P09—Early Onset of Cardiomyopathy Demonstrated by Tissue Doppler Imaging in a Child With Fabry Disease
2012
nearly significantly (from 83.7 [16.3] to 74.2 [23.0]; P 0.071). In the patients treated for 7 years (n 10), the mean (SD) scores on the 8 SF-36 parameters were 65.5 (17.6) at start of ERT and 56.7 (16.1) after 7 years (P 0.094). Also among the patients treated for 7 years, social functioning had worsened significantly (from 65.5 [17.6] to 56.7 [16.1]; P 0.03). No significant changes were seen in any of the other 7 domains tested. In the patients (n 10) considered not to need ERT and followed up for 1 year, no significant change was seen in the mean (SD) of the 8 SF-36 parameter scores (73.1 [32.3] vs 75.8 [29.4]; P 0.85), or in any of the 8 domains tested. Conclusions: According to our results from this open-label follow-up study, ERT stabilized quality of life for up to 7 years in these Finnish Fabry patients. However, worsening score in social functioning shows that ERT is not able to stop the worsening totally. Social functioning had worsened in 7 of 10 patients at 7 years and in 6 of 16 patients at 4 years.
Keywords:
- Correction
- Source
- Cite
- Save
- Machine Reading By IdeaReader
0
References
0
Citations
NaN
KQI