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Table 4 Variable interactions ‡ with QOL scores across time for sapropterin response groups and whole study sample †

From: Longitudinal quality of life analysis in a phenylketonuria cohort provided sapropterin dihydrochloride

 

Impact

Satisfaction

Total score

 

D

N

P

All

D

N

P

All

D

N

P

All

Phe tolerance (mg/day)

(+)***

(+)*

NS

(+)***

(+)*

NS

NS

(+)*

(+)**

NS

NS

(+)*

MF protein Rx (g/day)

NS

NS

NS

(−)*

(−)*

NS

NS

(−)*

(−)*

NS

NS

NS

Plasma Phe (μmoles/L)

(+)**

(+)*

NS

(+)**

(+)***

NS

NS

(+)**

(+)**

NS

NS

(+)*

  1. *p < 0.05, **p < 0.01, ***p < 0.001.
  2. ‡Interaction defined as “variable* time*response category” in longitudinal mixed regression analysis.
  3. †Results for Worries, Support, and Generally sub-scores not included since no significant changes occurred across time for those outcomes.
  4. (+) indicates positive association, (−) indicates negative association. NS = not statistically significant.
  5. D: Definitive Responders, N: Non-responders, P: Provisional Responders, All: Complete study sample.
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Health and Quality of Life Outcomes

ISSN: 1477-7525

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