Patient characteristics
| Variable . | Number of patients . | ||
|---|---|---|---|
| Group A . | Group B . | All . | |
| Male/female, n | 9/5 | 11/5 | 20/10 |
| Median age (range), y | 10 (5-21) | 9 (1-20) | 9 (1-21) |
| Donor, n | |||
| MSD/MFD | 5 | 4 | 9 |
| MUD | 6 | 9 | 15 |
| MMD/haplo | 3 | 3 | 6 |
| Stem cell source, n | |||
| BM | 9 | 10 | 19 |
| PBSCs | 3 | 4 | 7 |
| T-depleted PBSCs | 2 | 2 | 4 |
| Conditioning, n* | |||
| TBI-based | 7 | 0 | 7 |
| Busulfan-based | 1 | 0 | 1 |
| Fludarabine/melphalan | 5 | 12 | 17 |
| Other | 1 | 4 | 5 |
| Transplantation, n | |||
| First HSCT | 11 | 12 | 23 |
| Second HSCT | 3 | 4 | 7 |
| Risk score, n† | |||
| 1 | 3 | 12 | 15 |
| 2 | 6 | 4 | 10 |
| 3 | 5 | 0 | 5 |
| Disease status, n‡ | |||
| Standard risk | 5 | Not applicable | 5 |
| High risk | 9 | Not applicable | 9 |
| Variable . | Number of patients . | ||
|---|---|---|---|
| Group A . | Group B . | All . | |
| Male/female, n | 9/5 | 11/5 | 20/10 |
| Median age (range), y | 10 (5-21) | 9 (1-20) | 9 (1-21) |
| Donor, n | |||
| MSD/MFD | 5 | 4 | 9 |
| MUD | 6 | 9 | 15 |
| MMD/haplo | 3 | 3 | 6 |
| Stem cell source, n | |||
| BM | 9 | 10 | 19 |
| PBSCs | 3 | 4 | 7 |
| T-depleted PBSCs | 2 | 2 | 4 |
| Conditioning, n* | |||
| TBI-based | 7 | 0 | 7 |
| Busulfan-based | 1 | 0 | 1 |
| Fludarabine/melphalan | 5 | 12 | 17 |
| Other | 1 | 4 | 5 |
| Transplantation, n | |||
| First HSCT | 11 | 12 | 23 |
| Second HSCT | 3 | 4 | 7 |
| Risk score, n† | |||
| 1 | 3 | 12 | 15 |
| 2 | 6 | 4 | 10 |
| 3 | 5 | 0 | 5 |
| Disease status, n‡ | |||
| Standard risk | 5 | Not applicable | 5 |
| High risk | 9 | Not applicable | 9 |
MSD/MFD indicates HLA-matched sibling donor/HLA-matched family donor; MUD, HLA-matched unrelated donor; MMD, HLA-mismatched donor; haplo, HLA-haploidentical family donor; PBSCs, peripheral blood stem cells; and TBI, total body irradiation.
See Tables 2–3 for details.
Risk score was adapted from Matthes-Martin et al26 ; risk factors were age > 10 years, advanced disease, or alternative donor.
Disease status in malignant diseases: standard risk, complete remission (CR1/CR2); high risk, complete remission beyond CR2 or no remission (NR).