Individual characteristics of 6 children and 7 adults who experienced aHUS relapse after eculizumab discontinuation
| Patient . | Sex, age, y . | Complement gene variant . | At eculizumab discontinuation . | Precipitating factor of aHUS relapse . | At aHUS relapse . | At 3 mo after aHUS relapse and eculizumab restart . | At last follow-up . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| SCr, μmol/L (eGFR, mL/min/1.73 m2) . | UP/Cr, g/mmol . | Time from eculizumab discontinuation, mo . | SCr, μmol/L . | Plt × 109/L . | UP/Cr, g/mmol . | SCr, μmol/L (eGFR, mL/min/1.73 m2) . | UP/Cr, g/mmol . | Duration, mo . | SCr, μmol/L (eGFR, mL/min/1.73 m2) . | UP/Cr, g/mmol . | ||||
| 1 | M, 4 | CFI (p.Gly261Asp)/C3 (p.Thr1383Asn) | 34.0 (104) | 0.018 | Bacterial infection | 5.4 | 62 | 95 | 0.04 | 34.0 (107) | 0.01 | 18.0 | 46.0 (85) | 0.012 |
| 2* | F, 6. | CFH (p.Ser1191Trp) | 32.0 (128) | 0.03 | Flu-like illness | 11.5 | 92 | 63 | 1.28 | 33.0 (130) | 0.04 | 3.2 | 33.0 (130) | 0.04 |
| F, 7 | 34.0 (127) | 0.03 | Flu-like illness | 8.1 | 69 | 126 | 1.06 | 36.0 (127) | 0.03 | 18.3 | 38.0 (126) | 0.08 | ||
| 3† | F, 7 | MCP (persistently low CD46 level) | 52.0 (79) | 0.008 | Flu-like illness | 9.9 | 105 | 94 | 3.08 | 50 (78)‡ | 0.01 | 14.7 | 53.0 (85) | 0.009 |
| 4 | F, 8 | MCP (p.Asp33His)/MCP (p.Asp33His) | 34.0 (143) | 0.01 | Gastroenteritis | 20.5 | 188 | 56 | 5.66 | 32.0 (145) | 0.02 | 2.8 | 32.0 (145) | 0.02 |
| 5 | M, 9 | MCP (IVS2+2)/MCP (IVS2+2) | 39.1 (169) | 3.0 | Gastroenteritis | 13.4 | 214 | 72 | 1.21 | 32.8 (210) | 0.05 | 8.7 | 35.6 (199) | 0.05 |
| 6§ | M, 9 | None | 45.0 (109) | 0.01 | Tonsillitis | 17.2 | 45 | 62 | NA | 46 (108)‖ | NA | 15.0 | 47.0 (148) | 0.01 |
| 7 | F, 30 | C3 (p.Ala1094Ser) | 136.0 (42) | 0.08 | Sinusitis | 2.5 | 191 | 138 | 0.28 | 131.0 (44) | 0.15 | 18.5 | 148.5 (37) | 0.1 |
| 8 | F, 34 | CFH (p.Phe1199Leu) | 121.0 (47) | 0.03 | Tracheitis | 20.0 | 165 | 209 | 0.06 | 125.0 (45) | 0.03 | 6.6 | 129.0 (43) | 0.06 |
| 9 | F, 34 | MCP (p.Tyr117Stop) | 93.0 (63) | 0.13 | Diarrhea | 1.6 | 184 | 57 | 0.36 | 89.3 (66) | 0.1 | 23.7 | 77.8 (77) | 0.05 |
| 10 | F, 38 | MCP (IVS2+2)/MCP (IVS2+2) | 121.0 (46) | 0.22 | Viral tonsillitis | 2.5 | 163 | 113 | 0.37 | 145.0 (37) | 0.05 | 20.9 | 132.0 (41) | 0.08 |
| 11 | M, 44 | MCP (IVS2+2) | 245.0 (27) | 0.15 | — | 3.6 | 414 | 143 | 0.26 | 426.0 (14) | 0.16 | 10.8 | 881.0 (6)¶ | 0.21 |
| 12 | F, 53# | CFI (p.Pro50Ala) | 64.0 (89) | 0.01 | Pancreatitis | 3.7 | 802 | 30 | 0.25 | 69.0 (82) | NA | 21.1 | 64.0 (89) | 0.04 |
| 13 | F, 56 | CFH (p.Arg1215Stop) | 101 (52) | 0.02 | — | 22.1 | 232 | 235 | 0.26 | 168.0 (29) | 0.07 | 10.8 | 167.0 (29) | 0.04 |
