Patient and transplant characteristics
| Variables . | TCR group . | CD34 group . | P . |
|---|---|---|---|
| Number of patients | 14 | 40 | |
| Age, median (range), y | 7 (3-15.2) | 7 (2.4-17) | .63 |
| Male sex, n | 12 | 23 | .1 |
| Thalassemia, n | 11 | 34 | .68 |
| Sickle cell anemia, n | 3 | 4 | — |
| HbS-β thalassemia, n | — | 2 | — |
| Risk class | .08 | ||
| Class 1/2/3, n | 0/7/4 | 10/16/10 | — |
| Not applicable | 3 | 4 | — |
| Indications for transplantation, n | |||
| Chronic blood transfusion | 11 | 36 | .35 |
| Stroke | 1 | — | — |
| Recurrent vaso-occlusive crisis | 3 | 4 | — |
| Splenectomy, n | 2 | 11 | .47 |
| Median packed RBC units received pretransplant (range) | 55 (0-300) | 55 (7-310) | .53 |
| Median serum ferritin (range), ng/mL | 1391 (32-6649) | 1633 (125-8696) | .54 |
| Donor, n | .35 | ||
| Mother | 12 | 32 | — |
| Father | 2 | 4 | — |
| Brother | — | 4 | — |
| Graft type, n | — | ||
| CD34+ selection of PBSCs and BM | — | 32 | — |
| CD34+ selection of PBSCs and CD3+/CD19+ depletion of BM | — | 8 | — |
| TCRαβ+/CD19+ depletion of PBSCs | 14 | — | — |
| Donor–recipient sex, n | .06 | ||
| Female to male | 11 | 19 | — |
| Other combinations | 3 | 21 | — |
| Donor–recipient CMV status, n | |||
| Both positive | 10 | 24 | .53 |
| Any positive | 2 | 14 | .18 |
| Both negative | 2 | 2 | .27 |
| Number of mismatches, n | |||
| 5 | 10 | 20 | .21 |
| 4 | 3 | 12 | .73 |
| 3 | 1 | 6 | .66 |
| 2 | 0 | 2 | 1 |
| Conditioning regimen, n | |||
| BuTT10Cy200ATG preceded by HuAzFlu (day −59 to −12)* | 14 | 37 | — |
| BuTT10Cy200ATG preceded by Flu (day −17 to −12)* | — | 3 | — |
| Rituximab prophylaxis (yes/no) | 2/12 | 23/17 | .01 |
| GVHD prophylaxis (from day −2 to day +60), n | |||
| CSA + methylprednisolone | 12 | 40 | — |
| CSA + mycophenolate mofetil | 2 | — | — |
| Graft content, median (range) | |||
| CD34+ × 106/kg | 15.7 (8.1-39.2) | 16 (4.3-28.1) | .43 |
| CD3+ × 105/kg | — | 2.8 (0.06-31.6) | — |
| CD19+ × 106/kg | — | 0.2 (0.01-2.11) | — |
| TCRαβ+ CD3+ × 104/kg | 4 (1-10.0) | — | — |
| TCRγδ+ CD3+ × 106/kg | 9 (2.8-40.2) | — | — |
| CD3−CD16+CD56+ × 106/kg | 28.7 (9.8-194.3) | — | — |
| CD20+ × 106/kg | 0.06 (0.01-1.7) | — | — |
| Variables . | TCR group . | CD34 group . | P . |
|---|---|---|---|
| Number of patients | 14 | 40 | |
| Age, median (range), y | 7 (3-15.2) | 7 (2.4-17) | .63 |
| Male sex, n | 12 | 23 | .1 |
| Thalassemia, n | 11 | 34 | .68 |
| Sickle cell anemia, n | 3 | 4 | — |
| HbS-β thalassemia, n | — | 2 | — |
| Risk class | .08 | ||
| Class 1/2/3, n | 0/7/4 | 10/16/10 | — |
| Not applicable | 3 | 4 | — |
| Indications for transplantation, n | |||
| Chronic blood transfusion | 11 | 36 | .35 |
| Stroke | 1 | — | — |
| Recurrent vaso-occlusive crisis | 3 | 4 | — |
| Splenectomy, n | 2 | 11 | .47 |
| Median packed RBC units received pretransplant (range) | 55 (0-300) | 55 (7-310) | .53 |
| Median serum ferritin (range), ng/mL | 1391 (32-6649) | 1633 (125-8696) | .54 |
| Donor, n | .35 | ||
| Mother | 12 | 32 | — |
| Father | 2 | 4 | — |
| Brother | — | 4 | — |
| Graft type, n | — | ||
| CD34+ selection of PBSCs and BM | — | 32 | — |
| CD34+ selection of PBSCs and CD3+/CD19+ depletion of BM | — | 8 | — |
| TCRαβ+/CD19+ depletion of PBSCs | 14 | — | — |
| Donor–recipient sex, n | .06 | ||
| Female to male | 11 | 19 | — |
| Other combinations | 3 | 21 | — |
| Donor–recipient CMV status, n | |||
| Both positive | 10 | 24 | .53 |
| Any positive | 2 | 14 | .18 |
| Both negative | 2 | 2 | .27 |
| Number of mismatches, n | |||
| 5 | 10 | 20 | .21 |
| 4 | 3 | 12 | .73 |
| 3 | 1 | 6 | .66 |
| 2 | 0 | 2 | 1 |
| Conditioning regimen, n | |||
| BuTT10Cy200ATG preceded by HuAzFlu (day −59 to −12)* | 14 | 37 | — |
| BuTT10Cy200ATG preceded by Flu (day −17 to −12)* | — | 3 | — |
| Rituximab prophylaxis (yes/no) | 2/12 | 23/17 | .01 |
| GVHD prophylaxis (from day −2 to day +60), n | |||
| CSA + methylprednisolone | 12 | 40 | — |
| CSA + mycophenolate mofetil | 2 | — | — |
| Graft content, median (range) | |||
| CD34+ × 106/kg | 15.7 (8.1-39.2) | 16 (4.3-28.1) | .43 |
| CD3+ × 105/kg | — | 2.8 (0.06-31.6) | — |
| CD19+ × 106/kg | — | 0.2 (0.01-2.11) | — |
| TCRαβ+ CD3+ × 104/kg | 4 (1-10.0) | — | — |
| TCRγδ+ CD3+ × 106/kg | 9 (2.8-40.2) | — | — |
| CD3−CD16+CD56+ × 106/kg | 28.7 (9.8-194.3) | — | — |
| CD20+ × 106/kg | 0.06 (0.01-1.7) | — | — |
Az, azathioprine; ATG, anti-thymocyte globulin; Flu, fludarabine; Bu, busulfan; CD34 group, CD34+-selected and/or CD3+/CD19+-depleted grafts; CSA, cyclosporine; Cy, cyclophosphamide; HbS, hemoglobin S; Hu, hydroxyurea; RBC, red blood cell; TCR group, TCRαβ+/CD19+-depleted grafts; TT, thiotepa.
From July 2004 to June 2006, patients received 14 mg/kg total dose of oral busulfan and weight-based intravenous Bu thereafter.