Table 1.
Demographic and clinical characteristics of recipients of allogeneic BMT before the age of 22y
| Variable | All patients (N=789) | ALL (N=242) | AML/MDS (N=212) | SAA (N=102) | BMF syndrome (N=72) | CML (N=54) | Immunodeficieny (N=40) | Lymphoma* (N=23) | Other** (N=44) |
|---|---|---|---|---|---|---|---|---|---|
| Age at BMT in years | |||||||||
| Median (Range) | 11.3 (0.4–22.0) | 12.3 (0.5–22.0) | 13.7 (0.5–22.0) | 13.1 (0.5–21.5) | 9.0 (1.4–22.0) | 14.7 (1.1–21.9) | 2.5 (0.4–14.8) | 17.2 (5.7–21.9) | 4.1 (0.6–20.4) |
| Follow-up in years | |||||||||
| Median (Range) | 11.7 (2.0–45.3) | 8.9 (2.0–36.8) | 12.9 (2.0–43.0) | 14.6 (2.0–45.3) | 9.9 (2.2–23.0) | 12.3 (2.1–32.5) | 13.3 (2.2–38.1) | 10.7 (2.5–40.5) | 10.8 (2.2–28.5) |
| Age at BMT in years, n (%) | |||||||||
| 0–4 | 166 (21.0%) | 37 (15.3%) | 40 (18.9%) | 15 (14.7%) | 14 (19.4%) | 6 (11.1%) | 29 (72.5%) | 0 (0%) | 25 (56.8%) |
| 5–9 | 190 (24.1%) | 66 (27.3%) | 37 (17.5%) | 24 (23.5%) | 31 (43.1%) | 14 (25.9%) | 6 (15.0%) | 4 (17.4%) | 8 (18.2%) |
| 10–14 | 156 (19.8%) | 49 (20.3%) | 46 (21.7%) | 21 (20.6%) | 16 (22.2%) | 8 (14.8%) | 5 (12.5%) | 3 (13.0%) | 8 (18.2%) |
| 15–21 | 277 (35.1%) | 90 (37.2%) | 89 (42.0%) | 42 (41.2%) | 11 (15.3%) | 26 (48.1%) | 0 (0%) | 16 (69.6%) | 3 (6.8%) |
| Sex, n (%) | |||||||||
| Female | 357 (45.2%) | 97 (40.1%) | 103 (48.6%) | 57 (55.9%) | 34 (47.2%) | 32 (59.3%) | 10 (25.0%) | 7 (30.4%) | 17 (38.6%) |
| Race/ethnicity, n (%) | |||||||||
| Non-hispanic white | 590 (74.8%) | 172 (71.1%) | 164 (77.4%) | 75 (73.5%) | 55 (76.4%) | 42 (77.8%) | 33 (82.5%) | 13 (56.5%) | 36 (81.8%) |
| Hispanic | 109 (13.8%) | 51 (21.1%) | 28 (13.2%) | 15 (14.7%) | 6 (8.3%) | 3 (5.6%) | 0 (0%) | 5 (21.7%) | 1 (2.3%) |
| Black | 33 (4.2%) | 5 (2.1%) | 4 (1.9%) | 5 (4.9%) | 4 (5.6%) | 3 (5.6%) | 5 (12.5%) | 2 (8.7%) | 5 (11.4%) |
| Asian | 38 (4.8%) | 12 (5.0%) | 7 (3.3%) | 4 (3.9%) | 6 (8.3%) | 4 (7.4%) | 1 (2.5%) | 3 (13.0%) | 1 (2.3%) |
| Mixed/Other/Unknown | 19 (2.4%) | 2 (0.8%) | 9 (4.2%) | 3 (2.9%) | 1 (1.4%) | 2 (3.7%) | 1 (2.5%) | 0 (0%) | 1 (2.3%) |
| Year of BMT, n (%) | |||||||||
| <1990 | 230 (29.2%) | 66 (27.3%) | 74 (34.9%) | 42 (41.2%) | 1 (1.4%) | 19 (35.2%) | 12 (30.0%) | 10 (43.5%) | 6 (13.6%) |
| 1990–1999 | 230 (29.2%) | 79 (32.6%) | 67 (31.6%) | 26 (25.5%) | 10 (13.9%) | 16 (29.6%) | 16 (40.0%) | 1 (4.3%) | 15 (34.1%) |
| ≥2000 | 329 (41.7%) | 97 (40.1%) | 71 (33.5%) | 34 (33.3%) | 61 (84.7%) | 19 (35.2%) | 12 (30.0%) | 12 (52.2%) | 23 (52.3%) |
| Donor type, n (%) | |||||||||
| Related | 438 (55.5%) | 150 (62.0%) | 136 (64.2%) | 71 (69.6%) | 15 (20.8%) | 23 (42.6%) | 7 (17.5%) | 14 (60.9%) | 22 (50.0%) |
| Unrelated | 351 (44.5%) | 92 (38.0%) | 76 (35.8%) | 31 (30.4%) | 57 (79.2%) | 31 (57.4%) | 33 (82.5%) | 9 (39.1%) | 22 (50.0%) |
| Stem cell source, n (%) | |||||||||
| Cord blood | 103(13.1%) | 32 (13.2%) | 31 (14.6%) | 3 (2.9%) | 19 (26.4%) | 3 (5.6%) | 3 (7.5%) | 4 (17.4%) | 8 (18.2%) |
