Eculizumab regimen and clinical outcomes of the study population
| Patients sex, age (y) . | CAPS features . | Eculizumab regimen . | Complement activity (CH50) . | Platelet count . | CAPS outcome . | Long-term outcome . |
|---|---|---|---|---|---|---|
| F, 46 | Renal failure with cortical necrosis; colic and splenic infarcts; thrombocytopenia; MAHA; livedo reticularis | Started at day 45; 1 infusion of 900 mg | Before: 40 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 14 × 109/L; after C1: 89 × 109/L | Partial remission | No relapse; hemodialysis after CAPS; follow-up, 3 y |
| F, 42 | Renal and cardiac failures (LVEF 45%); deep vein and peripheral artery thrombosis; skin necrosis; thrombocytopenia; MAHA | Started at day 52; 900 mg/wk for 4 wk followed by 1 infusion of 1200 mg 7 d later | Before: normal; after administration: undetectable | Before C1: 66 × 109/L; after C1: 231 × 109/L | Symptoms worsened | Hemodialysis after eculizumab; death 4 mo later |
| M, 48 | Renal failure; diffuse alveolar hemorrhage; deep vein thrombosis; thrombocytopenia; livedo | Started at day 10; 1 infusion of 1200 mg | Before: 46 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 2 × 109/L; after C1: 4 × 109/L | Symptoms worsened | Improvement after rescue therapy with IVIG and rituximab; relapse 1 mo later; hemodialysis; follow-up, 5 y |
| F, 54 | Renal failure with cortical necrosis (hemodialysis); thrombocytopenia; MAHA | Started at day 3; 900 mg/wk for 4 wk | Data missing | Before C1: 92 × 109/L; after C1: 190 × 109/L | Symptoms worsened; renal graft removed; slow improvement | No relapse; hemodialysis; follow-up, 4 y |
| F, 78 | Renal failure with TMA; Multiple brain infarcts; livedo reticularis; thrombocytopenia; MAHA | Started at day 61: 900 mg/wk for 4 wk followed by 1200 mg every 15 d for 2 mo | Before: 50 U/mL (N); after administration: data missing | Before C1: 11 × 109/L; after C1: 142 × 109/L | Remission | No relapse; follow-up, 1 y |
| F, 61 | Renal failure (hemodialysis) and ischemic myocarditis (LVEF 55%); deep vein thrombosis; adrenal ischemic hemorrhage; liver and brain infarcts; livedo reticularis; thrombocytopenia | Started at day 100; 900 mg/wk for 4 wk followed by 1200 mg every 15 d for 2 mo | Before: 62 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 7 × 109/L; after C1: 70 × 109/L | Remission | CAPS relapse 1 y later; follow-up, 1 y |
| F, 25 | Diffuse alveolar hemorrhage; myocardial infarction (LVEF 52%); brain infarcts; livedo reticularis and skin necrosis; thrombocytopenia | Started at day 19’ 1 infusion of 900 mg | Before: 68 U/mL (N); after administration: data missing | Before C1: 106 × 109/L; after C1: 197 × 109/L | Remission | No relapse; follow-up, 1 y |
| M, 33 | Cardiac shock (LVEF 10%; ECMO); renal failure; intestinal ischemic ulcerations; adrenal ischemic hemorrhage; peripheral artery thrombosis; livedo reticularis and skin necrosis; thrombocytopenia; MAHA | Started at day 39 900 mg/wk for 4 wk | Before: 39 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 19 × 109/L; after C1: 43 × 109/L | Remission | No relapse; LVEF 39%; follow-up, 5 y |
| M, 67 | Renal (hemodialysis) and cardiac failures (LEVF 22%); deep vein thromboses; diffuse alveolar hemorrhage; thrombocytopenia; livedo reticularis | Started at day 25; 1 infusion of 1200 mg | Before: 60 U/mL (N); after administration: 33 U/mL (N) | Before C1: 219 × 109/L; after C1: 32 × 109/L | Symptoms worsened | Death 6 d later |
| F, 44 | Renal failure (hemodialysis); adrenal ischemic hemorrhage; thrombocytopenia MAHA | Started at day 21; 1 infusion of 900 mg | Before: 123 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 10 × 109/L; after C1: 30 × 109/L | Symptoms worsened | Death 11 d later |
| F, 58 | Renal and cardiac failures (LVEF 42%); liver and multiple brain infarcts; ischemic colitis; thrombocytopenia | Started at day 7; 900 mg/wk for 4 wk | Data missing | Before C1: 214 × 109/L; after C1: 248 × 109/L | Symptoms worsened | Death 33 d later |
