I have to start today with a word about the ghastly outbreak of E coli which has claimed many lives in Germany. I am sure the basic processes of outbreak control are in place - molecular epidemiology to distinguish patients with the outbreak strain from the background noise of ordinary E coli, plus case finding to ensure that we know the full extent of the outbreak in time (when was the very first case?) and time (is it really only Germany?). Thus we can avoid jumping to conclusions based on the first few known cases.
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The article of interest for today is here.
Usually, when we run a trial we need adequate statistical power, control groups, randomisation and so on. But sometimes, very occasionally, just a single patient is enough. This is one of those once-in-a-lifetime events. The intervention enabled a patient paraplegic from spinal cord injury to stand almost unaided. Unfortunately the report is almost incomprehensible but the basics are clear from the abstract and introduction, not to mention the videos.
Single patient studies are convincing when we know with certainty the natural history of the disease - in the case of spinal cord injury, permanent paralysis. We would also be convinced by any treatment which resulted in even a single patient recovering from rabies - a uniformly fatal disease once symptoms have developed.In the 1940s penicillin allowed recovery from what at the time were almost uniformly progressive staphylococcal infections.
So the hierarchy of evidence - case reports, case control studies, randomised trials - is appropriate 99.9% of the time but you also need to know when to lay it aside.