Table 1.
Demographic Data of the Study Patients (N = 25)
| Demographic | Value |
|---|---|
| Male sex, n/N (%) | 17/25 (68) |
| Age, yr, median (IQR) | 1.07 (.35–1.44) |
| Total number of transplantations | 28 |
| Patients with 2 transplantations | 3 |
| HLA match (including both bone marrow and cord blood), n/N (%) | |
| Matched unrelated | 16/28 (57) |
| Mismatched unrelated | 11/28 (39) |
| Matched related | 1/28 (4) |
| Stem cell source, n/N (%) | |
| Cord blood | 15/28 (54) |
| Bone marrow | 13/28 (46) |
| Conditioning regimen, n/N (%) | |
| Myeloablative | 18/28 (64) |
| Reduced intensity | 10/28 (36) |
| Conditioning regimen, n/N (%) | |
| Busulfan, cyclophosphamide | 13/28 (46) |
| Campath, fludarabine, melphalan | 8/28 (28) |
| Busulfan, cyclophosphamide, ATG | 3/28 (11) |
| Busulfan, fludarabine, ATG | 3/28 (11) |
| Busulfan, Campath, fludarabine | 1/28 (4) |
| GVHD prophylaxis, n/N(%) | |
| Calcineurin-based | 28/28 (100) |
| GVHD post-transplantation, n/N (%) | |
| GVHD at day 100 | 6/25 (24) |
| GVHD grade 1–2 | 4/6 (57) |
| GVHD grade 3–4 | 2/6 (33) |
| GVHD skin | 5/6 (83) |
| Stage 1–2 | 3/5 (60) |
| Stage 3–4 | 2/5 (40) |
| GVHD gut | 1/6 (17) |
| Stage 1–2 | 1/1 (100) |
| Stage 3–4 | 0/1 (0) |
| Hurler patients with both GVHD and a BSI | 2/6 (33) |
| Grade 1 | 1/2 (50) |
| Grade 4 | 1/2 (50) |
| Other transplantation complications, n/N (%) | |
| Engraftment syndrome | 8/25 (32) |
| Patients with engraftment syndrome and a BSI | 2/8 (25) |
| Patients with engraftment syndrome and TA-TMA | 0/8 (0) |
| TA-TMA | 9/25 (36) |
| Patients with TMA and a BSI | 7/9 (78) |
| Patients with TMA and a BSI treated with eculizumab | 3/7 (43) |
| Patients with GVHD and TA-TMA | 1/7 (14) |
ATG, antithymocyte globulin; MBI, mucosal barrier injury; LCBI, laboratory-confirmed bloodstream infection.