How can we determine the safety if anything we do in paediatric prescribing? For chronic conditions, we’re generally pretty sure that if we let it wind on, it will harm the child. If we treat it, we’ll be managing the disease but causing adversity. The balance is making this tip where the good stuff overwhelms the poor stuff.
I think the commonest, extreme, example is chemotherapy. These agents are intended to treat a cancer to save a life. To do this, they may cause sufficient immunosupression to produce a fatal infection, or mucosal erosions to give a fatal intestinal perforation, or a thrombotic event that produces a cerebral infarct and death. The carefully measured doses of these drugs are placed to make the tipping point in favour of benefit over harm; and we have improved survival in childhood cancer by this treatment approach.
Where the challenge becomes greater is where the condition we are treating may not be immediately life threatening, or where the side effects are less mortal and more morbid, or where a treatment is new and the uncertainties are greater. The challenges here are in determining what constitutes benefit over harm; how this might change in eyes of different patients, and how to make such decisions without enough evidence.
Let’s take a look at this latter question. There are some data, particularly within adults, that have shown that knowledge of expected side effects changes the reports of adverse experiences, so we need to see how the reports very between placebo and new treatment group … and yet some very rare events won’t be adequately captured in randomised trials of generally small numbers and short duration, so we’ll need to look at open, uncontrolled, observational series to do that. Or data from adults. (And we’ll also need to remember that we may not record things we don’t ask about and that some VERY NAUGHTY PEOPLE don’t report all trials.)
So:
- Always examine trial data for adverse effects; consider very much that lack of statistical significance is NOT evidence of no difference
- If you can’t find something reported, it doesn’t mean it hasn’t happened.
- Data from adult studies might be really really helpful. Whisper it – but adult & paed data often are not that different
- Listen to your patients and their families.
– Archi