Results of the modeling study fitting Poisson models (SHPM and C≥1PMs) to the 8 LDTAs with HSC frequency estimates, their SEs, and 95% confidence intervals
| LDTA no. . | Cell subset . | SHPM (C = 1) . | C≥1PMs . | f̃ . | |||||
|---|---|---|---|---|---|---|---|---|---|
| fc1* . | SE (fc1)† . | 95% CI (fc1) . | C‡ . | f̃§ . | SE (f̃)‖ . | 95% CI (f̃) . | fc1 . | ||
| 1a | Hoxa-9−/− 12 weeks | 6.11 × 10−6 (1/163 934) | 1.65 × 10−6 | 2.88 × 10−6 - 9.34 × 10−6 (1/347 222 - 1/107 066) | 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 | 3.59 × 10−5 (1/27 816) | 1.53 × 10−5 | 5.98 × 10−6 - 6.59 × 10−5 (1/167 179 - 1/15170) | 5.88 |
| 1b | Wild-type 12 weeks | 4.37 × 10−5 (1/22 883) | 1.11 × 10−5 | 2.2 × 10−5 - 6.55 × 10−5 (1/45 454 - 1/15 167) | 1, 2, 3, 4, 5, 6, 7, 8, 9 | 1.85 × 10−4 (1/5416) | 7.62 × 10−5 | 3.51 × 10−5 - 3.34 × 10−4 (1/28 449 - 1/2993) | 4.23 |
| 2a | Delta1-IgG 3 weeks | 1.25 × 10−4 (1/8000) | 1.94 × 10−5 | 8.73 × 10−5 - 1.63 × 10−4 (1/11 454 - 1/6135) | 1, 2, 3, 4 | 3.32 × 10−4 (1/3014) | 9.05 × 10−5 | 1.54 × 10−4 - 5.09 × 10−4 (1/6 477 - 1/1964) | 2.66 |
| 2b | Control-IgG 3 weeks | 3.79 × 10−5 (1/26 385) | 7.22 × 10−6 | 2.38 × 10−5 - 5.21 × 10−5 (1/42 016 - 1/19 193) | 1, 2, 3 | 7.25 × 10−5 (1/13 791) | 3.2 × 10−5 | 1.175 × 10−5 - 1.33 × 10−4 (1/85 106 - 1/7503) | 1.91 |
| 2c | Noncultured 3 weeks | 8.18 × 10−6 (1/122 249) | 1.36 × 10−6 | 5.52 × 10−6 - 1.08 × 10−5 (1/181 159 - 1/92 592) | 1, 2 | 1.08 × 10−5 (1/92 592) | 4.4 × 10−6 | 2.17 × 10−6 - 1.94 × 10−5 (1/459 559 - 1/51482) | 1.32 |
| 2d | Delta1-IgG 9 weeks | 9.91 × 10−5 (1/10 090) | 1.54 × 10−5 | 6.88 × 10−5 - 1.29 × 10−4 (1/14 534 - 1/7751) | 1, 2, 3 | 1.69 × 10−4 (1/5904) | 7.62 × 10−5 | 1.99 × 10−5 - 3.18 × 10−4 (1/50 176 - 1/3136) | 1.7 |
| 2e | Control-IgG 9 weeks | 5.62 × 10−5 (1/17 793) | 1.04 × 10−5 | 3.59 × 10−5 - 7.65 × 10−5 (1/27 855 - 1/13 071) | 1, 2, 3 | 1.29 × 10−4 (1/7692) | 3.075 × 10−5 | 6.97 × 10−5 - 1.9 × 10−4 (1/14 343 - 1/5255) | 2.29 |
| 2f | Noncultured 9 weeks | 1.58 × 10−5 (1/63 291) | 2.46 × 10−6 | 2.06 × 10−5 - 1.1 × 10−4 (1/48 543 - 1/9090) | 1 | NA | NA | NA | |
| LDTA no. . | Cell subset . | SHPM (C = 1) . | C≥1PMs . | f̃ . | |||||
|---|---|---|---|---|---|---|---|---|---|
| fc1* . | SE (fc1)† . | 95% CI (fc1) . | C‡ . | f̃§ . | SE (f̃)‖ . | 95% CI (f̃) . | fc1 . | ||
