Table 3.
Summary of clinical and radiological outcomes following surgical repair or iron-chelation therapy or both, reported in the time-period of year 2000 and November 2021 and limited to the English language (Supplemental table 1);
| Studies | Number of participants (n=168) | Outcomes (n, %) | |
|---|---|---|---|
| Clinical | Radiological | ||
| Deferiprone only | 63* | Improved 17 (27%) Stable 29 (46%) Worse 17 (27%) N/R 0 |
Improved 19 (42%) Stable 16 (36%) Worse 10 (22%) N/R 18 |
| Surgery only | 82 | Improved 24 (33%)** Stable 34 (47%)** Worse 14 (19%)** N/R 10 |
Improved 5 (25%) Stable 8 (40%) Worse 7 (35%) N/R 62 |
| Deferiprone and surgery combined | 19* | Improved 8 (73%) Stable 0 Worse 3 (27%) N/R 8 |
Improved 8 (100%) Stable 0 Worse 0 N/R 11 |
| Trientine only | 4 | Improved 0 Stable 2 (50%) Worse 2 (50%) N/R 0 |
Improved 0 Stable 1 (100%) Worse 0 N/R 3 |
| Side-effects of deferiprone (alone or combined with surgery; total n=82)† | (n, %)‡ | ||
| Fatigue | 10 (12%) | ||
| Neutropaenia; neutropaenic sepsis | 6 (7%) | ||
| Iron deficiency anaemia | 5 (6%) | ||
| Zinc deficiency | 5 (6%) | ||
| Joint pain | 4 (5%) | ||
| Abnormal liver function tests (transient) | 3 (4%) | ||
| Mouth ulcers | 2 (2%) | ||
| Transient nausea at start of treatment | 1 (1%) | ||
*
Outcomes for 2 patients who had surgery included into “deferiprone only” group 42;
†
side-effects not reported for trientine (4 individuals); N/R not reported;
‡
Including participants from ’deferiprone only’ and ’deferiprone and surgery combined’ cohorts (n=82);
**
Total at 99% due to rounding up; n, number; N/R not reported.