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N = 1 trials stand as another example of the potential "confirmatory" nature of individual observations, only in this case the design is experimental.
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As spontaneous regression of the disease may be observed in very young children, some investigators are evaluating the feasibility of observation only in infants presenting with localised but unresectable NB.
The first-line treatment regimen was observation only in 629 (80.4%) patients and, ultimately, 541 (69.2%) underwent observation only, but long-term prognosis was not poor in patients with NB MAC-LD.
For this reason study conditions had changed because before we allowed several exceptions (e.g. no observation while patient sleeps, observation only in 3/4 of an hour per hour of the intervention).
In patients with stage 4s disease (trial INES99.2), treatment can be very heterogeneous, with a possibility of observation only, in the absence of clinical symptoms, or necessity of upfront chemotherapy in the presence of clinical symptoms, leading to very different total treatment burdens.
With this longer follow-up period, high-risk patients randomly assigned to chemotherapy also exhibited a significantly lower probability of disease recurrence than those randomly assigned for observation only (in the per-protocol analysis, hazard ratio (HR) = 0.48, P = 0.019; in the intention-to-treat analysis, HR = 0.74, P = not significant).
For the statistical analysis of behavioural observations only behaviours occurring in more than 10% of the approaches were used.
It is important to realize that these observations only imply that in the population studied albumin levels are higher during winter, and not that the relation between mortality and lower serum albumin levels would be different in the MONDO population.
Scientific observations only have validity if others in the same game accept them.
Even if, sometimes, your suggestions and observations only land you a kick in the pants".
I exclude observations only for the North Caucasus region in the RLMS sample (Kabardino-Balkar republic) because of the poor regional data quality.
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Justyna Jupowicz-Kozak
CEO of Professional Science Editing for Scientists @ prosciediting.com