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By day 8 (the first assessment), physicians assessed disease activity as absent or minimal in 31 (88.6%) patients, and this response was maintained at the end of part 1.
In order to define outcome measures suitable for gene transfer trials, we prospectively assessed disease progression in MPS IIIA and IIIB subjects > 2 years old at three time points over one year (baseline, 6 and 12 months).
Torelli et al [39] assessed disease perception among a sample of 904 subjects; of 387 (123 males, 264 females) who reported headache in the past year, only one third perceived themselves as "headache sufferers", this perception being dependent on headache frequency in females only.
For these studies, the purpose of the study, the outcome assessed (disease symptom or lung function), and methods of measuring the exposure variable of interest (exposure to AFO) are included in TABLE 2. Self-reported outcomes such as wheeze, asthma, and depression were commonly used [42], [62], [68].
Two scales assessed disease severity and improvement.
Second, we assessed disease prevalence patterns over time.
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The study, which was conducted between 2002 and 2009, assessed disease-free and overall survival.
In patients with SSc, we assessed disease-related factors as possible determinants of macrovascular disease.
We also have not assessed disease-specific mortality in this analysis, instead using all-cause mortality, which would not be uncommon in studies of advanced disease.
Therefore, our group decided to develop and validate another disease-specific QOL questionnaire which covers subjectively assessed disease-specific QOL: the so-called Chronic Otitis Media Outcome Test 15 (COMOT-15) [ 2].
Significant herd effects were detected for total morbidity and all other assessed diseases.
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