Compilation of clinical parameters obtained at the beginning and end of study
| Patient . | Age/sex, y/F, M . | GAA size, kb . | Age at disease onset, y . | Ataxia duration, y . | DFP treatment, mg/kg/d-months . | ICARS 7 points, /100 . | Observations by parents/relatives and supporting staff . |
|---|---|---|---|---|---|---|---|
| A* | 15/F | G130V/2.1 | 12 | 3 | 30-6 | 29 → 26 | Improved gait and standing, no constipation |
| B | 23/F | 0.45/0.93 | 12 | 11 | 20-6 | 31 → 29 | Feels feet in swimming pool (therapy re-education), speech fluency, no constipation |
| C* | 14/M | 2/2.8 | 7 | 7 | 20-6 | 30 → 14 | Improved gait, writing, delicate movements, can move toes, feels feet, warming up feet |
| D* | 18/M | 2.2/3.7 | 10 | 8 | 20-6 | 31 → 27* | Improved gait and balance, warming up feet, good appetite, no constipation, no incontinence |
| E | 17/F | 2.5/2.6 | 9 | 8 | 20-6 | 39 → 37 | Improved gait, writing, delicate movements, can move toes, feels her feet, warming up feet |
| F | 13/M | 2.4/3.9 | 5 | 8 | 20-6 | 61 → 59 | No fatigue, delicate movements |
| G* | 20/F | 0.75/0.75 | 14 | 6 | 20-6 | 23 → 20 | Improved gait and balance, warming up feet, no incontinence |
| H* | 18/F | 1.6/3.6 | 11 | 7 | 30-6 | 23 → 20 | Improved gait and balance, no bending, no incontinence, no constipation |
| I | 20/F | 2.5/2.6 | 10 | 10 | 30-6 | 50 → 50 | Improved walking, warming up feet, no incontinence |
| Patient . | Age/sex, y/F, M . | GAA size, kb . | Age at disease onset, y . | Ataxia duration, y . | DFP treatment, mg/kg/d-months . | ICARS 7 points, /100 . | Observations by parents/relatives and supporting staff . |
|---|---|---|---|---|---|---|---|
| A* | 15/F | G130V/2.1 | 12 | 3 | 30-6 | 29 → 26 | Improved gait and standing, no constipation |
| B | 23/F | 0.45/0.93 | 12 | 11 | 20-6 | 31 → 29 | Feels feet in swimming pool (therapy re-education), speech fluency, no constipation |
| C* | 14/M | 2/2.8 | 7 | 7 | 20-6 | 30 → 14 | Improved gait, writing, delicate movements, can move toes, feels feet, warming up feet |
| D* | 18/M | 2.2/3.7 | 10 | 8 | 20-6 | 31 → 27* | Improved gait and balance, warming up feet, good appetite, no constipation, no incontinence |
| E | 17/F | 2.5/2.6 | 9 | 8 | 20-6 | 39 → 37 | Improved gait, writing, delicate movements, can move toes, feels her feet, warming up feet |
| F | 13/M | 2.4/3.9 | 5 | 8 | 20-6 | 61 → 59 | No fatigue, delicate movements |
| G* | 20/F | 0.75/0.75 | 14 | 6 | 20-6 | 23 → 20 | Improved gait and balance, warming up feet, no incontinence |
| H* | 18/F | 1.6/3.6 | 11 | 7 | 30-6 | 23 → 20 | Improved gait and balance, no bending, no incontinence, no constipation |
| I | 20/F | 2.5/2.6 | 10 | 10 | 30-6 | 50 → 50 | Improved walking, warming up feet, no incontinence |
The table depicts the age and sex of FA patients, the genetic definition of mutation in terms of size of GAA expansion in the frataxin gene, the age at onset of the disease, duration of ataxia, the doses of deferiprone (DFP) administration, and the ICARS scores prior to and at the end of the trial (indicated with arrow). The mean decrease ± SD in ICARS of all the 9 patients was 8.5 ± 4.5 points representing a relatively small but significant (P > .05 Student paired t test) decrease of 7.3% ± 1.1%.
Patients who showed greater than 10% changes in ICARS after 6-month DFP treatment relative to values attained before treatment.