Table 4.
TMA Biomarker Resolution by TA-TMA Risk Group
| Time Post-HSCT | LDH Resolved to Normal Level for Age, n/N (%) | LDH Resolved to <1.5 Times the Upper Normal Limit for Age, n/N (%) | Platelets ≥50 × 103/μ, n/N (%) | Platelet Transfusion-Independent, n/N (%) | RBC Transfusion-Independent, n/N (%) | Schistocytes Resolved, n/N (%) | Haptoglobin Recovered to Normal Level, n/N (%) | rUPCR Resolved to <2 mg/mg, n/N (%) |
|---|---|---|---|---|---|---|---|---|
| 100 days | ||||||||
| High risk TA-TMA | 28/51 (54.9) | 35/51 (68.6) | 28/51 (54.9) | 28/51 (54.9) | 25/51 (49) | 23/51 (45.1) | 33/51 64.7) | 23/51 (45.1) |
| Moderate risk TA-TMA | 27/37 (72.9) | 29/37 (78.3) | 22/37 (59.5) | 23/37 (62.2) | 21/37 (56.8) | 17/37 (45.9) | 29/37 (78.3) | 23/37 (62.2) |
| Low risk TA-TMA | 15/18 (83.3) | 16/18 (88.9) | 15/18 (83.3) | 15/18 (83.3) | 14/18 (77.8) | 15/18 (83.3) | 16/18 (88.9) | 15/18 (83.3) |
| 180 days | ||||||||
| High risk TA-TMA | 35/42 (88.1) | 39/42 (92.8) | 37/42 (88.1) | 37/42 (88.1) | 35/42 (83.3) | 30/42 (71.4) | 38/42 (90.5) | 34/42 (81) |
| Moderate risk TA-TMA | 31/36 (86) | 33/36 (91.7) | 27/36 (75) | 25/36 (69.4) | 25/36 (69.4) | 24/36 (66.7) | 31/36 (86) | 28/36 (77.8) |
| Low risk TA-TMA | 15/18 (83.3) | 18/18 (100) | 15/18 (83.3) | 15/18 (83.3) | 17/18 (94.4) | 17/18 (94.4) | 18/18 (100) | 18/18 (100) |
Biomarker resolution is listed for patients who were evaluated at a specific evaluation time point (100 days and 180 days). Time to response is calculated from TA-TMA diagnosis in the moderate-risk and low-risk groups and from the start of eculizumab therapy in the high-risk group. Biomarker resolution and transfusion independence had to be sustained for at least 14 days to count as resolved.