FFC#25/2015

Are CF guidelines credible? Evaluating methodological issues

FFC#25/2015

Are CF guidelines credible? Evaluating methodological issues

PRINCIPAL INVESTIGATOR

Cesare Braggion (Dipartimento di Medicina Pediatrica, Centro Regionale Fibrosi Cistica – Ospedale dei Bambini A. Meyer, Firenze)

RESEARCHERS

4

CATEGORY

AREA 5 Clinical and Epidemiological research

DURATION

1 year

GOAL

€ 22.000 €

RESULTS

110 documents were considered eligible for the analysis (58 consensus documents and 52 guidelines), then reduced to 40 as produced in the last 10 years and covering the most relevant clinical topics in CF field. A deep analysis of the data has shown on average a modest quality of consensus documents. Guidelines appeared to be better developed, but lacking in the field of applicability. This field represents a significant future investment framework for CF clinicians. Further 25 documents were selected regarding relevant questions in pulmonary disease and respiratory therapy from which 788 recommendations were extracted. In conclusion, the project graded guidelines in high, medium and low level of quality; a lot of documents with a low level of quality and discrepancies between recommendations were founded, so a synoptic table of evidence-based recommendation from valid guidelines was set up, to facilitate the implementation in daily practice.

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Tezacaftor, one of the components of Kaftrio, induces an accumulation of dihydroceramides both in vitro and in vivo in animal models