Baseline characteristics, prior treatment outcomes, and sirolimus response in ALPS patients.
| . | . | . | . | Response . | . | |||||
|---|---|---|---|---|---|---|---|---|---|---|
| 3 mo . | 6 mo . | 12 mo . | . | |||||||
| Disorder . | Lineage cytopenia . | Lymphoproliferative disease . | Prior therapy . | Heme . | Lymphoproliferation . | Heme . | Lymphoproliferation . | Heme . | Lymphoproliferation . | Time on sirolimus . |
| ALPS-FAS | A+N+T | Massive LAD splenomegaly | Corticosteroid (RT) | CR | CR | CR | CR | CR | CR | 8 y |
| IVIgG (PR) | ||||||||||
| MMF (NR) | ||||||||||
| Rituximab (RT) | ||||||||||
| ALPS-FAS | T+N | Massive LAD splenomegaly | Corticosteroid (RT) | CR | CR* | CR | CR* | CR | CR* | 6 y |
| MMF (NR) | ||||||||||
| ALPS-FAS | A+N+T | Massive LAD splenomegaly | Corticosteroid (RT) | CR* | CR* | CR* | CR* | CR* | CR* | 4.5 y |
| IVIgG (NR) | ||||||||||
| MMF (PR) | ||||||||||
| Rituximab (VTR) | ||||||||||
| ALPS-FAS | A+N+T | Massive LAD splenomegaly | Corticosteroid (RT) | CR | CR | CR | CR | CR | CR | 3 y |
| IVIgG (NR) | ||||||||||
| MMF (PR) | ||||||||||
| Rituximab (PR) | ||||||||||
| ALPS-FAS | A+N+T | Marked LAD splenomegaly | Corticosteroid (NR) | CR* | CR* | CR* | CR* | CR* | CR* | 2 y |
| IVIgG (NR) | ||||||||||
| G-CSF (NR) | ||||||||||
| ALPS-FAS | A+T | Marked splenomegaly | Corticosteroid (RT) | PR | CR | PR | CR | CR | CR | 1.5 y |
| IVIgG (RT) | ||||||||||
| ALPS-sFAS | A+N+T | Massive LAD and splenomegaly | Corticosteroids (RT) | CR | CR | CR | CR | CR | CR | 4.5 y |
| MMF (PR) | ||||||||||
| VCR (NR) | ||||||||||
| Pyrimethamine (NR) | ||||||||||
| ALPS-sFAS | A+N+T | Mild LAD and splenomegaly | Corticosteroid (NR) | CR | CR | CR | CR | CR | CR | 4 y |
| IVIgG (NR) | ||||||||||
| MMF (NR) | ||||||||||
| G-CSF (NR) | ||||||||||
| ALPS-sFAS | A+N+T | Massive LAD and splenomegaly | Corticosteroid (RT) | CR | CR* | CR | CR | CR | CR | 4 y |
| Depakote (PR) | ||||||||||
| ALPS-U | A+N+T | Massive LAD and splenomegaly | Corticosteroid (RT) | CR | CR | CR | CR | CR | CR | 3 y |
| IVIgG (NR) | ||||||||||
| MMF (PR) | ||||||||||
| 6-MP (NR) | ||||||||||
| Methotrexate (NR) | ||||||||||
| ALPS-U | A+N+T | Massive LAD and splenomegaly | Corticosteroid (NR) | CR | CR | CR | CR | CR | CR | 3 y |
| IVIgG (NR) | ||||||||||
| ALPS-U | A+N+T | Lymphadenopathy | Corticosteroid (RT) | CR | CR | CR | CR | CR | CR | 3 y |
| IVIgG (PR) | ||||||||||
| . | . | . | . | Response . | . | |||||
|---|---|---|---|---|---|---|---|---|---|---|
| 3 mo . | 6 mo . | 12 mo . | . | |||||||
| Disorder . | Lineage cytopenia . | Lymphoproliferative disease . | Prior therapy . | Heme . | Lymphoproliferation . | Heme . | Lymphoproliferation . | Heme . | Lymphoproliferation . | Time on sirolimus . |
