Patient clinical characteristics
| Patient no. . | Age (y), gender . | Diagnosis (trigger) . | Phase (wk) . | ADAMTS13 activity (%) . | ADAMTS13 inhibitor (%) . | PLT count (×103/μL) . | LDH (mg/dL) . | Cr (mg/dL) . | Genetic mutation . | Previous treatments . | Current treatment . | Response to treatment . | Nonviable cells (%) . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 47, F | aHUS (kidney stone) | Acute | 75 | NA | 9 | 2505 | 2.3 | CFHR3-CFHR1 homozygous deletion; CFH heterozygous c.2850G>T, p.Gln950His | — | — | — | NA |
| Remission (20) | NA | NA | 314 | 228 | 1.9 | PEx (×5) | — | No | 63 | ||||
| Eculizumab (×8) | Yes | ||||||||||||
| 2 | 52, F | aHUS (pancreatitis) | Acute | 47 | NA | 13 | 1276 | 3.0 | CFHR3-CFHR1 heterozygous deletion; ADAMTS13 heterozygous c.2195C>T, p.Ala732Val | — | Prednisone, PEx | No | NA |
| Remission (14) | NA | NA | 187 | 190 | 1.0 | Prednisone, PEx (×3) | Eculizumab (×14) | Yes | 24 | ||||
| 3 | 64, M | aHUS (unknown) | Acute | 59 | NA | 66 | 1288 | 2.5 | None | — | — | — | NA |
| Remission (1) | NA | NA | 237 | 216 | 1.3 | PEx (×14), rituximab | Eculizumab (×7) | Yes | 28 | ||||
| 4 | 38, F | aHUS (unknown) | Acute | >100 | NA | 78 | 858 | 6.8 | CFH NM_000186: heterozygous c.472G>A,p.Val158Ile; Heterozygous c.3079G>C,p.Ala1027Pro; CFHR3-CFHR1 heterozygous deletion; ADAMTS13 NM_139025: heterozygous c.2420+3G>A; heterozygous c.3677C>T,p.Thr1226Ile | — | — | — | 46 |
| Responding | >100 | <5 | 110 | 248 | 4.3 | Hemodialysis, PEx (×5), prednisone | Eculizumab (×2) | Yes | 39 | ||||
| 5 | 59, M | aHUS (Hodgkin disease) | Acute | 15 | 67 | 39 | 676 | 2.3 | None | PEx (×4) | — | No | 42 |
| Responding | 22 | 11 | 114 | 197 | 1.4 | PEx (×4) | Eculizumab (×2), brentuximab | Yes | 41 | ||||
| Remission (1) | NA | NA | 307 | 180 | 1.6 | PEx (×4) | Eculizumab (×7) | Yes | 34 | ||||
| Remission (12) | NA | NA | 330 | 170 | 1.4 | Eculizumab (×12) | — | Yes | 43 | ||||
| 6 | 65, F | aHUS (quinine) | Acute | 71 | NA | 24 | 209 | 7.8 | CFHR3-CFHR1 heterozygous deletion; CD46 NM_0022389: heterozygous c.424G>C, p.Glu142Gln | — | Hemodialysis, PEx | No | NA |
| Remission (36) | NA | NA | 445 | 168 | 1.1 | PEx (3), hemodialysis | None | No | 32 | ||||
| Eculizumab (×5) | Yes | ||||||||||||
| 7 | 35, F | aHUS (liver transplant) | Acute | NA | NA | NA | NA | NA | None | — | — | — | NA |
| Remission | NA | NA | 108 | 75 | 1.1 | — | Eculizumab (×77) | Yes | 42 | ||||
| 8 | 23, F | aHUS | Acute | 102 | NA | 62 | 2820 | 6.3 | None | — | — | — | NA |
| Remission (60) | NA | NA | 205 | 178 | 3.5 | Eculizumab (×37) | Hemodialysis | Yes | 49 | ||||
| 9 | 34, F | aHUS | Acute | 41 | NA | 17 | 1133 | 2.4 | CFHR5 heterozygous c.1357C>G,p.Pro453Ala; THBD heterozygous c.1456G>T,p.Asp486Tyr | — | — | — | — |
| Remission (105) | 101 | NA | 238 | NA | 0.8 | Prednisone, PEx, rituximab | — | Yes | 53 | ||||
