Table 1.
Demographic, clinical, analytical, and therapeutical characteristics of all patients, including responses
| Patient | 1 | 2 | 3 | 4 | 5 | 6 | 7 | 8 | 9 | 10 | 11 | 12 | Median, % |
| Gender | M | M | F | M | F | F | F | F | M | F | F | F | |
| Age (years) | 55 | 45 | 36 | 57 | 53 | 25 | 46 | 66 | 36 | 61 | 44 | 71 | 49.5 |
| TTP episode | 1st | 1st | 1st | 1st | 2nd | 1st | 4th | 1st | 1st | 2nd | 1st | 2nd | |
| Symptoms | Neurol. | Neurol. Bleeding | Neurol. Bleeding | Neurol. | Bleeding | Neurol. |
Neurol. Bleeding |
Bleeding | Abdominal pain |
Neurol. Bleeding |
Bleeding |
Bleeding Weakness |
|
| Hemoglobin (g/dL) | 7.0 | 8.8 | 8.9 | 7.8 | 10.6 | 8.5 | 10.7 | 7.9 | 7.7 | 8.8 | 5.3 | 9.4 | 8.65 |
| Platelets (× 109/L) | 11 | 17 | 27 | 6 | 28 | 11 | 11 | 5 | 14 | 11 | 5 | 8 | 11 |
| LDH (U/L) | 1130 | 1333 | 589 | 2649 | 402 | 1779 | 646 | 1843 | 401 | 322 | 1081 | 1117 | 1099 |
| Bilirubin (mg/dL) | 3.10 | 1.40 | 0.80 | 2.92 | 0.52 | 2.76 | 1.86 | 3.55 | 4.0 | 1.55 | 0.77 | 4 | 2.31 |
| Haptoglobin (mg/dL) | < 6 | < 6 | < 6 | < 6 | < 6 | < 6 | 114 | - | 8 | - | 8 | 38 | 23 |
| Schistocytes (%) | 7 | 10 | 7 | 15 | 7 | 1.5 | 5 | 12 | >10 | 6 | >10 | 5 | 7 |
| ADAMTS 13 (%) | 0.5 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0.4 | 0 |
| Anti-ADAMTS 13 antibodies | + | + | + | + | + | + | + | + | + | + | + | + | 100% |
| Plasma exchange (days) | 23 | 10 | 8 | 19 | 10 | 8 | 11 | NA | NA | 4 | 12 | 9 | 10 |
| Steroids: PDN, MTPDN (1–2 mg/kg/d) | MTPDN | PDN | PDN | MTPDN | PDN | PDN | PDN |
PDN MTPDN |
MTPDN | MTPDN | PDN |
MTPDN PDN |
|
| High-dose steroids MTPDN (1 g/d × 3) | Yes | No | No | No | No | No | No | Yes | No | No | No | No | 16.6% |
| Rituximab (375 mg/m2/week × 4) | Yes | Yes | No | Yes | Yes | No | Yes | Yes | Yes | No | No | Yes | 66.6% |
| Time to platelet normalization after starting rituximab (days) | 8 | 2 | - | 10 | 3 | - | 7 | 76 | 6 | - | - | 5 | 6.5 |
| Days of high-dose NAC (150 mg/kg/d) | 10 | 10 | 10 | 10 | 10 | 10 | 10 | 10 | 10 | 4 | 10 | 10 | 10 |
| Time to platelet normalization after starting NAC (days) | 3 | 9 | 3 | 10 | 4 | 6 | 9 | 9 | 5 | 5 | 1 | 5 | 5.5 |
| Other treatments | No | No | No | No | No | No | No | Borte-zomib | MMF | No | No | No | 16.6% |
| Time to platelet normalization (days) | 15 | 10 | 4 | 11 | 5 | 7 | 10 | 85 | 6 | 6 | 2 | 6 | 6,5 |
| Maintenance NAC | NA | Yes | Yes | NA | Yes | No | No | No | No | No | No | No | 25% |
| Exacerbation | Yes | No | No | No | No | No | Yes | Yes | Yes | No | Yes | Yes | 50% |
| Refractory | Yes | No | No | No | No | No | No | Yes | No | No | No | No | 16.6% |
| Relapse | NA | No | No | No | Yes | Yes | No | No | No | No | No | No | 16.6% |
| Rituximab-treated ADAMTS 13 relapse | NA | No | No | No | No | No | Yes | No | Yes | No | No | No | 16.6% |
| Follow-up (months) | NA | 50 | 50 | NA | 37 | 36 | 32 | 26 | 22 | 9 | 8 | 1 | 29 |
Platelet normalization was defined by a platelet count above 150 × 109/L. In the last column, results are represented as median for quantitative variables and as n (%) for qualitative variables
M male, F female, Neurol neurological, PDN prednisone, MTPDN methyprednisolone, NA not available, MMF micophenolate mofetil