Table 3. Evaluation of HSCT outcome... | Rockefeller University Press

Table 3.

Evaluation of HSCT outcome using molecular diagnostic assays

				Pretransplant			∼1.5 years after transplant
	Mother	Father	Unaffected sibling	Sister	Brother	Ref. int.	Sister	Brother	Ref. int.
White blood cell counts
WBC	3.93	7.51	5.26	3.05^{^*}	2.54^{^*}	3.5–8.8	4.21	3.98	3.5–8.8
Neutrophils	2.22	3.73	2.87	1.57^{^*}	1^{^*}	1.8–7.5	2.91	1.7	1.6–5.9
Lymphocytes	1.26	2.73	1.7	0.924	1.02	0.8–4.5	0.74^{^*}	0.86^{^*}	1.1–3.5
Monocytes	0.307	0.664	0.494	0.307	0.455	0.1–1.0	0.25	0.28	0.2–0.8
Eosinophils	0.115	0.297	0.188	0.244	0.041	0.04–0.4	0.23	0.93	<0.5
Basophils	0.026	0.084	0	0.012	0.032	0–0.1	0.02	0.01	<0.1
CD3⁺ (T cells)				0.52^{^*}	0.11^{^*}	0.65–1.57	0.43^{^*}	0.38^{^*}	0.65–1.57
CD3⁺CD4⁺ (Th cells)				0.46^{^*}	0.07^{^*}	0.49–1.34	0.32^{^*}	0.33^{^*}	0.41–1.02
CD3⁺CD8⁺ (Tc cells)				0.06^{^*}	0.02^{^}	0.19–0.8	0.1^{^*}	0.03^{^*}	0.17–0.8
CD4⁺/CD8⁺ ratio				7.58^{^*}	3.31	1.13–3.93	3.23	9.83^{^*}	0.79–3.63
T cell proliferation (%)	90 ± 2	92 ± 10	91 ± 0	87 ± 2	20 ± 3^{^*}	78–94 (n = 300)	84 ± 1	84 ± 1	78–94 (n = 300)
IR sensitivity (CDA index %)
Dose 1 (low)^{^#}	67 ± 2	74 ± 1	72 ± 4	42 ± 10^{^*}	21 ± 4^{^*}	49–95 (n = 28)	61 ± 6	56 ± 7	50–97 (n = 40)
Dose 2 (high)^{^#}	13 ± 0	18 ± 2	24 ± 4	3 ± 0^{^*}	4 ± 0.3^{^*}	11–41 (n = 28)	12 ± 1^{^*}	11 ± 1^{^*}	15–47 (n = 40)
DSB repair (%)
NHEJ							19.8 ± 0.3	20.1 ± 0.3	7.4–36 (n = 12)
HR							1.9 ± 0.1	2.1 ± 0.1	0.4–9 (n = 11)
Chimerism (% patient)
CD3⁺							3.40%	3.60%
CD19⁺							3.20%	0.40%
CD33⁺							85.80%	80.80%

				Pretransplant			∼1.5 years after transplant
	Mother	Father	Unaffected sibling	Sister	Brother	Ref. int.	Sister	Brother	Ref. int.
White blood cell counts
WBC	3.93	7.51	5.26	3.05^{^*}	2.54^{^*}	3.5–8.8	4.21	3.98	3.5–8.8
Neutrophils	2.22	3.73	2.87	1.57^{^*}	1^{^*}	1.8–7.5	2.91	1.7	1.6–5.9
Lymphocytes	1.26	2.73	1.7	0.924	1.02	0.8–4.5	0.74^{^*}	0.86^{^*}	1.1–3.5
Monocytes	0.307	0.664	0.494	0.307	0.455	0.1–1.0	0.25	0.28	0.2–0.8
Eosinophils	0.115	0.297	0.188	0.244	0.041	0.04–0.4	0.23	0.93	<0.5
Basophils	0.026	0.084	0	0.012	0.032	0–0.1	0.02	0.01	<0.1
CD3⁺ (T cells)				0.52^{^*}	0.11^{^*}	0.65–1.57	0.43^{^*}	0.38^{^*}	0.65–1.57
CD3⁺CD4⁺ (Th cells)				0.46^{^*}	0.07^{^*}	0.49–1.34	0.32^{^*}	0.33^{^*}	0.41–1.02
CD3⁺CD8⁺ (Tc cells)				0.06^{^*}	0.02^{^}	0.19–0.8	0.1^{^*}	0.03^{^*}	0.17–0.8
CD4⁺/CD8⁺ ratio				7.58^{^*}	3.31	1.13–3.93	3.23	9.83^{^*}	0.79–3.63
T cell proliferation (%)	90 ± 2	92 ± 10	91 ± 0	87 ± 2	20 ± 3^{^*}	78–94 (n = 300)	84 ± 1	84 ± 1	78–94 (n = 300)
IR sensitivity (CDA index %)
Dose 1 (low)^{^#}	67 ± 2	74 ± 1	72 ± 4	42 ± 10^{^*}	21 ± 4^{^*}	49–95 (n = 28)	61 ± 6	56 ± 7	50–97 (n = 40)
Dose 2 (high)^{^#}	13 ± 0	18 ± 2	24 ± 4	3 ± 0^{^*}	4 ± 0.3^{^*}	11–41 (n = 28)	12 ± 1^{^*}	11 ± 1^{^*}	15–47 (n = 40)
DSB repair (%)
NHEJ							19.8 ± 0.3	20.1 ± 0.3	7.4–36 (n = 12)
HR							1.9 ± 0.1	2.1 ± 0.1	0.4–9 (n = 11)
Chimerism (% patient)
CD3⁺							3.40%	3.60%
CD19⁺							3.20%	0.40%
CD33⁺							85.80%	80.80%

Reference interval for the T cell proliferation, CDA, and DNA repair assay is based on the 2.5th and 97.5th percentile of controls, and “n” indicates the number of controls. WBC, white blood cells.

Indicates values outside the reference interval (also indicated in bold).

Estimated IR doses used were 1.0 Gy (dose 1) and 3.5 Gy (dose 2) for both parents, siblings, and patients prior to patient transplantation; for posttransplant samples, IR doses were 0.36 Gy (dose 1) and 1.95 Gy (dose 2).

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