| 10 | 24, F | TTP | Acute | 6 | 82 | 111 | 556 | 1.0 | CFHR3-CFHR1 homozygous deletion | — | Prednisone, PEx (×10), Rituximab (×3) | — | NA |
| Responding | 100 | 23 | 303 | 744 | 1.1 | Prednisone, PEx (×10), rituximab (×3) | Azathioprine 100 mg/d prednisone | Yes | 2 | ||||
| 11 | 42, M | TTP | Acute | <5 | 59 | 8 | 1408 | 1.4 | None | — | Prednisone | — | 8 |
| Remission (3) | <5 | 31 | 156 | 195 | 1.0 | Prednisone, PEx (×20), rituximab | — | Yes | 17 | ||||
| 12 | 23, M | TTP | Acute | 9 | NA | 26 | 732 | 1.0 | CFHR3-CFHR1 heterozygous deletion;ADAMTS13 heterozygous c.2384C>T, p.Ala795Val; heterozygous c.1368G>T, p.Gln456His | — | None | — | NA |
| Remission (1) | 9 | 33 | 209 | 113 | 1.1 | — | PEx (×11) | Yes | 1 | ||||
| Remission (8) | 8 | NA | 228 | 126 | 1.1 | PEx (×11) | Prednisone | Yes | 2 | ||||
| 13 | 50, F | TTP | Acute | <5 | 90 | 7 | NA | 0.8 | None | PEx, rituximab (×4), prednisone, azathioprine | PEx | — | NA |
| Remission (24) | NA | NA | 173 | 186 | 0.8 | PEx, rituximab (×4), prednisone, azathioprine | — | Yes | 12 | ||||
| 14 | 64, F | TTP | Acute | < 5 | 18 | 7 | 1109 | 1.1 | None | Prednisone | PEx (×7) | No | NA |
| Remission | NA | NA | 324 | NA | 1.0 | Prednisone, PEx(×12), rituximab (×4) | None | Yes | 8 | ||||
| 15 | 42, F | TTP | Acute | <5 | 57 | 61 | 331 | 0.8 | None | — | Prednisone | NA | |
| Remission | 40 | 20 | 365 | NA | 1.0 | Prednisone (×4), rituximab | — | Yes | 7 | ||||
| 16 | 35, F | TTP | Acute | <5 | 96 | 5 | 2200 | NA | NA | — | — | — | 3 |
| 17 | 45, F | TTP | Acute | <5 | 75 | 12 | 1100 | NA | NA | — | — | — | 1 |
| 18 | 48, F | TTP | Acute | <4 | NA | 25 | 521 | 3.3 | NA | Prednisone, PEx | — | — | NA |
| Remission | 52 | NA | 310 | 136 | 1.1 | Prednisone, PEx, rituximab | — | Yes | 17 | ||||
| 19 | 50, M | TTP | Acute | 6 | 26 | 28 | 574 | 0.8 | NA | Prednisone | Prednisone | — | — |
| Remission | >100 | 14 | 335 | 618 | 1.2 | Prednisone, rituximab | — | Yes | 1 | ||||
| 20 | 57, M | TTP | Acute | 4 | 10 | 7 | 1231 | 1.3 | NA | — | PEx | — | NA |
| Remission | 64 | 9 | 241 | 165 | NA | NA | PEx, rituximab | — | Yes | 14 | |||
| 21 | 19, F | TTP | Acute | 8 | NA | 56 | 750 | 1.1 | NA | — | PEx, Prednisone | — | 1 |
| 22 | 85, F | Stec-HUS | Acute | 59 | 27 | 6 | 1662 | 3.0 | None | — | None | NA | NA |
| Remission | NA | 33 | 124 | 465 | 1.6 | PEx, prednisolone | Eculizumab (×3) | Yes | 24 | ||||
| 23 | 31, M | aPIGN | Acute | 84 | NA | 234 | 138 | 2.2 | CFHR3-CFHR1 heterozygous deletion; CFH NM_000186: heterozygous c.2850G>T,p.Gln950His ADAMTS13 NM_139205: heterozygous c.2758A>G,p.Met920Val | — | Prednisone | No | 19 |
| 24 | 36, F | DIC | Acute | NA | NA | 120 | 333 | 1.9 | NA | — | None | No | 6 |
| 25 | 69, F | DIC | Acute | 53 | 6 | 44 | 360 | 1.2 | NA | — | None | No | 9 |