| 1a | Hoxa-9−/− 12 weeks | 6.11 × 10−6 (1/163 934) | 1.65 × 10−6 | 2.88 × 10−6 - 9.34 × 10−6 (1/347 222 - 1/107 066) | 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11 | 3.59 × 10−5 (1/27 816) | 1.53 × 10−5 | 5.98 × 10−6 - 6.59 × 10−5 (1/167 179 - 1/15170) | 5.88 |
| 1b | Wild-type 12 weeks | 4.37 × 10−5 (1/22 883) | 1.11 × 10−5 | 2.2 × 10−5 - 6.55 × 10−5 (1/45 454 - 1/15 167) | 1, 2, 3, 4, 5, 6, 7, 8, 9 | 1.85 × 10−4 (1/5416) | 7.62 × 10−5 | 3.51 × 10−5 - 3.34 × 10−4 (1/28 449 - 1/2993) | 4.23 |
| 2a | Delta1-IgG 3 weeks | 1.25 × 10−4 (1/8000) | 1.94 × 10−5 | 8.73 × 10−5 - 1.63 × 10−4 (1/11 454 - 1/6135) | 1, 2, 3, 4 | 3.32 × 10−4 (1/3014) | 9.05 × 10−5 | 1.54 × 10−4 - 5.09 × 10−4 (1/6 477 - 1/1964) | 2.66 |
| 2b | Control-IgG 3 weeks | 3.79 × 10−5 (1/26 385) | 7.22 × 10−6 | 2.38 × 10−5 - 5.21 × 10−5 (1/42 016 - 1/19 193) | 1, 2, 3 | 7.25 × 10−5 (1/13 791) | 3.2 × 10−5 | 1.175 × 10−5 - 1.33 × 10−4 (1/85 106 - 1/7503) | 1.91 |
| 2c | Noncultured 3 weeks | 8.18 × 10−6 (1/122 249) | 1.36 × 10−6 | 5.52 × 10−6 - 1.08 × 10−5 (1/181 159 - 1/92 592) | 1, 2 | 1.08 × 10−5 (1/92 592) | 4.4 × 10−6 | 2.17 × 10−6 - 1.94 × 10−5 (1/459 559 - 1/51482) | 1.32 |
| 2d | Delta1-IgG 9 weeks | 9.91 × 10−5 (1/10 090) | 1.54 × 10−5 | 6.88 × 10−5 - 1.29 × 10−4 (1/14 534 - 1/7751) | 1, 2, 3 | 1.69 × 10−4 (1/5904) | 7.62 × 10−5 | 1.99 × 10−5 - 3.18 × 10−4 (1/50 176 - 1/3136) | 1.7 |
| 2e | Control-IgG 9 weeks | 5.62 × 10−5 (1/17 793) | 1.04 × 10−5 | 3.59 × 10−5 - 7.65 × 10−5 (1/27 855 - 1/13 071) | 1, 2, 3 | 1.29 × 10−4 (1/7692) | 3.075 × 10−5 | 6.97 × 10−5 - 1.9 × 10−4 (1/14 343 - 1/5255) | 2.29 |
| 2f | Noncultured 9 weeks | 1.58 × 10−5 (1/63 291) | 2.46 × 10−6 | 2.06 × 10−5 - 1.1 × 10−4 (1/48 543 - 1/9090) | 1 | NA | NA | NA | |
NA indicates not available (the SHPM is clearly the best expected model).
Maximum likelihood estimate of the HSC frequency obtained on fitting the SHPM to the data.
SE(fc1) indicates conditional standard error of fc1; based on standard normal distribution of fc1 95% CI (fc1) is given by the endpoints fc1 ± 1.96 × SE (fc1).
Value(s) of C corresponding to the set P of plausible C≥1PMs, C being the minimum number of HSCs necessary to promote detectable repopulation in recipients (positive outcome).
f̃ indicates the C≥1PM model–averaged HSC frequency estimate in the set P of plausible C≥1PMs.
SE (f̃) indicates the unconditional standard error of f̃; 95% CI (f̃) is given by the endpoints f̃ ±1.96× SE (f̃); see supplemental data.