Patient, donor, and transplant characteristics
| . | MUD . | MMUD . | Haploidentical . | P . |
|---|---|---|---|---|
| Number of patients | 127 | 118 | 98 | |
| Sex | ||||
| Male | 74 (58%) | 71 (60%) | 65 (66%) | .450 |
| Female | 53 (42%) | 47 (40%) | 33 (34%) | |
| Median age at diagnosis (range), y | 5.7 (0.2-17.4) | 8.8 (0.3-17.5) | 6.6 (0.1-17.3) | .113 |
| Diagnosis | ||||
| ALL | 84 (66%) | 86 (73%) | 68 (68%) | .513 |
| AML | 43 (34%) | 32 (27%) | 30 (32%) | |
| ALL phenotype | ||||
| B-cell precursor | 71 (85%) | 67 (78%) | 53 (78%) | .474 |
| T-cell precursor | 13 (15%) | 19 (22%) | 15 (22%) | |
| AML FAB classification | ||||
| AML M0 | 1 (2%) | 2 (6%) | 3 (10%) | .623 |
| AML M1 | 7 (17%) | 6 (19%) | 4 (13%) | |
| AML M2 | 13 (30%) | 4 (13%) | 6 (20%) | |
| AML M3 | 1 (2%) | 1 (3%) | 1 (3%) | |
| AML M4 | 5 (12%) | 4 (13%) | 5 (17%) | |
| AML M5 | 12 (28%) | 13 (40%) | 8 (27%) | |
| AML M6 | 3 (7%) | 1 (3%) | 0 (0%) | |
| AML M7 | 1 (2%) | 1 (3%) | 3 (10%) | |
| Disease phase at HSCT | ||||
| ALL | ||||
| CR1 | 35 (42%) | 39 (45%) | 19 (28%) | <.001 |
| CR2 S1-S2 | 35 (42%) | 28 (33%) | 22 (32%) | |
| CR2 S3-S4 | 14 (16%) | 18 (21%) | 17 (25%) | |
| Other CR | 0 | 1 (1%) | 10 (15%) | |
| AML | ||||
| CR1 | 33 (77%) | 26 (81%) | 23 (77%) | .803 |
| CR2 | 9 (21%) | 6 (19%) | 7 (23%) | |
| Other CR | 1 (2%) | 0 (0%) | 0 (0%) | |
| Median age at HSCT (range), y | 5.7 (1.0-18.0) | 10.3 (0.4-18.1) | 9.4 (0.9-18.2) | .106 |
| Stem cell source | ||||
| BM | 97 (76%) | 90 (76%) | 0 (0%) | <.001 |
| PBSC | 30 (24%) | 28 (24%) | 98 (100%) | |
| Conditioning regimen | ||||
| TBI-based | 64 (50%) | 68 (58%) | 72 (74%) | .016 |
| Busulfan-based | 54 (43%) | 43 (36%) | 18 (18%) | |
| Treosulfan-based | 8 (6%) | 6 (5%) | 7 (7%) | |
| Other chemotherapy | 1 (1%) | 1(1%) | 1 (1%) | |
| GVHD prophylaxis | ||||
| Cs-A+MTX | 2 (2%) | 2 (2%) | 0 (0%) | <.001 |
| Cs-A+MTX+ATLG | 112 (89%) | 97 (82%) | 0 (0%) | |
| Cs-A+MTX+ATLG+MMF | 0 (0%) | 6 (5%) | 0 (0%) | |
| Tacrolimus+MTX+ATLG | 12 (9%) | 13 (11%) | 0 (0%) | |
| αβ+ and CD19+ negative selection + ATLG | 0 (0%) | 0 (0%) | 98 (100%) | |
| Cell dose infused | ||||
| Total nucleated cell dose (range), ×108/kg | 5.5 (1.7-48) | 5.4 (2.2-43) | 10.2 (3-52) | <.001 |
| CD34+ cells, ×106/kg | 5.2 (1.0-40) | 4.9 (2.1-39) | 14.4 (5.5-56) | <.001 |
| CD3+ αβ+ T lymphocytes, ×106/kg | — | — | 0.04 (0.00-0.99) | — |
| CD3+ γδ+ T lymphocytes, ×106/kg | — | — | 8.1 (1.0-64.6) | — |
| . | MUD . | MMUD . | Haploidentical . | P . |
|---|---|---|---|---|
| Number of patients | 127 | 118 | 98 | |
| Sex | ||||
| Male | 74 (58%) | 71 (60%) | 65 (66%) | .450 |
| Female | 53 (42%) | 47 (40%) | 33 (34%) | |
| Median age at diagnosis (range), y | 5.7 (0.2-17.4) | 8.8 (0.3-17.5) | 6.6 (0.1-17.3) | .113 |
| Diagnosis | ||||
| ALL | 84 (66%) | 86 (73%) | 68 (68%) | .513 |
| AML | 43 (34%) | 32 (27%) | 30 (32%) | |
| ALL phenotype | ||||
