Figures
- FIGURE 1
The 20-year overall survival was significantly higher in the newborn screening (NBS) group than in the symptoms group for each of the subgroups defined according to severity. In a) the severe group it was 85% in the NBS versus 64% in the symptoms group (p=0.007); in b) the moderate group it was 94% in the NBS versus 86% in the symptoms group (p=0.016); and in c) the mild group it was 98% in the NBS versus 93% in the symptoms group (p=0.6). Survival probability is presented on a scale from 0.5 to 1.0 for readability. HR: hazard ratio; OS: overall survival.
Tables
- TABLE 1
Overall survival of children with cystic fibrosis diagnosed by neonatal screening or symptoms or with meconium ileus
Patients Events# 20-year survival % (95% CI) 30-year survival % (95% CI) p-value¶ NBS 342 (100) 35 92.35 (88.03–95.16) 80.11 (71.42–86.40) Sex Male 167 (49) 15 91.86 (85.26–95.58) 84.95 (74.51–91.35) 0.3 Female 175 (51) 20 92.90 (86.14–96.43) 73.70 (57.27–84.61) Pancreatic status at birth+ Sufficient 60 (18) 1 96.15 (75.69–99.45) 96.15 (75.69–99.45) 0.10 Insufficient 281 (82) 34 91.75 (86.96–94.84) 78.34 (68.99–85.18) Birth cohort 1971–1980 30 (9) 10 74.90 (54.33–87.20) 67.20 (46.26–81.47) 0.2 1981–1990 92 (27) 18 93.34 (85.78–96.95) 79.89 (67.80–87.83) 1991–2000 119 (35) 6 96.40 (90.68–98.63) 2001–2014 101 (29) 1 Symptoms 143 (100) 50 82.98 (75.49–88.35) 71.03 (62.21–78.15) Sex Male 74 (52) 27 76.93 (65.10–85.19) 69.58 (56.69–79.32) 0.43 Female 69 (48) 23 89.29 (78.83–94.75) 72.82 (59.89–82.19) Pancreatic status at birth+ Sufficient 48 (34) 0 0.00 0.00 <0.001 Insufficient 94 (66) 49 75.40 (65.07–83.06) 58.50 (47.32–68.10) Birth cohort 1971–1980 96 (67) 46 77.08 (67.32–84.27) 65.22 (54.70–73.87) 0.06 1981–1990 31 (22) 4 96.55 (77.95–99.51) 84.69 (63.79–94.05) 1991–2000 10 (7) 0 100.00 2001–2011 6 (4) 0 Meconium ileus 101 (100) 18 90.64 (82.04–95.24) 71.77 (56.47–82.49) Data are presented as n (%) or n, unless otherwise stated. NBS: newborn screening. #: death due to any cause is considered an event; ¶: log-rank test; +: data were missing in one case.
- TABLE 2
Survival analysis of cystic fibrosis (CF) patients according to clinical severity in the first 3 years of life and diagnosis
Patients Events 10-year survival %
(95% CI)15-year survival %
(95% CI)20-year survival %
(95% CI)p-value Overall survival# Severe NBS 95 23 98.84 (92.03–99.84) 92.56 (84.17–96.59) 84.93 (74.29–91.41) 0.007§ Symptoms 34 21 87.88 (70.86–95.27) 72.73 (54.13–84.77) 63.64 (44.95–77.46) Moderate NBS 80 4 100.00 98.46 (89.58–99.78) 94.48 (83.72–98.20) 0.016§ Symptoms 40 12 100.00 88.89 (73.05–95.68) 85.93 (69.41–93.89) Mild NBS 149 6 99.29 (95.07–99.90) 99.29 (95.07–99.90) 98.21 (92.83–99.56) 0.6§ Symptoms 58 12 96.55 (86.91–99.13) 96.55 (86.91–99.13) 92.80 (81.92–97.24) Cumulative incidence of CF-related death¶ Severe NBS 95 13 1.16 (0.17–8.16) 6.22 (2.66–14.55) 12.36 (6.66–22.92) 0.0001ƒ Symptoms 34 19 12.12 (4.84–30.38) 27.27 (15.62–47.61) 36.36 (23.16–57.11) Moderate NBS 80 3 0.00 0.00 3.98 (1.02–15.48) 0.01ƒ Symptoms 40 10 0.00 11.11 (4.41–27.99) 14.07 (6.24–31.75) Mild NBS 149 4 0.71 (0.10–5.00) 0.71 (0.10–5.00) 1.79 (0.44–7.28) 0.2ƒ Symptoms 58 9 3.45 (0.88–13.46) 3.45 (0.88–13.46) 7.20 (2.80–18.53) Event-free survival+ Severe NBS 95 33 98.84 (92.03–99.84) 88.68 (79.34–93.95) 79.76 (68.53–87.33) 0.07§ Symptoms 34 25 87.88 (70.86–95.27) 72.73 (54.13–84.77) 63.64 (44.95–77.46) Moderate NBS 80 6 100.00 98.46 (89.58–99.78) 92.68 (81.55–97.21) 0.013§ Symptoms 40 15 100.00 88.89 (73.05–95.68) 85.93 (69.41–93.89) Mild NBS 149 8 99.29 (95.07–99.90) 99.29 (95.07–99.90) 95.78 (88.94–98.43) 0.8§ Symptoms 58 14 96.55 (86.91–99.13) 96.55 (86.91–99.13) 92.80 (81.92–97.24) Data are presented as n, unless otherwise stated. NBS: newborn screening. #: death due to any cause is considered an event; ¶: CF-related death is considered an event; +: death due to any cause and lung transplant were considered events; §: log-rank test; ƒ: Gray test.
Supplementary Material
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FIGURE S1 Cumulative incidence of death due to cystic fibrosis 00109-2017_figureS1
TABLE S1 Univariate and multivariate analysis for severe and moderate groups 00109-2017_tableS1
