Is Neonatale Screening op Mucoviscidose aangewezen in België?

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18 Cystic Fibrosis Neonatal Screening KCE Reports 132 Key points: • CF NBS should be followed by immediate follow-up and appropriate management. • CF-NBS decreases the median and mean age at diagnosis. This finding has been confirmed by all studies on this subject, RCT as well as non-RCT. • Patients diagnosed by CF-NBS followed by immediate treatment and followup have a long term nutritional advantage over clinically diagnosed patients. This has been confirmed by RCT and non-RCT. Data on the advantage for fat soluble vitamin status and cognitive function are based on low number of patients. • RCTs have not shown an advantage for respiratory disease after early diagnosis in the context of screening but this may be linked to nonsegregation of patients after screening in this trial leading to more Pseudomonas infections. Non-RCT and register data do however suggest that with current standards of care, NBS will improve pulmonary status and decrease the burden of therapy. • NBS possibly decreases CF mortality based on RCT and non-RCT data. Since mortality below the age of 10 years is low, these data are only based on low numbers of deceased CF patients.
KCE Reports 132 Cystic Fibrosis Neonatal Screening 19 3 CF NBS – POSSIBLE HARMS 3.1 RISK FOR PSEUDOMONAS ACQUISITION CF NBS is universally coupled with immediate referral to a specialized CF centre. It is long known that without currently widely accepted standards of care, centralized CFcare might be associated with an increased isolation rate of PA in respiratory secretions 88, 89 as well as with outbreaks of infection with multiresistant strains of this pathogen. 90-92 This is especially worrying as chronic PA colonization has been associated with the loss of nearly a third of life expectancy, 93 a less favourable FEV1 and an accelerated decline of this parameter, 94, 95 an increased treatment load affecting quality of life and much higher treatment costs. 96 The earlier acquisition of PA in the SG compared to the CG in one of the 2 centres involved in the Wisconsin RCT raised concern but was shown to be primarily due to absence of cohort segregation for PA positive and negative CF patients. 78 Cross sectional data after 9 years follow-up showed a PA prevalence of 56.7 % in the SG versus 46.3 % in the CG, this difference not being significant. When looking at the 2 centres separately the median PA free survival time was significantly shorter only in 1 centre for the SG compared to the CG (52 weeks versus 234 weeks; p = 0,0003). In the other centre the median time to Pa acquisition was 130 weeks in the CG versus 289 weeks in the SG. 84 This finding of earlier acquisition of PA in CF NB screened patients was not confirmed in subsequent studies. 67, 97 In fact, several studies including long term data from the US CF registry suggests that the risk for acquiring PA is lower in children detected by CF 70, 82, 98 NBS. This is not surprising. It is actually now realized that a link between attendance at a centre and earlier acquisition of PA is not a fatality, neither during regular specialist CF care nor in the context of neonatal screening. 68, 97 In reality it is largely manageable with a policy of patient segregation based on bacteriology (applied to ambulatory as well as hospitalised care), regular systematic bacteriological surveillance and early intervention once this organism is detected. 99-103 Such policies have been put into practice in the 6 Belgian centres studied (the 7 th centre was not included in this study), for more than 17 years in one centre, for more than 7 years in 4 others, and only recently in the 6 th centre, contributing to a low number of clusters 104 and a rather low (though variable in different centres) prevalence of PA in our country. 105 Moreover, another Belgian study showed that “early” referral of CF patient to an accredited CF centre (defined as a delay less than 2 years) was associated with a significantly better FEV1 (86.7% pred. ± 19.4 vs 77.2% pred. ± 22.4, p
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KCE reports 1. Effectiviteit en kos
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73. Financiering van het zorgprogra
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Is Neonatale Screening op Mucoviscidose aangewezen in België?

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