Table 1. Demographics and baseline... | American Society of Hematology

Table 1.

Demographics and baseline clinical characteristics of participants in the primary efficacy set

	Primary efficacy set N = 42
Sex, n (%)
Male	24 (57.1)
Female	18 (42.9)
Genotype, n (%)
β^S/β^S	39 (92.9)
β^S/β⁰	2 (4.8)
β^S/β⁺	1 (2.4)
Age at screening, mean (SD), y	20.9 (5.9)
≥12 to <18 years, n (%)	12 (28.6)
≥18 to ≤35 years, n (%)	30 (71.4)
Historical VOC episodes/year,∗ mean (SD)	4.2 (3.1)
Historical in-patient hospitalizations for severe VOCs/year,∗ mean (SD)	2.8 (2.1)
Duration of follow-up after exa-cel infusion,† median (range), months	33.6 (17.2-62.2)

	Primary efficacy set N = 42
Sex, n (%)
Male	24 (57.1)
Female	18 (42.9)
Genotype, n (%)
β^S/β^S	39 (92.9)
β^S/β⁰	2 (4.8)
β^S/β⁺	1 (2.4)
Age at screening, mean (SD), y	20.9 (5.9)
≥12 to <18 years, n (%)	12 (28.6)
≥18 to ≤35 years, n (%)	30 (71.4)
Historical VOC episodes/year,∗ mean (SD)	4.2 (3.1)
Historical in-patient hospitalizations for severe VOCs/year,∗ mean (SD)	2.8 (2.1)
Duration of follow-up after exa-cel infusion,† median (range), months	33.6 (17.2-62.2)

n: number of participants.

Annualized rate = total number of events/number of years.

Post–exa-cel infusion follow-up duration (months) = (data cutoff date – exa-cel infusion date + 1)/30.