| Peripheral blood stem cells | 77 (9.8%) | 35 (14.5%) | 27 (12.7%) | 4 (3.9%) | 0(0%) | 4 (7.4%) | 2 (5.0%) | 3 (13.0%) | 2 (4.5%) |
| Bone marrow | 609 (77.2%) | 175 (72.3%) | 154 (72.6%) | 95 (93.1%) | 53 (73.6%) | 47 (87.0%) | 35 (87.5%) | 16 (69.6%) | 34 (77.3%) |
| Total body irradiation, n (%) | |||||||||
| Yes | 572 (72.5%) | 233 (96.3%) | 158(74.5%) | 32 (31.4%) | 58 (80.6%) | 46 (85.2%) | 8 (20.0%) | 22 (95.7%) | 15 (34.1%) |
| Conditioning intensity***, 20, n (%) | |||||||||
| Myelooablative | 542 (69.0%) | 225 (93.0%) | 145 (68.4%) | 71 (71.0%) | 9 (12.5%) | 48 (88.9%) | 9 (23.7%) | 15 (65.2%) | 20 (45.5%) |
| NMA/Reduced intensity | 243 (31.0%) | 17 (7.0%) | 67 (31.6%) | 29 (28.4%) | 63 (87.5%) | 6 (11.1%) | 29 (72.5%) | 8 (24.8%) | 24 (54.5%) |
| cGVHD status, n (%) | |||||||||
| No | 505 (64.0%) | 142 (58.7%) | 124 (58.5%) | 69 (67.7%) | 64 (88.9%) | 26 (48.2%) | 30 (75.0%) | 13 (56.5%) | 37 (84.1%) |
| Yes | 262 (33.2%) | 94 (38.8%) | 81 (38.2%) | 30 (29.4%) | 7 (9.7%) | 27 (50.0%) | 8 (20.0%) | 9 (39.1%) | 6 (13.6%) |
| Missing | 22 (2.8%) | 6 (2.5%) | 7 (3.3%) | 3 (2.9%) | 1 (1.4%) | 1 (1.9%) | 2 (5.0%) | 1 (4.3%) | 1 (2.3%) |
| Pre-BMT treatment, n (% of patients with pre-BMT data available) # | |||||||||
| Alkylators | 124 (33.4%) | 88 (61.5%) | 10 (8.2%) | 0 (0%) | 0 (0%) | 8 (17.8%) | 2 (66.7%) | 14 (77.8%) | 2 (28.6%) |
| Anthracyclines | 267 (72.0%) | 130 (90.9%) | 116 (95.1%) | 0 (0%) | 0 (0%) | 4 (8.9%) | 2 (66.7%) | 14 (77.8%) | 1 (14.3%) |
| Antimetabolites | 285 (76.8%) | 140 (97.9%) | 116 (95.1%) | 0 (0%) | 0 (0%) | 9 (20.0%) | 2 (66.7%) | 14 (77.8%) | 4 (57.1%) |
| Topoisomerase inhibitors | 126 (34.0%) | 53 (37.1%) | 60 (49.2%) | 0 (0%) | 0 (0%) | 1 (2.2%) | 2 (66.7%) | 6 (33.3%) | 4 (57.1%) |
| Plant alkaoids | 176 (47.4%) | 139 (97.2%) | 15 (12.3%) | 0 (0%) | 0 (0%) | 3 (6.7%) | 2 (66.7%) | 15 (83.3%) | 2 (28.6%) |
| Radiotherapy | 63 (16.9%) | 49 (34.0%) | 6 (4.9%) | 0 (0%) | 0 (0%) | 1 (2.2%) | 1 (33.3%) | 5 (27.8%) | 1 (14.3%) |
| Deceased, n (%) | |||||||||
| Yes | 249 (31.6%) | 92 (38.0%) | 69 (32.5%) | 19 (18.6%) | 19 (26.4%) | 19 (35.2%) | 6 (15.0%) | 6 (26.1%) | 19 (43.2%) |
*
Lymphoma includes non-Hodgkin lymphoma (N=20) and Hodgkin lymphoma (N=3);
**
Other include hemophagocytic lymphohistiocytosis (N=10), neuroblastoma (N=8), recessive dystrophic epidermolysis bullosa (N=9), sickle cell disease (N=5), Langerhans cell histiocytosis (N=3), thalassemia (N=2), other leukemia (N=5), other (N=2);
***
Information on conditioning missing for 2 patients with SAA and 2 patients with immunodeficiency;
#
Pre-BMT treatment data available for 372 of 789 BMT recipients (47%); of the 540 BMT recipients alive at study participation, 304 (56%) had pre-BMT data available. ALL=acute lymphoblastic leukemia, AML/MDS=acute myeloid leukemia/myelodysplastic syndrome, SAA=severe aplastic leukemia, BMF=bone marrow failure, CML=chronic myeloid leukemia, NMA=non-myeloablative, cGVHD=chronic graft vs. host disease