| Patient no. . | Age (y), gender . | Diagnosis (trigger) . | Phase (wk) . | ADAMTS13 activity (%) . | ADAMTS13 inhibitor (%) . | PLT count (×103/μL) . | LDH (mg/dL) . | Cr (mg/dL) . | Genetic mutation . | Previous treatments . | Current treatment . | Response to treatment . | Nonviable cells (%) . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 47, F | aHUS (kidney stone) | Acute | 75 | NA | 9 | 2505 | 2.3 | CFHR3-CFHR1 homozygous deletion; CFH heterozygous c.2850G>T, p.Gln950His | — | — | — | NA |
| Remission (20) | NA | NA | 314 | 228 | 1.9 | PEx (×5) | — | No | 63 | ||||
| Eculizumab (×8) | Yes | ||||||||||||
| 2 | 52, F | aHUS (pancreatitis) | Acute | 47 | NA | 13 | 1276 | 3.0 | CFHR3-CFHR1 heterozygous deletion; ADAMTS13 heterozygous c.2195C>T, p.Ala732Val | — | Prednisone, PEx | No | NA |
| Remission (14) | NA | NA | 187 | 190 | 1.0 | Prednisone, PEx (×3) | Eculizumab (×14) | Yes | 24 | ||||
| 3 | 64, M | aHUS (unknown) | Acute | 59 | NA | 66 | 1288 | 2.5 | None | — | — | — | NA |
| Remission (1) | NA | NA | 237 | 216 | 1.3 | PEx (×14), rituximab | Eculizumab (×7) | Yes | 28 | ||||
| 4 | 38, F | aHUS (unknown) | Acute | >100 | NA | 78 | 858 | 6.8 | CFH NM_000186: heterozygous c.472G>A,p.Val158Ile; Heterozygous c.3079G>C,p.Ala1027Pro; CFHR3-CFHR1 heterozygous deletion; ADAMTS13 NM_139025: heterozygous c.2420+3G>A; heterozygous c.3677C>T,p.Thr1226Ile | — | — | — | 46 |
| Responding | >100 | <5 | 110 | 248 | 4.3 | Hemodialysis, PEx (×5), prednisone | Eculizumab (×2) | Yes | 39 | ||||
| 5 | 59, M | aHUS (Hodgkin disease) | Acute | 15 | 67 | 39 | 676 | 2.3 | None | PEx (×4) | — | No | 42 |
| Responding | 22 | 11 | 114 | 197 | 1.4 | PEx (×4) | Eculizumab (×2), brentuximab | Yes | 41 | ||||
| Remission (1) | NA | NA | 307 | 180 | 1.6 | PEx (×4) | Eculizumab (×7) | Yes | 34 | ||||
| Remission (12) | NA | NA | 330 | 170 | 1.4 | Eculizumab (×12) | — | Yes | 43 | ||||
| 6 | 65, F | aHUS (quinine) | Acute | 71 | NA | 24 | 209 | 7.8 | CFHR3-CFHR1 heterozygous deletion; CD46 NM_0022389: heterozygous c.424G>C, p.Glu142Gln | — | Hemodialysis, PEx | No | NA |
| Remission (36) | NA | NA | 445 | 168 | 1.1 | PEx (3), hemodialysis | None | No | 32 | ||||
| Eculizumab (×5) | Yes | ||||||||||||
| 7 | 35, F | aHUS (liver transplant) | Acute | NA | NA | NA | NA | NA | None | — | — | — | NA |
| Remission | NA | NA | 108 | 75 | 1.1 | — | Eculizumab (×77) | Yes | 42 | ||||
| 8 | 23, F | aHUS | Acute | 102 | NA | 62 | 2820 | 6.3 | None | — | — | — | NA |
| Remission (60) | NA | NA | 205 | 178 | 3.5 | Eculizumab (×37) | Hemodialysis | Yes | 49 | ||||
| 9 | 34, F | aHUS | Acute | 41 | NA | 17 | 1133 | 2.4 | CFHR5 heterozygous c.1357C>G,p.Pro453Ala; THBD heterozygous c.1456G>T,p.Asp486Tyr | — | — | — | — |
| Remission (105) | 101 | NA | 238 | NA | 0.8 | Prednisone, PEx, rituximab | — | Yes | 53 | ||||
| 10 | 24, F | TTP | Acute | 6 | 82 | 111 | 556 | 1.0 | CFHR3-CFHR1 homozygous deletion | — | Prednisone, PEx (×10), Rituximab (×3) | — | NA |
| Responding | 100 | 23 | 303 | 744 | 1.1 | Prednisone, PEx (×10), rituximab (×3) | Azathioprine 100 mg/d prednisone | Yes | 2 | ||||
