Table 1.
Clinical information on 20 patients with heterozygous MCP mutation and aHUS
| Family Nr. | Gender | Age of onset (yrs) | Age at first relapse (yrs) | Time until first relapse (yrs) | Nr. of relapses | Best GFR after FM | Best GFR last follow-up | Years of follow-up | Eculizumab |
|---|---|---|---|---|---|---|---|---|---|
| 1 | m | 1.6 | 6.8 | 5.3 | 2 | 148 | 5 | 7.8 | / |
| 2 | m | 15.3 | / | / | 0 | 98 | 98 | 0.2 | / |
| 3 | m | 5.8 | 7.6 | 1.8 | 1 | 70 | 118 | 1.8 | / |
| 4 | m | 1.9 | 2.7 | 0.7 | 11 | 102 | 105 | 14.8 | Long-term after the eleventh relapse |
| 5 | f | 4.4 | / | / | 0 | 148 | 148 | 2.3 | / |
| 6 | m | 0.6 | 2.3 | 1.8 | 1 | 67 | 82 | 2.3 | Long-term after FM |
| 7 | f | 4.8 | / | / | 0 | 108 | 105 | 0.3 | / |
| 8 | m | 8 | 14 | 6 | 1 | 69 | 94 | 6 | / |
| 9 | m | 1.2 | / | / | 0 | 114 | 125 | 2.8 | / |
| 10 | m | 14.1 | / | / | 0 | 97 | 85 | 2.4 | / |
| 11 | f | 2.8 | / | / | 0 | 144 | 112 | 2.3 | Long-term after FM |
| 12 | f | 6 | / | / | 0 | 150 | 151 | 6.2 | / |
| 13 | m | 3 | 6.8 | 3.8 | 3 | 113 | 124 | 9.8 | Long-term after the third relapse |
| 14 | m | 3.2 | 10.7 | 7.5 | 2 | 63 | 85 | 13.8 | / |
| 15 | m | 12.3 | / | / | 0 | 80 | 80 | 0.5 | / |
| 16 | m | 4.8 | 14.3 | 9.4 | 1 | 70 | 82 | 9.5 | After the first relapse, on-demand |
| 17 | m | 4.1 | 5.8 | 1.6 | 2 | 151 | 133 | 3.3 | After the first relapse, on-demand |
| 18 | m | 10.7 | / | / | 0 | 108 | 124 | 1.8 | After FM, on-demand |
| 19 | f | 4.1 | / | / | 0 | 96 | 90 | 1.4 | / |
| 20 | m | 6.1 | 7.5 | 1.4 | 7 | 69 | 93 | 15.9 | / |
GFR glomerular filtration rate, f female, FM first manifestation, m male, Nr number, Y yes, yrs years. GFR in ml/min/1.73 m2
/, not applicable