Table 1.
Cytopenia treatment outcomes in ALPS patients.
| Patients | MMF (n = 64) | Sirolimus (n = 14) | Rituximab (n = 26) | Hydroxychloroquine (n = 4) | TPO mimetics (Eltrombopag and Nplate) (n = 4) |
|---|---|---|---|---|---|
| ALPS-FAS | 45 | 12 | 20 | 4 | 3 |
| ALPS-U | 19 | 2 | 6 | 1 | |
| Gender ratio (male/female) | 49:15 | 10:4 | 20:6 | 2:2 | 3:1 |
| Median age at start of medication (range) | 10 years (0.5–34 years) | 2 years (1–37 years) | 13 years (1–47 years) | 17 years (12–30 years) | 19 years (9–20 years) |
| Median duration of follow-up on medication (range, years) | 4.5 years (0.5–l5 years) | (0.5–6 years) | NA | 3 years (0.5–7 years) | 2.25 (0.5–4 years) |
| Long-term responders | 44 | 10 | 16a | 2 | 2 |
| Non-responders (requiring other interventions) | 20 | 4 | 10 | 2 | 2 |
aRemission lasted at least 6 months after one course of rituximab 375 mg/M2 weekly × 4.