Table 2.
Non-ALPS patient baseline characteristics, prior treatment outcomes, and sirolimus response.
| Response | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| 3 mo | 6 mo | 12 mo | ||||||||
| Disorder | Lineage cytopenia | Lymphoproliferative disease | Prior therapy | Heme | Lymphoproliferation | Heme | Lymphoproliferation | Heme | Lymphoproliferation | Time on sirolimus |
| ES | A+N+T | None | Corticosteroids (PR) | PR | N/A | NR | NR | 15 wk | ||
| IVIgG (NR) | ||||||||||
| MMF (PR) | ||||||||||
| Rituximab (NR) | ||||||||||
| ES | A+N+T | Marked LAD and splenomegaly | Corticosteroids (PR) | PR | PR | Off study | Off study | 5 mo | ||
| IVIgG (NR) | ||||||||||
| MMF (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
| ES | A+N+T | Mild splenomegaly | Corticosteroids (PR) | CR | CR | CR | CR | CR | CR | 2 y |
| IVIgG (NR) | ||||||||||
| MMF (NR) | ||||||||||
| Rituximab (allergy) | ||||||||||
| ES | A+N+T | Mild splenomegaly | Corticosteroids (NR) | CR | CR | CR | CR | CR | CR | 2 y |
| IVIgG (NR) | ||||||||||
| MMF (PR) | ||||||||||
| ES | N+T | None | Corticosteroids (RT) | NR | Off study | Off study | N/A | |||
| ES | A+N+T | Mild LAD and splenomegaly | Corticosteroids (RT) | NR | Off study | Off study | N/A | |||
| G-CSF (CR) | ||||||||||
| ES | N+T | None | Corticosteroids (RT) | CR | N/A | CR | CR | 1 y | ||
| IVIgG (PR) | ||||||||||
| ES | N+T | Mild splenomegaly, marked cervical LAD | Corticosteroids (RT) | PR | CR | PR | CR | CR | CR | 1 y |
| IVIgG (NR) | ||||||||||
| CVID | A+N+T | None | Corticosteroid (RT) | CR | N/A | CR | CR | 4.5 y | ||
| IVIgG (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
| CVID | N+T | None | Corticosteroids (RT) | CR | N/A | CR | CR | 3.5 y | ||
| IVIgG (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
| SLE | N+T | None | Corticosteroids (NR) | CR | N/A | CR | CR | 3 y | ||
| IVIgG (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
| SLE | A+N+T | None | Corticosteroids (RT) | PR | N/A | CR | CR | 2.5 y | ||
| IVIgG (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
| ITP | T | None | Corticosteroids (NR) | NR | Off study | Off study | 1 mo | |||
| IVIgG (NR) | ||||||||||
| Splenectomy | ||||||||||
| ITP | T | None | Corticosteroids (NR) | PR | N/A | PR | N/A | Off study | 6 mo | |
| IVIgG (NR) | ||||||||||
| MMF (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
| 6-MP (NR) | ||||||||||
| Hydroxycholoroquine (NR) | ||||||||||
| ITP | T | None | Corticosteroids (NR) | NR | Off study | Off study | 2 mo | |||
| IVIgG (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
| Rh(D) IVIG (NR) | ||||||||||
| Romiplostim (NR) | ||||||||||
| ITP | T | None | Corticosteroids (NR) | NR | Off study | Off study | 3 mo | |||
| IVIgG (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
| Rh(D) IVIgG (NR) | ||||||||||
| Romiplostim (NR) | ||||||||||
| AIHA | A | None | Corticosteroids (RT) | PR | N/A | PR | N/A | PR | N/A | Intermittent 4 y |
| IVIgG (NR) | ||||||||||
| MMF (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
| 6-MP (NR) | ||||||||||
| Splenectomy | ||||||||||
| AIHA | A | Marked LAD and splenomegaly | Corticosteroids (RT) | PR | CR | CR | CR | CR | CR | 1 y |
| IVIgG (NR) | ||||||||||
| MMF (NR) | ||||||||||
| Rituximab (NR) | ||||||||||
N/A, not applicable; other abbreviations are explained in Table 1.