WASP expression and response to therapy
. | WASP . | . | . | |
|---|---|---|---|---|
. | Positive . | Negative . | P . | |
| Stem cell transplantation, n | 3 | 12 | — | |
| Mean age, y | 12.4 | 12.8 | .017 | |
| Age range, y | 0.25-19 | 0.5-14 | — | |
| Alive, n | 3 | 11 | — | |
| Dead, n | 0 | 1 | — | |
| Splenectomy, n | 5 | 5 | — | |
| Mean age at present, y | 17.0 | 5.0 | .017 | |
| Range, y | 0-59 | 1.0-8.2 | — | |
| Mean age at splenectomy, y | 8.8 | 6.0 | — | |
| Range, y | 2-23 | 0.3-9 | — | |
| Alive, n | 5 | 2 | — | |
| Dead, n | 0 | 3† | — | |
| Good response, n (%)* | 2 (40) | 3 (60) | — | |
| Partial response, n (%)* | 3 (60) | 2 (40) | — | |
| No response, n | 0 | 0 | — | |
| Pneumococcal sepsis/meningitis after splenectomy, n | 2 | 3 | — | |
| High-dose IVIG, n | 13 | 11 | — | |
| Good response, n* | 0 | 0 | — | |
| Transient response, n (%) | 4 (31) | 2 (18) | — | |
| No response, n (%) | 9 (69) | 9 (82) | — | |
| Steroids, n | 15 | 11 | — | |
| Good response, n* | 0 | 0 | — | |
| Transient response, n (%) | 8 (53) | 2 (18) | — | |
| No response, n (%) | 7 (47) | 9 (82) | — | |
. | WASP . | . | . | |
|---|---|---|---|---|
. | Positive . | Negative . | P . | |
| Stem cell transplantation, n | 3 | 12 | — | |
| Mean age, y | 12.4 | 12.8 | .017 | |
| Age range, y | 0.25-19 | 0.5-14 | — | |
| Alive, n | 3 | 11 | — | |
| Dead, n | 0 | 1 | — | |
| Splenectomy, n | 5 | 5 | — | |
| Mean age at present, y | 17.0 | 5.0 | .017 | |
| Range, y | 0-59 | 1.0-8.2 | — | |
| Mean age at splenectomy, y | 8.8 | 6.0 | — | |
| Range, y | 2-23 | 0.3-9 | — | |
| Alive, n | 5 | 2 | — | |
| Dead, n | 0 | 3† | — | |
| Good response, n (%)* | 2 (40) | 3 (60) | — | |
| Partial response, n (%)* | 3 (60) | 2 (40) | — | |
| No response, n | 0 | 0 | — | |
| Pneumococcal sepsis/meningitis after splenectomy, n | 2 | 3 | — | |
| High-dose IVIG, n | 13 | 11 | — | |
| Good response, n* | 0 | 0 | — | |
| Transient response, n (%) | 4 (31) | 2 (18) | — | |
| No response, n (%) | 9 (69) | 9 (82) | — | |
| Steroids, n | 15 | 11 | — | |
| Good response, n* | 0 | 0 | — | |
| Transient response, n (%) | 8 (53) | 2 (18) | — | |
| No response, n (%) | 7 (47) | 9 (82) | — | |
WASP-positive numbers represent WASP detectable by Western blot. WASP-negative numbers represent WASP found absent or truncated by Western blot. — indicates not applicable.
Good response indicates a platelet count greater than 100 × 109/L; partial response, a platelet count less than 100 × 109/L.
Two patients died of ICH, and 1 died of complications related to BMT.