Table 1. Definitions of dialysis status and outcome measures.
| Dialysis status | Definition | |
|---|---|---|
| At baseline | Recorded as “yes” for patients who received dialysis within: • 5 days before study drug initiation (trials ALXN-aHUS-311 and ALXN-aHUS-312 (ravulizumab), and C10-003 and C10-004 (eculizumab)) • 7 days before study drug initiation (trial C08-002 (eculizumab)) |
|
| At endpoint | Recorded as “yes” for patients who received dialysis within: • 5 days before endpoint measure (trials ALXN-aHUS-311 and ALXN-aHUS-312 (ravulizumab)) • 7 days before endpoint measure (trials C08-002, C10-003, and C10-004 (eculizumab)) |
|
| Outcome measure | Improvement/normalization | Normal range |
| SCr concentration for non-dialysis patients | An improvement (decrease) in SCr of ≥ 25% from baseline sustained in ≥ 2 consecutive measures, ≥ 4 weeks apart Response to have been achieved at any time during the 26-week period |
74 mmol/L ≤ observation ≤ 107 mmol/L |
| Platelet count | Platelet count ≥ 150 (×109/L) sustained in ≥ 2 consecutive measures, ≥ 4 weeks apart Response to have been achieved at any time during the 26-week period |
Observation ≥ 150 (×109/L) |
| LDH concentration | LDH concentration < 246 U/L sustained in ≥ 2 consecutive measures, ≥ 4 weeks apart Response to have been achieved at any time during the 26-week period |
Observation < 246 U/L |
| eGFR | An improvement of ≥ 15 mL/min/1.73m2 in eGFR, from baseline to 26 weeks | Observation ≥ 60 mL/min/1.73m2 |
| FACIT-F subscale score | Improvement was derived using a threshold of a 3-point improvement from baseline to 26 weeks Higher scores indicate less fatigue |
0 – 52 points |
| EQ-5D VAS score | Improvement was derived using a threshold of a 10-point improvement from baseline to 26 weeks Higher scores indicate better quality of life |
|
| cTMA response | Criteria met simultaneously for SCr improvement, LDH normalization and platelet count normalization | See criteria for SCr improvement, LDH normalization, and platelet count normalization in the respective table rows above |
cTMA = complete thrombotic microangiopathy; eGFR = estimated glomerular filtration rate; EQ-5D = 5-dimension EuroQol questionnaire; FACIT-F = Functional Assessment of Chronic Illness Therapy-Fatigue; LDH = lactate dehydrogenase; SCr = serum creatinine; VAS = visual analog scale.