Table 1.
Patient-, donor-characteristics, and transplant outcomes.
| Haplo-HSCT N = 33 | UCBT N = 24 | p | |
|---|---|---|---|
| RECIPIENT | |||
| Male, N (%) | 23 (70%) | 14 (58%) | 0.411 |
| Median age, years (range) | 7.7 (3–17) | 4.7 (1–16) | <0.001 |
| Hematological malignancies | 27 (82%) | 19 (79%) | 0.999 |
| Acute lymphoblastic leukemia | 20 | 11 | |
| Acute myeloid leukemia | 3 | 6 | |
| Myelodysplastic syndromes | 3 | 0 | |
| Juvenile myelomonocytic leukemia | 1 | 2 | |
| Other diagnosis | |||
| Hemophagocytic lymphohistiocytosis | 0 | 5 | |
| Fanconi anemia | 3 | 0 | |
| Congenital amegakaryocytic thrombocytopenia | 2 | 0 | |
| Blackfan-Diamond anemia | 1 | 0 | |
| TRANSPLANTATION | |||
| Source of cells PB/CB | 33/0 | 0/24 | <0.001 |
| Conditioning regimena: TBI/chemo-based | 24/9 | 9/15 | 0.014 |
| GvHD prophylaxisb: TCD/CsA + steroids | 33/0 | 0/24 | <0.001 |
| Infused CD34+ cells: Median (range) × 106/Kg | 22 (8.7–41) | ||
| Infused nucleated cells: Median (range) × 107/Kg | 5.05 (1.4–12.5) | ||
| CLINICAL OUTCOMES | |||
| Acute GvHD | |||
| Grade (I/II/III/IV) | 6/4/1/0 | 4/8/1/0 | |
| Grade II–IV | 5 (15%) | 9 (37%) | 0.067 |
| Chronic GvHD | 6 (18%) | 2 (8%) | 0.446 |
| Relapse | 8 (24%) | 3 (12%) | 0.326 |
| Serious infectionsc | 20 (61%) | 13 (54%) | 0.786 |
Haplo-HSCT indicates HLA-Haploidentical Hematopoietic Stem Cell Transplantation; UCBT, Unrelated Cord Blood Transplantation; CsA, cyclosporin A; GvHD, graft-versus-host disease; PB, peripheral blood; TBI, total body irradiation.
TBI-based conditioning regimen was employed in 33 children and consisted of: fractionated TBI (12 Gy over 6 fractions in 3 days), Thiotepa (10 mg/Kg in 2 doses) and fludarabine (160 mg/m2 over 4 days). Chemotherapy-based conditioning regimen were as follows: 12 patients received Busulfan (16 mg/Kg in 16 doses over 4 days), Cyclophosphamide (120 mg/Kg in 2 days) and melphalan (140 mg/m2 in single dose); 7 children received Busulfan (16 mg/Kg in 16 doses over 4 days), Thiotepa (10 mg/Kg in 2 doses) and fludarabine (160 mg/m2 over 4 days); and 5 children were given Treosulfan (14 gr/m2 for 3 consecutive days), Thiotepa (10 mg/Kg in 2 doses) and fludarabine (160 mg/m2 over 4 days).
Patients receiving Haplo-HSCT were transplanted with CD34+ selected cells and were not given any immune-suppressive drug after transplantation. Patients transplanted with cord blood cells received a combination of Cyclosporine-A (3 mg/Kg/day) and steroids [methylprednisolone (2 mg/Kg/day)] as GvHD prophylaxis.
Reactivation of viral infections (i.e., cytomegalovirus and Epstein-Barr virus) and proven/probable invasive aspergillosis.