Monday, 11 October 2021

Not Statistical But 'Clinically Meaningful'?

It's difficult to properly evaluate a medical study that, whilst failing to show significant effects, did apparently generate some striking cases. You wouldn't be able to proceed with a new vaccine on this basis! London's Cancer Research Institute and the Royal Marsden NHS Foundation Trust studied almost 1000 terminally-ill head and neck cancer patients. The patients were given a combination of 2 drugs (nivolumab and ipilimumab, designed to activate the body's immune system). The researchers looked at the effects of the drug combination on the size(s) of the patient's tumour(s) (https://www.theguardian.com/society/2021/oct/11/new-cancer-treatment-destroys-tumours-in-terminally-ill-finds-trial). The group of patients was apparently large but they must have been somewhat heterogeneous. They would have varied in terms of the type of their tumour, its location, how long it had been present etc. There are lots of different tissues in the head and the neck. The patients would also differ in terms of age, gender, general health etc. Although the comparisons did not reach significance, some patients lived months or years longer than expected. In a few exceptional cases, the tumours disappeared. The drug combination also had much milder side effects than those produced by extreme chemotherapy. Extreme chemotherapy has been all that was available for terminally ill cancer patients. It is worth describing the study as 'clinically meaningful'. More work is, however, required. Perhaps the drug combination is more effective against certain types of tumour or tumours in particular locations?

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