| 11 | 42, M | TTP | Acute | <5 | 59 | 8 | 1408 | 1.4 | None | — | Prednisone | — | 8 |
| Remission (3) | <5 | 31 | 156 | 195 | 1.0 | Prednisone, PEx (×20), rituximab | — | Yes | 17 | ||||
| 12 | 23, M | TTP | Acute | 9 | NA | 26 | 732 | 1.0 | CFHR3-CFHR1 heterozygous deletion;ADAMTS13 heterozygous c.2384C>T, p.Ala795Val; heterozygous c.1368G>T, p.Gln456His | — | None | — | NA |
| Remission (1) | 9 | 33 | 209 | 113 | 1.1 | — | PEx (×11) | Yes | 1 | ||||
| Remission (8) | 8 | NA | 228 | 126 | 1.1 | PEx (×11) | Prednisone | Yes | 2 | ||||
| 13 | 50, F | TTP | Acute | <5 | 90 | 7 | NA | 0.8 | None | PEx, rituximab (×4), prednisone, azathioprine | PEx | — | NA |
| Remission (24) | NA | NA | 173 | 186 | 0.8 | PEx, rituximab (×4), prednisone, azathioprine | — | Yes | 12 | ||||
| 14 | 64, F | TTP | Acute | < 5 | 18 | 7 | 1109 | 1.1 | None | Prednisone | PEx (×7) | No | NA |
| Remission | NA | NA | 324 | NA | 1.0 | Prednisone, PEx(×12), rituximab (×4) | None | Yes | 8 | ||||
| 15 | 42, F | TTP | Acute | <5 | 57 | 61 | 331 | 0.8 | None | — | Prednisone | NA | |
| Remission | 40 | 20 | 365 | NA | 1.0 | Prednisone (×4), rituximab | — | Yes | 7 | ||||
| 16 | 35, F | TTP | Acute | <5 | 96 | 5 | 2200 | NA | NA | — | — | — | 3 |
| 17 | 45, F | TTP | Acute | <5 | 75 | 12 | 1100 | NA | NA | — | — | — | 1 |
| 18 | 48, F | TTP | Acute | <4 | NA | 25 | 521 | 3.3 | NA | Prednisone, PEx | — | — | NA |
| Remission | 52 | NA | 310 | 136 | 1.1 | Prednisone, PEx, rituximab | — | Yes | 17 | ||||
| 19 | 50, M | TTP | Acute | 6 | 26 | 28 | 574 | 0.8 | NA | Prednisone | Prednisone | — | — |
| Remission | >100 | 14 | 335 | 618 | 1.2 | Prednisone, rituximab | — | Yes | 1 | ||||
| 20 | 57, M | TTP | Acute | 4 | 10 | 7 | 1231 | 1.3 | NA | — | PEx | — | NA |
| Remission | 64 | 9 | 241 | 165 | NA | NA | PEx, rituximab | — | Yes | 14 | |||
| 21 | 19, F | TTP | Acute | 8 | NA | 56 | 750 | 1.1 | NA | — | PEx, Prednisone | — | 1 |
| 22 | 85, F | Stec-HUS | Acute | 59 | 27 | 6 | 1662 | 3.0 | None | — | None | NA | NA |
| Remission | NA | 33 | 124 | 465 | 1.6 | PEx, prednisolone | Eculizumab (×3) | Yes | 24 | ||||
| 23 | 31, M | aPIGN | Acute | 84 | NA | 234 | 138 | 2.2 | CFHR3-CFHR1 heterozygous deletion; CFH NM_000186: heterozygous c.2850G>T,p.Gln950His ADAMTS13 NM_139205: heterozygous c.2758A>G,p.Met920Val | — | Prednisone | No | 19 |
| 24 | 36, F | DIC | Acute | NA | NA | 120 | 333 | 1.9 | NA | — | None | No | 6 |
| 25 | 69, F | DIC | Acute | 53 | 6 | 44 | 360 | 1.2 | NA | — | None | No | 9 |
aPIGN, atypical postinfectious glomerulonephritis; CFHR, complement factor H–related proteins; Cr, creatinine; F, female; LDH, lactate dehydrogenase; M, male; NA, not available; PAI-1, plasminogen activator inhibitor-1, plasma exchange; PLT, platelet; Stec-HUS, Shiga-toxin–associated hemolytic uremic syndrome; THBD, thrombomodulin; PEx, plasma exchange.