| B-cell precursor | 71 (85%) | 67 (78%) | 53 (78%) | .474 |
| T-cell precursor | 13 (15%) | 19 (22%) | 15 (22%) | |
| AML FAB classification | ||||
| AML M0 | 1 (2%) | 2 (6%) | 3 (10%) | .623 |
| AML M1 | 7 (17%) | 6 (19%) | 4 (13%) | |
| AML M2 | 13 (30%) | 4 (13%) | 6 (20%) | |
| AML M3 | 1 (2%) | 1 (3%) | 1 (3%) | |
| AML M4 | 5 (12%) | 4 (13%) | 5 (17%) | |
| AML M5 | 12 (28%) | 13 (40%) | 8 (27%) | |
| AML M6 | 3 (7%) | 1 (3%) | 0 (0%) | |
| AML M7 | 1 (2%) | 1 (3%) | 3 (10%) | |
| Disease phase at HSCT | ||||
| ALL | ||||
| CR1 | 35 (42%) | 39 (45%) | 19 (28%) | <.001 |
| CR2 S1-S2 | 35 (42%) | 28 (33%) | 22 (32%) | |
| CR2 S3-S4 | 14 (16%) | 18 (21%) | 17 (25%) | |
| Other CR | 0 | 1 (1%) | 10 (15%) | |
| AML | ||||
| CR1 | 33 (77%) | 26 (81%) | 23 (77%) | .803 |
| CR2 | 9 (21%) | 6 (19%) | 7 (23%) | |
| Other CR | 1 (2%) | 0 (0%) | 0 (0%) | |
| Median age at HSCT (range), y | 5.7 (1.0-18.0) | 10.3 (0.4-18.1) | 9.4 (0.9-18.2) | .106 |
| Stem cell source | ||||
| BM | 97 (76%) | 90 (76%) | 0 (0%) | <.001 |
| PBSC | 30 (24%) | 28 (24%) | 98 (100%) | |
| Conditioning regimen | ||||
| TBI-based | 64 (50%) | 68 (58%) | 72 (74%) | .016 |
| Busulfan-based | 54 (43%) | 43 (36%) | 18 (18%) | |
| Treosulfan-based | 8 (6%) | 6 (5%) | 7 (7%) | |
| Other chemotherapy | 1 (1%) | 1(1%) | 1 (1%) | |
| GVHD prophylaxis | ||||
| Cs-A+MTX | 2 (2%) | 2 (2%) | 0 (0%) | <.001 |
| Cs-A+MTX+ATLG | 112 (89%) | 97 (82%) | 0 (0%) | |
| Cs-A+MTX+ATLG+MMF | 0 (0%) | 6 (5%) | 0 (0%) | |
| Tacrolimus+MTX+ATLG | 12 (9%) | 13 (11%) | 0 (0%) | |
| αβ+ and CD19+ negative selection + ATLG | 0 (0%) | 0 (0%) | 98 (100%) | |
| Cell dose infused | ||||
| Total nucleated cell dose (range), ×108/kg | 5.5 (1.7-48) | 5.4 (2.2-43) | 10.2 (3-52) | <.001 |
| CD34+ cells, ×106/kg | 5.2 (1.0-40) | 4.9 (2.1-39) | 14.4 (5.5-56) | <.001 |
| CD3+ αβ+ T lymphocytes, ×106/kg | — | — | 0.04 (0.00-0.99) | — |
| CD3+ γδ+ T lymphocytes, ×106/kg | — | — | 8.1 (1.0-64.6) | — |
Data are expressed as median and range or as absolute number and column percentage, as appropriate. Indications for hematopoietic stem cell transplantation in CR1 of patients with ALL were high level of minimal residual disease at end of induction therapy (ie, >1 × 10−3 at day +78 after beginning of treatment), high-risk infant ALL, t(4;11), hypodiploidy (≤43 chromosomes), and hyperleukocytosis T ALL with poor response to the steroid prephase. Indications for hematopoietic stem cell transplantation in CR1 of patients with AML were t(10;11), t(6;11), t(6;9), t(5;11), complex karyotype (≥3 either numeric or structural aberrations), FLT3-ITD with high allelic ratio, M7 AML, CBFA2T3-GLIS2 fusion transcript, and not being in morphological CR after the first of the 2 induction courses.
BM, bone marrow; CR, complete remission; CsA, cyclosporine-A; MMF, mycophenolate mofetil; MTX, methotrexate; PBSC, peripheral blood stem cells.