Table 4.
Patient Number | Months since Infusion | No. of Transfusions (Annualized) | No. of Severe Sickle Cell Clinical Events† | |||
---|---|---|---|---|---|---|
Prestudy | After Engraftment | Prestudy | After Gene Therapy (<5 mo) | After Gene Therapy (≥5 mo) | ||
2 | 29 | 12.5 | 0 | 0 | 0 | 0 |
3 | 19 | 10.5 | 5.7 | 0 | 0 | 0 |
4 | 20 | 2 | 0 | 13 | 5 | 1‡ |
6 | 16 | 3 | 0 | 6 | 0 | 0 |
7 | 12 | 11 | 0 | 0 | 0 | 0 |
8 | 7 | 1 | 0 | 3 | 0 | 0 |
Prestudy events represent the 2 years preceding study enrollment. Events that occurred after gene therapy represent the time from engraftment to the most recent follow-up.
Events include vaso-occlusive crisis pain requiring an emergency department visit or admission for opioid treatment, admission for acute chest syndrome, and priapism events requiring an emergency department visit or admission for a procedural intervention.
The most recent severe event was an emergency department visit that occurred 8 months after gene therapy.