. 2023 Feb 25;43(5):951–964. doi: 10.1007/s10875-022-01426-y

Table 1.

Patient demographics and clinical status

Patient	Sex	Age range, years^a	Race	Population	Clinical status^b		Previous therapy
Patient	Sex	Age range, years^a	Race	Population	Start	End	Previous therapy
1	M	< 10	White	As-treated	100	100	-
2	M	19 − 30	Other	As-treated/Completer	40	50	-
3	M	19 − 30	Other	As-treated/Completer	100	100	-
4	M	31 − 40	Black	As-treated/Completer	100	100	Exchange transfusion
5	F	19 − 30	White	As-treated/Completer	100	80	Gene therapy
6	F	10 − 18	White	As-treated/Completer	90	90	-
7	M	10 − 18	White	As-treated/Completer	70	70	-

^aAge ranges patients fall into were provided in lieu of an exact age to protect patient anonymity

^bClinical status at the start and end of the study was determined by measuring LPS (patients < 16 years old) or KPS (patients ≥ 16 years old) F, female; KPS, Karnofsky Performance Scale [23]; LPS, Lansky Performance Scale [22]; M, male