Table 1. Demographic and Clinical Characteristics of Individuals Who Lived 2 Years or More After Allogeneic BMT Performed in Childhood.
| Variable | Patients, No. (%) (Nā=ā1388) |
|---|---|
| Treating institution | |
| University of Minnesota | 950 (68.4) |
| City of Hope | 438 (31.6) |
| Sex | |
| Male | 829 (59.7) |
| Female | 559 (40.3) |
| Race/ethnicity | |
| Non-Hispanic white | 982 (70.7) |
| Hispanic | 213 (15.3) |
| Non-Hispanic black | 71 (5.1) |
| Other | 96 (6.9) |
| Unknown | 26 (1.9) |
| Age at BMT, y | |
| ā„4 | 379 (27.3) |
| 5-9 | 362 (26.1) |
| 10-14 | 266 (19.2) |
| 15-21 | 381 (27.4) |
| Time period of BMT | |
| <1990 | 323 (23.3) |
| 1990-1999 | 407 (29.3) |
| 2000-2010 | 658 (47.4) |
| Type of donor | |
| Related | 803 (57.9) |
| Unrelated | 585 (42.1) |
| Source of stem cells | |
| Bone marrow | 1019 (73.4) |
| Cord blood | 253 (18.2) |
| PBSCs | 116 (8.4) |
| Primary disease | |
| ALL | 348 (25.1) |
| AML or MDS | 326 (23.5) |
| Inborn errors of metabolism | 192 (13.8) |
| Severe aplastic anemia | 147 (10.6) |
| Fanconi anemiaa | 115 (8.3) |
| Chronic myelogenous leukemia | 90 (6.5) |
| Immune disorders | 55 (4.0) |
| Sickle cell disease or thalassemia | 26 (1.9) |
| Other malignant diseaseb | 64 (4.6) |
| Other nonmalignant diseasec | 25 (1.8) |
| Conditioning regimen | |
| Cyclophosphamide | 1118 (80.5) |
| Total body irradiation | 892 (64.3) |
| Antithymocyte globulin | 563 (40.6) |
| Busulfan | 355 (25.6) |
| Fludarabine | 251 (18.1) |
| Etoposide | 216 (15.6) |
| Melphalan | 68 (4.9) |
| Cytarabine | 58 (4.2) |
| Other chemotherapy | 126 (9.1) |
| Other radiotherapy | 84 (6.1) |
| Total body irradiation plus cyclophosphamide | 691 (49.8) |
| Busulfan plus cyclophosphamide | 326 (23.5) |
| Disease status at BMT | |
| Standard risk of relapsed | 742 (53.5) |
| High risk of relapse | 642 (46.3) |
| Chronic GvHD prophylaxis | |
| Yes | 1361 (98.1) |
| Cyclosporine | 953 (68.7) |
| Methotrexate | 777 (56.0) |
| Systemic corticosteroids | 582 (41.9) |
| Mycophenolic acid | 206 (14.8) |
| T-cell depletion | 190 (13.7) |
| Tacrolimus or sirolimus | 99 (7.1) |
| No. of deaths | 295 (21.3) |
Abbreviations: ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; BMT, blood or marrow transplantation; GvHD, graft-vs-host disease; MDS, myelodysplastic syndrome; PBSC, peripheral blood stem cell.
Includes Fanconi anemia, Diamond Blackfan anemia, and Shwachman-Diamond syndrome.
Includes 27 patients with non-Hodgkin lymphoma, 7 with Hodgkin lymphoma, 8 with neuroblastoma, 2 with unspecified leukemia, and 20 with histiocytosis.
Includes 1 patient with megakaryocytosis, 3 with dyskeratotis congenita, 8 with epidermolysis bullosa, 1 with Kostmann agranulocytosis, 11 with osteopetrosis, and 1 with paroxysmal nocturnal hemoglobinuria.
Standard risk: first or second complete remission after acute leukemia or lymphoma or first chronic phase of chronic myelogenous leukemia or severe aplastic anemia; all others, high risk.