Patient, donor, and transplantation characteristics
| Patients . | (n = 80) . |
|---|---|
| Sex | |
| Male | 55 (69%) |
| Female | 25 (31%) |
| Median (range) age at diagnosis, y | 6.6 (0.4-16.8) |
| Median (range) age at transplantation, y | 9.7 (0.9-20.9) |
| Disease | |
| ALL | 56 (70%) |
| AML | 24 (30%) |
| ALL phenotype | |
| BCP | 41 (73%) |
| T | 15 (27%) |
| ALL recurrent molecular lesions | |
| t(4;11)(AF4/MLL) | 3 |
| t(9;22)(BCR/ABL) | 2 |
| SIL-TAL | 1 |
| t(12;21)(TEL/AML1) | 2 |
| Hypodiploid | 1 |
| AML recurrent molecular/cytogenetic lesions | |
| MLL/FLT3-ITD | 5 |
| 7- | 1 |
| Complex karyotype | 3 |
| inv(16) (MYH11-CBFB) | 2 |
| Other | 1 |
| Disease status at transplantation | |
| ALL | |
| CR1* | 15 (19%) |
| CR2† | 37 (46%) |
| ≥CR3 | 4 (5%) |
| AML | |
| CR1‡ | 16 (20%) |
| CR2 | 8 (10%) |
| CMV serology (donor/recipient) | |
| Neg/Neg | 5 (6%) |
| Neg/Pos | 7 (9%) |
| Pos/Neg | 11 (14%) |
| Pos/Pos | 57 (71%) |
| Conditioning regimens§ | |
| TBI+TT+Flu | 40 (50%) |
| TBI+TT+L-PAM | 20 (25%) |
| TT+Bu+Flu | 13 (16%) |
| Bu+Cy+L-PAM | 7 (9%) |
| Donor characteristics | |
| Age, y | 41.5 (27-55) |
| Type of donor | |
| Mother | 46 (58%) |
| Father | 34 (42%) |
| Sex mismatch | 49 (61%) |
| Female donor → Male recipient | 35/49 (71%) |
| NK alloreactivity (KIR/KIR-L model) yes/no | 36 (45%)/44 (55%) |
| KIR genotype A/A vs B/X | 16 (20%)/64 (80%) |
| Donor B content value 0-1 vs ≥2 | 44 (55%)/36 (45%) |
| Donor KIR2DS1 “educated and useful”‖ yes/no | 28 (35%)/52 (65%) |
| Cell dose infused, median (range) | |
| CD34+ cells × 106/kg | 13.93 (6-40.44) |
| αβ+ T cells × 106/kg | 0.047 (0.002-0.099) |
| γδ+ T cells × 106/kg | 8.1 (0.86-56.7) |
| NK cells × 106/kg | 34.6 (3.84-146.1) |
| CD20+ B cells × 106/kg | 0.09 (0.05-0.48) |
| Patients . | (n = 80) . |
|---|---|
| Sex | |
| Male | 55 (69%) |
| Female | 25 (31%) |
| Median (range) age at diagnosis, y | 6.6 (0.4-16.8) |
| Median (range) age at transplantation, y | 9.7 (0.9-20.9) |
| Disease | |
| ALL | 56 (70%) |
| AML | 24 (30%) |
| ALL phenotype | |
| BCP | 41 (73%) |
| T | 15 (27%) |
| ALL recurrent molecular lesions | |
| t(4;11)(AF4/MLL) | 3 |
| t(9;22)(BCR/ABL) | 2 |
| SIL-TAL | 1 |
| t(12;21)(TEL/AML1) | 2 |
| Hypodiploid | 1 |
| AML recurrent molecular/cytogenetic lesions | |
| MLL/FLT3-ITD | 5 |
| 7- | 1 |
| Complex karyotype | 3 |
| inv(16) (MYH11-CBFB) | 2 |
| Other | 1 |
| Disease status at transplantation | |
| ALL | |
| CR1* | 15 (19%) |
| CR2† | 37 (46%) |
| ≥CR3 | 4 (5%) |
| AML | |
| CR1‡ | 16 (20%) |
| CR2 | 8 (10%) |
| CMV serology (donor/recipient) | |
| Neg/Neg | 5 (6%) |
| Neg/Pos | 7 (9%) |
| Pos/Neg | 11 (14%) |
| Pos/Pos | 57 (71%) |
| Conditioning regimens§ | |
| TBI+TT+Flu | 40 (50%) |
| TBI+TT+L-PAM | 20 (25%) |
| TT+Bu+Flu | 13 (16%) |
| Bu+Cy+L-PAM | 7 (9%) |
| Donor characteristics | |
| Age, y | 41.5 (27-55) |
| Type of donor | |
| Mother | 46 (58%) |
| Father | 34 (42%) |
| Sex mismatch | 49 (61%) |
| Female donor → Male recipient | 35/49 (71%) |
| NK alloreactivity (KIR/KIR-L model) yes/no | 36 (45%)/44 (55%) |
| KIR genotype A/A vs B/X | 16 (20%)/64 (80%) |
| Donor B content value 0-1 vs ≥2 | 44 (55%)/36 (45%) |
| Donor KIR2DS1 “educated and useful”‖ yes/no | 28 (35%)/52 (65%) |
| Cell dose infused, median (range) | |
| CD34+ cells × 106/kg | 13.93 (6-40.44) |
| αβ+ T cells × 106/kg | 0.047 (0.002-0.099) |
| γδ+ T cells × 106/kg | 8.1 (0.86-56.7) |
| NK cells × 106/kg | 34.6 (3.84-146.1) |
| CD20+ B cells × 106/kg | 0.09 (0.05-0.48) |
BCP, B-cell precursors; BU, busulfan; CMV, cytomegalovirus; CY, cyclophosphamide; Flu, fludarabine; L-PAM, melphalan; Neg, negative; Pos, positive; TT, thiotepa.
Of the 15 patients with ALL transplanted in CR1, 9 had high level of minimal residual disease at the end of induction therapy (ie, >1 × 10−3 at day +78 after beginning of treatment), 2 had high-risk infant ALL, 1 had t(4;11), and 3 had hyperleukocytosis T ALL with poor response to the steroid prephase.
Of the 16 patients with AML transplanted in CR1, 3 had t(10;11), 2 a complex karyotype, 3 had FLT3-ITD with high allelic ratio, 2 had M7 AML, and 6 were not in morphological CR after the first of the 2 induction courses.
Of the 37 patients with ALL transplanted in CR2, 21 (57%) and 16 (43%) patients belonged to the S2 and S3/S4 Berlin-Frankfurt-Münster classification of first relapse ALL, respectively.59
TBI (12 Gy over 3 d in 6 fractions of 200 cGy each) was employed in 50 children with ALL and in 10 children with AML; all these patients were older than 3 y.
HLA-C C1pos donor and HLA-C C2pos patient.