Table 1 Summary data of bone marrow harvests for GT

From: Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy

Disease

Age at GT (years)

Weight (kg)

Harvested volume (ml)

Volume/kg

Collected TNC (×108)/kg

CD34+ (%)

Collected CD34+ cells (×106)/kg

Selected CD34+ cells (×106)/kg

Infused CD34+ cells (×106)/kg

ADA-SCID (n = 23)

1.7 (0.5–6.0)

10.1 (5.9–26.5)

303.0 (190.0–1167.5)

31.2 (14.2–48.6)

4.2 (2.9–6.8)

5.7 (2.0–6.7)a

28.9 (9.8–43.1)a

13.8 (4.4–22.9)

10.7 (3.7–19.7)

WAS (n = 6)

2.1 (0.9–6.0)

12.7 (8.0–26.3)

424.3 (275.0–773.0)

34.2 (29.4–36.5)

5.7 (3.5–6.6)

2.9 (1.8–5.2)

15.8 (10.8–18.7)

9.1 (4.5–14.5)

9.0 (3.7–14.1)

MLD (n = 28)

1.3 (0.5–11.4)

11 (7.3–25.3)

420.0 (194.0–1132.0)

36.9 (17.6–56.6)

6.3 (3.4–9.4)

3.0 (1.0–7.7)

16.3 (5.6–65.9)

9.6 (3.9–34.7)

10.3 (3.8–25.9)

Total (n = 57)

1.5 (0.5–11.4)

10.6 (5.9–26.5)

348.0 (190.0–1167.5)

34.2 (14.2–56.6)

5.1 (2.9–9.4)

3.1 (1.0–7.7)

17.3 (5.6–65.9)

10.4 (3.9–34.7)

10.3 (3.7–25.9)

  1. Data indicate median and range
  2. aData available for the last consecutive eight ADA-SCID patients