Treatment of dysferlinopathy with deflazacort: a double-blind, placebo-controlled clinical trial

Table 2 Health-related quality of life assessment with the sf-36: baseline data and changes during study phases

SF-36 scale	Baseline^†	Natural history	Deflazacort (pooled)	Placebo (pooled)	p-value
SF-36 scale	Baseline^†	End of year 1 – begin*	End – begin of crossover*	End – begin of crossover*	Deflazacort vs. placebo^‡
Subscales
Physical functioning	38.3 ± 19.8 (5–85)	−0.1 ± 13.4	−6.8 ± 9.3	+1.5 ± 11.1	0.118
Role Physical	70.6 ± 36.7 (0–100)	+11.8 ± 46.9	−22.4 ± 32.2	+6.6 ± 38.0	0.114
Role Emotional	91.7 ± 22.8 (33–100)	+6.3 ± 25.0	−14.8 ± 46.1	+13.0 ± 34.6	0.037
Social functioning	86.8 ± 12.5 (62–100)	0 ± 12.9	−5.9 ± 23.0	−3.3 ± 14.3	0.963
Mental health	79.8 ± 12.7 (44–96)	−0.2 ± 5.6	−9.1 ± 9.4	+2.9 ± 14.3	0.009
Bodily pain	73.2 ± 16.2 (12–100)	+7.1 ± 21.5	−1.9 ± 25.4	+3.2 ± 18.8	0.394
Vitality	60.0 ± 16.3 (40–95)	+0.3 ± 11.3	−7.4 ± 16.1	+1.8 ± 11.2	0.096
General health perception	45.5 ± 14.4 (25–82)	+6.0 ± 10.9	−4.3 ± 11.6	−1.7 ± 11.0	0.965
Standardized component Scales
Physical	36.4 ± 8.0 (19–50.4)	+1.5 ± 5.5	−2.5 ± 6.6	+0.4 ± 5.3	0.3123
Mental	56.2 ± 5.8 (40–65)	+0.6 ± 4.6	−4.2 ± 10.7	+1.6 ± 8.7	0.0922

†: The column shows arithmetic mean ± standard deviations (minimum and maximum) values. The scoring of the SF-36 scales ranges from 0 = very bad to 100 = very good quality of life in the different domains of the scale.
*: The column shows arithmetic mean ± standard deviations for changes between end and begin of each study period. Deflazacort and placebo values are pooled across both crossover periods.
‡: p-values associated with the treatment effect in Lehmacher’s (1991) proposal for the non-parametric analysis of crossover-data (deflazacort vs. placebo). With the exception of “role emotional”, there were no carry-over effects detected. No period effects were identified.

ISSN: 1750-1172