Table 4.
Management and long-term outcomes
| Patient | Age now | Duration of FU | Maintenance treatment | TMA relapses | Management of relapses | Clinical characteristics at last FU |
ESRD (age) | Transplanted (age) | |||
|---|---|---|---|---|---|---|---|---|---|---|---|
| eGFR (ml/min per 1.73 m2) (CKD stage) | Proteinuria urine ACR or PCR (mg/mmol) | Hematuria (dipstick positive) | Hypertension (number of drugs) | ||||||||
| NCL25 | 47 yr | 45 yr | None | NA | na | 63 (CKD G2) | NA | NA | NA | No | No |
| NCL26 | 37 yr | 35 yr | None | Multiple | PEX | 30 (CKD G3b) | NA | NA | Yes (2) | No | No |
| NCL27 | 32 yr | 31 yr | None | No | na | ESRD | NA | Yes | Yes | Yes (28 yr) | No |
| NCL28 | 27 yr | Lost to FU | None | Multiple | NA | NA | NA | NA | NA | NA | NA |
| NCL29 | 26 yr | 25 yr | Eculizumab 2013–2014 | No | na | Transplant: creatinine level 106 μmol/l | PCR 0.06 | NA | NA | Yes (22 yr) | Yes (24 yr) |
| NCL30 | 18 yr | 16 yr | None | 3 | Supportive | 79 (CKD G2 A3) | ACR 275 | 2+ | Yes (1) | No | No |
| NCL31 | 15 yr 6 mo | 15 yr | Eculizumab | 2, while on eculizumab | FFP, PEX, eculizumab | 42 (CKD G3b A3) | ACR 262 | 1+ | Yes (1) | No | No |
| NCL32 | 14 yr | 14 yr | Eculizumab 2013–2016 | 0 | na | 34 (CKD G3b A3) | ACR 182 | 1+ | Yes (2) | No | No |
| NCL33 | 13 yr 8 mo | 13 yr | None | 3 | Supportive, FFP (1 yr), PEX | 42 (CKD G3b A3) | ACR 409 | 3+ | Yes (3) | No | No |
| NCL34 | 13 yr 7 mo | 5 yr 7 mo | None | 0 | na | 88 (CKD G1 A2) | ACR 6.4 | No | No | No | No |
| NCL35 | 11 yr 2 mo | 11 yr | Eculizumab | 1, before eculizumab | FFP, PEX, eculizumab | 174 (CKD G1 A3) | ACR 378 | 1+ | Yes (2) | No | No |
| NCL36 | 10 yr 6 mo | 10 yr | PEX 2009–2012 Eculizumab 2012–2018 |
3, before eculizumab | PEX | 103 (CKD G1 A3) | PCR 186 | Yes | Yes (2) | No | No |
| NCL37 | 14 yr 8 mo | 12 yr | None | na No NS relapse |
na | 90 (CKD G1 A3) | PCR 67 | Yes | Yes (1) | No | No |
| NCL38 | 5 yr 7 mo | 4 yr 5 mo | None | 1 | Supportive, FFP, PEX | Creatinine level 23 μmol/l (CKD G1 A3) | PCR 284 | No | Yes (4) | No | No |
| NCL39 | 4 yr 7 mo | 2 yr 8 mo | None | 0 | na | 74 (CKD G2 A3) | ACR 74 | 1+ | Yes (1) | No | No |
| NCL40 | 2 yr | 1 yr 9 mo | Eculizumab 2018 for 3 mo | 1, 6 wk after discontinuing eculizumab | Supportive | >90 (CKD G1 A1) | ACR <50 | NA | No | No | No |
ACR, albumin/creatinine ratio; CKD, chronic kidney disease; eGFR, estimated glomerular filtration rate; ESRD, end-stage renal disease; FFP, fresh frozen plasma; FU, follow-up; NA, not available; na, not applicable; NS, nephrotic syndrome; PCR, protein/creatinine ratio; PEX, plasma exchange; TMA, thrombotic microangiopathy.
The eGFR was calculated as follows: for children (<18 yr), the Schwartz formula was used: eGFR (ml/min per 1.73 m2) = (0.55 × height [cm] × K [constant])/serum creatinine level (μmol/l) × 0.0113 (correction factor for mg/dl); in the first year of life, for preterm babies, K = 0.33 and for full-term infants, K = 0.45; for infants and children between ages of 1 and 12 yr, K = 0.55; and for adolescent boys, K = 0.7. For adults, the Chronic Kidney Disease Epidemiology Collaboration equation was used: 141 × min(Scr/κ, 1)α × max(Scr/κ, 1)−1.209 × 0.993Age × 1.018 [if female] × 1.159 [if black], where Scr is serum creatinine level in mg/dl, κ is 0.7 for females and 0.9 for males, α is −0.329 for females and −0.411 for males, min indicates the minimum of Scr/κ or 1, and max indicates the maximum of Scr/κ or 1.