Is Neonatale Screening op Mucoviscidose aangewezen in België?

16 Cystic Fibrosis Neonatal Screening KCE Reports 132
In the observational study from Connecticut, 68 FEV1 was higher at all ages in the SG
(p=0.03) but only when controlling for age, gender, inhaled tobramycin and Pa
colonization. In a univariate analysis, FEV1 was significantly better in SG versus CG at age
15 (90% vs. 74% p=0.15). FEV1 decline was less in the SG between age 6-15 years
(p=0.01). Brasfield X-rays scores and age at acquisition of PA infections were not
different.
For patient data in the USA CF registry, an FEV1 below 70% was 1.32 times more likely
in CG (0.77-1.71; p=0.11) but this difference compared to the SG did not reach
statistical significance. 82 Recent cross sectional data (2002 registry data on 14 647
patients) show an advantage for FEV1 in the SG in age category 6-10 (97% vs. 89% , p<
0.05, CI not stated) and 11-20 years (87% vs. 82% p< 0.05, CI not stated). 69
The UK registry data (cross sectional data analysis in 2002; n=183 screen and n=950
CG) documented no LF differences between SC and CG. X-ray scores (Northern
score, lower is less disease) were significantly lower in the SG aged 1-3 (p=0.05) and 7-
9 years (P=0.05). Shwachman scores were higher for NS in the age range of 1-3
(p=0.05) and 7-9 (p=0.05) years.
2.2.2.4 Complications
In the US CF patient registry the proportion of patients with more than 1
hospitalisation for complications was 70% in CG versus 29% in SG (p< 0.0001). Patients
studied were diagnosed under age 1 and born in the period 2000-2002. 69
2.2.2.5 Burden of therapy
Burden of therapy is related to severity of CF lung disease especially when looking at
the need for intravenous (IV) antibiotics and hospital admission. 83
In the Wisconsin trial, there was no difference in the use of intravenous or inhaled
antibiotics, nor for the days spent in hospital per year between the groups. 84 This may
be due to the earlier acquisition of PA in a subset of the screened group.
Most observational studies report lower treatment burden in CF patients after
screening.
In the UK trial, patients in the SG were on average 1.3 (sd 1.9) times admitted in
hospital in the first year while this was 3.2 (SD 2.7) times for the CG (CI not stated; p<
0.01). 55
In the French study, the proportion of children requiring at least one hospital admission
before age 10 years was 86% (CG) versus 49% (P< 0.001) (SG). 67 Despite this
difference, use of IV and inhaled antibiotics was not different.
In the UK registry data treatment burden was much higher in the CG, despite
comparable LF. This may suggest that LF is maintained in the CG at the cost of more
70, 85
treatment burden.
Again in the UK registry, cross sectional data on treatment intensity have been
reported for CF patients born after 1994 and younger than 10 years (NS n=184; CG
n=950). 85 Long term therapies (prescribed for longer than 3 months) were fewer in SG
vs. CG (2 versus 3; p=0.005). In the first 3 years of life, 42% of the CG needed IV
antibiotics versus 20% of the SG (p=0.05). At age 7-9 the figures were 38% versus 15 %
respectively (p= 0.05).
In the year of diagnosis 22% (CI 18-28) of SG were hospitalized versus 64% (CI 60-67)
of the CG according to the data of the US CF registry (OR 3.7 (95%CI 2.3-5.9) and p<
0.001). 69