D | 1.04% | – | ||
R553X | 0.52% | 0.3% | ||
W1282X | 0.52% | – | ||
Shwachman-Kulczycki score | Excellent or good | 57.8% | 36.2% |
0.005
|
Mild | 26.5% | 36.2% | ||
Moderate or severe | 15.7% | 27.6% | ||
Deaths | 18 | 31 | 1 | |
Fecal balance | 67.9% | 80.0% |
0.031
|
Decade | Onset of digestive symptoms | p-value | OR | 95% CI | ||
---|---|---|---|---|---|---|
Presence | Absence | Total | ||||
1990–2000 | 62 (59.6%) | 42 (40.4%) | 104 |
<0.001
|
0.295
|
0.17–0.51
|
2000–2010 | 151 (83.4%) | 30 (16.6%) | 181 | 1 | – | |
Decade | Meconium ileus | p | OR | 95%CI | ||
Presence | Absence | Total | ||||
1990–2000 | 6 (5.8%) | 98 (94.2%) | 104 |
0.021
|
0.35
|
0.11–0.91
|
2000–2010 | 27 (14.9%) | 181 | 1 | – | ||
Decade | Diabetes mellitus | p | OR | 95%CI | ||
Presence | Absence | Total | ||||
1990–2000 | 5 (4.8%) | 99 (95.2%) | 104 |
0.001
|
0.228
|
0.07–0.61
|
2000–2010 | 33 (18.2%) | 148 (81.8%) | 181 | 1 | – | |
Decade | Fecal fat balance | p | OR | 95%CI | ||
Presence | Absence | Total | ||||
1990–2000 | 71 (68.3%) | 33 (31.7%) | 104 |
0.031
|
0.534
|
(0.31–0.93)
|
2000–2010 | 145 (80.1%) | 36 (24.2%) | 181 | 1 | – | |
Decade | Weight percentile | p | OR | 95%CI | ||
P < 10 | P ≥ 10 | Total | ||||
1990–2000 | 73 (70.2%) | 31 (29.8%) | 104 |
<0.001
|
4.206
|
2.34–7.67
|
2000–2010 | 35 (35.9%) | 63 (64.1%) | 98 | 1 | – | |
Decade | Height percentile | p | OR | 95%CI | ||
P < 10 | P ≥ 10 | Total | ||||
1990–2000 | 59 (56.7%) | 45 (43.3%) | 104 |
0.025
|
1.895
|
1.08–3.34
|
2000–2010 | 40 (40.9%) | 58 (59.1%) | 98 | 1 | – |
Decade | Pseudomonas aeruginosa* | p-value | OR | 95% CI | ||
---|---|---|---|---|---|---|
Presence | Absence | Total | ||||
1990–2000 | 79 (76.0%) | 25 (24.0%) | 104 |
0.001
|
2.495
|
1.42–4.48
|
2000–2010 | 101 (55.8%) | 80 (44.2%) | 181 | 1 | – | |
Decade |
Burkholderia cepacia
| p-value | OR | 95%CI | ||
Presence | Absence | Total | ||||
1990–2000 | 5 (4.8%) | 99 (95.2%) | 104 |
0.016
|
0.302
|
0.09–0.84
|
2000–2010 | 26 (14.4%) | 155 (85.6%) | 181 | 1 | – | |
Decade | Mucoid and nonmucoid P. aeruginosa# | p-value | OR | 95%CI | ||
Presence | Absence | Total | ||||
1990–2000 | 53 (51.0%) | 51 (49.0%) | 104 |
<0.001
|
3.645
|
2.11–6.38
|
2000–2010 | 40 (22.1%) | 141 (77.9%) | 181 | 1 | – |
Decade | Spirometry | Total | |||||||
---|---|---|---|---|---|---|---|---|---|
ORD | OR (95% CI) | MRD | OR (95% CI) | RVD | OR (95% CI) | Normal | OR (95% CI) | ||
1990–2000 | 14 (25.0%) | 2.058 (0.87–4.79) | 16 (29.8%) |
17.58 (4.70–98.97)
| 10 (18.3%) |
0.231 (0.10–0.51)
| 15 (26.9%) | 0.719 (0.33–1.50) | 55 |
2000–2010 | 19 (14.2%) | 1 | 3 (2.2%) | 1 | 66 (49.3%) | 1 | 46 (34.3%) | 1 | 134 |
Decade
|
Clinical score
|
Total
| |||||||
Moderate/Severe
|
OR (95% CI)
|
Mild
|
OR (95% CI)
|
Excellent/Good
|
OR (95% CI)
| ||||
1990–2000 | 13 (15.4%) |
0.491 (0.24–0.96)
| 22 (26.9%) | 0.635 (0.34–1.18) | 48 (57.7%) |
2.403 (1.40–4.16)
| 83 | ||
2000–2010 | 44 (27.5%) | 1 | 58 (36.3%) | 1 | 58 (36.3%) | 1 | 160 | ||
Decade
|
F508del mutation genotype
|
Total
| |||||||
Homozygotes
|
OR (95% CI)
|
Heterozygotes
|
OR (95% CI)
|
Without F508del
|
OR (95% CI)
| ||||
1990–2000 | 19 (20.2%) | 0.647 (0.34–1.21) | 59 (61.5%) |
5.445 (3.09–9.74)
| 18 (18.3%) |
0.235 (0.13–0.42)
| 96 | ||
2000–2010 | 50 (27.6%) | 1 | 41 (22.7%) | 1 | 90 (49.7%) | 1 | 181 |
Age groups | Decades | p-value | OR (95% CI) | |
---|---|---|---|---|
1990–2000 | 2000–2010 | |||
Newborn | 0 (0.0%) | 0 (0.0%) | <0.001 | - |
Infant | 6 (7.0%) | 6 (3.4%) | 2.131 (0.63–7.19) | |
Preschool | 25 (29.1%) | 24 (13.6%) |
2.602 (1.38–4.94)
| |
Academic | 18 (20.9%) | 52 (29.4%) | 0.636 (0.35–1.17) | |
Adolescent | 27 (31.4%) | 40 (22.6%) | 1.567 (0.88–2.79) | |
Adult | 10 (11.6%) | 55 (31.0%) |
0.293 (0.13–0.60)
|