Table 1. Summary of engraftment results in NSGS mice.
| Donor | Diagnosis | Injected Fraction | Injected cell number | Calculated LSC number | Engraftment (% CD45+/CD33+ cells) |
|---|---|---|---|---|---|
| #10 | CMML-1 | Bulk (n=5) | 3.0 x 106 | > 2.8 | 94.9 ± 0.8 % |
| CD34+ (n=3) | 0.3 x 106 | > 30.6 | 63.8 ± 3.5 % | ||
| CD34– (n=3) | 1.0 x 106 | n.a. | 0.4 ± 0.1 % | ||
| # 18 | CMML-2 | Bulk (n=5) | 3.0 x 106 | > 7.0 | 62.4 ± 19.2 % |
| CD34+ (n=3) | 0.3 x 106 | > 20.4 | 25.8 ± 24.4 % | ||
| CD34– (n=3) | 1.0 x 106 | n.a. | 1.3 ± 0.2 % | ||
| #21 | sAML | Bulk (n=5) | 1.2 x 106 | > 26.1 | 75.2 ± 37.0 % |
| CD34+ (n=5) | 0.4 x 106 | > 16.3 | 19.5 ± 41.8 % | ||
| CD34– (n=5) | 0.4 x 106 | n.a. | 0.4 ± 0.2 % | ||
| #23 | sAML | Bulk (n=5) | 7.0 x 106 | > 261.3 | 94.4 ± 3.0 % |
| CD34+ (n=3) | 1.6 x 106 | > 163.2 | 84.3 ± 1.9 % | ||
| CD34– (n=3) | 1.5 x 106 | n.a. | 0.6 ± 0.2 % |
CD3-depleted bulk BM cells or FACS-purified CD34+ or CD34– cell fractions obtained from patients with CMML or sAML were injected i.v. into NSGS mice. For detailed patient characteristics, see Supplementary Table S2. After 6 weeks (donor 10), 12 weeks (donors 18 and 23) or 25 weeks (donor 21), mice were sacrificed and engraftment evaluated by flow cytometry. The table shows a summary of these experiments including number of mice injected, number of cells injected, calculated number of LSC injected (based on LSC frequency calculations shown in Supplementary Table S3) and engraftment levels (expressed as mean±S.D. of human CD45+/CD33+ cells in the murine BM). Abbreviations: CMML, chronic myelomonocytic leukemia; sAML, post-CMML secondary acute myeloid leukemia; LSC, leukemic stem cell; n.a., not applicable.