Table 3. Deficient in vivo multilineage... | American Society of Hematology

Table 3.

Deficient in vivo multilineage reconstitution ability of MDS BM CD34⁺ cells in NOD-SCID mice

Cells	Number of cells transplanted	Mice^3-150	Human reconstitution
Cells	Number of cells transplanted	Mice^3-150	% human engraftment	Multilineage engraftment, positive mice
Patient no. 1
CD34⁺	200 000	N/S	0	0/2
CD34⁺CD38⁺	2 500 000	N/S	0	0/1
CD34⁺CD38⁻	17 500	N/S	0.23^3-151	0/3
Patient no. 9
CD34⁺CD38⁺	200 000	β₂N/S	0	0/2
CD34⁺CD38⁻	20 000	β₂N/S	0	0/5
Patient no. 10
CD34⁺	1 000 000	β₂N/S	0	0/2
CD34⁺	2 500 000	N/S	0	0/2
Total MDS, patients 1, 9, 10	—		—	0/17
Normal BM populations
First: CD34⁺	500 000	N/S	5.0	1/4
Second:
CD34⁺	168 000	N/S	2.8-0.8	2/5
CD34⁺CD38⁻	10 000	N/S	10.6	1/6
Third: CD34⁺CD38⁻	14 000	β₂N/S	0.2-0.5	2/2
Total, normal BM	—		—	6/17

Cells	Number of cells transplanted	Mice^3-150	Human reconstitution
Cells	Number of cells transplanted	Mice^3-150	% human engraftment	Multilineage engraftment, positive mice
Patient no. 1
CD34⁺	200 000	N/S	0	0/2
CD34⁺CD38⁺	2 500 000	N/S	0	0/1
CD34⁺CD38⁻	17 500	N/S	0.23^3-151	0/3
Patient no. 9
CD34⁺CD38⁺	200 000	β₂N/S	0	0/2
CD34⁺CD38⁻	20 000	β₂N/S	0	0/5
Patient no. 10
CD34⁺	1 000 000	β₂N/S	0	0/2
CD34⁺	2 500 000	N/S	0	0/2
Total MDS, patients 1, 9, 10	—		—	0/17
Normal BM populations
First: CD34⁺	500 000	N/S	5.0	1/4
Second:
CD34⁺	168 000	N/S	2.8-0.8	2/5
CD34⁺CD38⁻	10 000	N/S	10.6	1/6
Third: CD34⁺CD38⁻	14 000	β₂N/S	0.2-0.5	2/2
Total, normal BM	—		—	6/17

Irradiated nonobese diabetic–severe combined immunodeficiency (NOD-SCID) mice received transplants of the indicated bone marrow (BM) cell types and numbers from patients with myelodysplastic syndrome (MDS) and healthy controls, and were investigated for human reconstitution 6 to 8 weeks later (“Patients, materials, and methods”). Data are presented as the number of mice positive for human reconstitution with both B cells and myeloid cells. Also shown is the percent of human engraftment (representing total human reconstitution independent of whether both myeloid and lymphoid reconstitution were observed). Note that no multilineage engraftment was seen in any mouse that received a transplant with CD34 subpopulations from patients with MDS, in spite of having received a transplant with as high or a higher number of cells than was used in parallel for normal BM CD34⁺ populations (positive control).

F3-150

N/S indicates NOD-SCID mice and β₂N/S indicates β₂-microglobulin–deficient NOD-SCID mice.

F3-151

No myeloid reconstitution, only CD45⁺CD19⁺ cells with disomy 8, most likely representing normal B cells.

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