An overview based on a contemporary clinical question: eculizumab and paroxysmal nocutrnal haemoglobinuria
- introduce a difficult clinical issue
- formulate a treatment question
- consider what evidence we need to answer our quesiton
- Look at some EBP resources
- do a systematic search
- resources to help with clinical appraisal
PNH rare acquired haemolytic anamia stemn cell mutation cauding a lack of compleement inhibitor on RBCs
- haemolutic anaemia
- free hg in blood
Eculizumab monocloncal anitbody bind and inhibits complement activation effects prevent haeolusis and haemolutic anameia
but susceptible to infection especially meningococcal
current reccomensation is to give it
grad 1b reccomentation
grade 1 is strong recommensation and that the goverment believes that if you follow it you will do more harm than not
grade b means best estimates of the critical benefits and risks come from RCTs with important limitations
why did pharmac turn it down.
they daid that most evidence was from observational studies.
5 a’s of evidence based practive
tools for finding information
there was a lack of evidence that it prevented thrombosis and therefore did not make financial sense as it was being looked to for the purpose of anti thrombotic