Table 1.
Baseline characteristics.
| Characteristics | Total (N = 44) | AIHA (N = 37) | ES (N = 7) | P value |
|---|---|---|---|---|
| Male sex, No. (%) | 9 (20.5) | 9 (24.3) | 0 (0) | 0.148 |
| Age, y, median (range) | 44 (18–86) | 45 (18–86) | 43 (30–72) | 0.441 |
| Time from diagnosis to sirolimus treatment, m, median (range) | 15 (1–212) | 15 (1–212) | 58 (8–83) | 0.150 |
| Diagnosis, No. (%) | ||||
| wAIHA | 37 (84.1) | – | – | – |
| ES | 7 (15.9) | – | – | – |
| Type of antibody, No. (%) | 0.480 | |||
| Warm (IgG) | 33 (75.0) | 27 (73.0) | 6 (85.7) | |
| Negative | 11 (25.0) | 10 (27.0) | 1 (14.3) | |
| Disease status, No. (%) | 0.104 | |||
| Refractory to glucocorticoid | 25 (56.8) | 23 (62.2) | 2 (28.6) | |
| Relapsed to glucocorticoid | 19 (43.2) | 14 (37.8) | 5 (71.4) | |
| Previous treatment, No. (%) | ||||
| Glucocorticoid | 44 (100) | 37 (100) | 7 (100) | 1.000 |
| Rituximab | 6 (13.6) | 5 (13.5) | 1 (14.3) | 0.975 |
| CsA | 13 (29.5) | 10 (27.0) | 3 (42.9) | 0.405 |
| CTX | 10 (22.7) | 7 (18.9) | 3 (42.9) | 0.171 |
| IVIG | 5 (11.4) | 4 (10.8) | 1 (14.3) | 0.793 |
| Azathioprine | 5 (11.4) | 4 (10.8) | 1 (14.3) | 0.793 |
| Two or more lines of previous therapies | 26 (59.1) | 20 (54.1) | 6 (85.7) | 0.122 |
| Haemoglobin, g/L, median (range) | 85 (30–107) | 85 (30–106) | 83 (44–107) | 0.759 |
| Platelet, ×109/L, median (range) | 189 (17–414) | 211 (102–414) | 57 (17–69) | 3.55 × 10-5 |
| Absolute neutrophil count, ×109/L, median (range) | 2.95 (0.90–12.61) | 2.95 (0.90–8.73) | 3.40 (1.21–12.61) | 0.360 |
| Absolute reticulocyte count, ×109/L, median (range) | 195.2 (75.0–875.2) | 202.0 (75.0–875.0) | 188.4 (111.4–371.6) | 0.656 |
| Lactate dehydrogenase, U/L, median (range) | 256 (114–491) | 259 (138–405) | 224 (114–491) | 0.890 |
| Serum total bilirubin, μmol/L, median (range) | 30.1 (8.5–286.6) | 34.5 (8.5–137.3) | 18.7 (12.5–286.6) | 0.285 |
| Serum indirect bilirubin, μmol/L, median (range) | 20.3 (5.7–251.3) | 24.3 (5.7–128.1) | 13.4 (9.0–251.3) | 0.292 |
| Serum ferritin, ng/mL, median (range) | 364 (43–1661) | 419 (43–1661) | 171 (47–600) | 0.188 |
| With glucocorticoid tapering, No. (%) | 13 (29.5) | 13 (35.1) | 0 | 0.065 |
| Duration of glucocorticoid tapering, wks, median (range) | 8 (2–19) | 8 (2–19) | – | – |
| Sirolimus plasma trough concentration, ng/mL, median (range)* | 4.5 (2.0–8.1) | 4.5 (2.0–8.1) | 4.3 (3.5–8.1) | 0.742 |
| Duration of sirolimus treatment, m, median (range) | 23 (6–80) | 23 (6–80) | 23 (6–38) | 0.712 |
| Duration of follow-up, m, median (range) | 25 (12–80) | 26 (12–80) | 23 (12–38) | 0.688 |
wAIHA: warm autoimmune haemolytic anaemia; ES: Evans syndrome; IgG: immunoglobulin G; CsA: cyclosporin A; CTX: cyclophosphamide; IVIG: intravenous immunoglobulin.
*
Based on the average sirolimus plasma trough concentration of the first 6 months after sirolimus of each patient.