TABLE II.
Characteristics, outcomes, and complications of HSCT
| No. patients | Percent | |
|---|---|---|
| Characteristics | ||
| Tot no. of patients | 58 | |
| Age (y) at transplant, median (range) | 1.4 (0.2-18.8) | |
| Patients who received multiple transplants | ||
| 2 HSCT | 7 | |
| 3 HSCT | 1 | |
| Conditioning | ||
| Full | 27 | 47 |
| RIT | 31 | 53 |
| Donor-related; unrelated | ||
| Matched | 10; 21 | 17; 36 |
| 1 MM | 1; 13 | 2; 22 |
| 2 MM | 0; 6 | 0; 10 |
| 3 MM | 1; 1 | 2; 2 |
| Haplo | 5; 0 | 9; 0 |
| HSC source | ||
| BM | 35 | 60 |
| PB | 11 | 19 |
| CB | 12 | 21 |
| Cell doses | ||
| BM (TNC × 108/kg), median (range) | 7.1 (0.01-91.3) | |
| PB (CD34 × 106/kg), median (range) | 11.4 (4.3-40) | |
| CB (TNC × 107/kg), median (range) | 9.8 (0.6-42) | |
| Serotherapy | ||
| ATG | 22 | 38 |
| Alm | 27 | 46 |
| None | 9 | 16 |
| GvHD prophylaxis | ||
| CSA + MMF | 18 | 31 |
| CSA + steroids | 10 | 17 |
| CSA | 6 | 10 |
| MTX + CSA (with or without short course of steroids) | 8 | 14 |
| MTX + FK506 1 steroids | 7 | 12 |
| Others | 9 | 16 |
| Bone marrow recovery | ||
| Neutrophils (days after HSCT), median (range) | 16 (3-33) | |
| Platelets (days after HSCT), median (range) | 20 (5-114) | |
| Immunoreconstitution | ||
| Patients with T cells > 1000/mmc at 1 y | 22 of 33 | |
| Positive PHA response (months after HSCT), median (range) | 14.5 (3-60) | |
| Independence from IVIg substitution (months after HSCT), median (range) | 7 (1-48) | |
| Use of donor stem cell boost | 3 | |
| Use of donor lymphocytes infusion | 3 | |
| Complications | ||
| Transplant-related toxicity* | 11 | 20 |
| Infections | 46 | 79 |
| Tot GvHD, | 21 | 36 |
| aGvHD (grade I-IV) | 19 | 33 |
| aGvHD (grade III-IV) | 9 | 16 |
| cGvHD | 6 | 10 |
| No GvHD | 37 | 64 |
| Deaths | 15 | 26 |
Alm, Alemtuzumab; aGvHD, acute graft-versus-host disease; ATG, antithymocyte globulin; BM, bone marrow; CB, cord blood; cGvHD, chronic graft-versus-host disease; CSA, cyclosporine; FK506, tacrolimus; Full, full conditioning regimen; IVIg, intravenous immunoglobulin; MM, mismatch; MMF, mycophenolate mofetil; MTX, methotrexate; PB, peripheral blood; PHA, phytohemagglutinine; TNC, total nucleated cells.
*
Toxicity after HSCT consisted of mucositis, pneumonitis, posterior reversible encephalopathy, undefined hypertrophic cardiomyopathy, nephropathy, and hepatic sinusoidal obstruction syndrome.