Table 2. SMR and AER Among 1388 Individuals Who Lived 2 Years or More After Allogeneic BMT in Childhood.
| Variable | Entire Cohort, No. of Patients | All-Cause Mortality (95% CI) | |
|---|---|---|---|
| SMR | AER | ||
| All patients | 1388 | 14.4 (12.8 to 16.1) | 12.0 (10.5 to 13.5) |
| Sex | |||
| Male | 829 | 11.2 (9.6 to 13.0) | 11.5 (9.6 to 13.4) |
| Female | 559 | 23.6 (19.6 to 28.0) | 12.7 (10.4 to 15.1) |
| Age at BMT, y | |||
| ≤4 | 379 | 20.3 (15.8 to 25.7) | 9.6 (7.1 to 12.0) |
| 5-9 | 362 | 22.8 (17.9 to 28.4) | 12.9 (9.8 to 16.0) |
| 10-14 | 266 | 12.8 (9.8 to 16.3) | 11.9 (8.6 to 15.2) |
| 15-21 | 381 | 10.3 (8.4 to 12.5) | 13.8 (10.8 to 16.9) |
| Time period of BMT | |||
| <1990 | 323 | 12.6 (10.6 to 14.9) | 15.0 (12.3 to 17.8) |
| 1990-1999 | 407 | 13.6 (10.9 to 16.6) | 10.3 (7.9 to 12.6) |
| 2000-2010 | 658 | 20.8 (16.4 to 25.9) | 10.4 (7.9 to 12.9) |
| Type of donor | |||
| Related | 803 | 12.8 (11.1 to 14.7) | 12.6 (10.7 to 14.4) |
| Unrelated | 585 | 19.9 (16.0 to 24.3) | 11.0 (8.6 to 13.3) |
| Source of stem cells | |||
| Bone marrow | 1019 | 13.4 (11.8 to 15.2) | 12.1 (10.4 to 13.7) |
| Cord blood | 253 | 21.2 (14.3 to 30.0) | 9.3 (5.7 to 12.8) |
| PBSC | 116 | 22.5 (14.5 to 32.9) | 17.8 (10.2 to 25.4) |
| Primary diagnosis | |||
| ALL | 348 | 18.5 (15.1 to 22.4) | 17.3 (13.7 to 20.9) |
| AML or MDS | 326 | 13.3 (10.5 to 16.7) | 11.6 (8.6 to 14.5) |
| Inborn errors of metabolism | 192 | 28.2 (20.6 to 37.5) | 14.6 (10.1 to 19.2) |
| Severe aplastic anemia | 147 | 4.6 (2.8 to 7.0) | 4.3 (1.8 to 6.9) |
| Fanconi anemiaa | 115 | 21.0 (11.6 to 34.6) | 9.3 (4.0 to 14.6) |
| Chronic myelogenous leukemia | 90 | 11.8 (7.3 to 17.9) | 11.5 (5.8 to 17.1) |
| Other malignant diseaseb | 64 | 14.0 (7.9 to 22.7) | 13.8 (6.0 to 21.6) |
| Immune disorders | 55 | 9.3 (4.0 to 18.0) | 5.6 (1.0 to 10.2) |
| Sickle cell disease or thalassemia | 26 | 24.1 (6.0 to 62.5) | 9.1 (–1.6 to 19.7) |
| Other nonmalignant diseasec | 25 | 37.9 (15.1 to 76.8) | 19.9 (3.6 to 36.3) |
| Disease status at BMT | |||
| Standard risk of relapsed | 742 | 10.3 (9.4 to 11.3) | 9.9 (8.1 to 11.7) |
| High risk of relapse | 642 | 23.3 (21.2 to 25.6) | 15.1 (12.6 to 17.6) |
| Overall survival after BMT, y | |||
| 2-5 | 142 | 522.0 (439.9 to 613.6) | 310.8 (259.0 to 362.6) |
| 6-9 | 337 | 35.9 (26.3 to 47.6) | 15.0 (10.5 to 19.6) |
| 10-14 | 281 | 14.5 (10.0 to 20.0) | 7.9 (5.0 to 10.9) |
| 15-19 | 218 | 9.1 (6.1 to 13.0) | 6.0 (3.5 to 8.5) |
| 20-24 | 164 | 5.8 (3.6 to 8.8) | 4.3 (2.0 to 6.6) |
| ≥25 | 246 | 2.9 (2.0 to 4.1) | 2.6 (1.2 to 3.9) |
Abbreviations: AER, absolute excess risk; ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; BMT, blood or marrow transplantation; MDS, myelodysplastic syndrome; PBSC, peripheral blood stem cells; SMR, standardized mortality ratio.
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.
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.