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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).
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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.
The observation only seen in MKN7 suggests that PD98059 treatment may partially act through the JAK-STAT pathway in MKN7.
Another observation only made in patients treated with CYC was a significant increase of circulating CD8+CD44+CD62L− effector T cells (19.9 (2.2 to 157.6) versus 35.5 (4.9 to 227.8 /μl; P = 0.0384).
Compatibly with this observation, only two stretches in the primary sequence of Comm were shown to be evolutionarily conserved: the so called LPSY sorting motif and the transmembrane and juxtamembrane region [12], [22].
Although the present study includes patients more frequently treated with observation only than those in the Kitada et al report, the prognosis and 10-year radiographic deterioration rates were very similar.
All patients received pegloticase in the OLE study, except for two patients who chose observation only after receiving monthly pegloticase in the RCTs.
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