| Patients sex, age (y) . | CAPS features . | Eculizumab regimen . | Complement activity (CH50) . | Platelet count . | CAPS outcome . | Long-term outcome . |
|---|---|---|---|---|---|---|
| F, 46 | Renal failure with cortical necrosis; colic and splenic infarcts; thrombocytopenia; MAHA; livedo reticularis | Started at day 45; 1 infusion of 900 mg | Before: 40 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 14 × 109/L; after C1: 89 × 109/L | Partial remission | No relapse; hemodialysis after CAPS; follow-up, 3 y |
| F, 42 | Renal and cardiac failures (LVEF 45%); deep vein and peripheral artery thrombosis; skin necrosis; thrombocytopenia; MAHA | Started at day 52; 900 mg/wk for 4 wk followed by 1 infusion of 1200 mg 7 d later | Before: normal; after administration: undetectable | Before C1: 66 × 109/L; after C1: 231 × 109/L | Symptoms worsened | Hemodialysis after eculizumab; death 4 mo later |
| M, 48 | Renal failure; diffuse alveolar hemorrhage; deep vein thrombosis; thrombocytopenia; livedo | Started at day 10; 1 infusion of 1200 mg | Before: 46 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 2 × 109/L; after C1: 4 × 109/L | Symptoms worsened | Improvement after rescue therapy with IVIG and rituximab; relapse 1 mo later; hemodialysis; follow-up, 5 y |
| F, 54 | Renal failure with cortical necrosis (hemodialysis); thrombocytopenia; MAHA | Started at day 3; 900 mg/wk for 4 wk | Data missing | Before C1: 92 × 109/L; after C1: 190 × 109/L | Symptoms worsened; renal graft removed; slow improvement | No relapse; hemodialysis; follow-up, 4 y |
| F, 78 | Renal failure with TMA; Multiple brain infarcts; livedo reticularis; thrombocytopenia; MAHA | Started at day 61: 900 mg/wk for 4 wk followed by 1200 mg every 15 d for 2 mo | Before: 50 U/mL (N); after administration: data missing | Before C1: 11 × 109/L; after C1: 142 × 109/L | Remission | No relapse; follow-up, 1 y |
| F, 61 | Renal failure (hemodialysis) and ischemic myocarditis (LVEF 55%); deep vein thrombosis; adrenal ischemic hemorrhage; liver and brain infarcts; livedo reticularis; thrombocytopenia | Started at day 100; 900 mg/wk for 4 wk followed by 1200 mg every 15 d for 2 mo | Before: 62 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 7 × 109/L; after C1: 70 × 109/L | Remission | CAPS relapse 1 y later; follow-up, 1 y |
| F, 25 | Diffuse alveolar hemorrhage; myocardial infarction (LVEF 52%); brain infarcts; livedo reticularis and skin necrosis; thrombocytopenia | Started at day 19’ 1 infusion of 900 mg | Before: 68 U/mL (N); after administration: data missing | Before C1: 106 × 109/L; after C1: 197 × 109/L | Remission | No relapse; follow-up, 1 y |
| M, 33 | Cardiac shock (LVEF 10%; ECMO); renal failure; intestinal ischemic ulcerations; adrenal ischemic hemorrhage; peripheral artery thrombosis; livedo reticularis and skin necrosis; thrombocytopenia; MAHA | Started at day 39 900 mg/wk for 4 wk | Before: 39 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 19 × 109/L; after C1: 43 × 109/L | Remission | No relapse; LVEF 39%; follow-up, 5 y |
| M, 67 | Renal (hemodialysis) and cardiac failures (LEVF 22%); deep vein thromboses; diffuse alveolar hemorrhage; thrombocytopenia; livedo reticularis | Started at day 25; 1 infusion of 1200 mg | Before: 60 U/mL (N); after administration: 33 U/mL (N) | Before C1: 219 × 109/L; after C1: 32 × 109/L | Symptoms worsened | Death 6 d later |
| F, 44 | Renal failure (hemodialysis); adrenal ischemic hemorrhage; thrombocytopenia MAHA | Started at day 21; 1 infusion of 900 mg | Before: 123 U/mL (N); after administration: <13 U/mL (undetectable) | Before C1: 10 × 109/L; after C1: 30 × 109/L | Symptoms worsened | Death 11 d later |
| F, 58 | Renal and cardiac failures (LVEF 42%); liver and multiple brain infarcts; ischemic colitis; thrombocytopenia | Started at day 7; 900 mg/wk for 4 wk | Data missing | Before C1: 214 × 109/L; after C1: 248 × 109/L | Symptoms worsened | Death 33 d later |
C1, first eculizumab administration; ECMO, extracorporeal membrane oxygenation; LVEF, left ventricular ejection fraction; N, normal value; TMA, thrombotic microangiopathy.