| Patient . | Sex, age, y . | Complement gene variant . | At eculizumab discontinuation . | Precipitating factor of aHUS relapse . | At aHUS relapse . | At 3 mo after aHUS relapse and eculizumab restart . | At last follow-up . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| SCr, μmol/L (eGFR, mL/min/1.73 m2) . | UP/Cr, g/mmol . | Time from eculizumab discontinuation, mo . | SCr, μmol/L . | Plt × 109/L . | UP/Cr, g/mmol . | SCr, μmol/L (eGFR, mL/min/1.73 m2) . | UP/Cr, g/mmol . | Duration, mo . | SCr, μmol/L (eGFR, mL/min/1.73 m2) . | UP/Cr, g/mmol . | ||||
| 1 | M, 4 | CFI (p.Gly261Asp)/C3 (p.Thr1383Asn) | 34.0 (104) | 0.018 | Bacterial infection | 5.4 | 62 | 95 | 0.04 | 34.0 (107) | 0.01 | 18.0 | 46.0 (85) | 0.012 |
| 2* | F, 6. | CFH (p.Ser1191Trp) | 32.0 (128) | 0.03 | Flu-like illness | 11.5 | 92 | 63 | 1.28 | 33.0 (130) | 0.04 | 3.2 | 33.0 (130) | 0.04 |
| F, 7 | 34.0 (127) | 0.03 | Flu-like illness | 8.1 | 69 | 126 | 1.06 | 36.0 (127) | 0.03 | 18.3 | 38.0 (126) | 0.08 | ||
| 3† | F, 7 | MCP (persistently low CD46 level) | 52.0 (79) | 0.008 | Flu-like illness | 9.9 | 105 | 94 | 3.08 | 50 (78)‡ | 0.01 | 14.7 | 53.0 (85) | 0.009 |
| 4 | F, 8 | MCP (p.Asp33His)/MCP (p.Asp33His) | 34.0 (143) | 0.01 | Gastroenteritis | 20.5 | 188 | 56 | 5.66 | 32.0 (145) | 0.02 | 2.8 | 32.0 (145) | 0.02 |
| 5 | M, 9 | MCP (IVS2+2)/MCP (IVS2+2) | 39.1 (169) | 3.0 | Gastroenteritis | 13.4 | 214 | 72 | 1.21 | 32.8 (210) | 0.05 | 8.7 | 35.6 (199) | 0.05 |
| 6§ | M, 9 | None | 45.0 (109) | 0.01 | Tonsillitis | 17.2 | 45 | 62 | NA | 46 (108)‖ | NA | 15.0 | 47.0 (148) | 0.01 |
| 7 | F, 30 | C3 (p.Ala1094Ser) | 136.0 (42) | 0.08 | Sinusitis | 2.5 | 191 | 138 | 0.28 | 131.0 (44) | 0.15 | 18.5 | 148.5 (37) | 0.1 |
| 8 | F, 34 | CFH (p.Phe1199Leu) | 121.0 (47) | 0.03 | Tracheitis | 20.0 | 165 | 209 | 0.06 | 125.0 (45) | 0.03 | 6.6 | 129.0 (43) | 0.06 |
| 9 | F, 34 | MCP (p.Tyr117Stop) | 93.0 (63) | 0.13 | Diarrhea | 1.6 | 184 | 57 | 0.36 | 89.3 (66) | 0.1 | 23.7 | 77.8 (77) | 0.05 |
| 10 | F, 38 | MCP (IVS2+2)/MCP (IVS2+2) | 121.0 (46) | 0.22 | Viral tonsillitis | 2.5 | 163 | 113 | 0.37 | 145.0 (37) | 0.05 | 20.9 | 132.0 (41) | 0.08 |
| 11 | M, 44 | MCP (IVS2+2) | 245.0 (27) | 0.15 | — | 3.6 | 414 | 143 | 0.26 | 426.0 (14) | 0.16 | 10.8 | 881.0 (6)¶ | 0.21 |
| 12 | F, 53# | CFI (p.Pro50Ala) | 64.0 (89) | 0.01 | Pancreatitis | 3.7 | 802 | 30 | 0.25 | 69.0 (82) | NA | 21.1 | 64.0 (89) | 0.04 |
| 13 | F, 56 | CFH (p.Arg1215Stop) | 101 (52) | 0.02 | — | 22.1 | 232 | 235 | 0.26 | 168.0 (29) | 0.07 | 10.8 | 167.0 (29) | 0.04 |
eGFR determined using the Modification of Diet in Renal Disease formula in adults and the Schwartz formula in children.
NA, not available; Plt, platelet; SCr, serum creatinine; UP/Cr, urinary protein/creatinine ratio.
*Patient was included twice in the study; †Patient had a familial form of aHUS; ‡Data at <1 mo after relapse; §Patient was diagnosed with hereditary ADAMTS13 deficiency after completion of the study; ‖Data at 6 mo after relapse; ¶Patient reached end-stage renal disease and subsequently underwent kidney transplantation; #Kidney transplantation patient.