| ALPS-FAS | A+N+T | Massive LAD splenomegaly | Corticosteroid (RT) | CR | CR | CR | CR | CR | CR | 8 y |
| IVIgG (PR) | ||||||||||
| MMF (NR) | ||||||||||
| Rituximab (RT) | ||||||||||
| ALPS-FAS | T+N | Massive LAD splenomegaly | Corticosteroid (RT) | CR | CR* | CR | CR* | CR | CR* | 6 y |
| MMF (NR) | ||||||||||
| ALPS-FAS | A+N+T | Massive LAD splenomegaly | Corticosteroid (RT) | CR* | CR* | CR* | CR* | CR* | CR* | 4.5 y |
| IVIgG (NR) | ||||||||||
| MMF (PR) | ||||||||||
| Rituximab (VTR) | ||||||||||
| ALPS-FAS | A+N+T | Massive LAD splenomegaly | Corticosteroid (RT) | CR | CR | CR | CR | CR | CR | 3 y |
| IVIgG (NR) | ||||||||||
| MMF (PR) | ||||||||||
| Rituximab (PR) | ||||||||||
| ALPS-FAS | A+N+T | Marked LAD splenomegaly | Corticosteroid (NR) | CR* | CR* | CR* | CR* | CR* | CR* | 2 y |
| IVIgG (NR) | ||||||||||
| G-CSF (NR) | ||||||||||
| ALPS-FAS | A+T | Marked splenomegaly | Corticosteroid (RT) | PR | CR | PR | CR | CR | CR | 1.5 y |
| IVIgG (RT) | ||||||||||
| ALPS-sFAS | A+N+T | Massive LAD and splenomegaly | Corticosteroids (RT) | CR | CR | CR | CR | CR | CR | 4.5 y |
| MMF (PR) | ||||||||||
| VCR (NR) | ||||||||||
| Pyrimethamine (NR) | ||||||||||
| ALPS-sFAS | A+N+T | Mild LAD and splenomegaly | Corticosteroid (NR) | CR | CR | CR | CR | CR | CR | 4 y |
| IVIgG (NR) | ||||||||||
| MMF (NR) | ||||||||||
| G-CSF (NR) | ||||||||||
| ALPS-sFAS | A+N+T | Massive LAD and splenomegaly | Corticosteroid (RT) | CR | CR* | CR | CR | CR | CR | 4 y |
| Depakote (PR) | ||||||||||
| ALPS-U | A+N+T | Massive LAD and splenomegaly | Corticosteroid (RT) | CR | CR | CR | CR | CR | CR | 3 y |
| IVIgG (NR) | ||||||||||
| MMF (PR) | ||||||||||
| 6-MP (NR) | ||||||||||
| Methotrexate (NR) | ||||||||||
| ALPS-U | A+N+T | Massive LAD and splenomegaly | Corticosteroid (NR) | CR | CR | CR | CR | CR | CR | 3 y |
| IVIgG (NR) | ||||||||||
| ALPS-U | A+N+T | Lymphadenopathy | Corticosteroid (RT) | CR | CR | CR | CR | CR | CR | 3 y |
| IVIgG (PR) | ||||||||||
A, anemia; G-CSF, granulocyte colony-stimulating factor; LAD, lymphadenopathy; N, neutropenia; 6-MP, mercaptopurine; T, thrombocytopenia; VCR, vincristine; VTR, very transient response. Prior therapy responses are listed in parentheses: CR, responds but toxicity (RT), PR, PR but relapse (PR-R), and no response (NR).
Designate the patients who remain in a durable CR but have occasional, mild, transient disease flares, manifested as dropping blood counts or the development of lymphadenopathy with viral illness. These “flares” are mild and do not require the use of additional immune suppression, as they self-resolve following the resolution of the infection