Patient characteristics at study entry (N = 51)
| . | Values . |
|---|---|
| Median age, y (range) | 9.8 (0.9-20.7) |
| Median time from transplantation to GVHD, mo (range) | 5.0 (1.4-26.1) |
| Median time from GVHD to study entry, mo (range) | 6.2 (0.1-42.1) |
| Median time from transplantation to study entry, mo (range) | 12.2 (3.4-47.7) |
| Sex | |
| Male, n (%) | 27 (53) |
| Female, n (%) | 24 (47) |
| Diagnoses | |
| Acute myeloid leukemia/myelodysplasia, n (%) | 13 (25) |
| Chronic myelogenous leukemia, n (%) | 3 (6) |
| Acute lymphoblastic leukemia, n (%) | 15 (29) |
| Metabolic disorder, n (%) | 6 (12) |
| Hemoglobinopathy, n (%) | 3 (6) |
| Immune deficiency, n (%) | 1 (2) |
| Bone marrow failure syndrome, n (%) | 8 (16) |
| Familial hematophagocytic lymphohistiocytosis, n (%) | 2 (4) |
| Transplantation regimen | |
| Myeloablative, n (%) | 37 (73) |
| Reduced intensity/reduced toxicity, n (%) | 14 (27) |
| Stem cell source | |
| HLA-identical sibling bone marrow, n (%) | 3 (6) |
| HLA-identical sibling PBSC, n (%) | 7 (14) |
| HLA-identical sibling cord blood, n (%) | 1 (2) |
| Unrelated cord blood, n (%)* | 18 (35) |
| Unrelated donor bone marrow, n (%)† | 9 (17) |
| Unrelated donor PBSC, n (%)‡ | 10 (20) |
| Nongenotypically matched family member donor bone marrow (HLA 6:6), n (%) | 1 (2) |
| Nongenotypically matched family member donor PBSC, n (%)§ | 2 (4) |
| . | Values . |
|---|---|
| Median age, y (range) | 9.8 (0.9-20.7) |
| Median time from transplantation to GVHD, mo (range) | 5.0 (1.4-26.1) |
| Median time from GVHD to study entry, mo (range) | 6.2 (0.1-42.1) |
| Median time from transplantation to study entry, mo (range) | 12.2 (3.4-47.7) |
| Sex | |
| Male, n (%) | 27 (53) |
| Female, n (%) | 24 (47) |
| Diagnoses | |
| Acute myeloid leukemia/myelodysplasia, n (%) | 13 (25) |
| Chronic myelogenous leukemia, n (%) | 3 (6) |
| Acute lymphoblastic leukemia, n (%) | 15 (29) |
| Metabolic disorder, n (%) | 6 (12) |
| Hemoglobinopathy, n (%) | 3 (6) |
| Immune deficiency, n (%) | 1 (2) |
| Bone marrow failure syndrome, n (%) | 8 (16) |
| Familial hematophagocytic lymphohistiocytosis, n (%) | 2 (4) |
| Transplantation regimen | |
| Myeloablative, n (%) | 37 (73) |
| Reduced intensity/reduced toxicity, n (%) | 14 (27) |
| Stem cell source | |
| HLA-identical sibling bone marrow, n (%) | 3 (6) |
| HLA-identical sibling PBSC, n (%) | 7 (14) |
| HLA-identical sibling cord blood, n (%) | 1 (2) |
| Unrelated cord blood, n (%)* | 18 (35) |
| Unrelated donor bone marrow, n (%)† | 9 (17) |
| Unrelated donor PBSC, n (%)‡ | 10 (20) |
| Nongenotypically matched family member donor bone marrow (HLA 6:6), n (%) | 1 (2) |
| Nongenotypically matched family member donor PBSC, n (%)§ | 2 (4) |
GVHD indicates graft-versus-host disease; HLA, human leukocyte antigen; and PBSC, peripheral blood stem cell.
HLA-matching: HLA 4:6 (N = 5), HLA 5:6 (N = 11), HLA 6:6 (N = 2).
HLA- matching: HLA 5:6 (N = 1), HLA 6:6 (N = 8).
HLA- matching: HLA 5:6 (N = 4), HLA 6:6 (N = 6).
HLA-matching: HLA 5:6 (N = 1), HLA 6:6 (N = 1).