Table 3 Severity factors for successful steroid-free maintenance therapy in patients with anti-HMGCR myopathy stratified by the use of steroid-sparing immunosuppressants in monotherapy or in combination, with or without IVIG (N = 41)
Successful maintenance with SSI monotherapy, n = 23 | Unsuccessful or unevaluable maintenance with SSI monotherapy | |||||
|---|---|---|---|---|---|---|
Overall, n = 18 | Remission with SSI monotherapy and IVIG, n = 5 | Remission with SSI combination therapy (± IVIG), n = 5 | Unsuccessful maintenance with SSI therapies, n = 6* | Maintenance with SSI therapy not evaluable, n = 2 | ||
Weakness at treatment onset, n (%) | 21 (91) | 16 (89) | 5 (100) | 5 (100) | 5 (83) | 1 (50) |
Age at treatment onset, median (range) years | 70.5 (50.1–83.6) | 67.5 (44.0–83.1) | 69.4 (56.7–78.4) | 66.2 (44.0–78.8) | 63.0 (46.5–74.8) | 67.5 (73.0–83.1) |
CK at treatment onset, median (range) UI/L | 5380 (696–23,000) | 8234 (1556–14,098) | 4750 (2770–14,098) | 8300 (1556–11,755) | 6737 (2267–13,339) | 6327 (2832–9821) |
Severity score, mean (SD) | 2.2 ± 1.1 | 2.7 ± 1.2 | 2.8 ± 0.8 | 3.4 ± 0.9 | 2.5 ± 1.4 | 1.5 ± 2.1 |
Severity score ≥ 3, n (%) | 8 (35) | 11 (61) | 3 (60) | 4 (80) | 3 (50) | 1 (50) |
Delay from first increased serum CK (< 500 UI/L) to treatment, median (range) months | 1.7 (0–24.9) | 12.7 (0–95.0) | 14 (0–95.0) | 13.4 (0.4–26.0) | 6.6 (0–42.2) | 0 and 79.0 |
IVIG at last follow-up | 0 | 11 (61) | 5 (100) | 2 (40) | 3 (50) | 1 (50) |
Corticosteroid dosage at last follow-up | ||||||
No corticosteroids | 22 (96) | 11 (61) | 4 (80) | 4 (80) | 3 (50) | 0 |
Prednisone ≤ 5 mg per day | 1 (4) | 4 (22) | 1 (20) | 1 (20) | 1 (17) | 1 (50) |
Prednisone > 5 mg per day | 0 | 3 (17) | 0 | 0 | 2 (33) | 1 (50) |
Drug-free remission, n (%) | 4 (17) | 0 | 0 | 0 | 0 | 0 |
Normal strength at last follow-up, n (%) | 20 (87) | 8 (44) | 2 (40) | 3 (60) | 2 (33) | 1 (50) |