Table 1.
Studies of empiric allogeneic HSCT in the treatment of pediatric hypodiploid ALL
| Study | Design | Study years | Population, N (n with HSCT) | CR (% of patients) | Median age (range), y | N of chromosomes (% of patients) | EOI MRD (% of patients) | Outcomes (95% CI) |
|---|---|---|---|---|---|---|---|---|
| Mehta et al7 (CIBMTR) | Retrospective, nonrandomized, multicenter | 1990-2010 | 78 (78) | CR1 (55) | 10 (3-18) | ≤43 (50) | NR | 5-y LFS: 51% |
| CR2 (38) | 44 (15) | ≤43 ch, 37% (23-51) vs 44-45, 64% (48-76); P = .01 | ||||||
| CR3 (7) | 45 (35) | 5-y OS: 56% | ||||||
| ≤43 ch, 38% (24-52) vs 44-45, 71% (56-82); P = .001 | ||||||||
| Pui et al4 (Ponte di Legno) | Retrospective, nonrandomized, multicenter | 1997-2013 | 272 (42 of 228)* | CR1 (100) | 9.8 (0.6-19.5) | 25-29 (37) | <10−4 (54) | 5-y EFS: 55.1% (49.3-61.5) |
| 30-39 (43) | 10−4-10−3 (16) | Favorable: 44 ch, 74% (61-89); P = .021 | ||||||
| 40-43 (5) | ≥10−3 (30) | MRD <10−4, 75% (66-85); P = .003 | ||||||
| 44 (15) | 5-y DFS: | |||||||
| HSCT vs no HSCT*: 59.8% (45.7-78.2) vs 53% (45.9-61.2); P = .47 | ||||||||
| MRD <10−4: 70% (46.7-100) vs 73.6% (63.3-85.7); P = .81 | ||||||||
| MRD ≥10−3: 55.9% (37.2-84) vs 40.3% (27.2-59.7); P = .29 | ||||||||
| 30-39 ch, 63.5 (43.2-93.3) vs 61.6 (51.8-73.1); P = .89 | ||||||||
| 25-29 ch, 50.8 (32.5-79.4) vs 44 (34.2-56.6); P = .60 | ||||||||
| 5-y OS: | ||||||||
| HSCT vs no HSCT*: 68.9% (55.8-85.2) vs 57.7% (50.7-65.7); P = .21 | ||||||||
| McNeer et al2 (COG) | Retrospective, nonrandomized, multicenter | 2003-2011 | 131 (61 of 113) | CR1 (100) | 10 (1-30)† | 25-29 (42) | <0.01% (68) | 5-y EFS: 52.2% ± 4.9% |
| 30-39 (36) | ≥0.01%, 32% | HSCT vs no HSCT: 56.4% ± 7.3% vs 48.8% ± 7.8%; P = .62 | ||||||
| 40-43 (2) | NCI SR: 68.8% ± 10.3% vs 57.1% ± 13.2%; P = .64 | |||||||
| Masked (20) | NCI HR: 48.3% ± 9.0% vs 44.4% ± 9.2%; P = .75 | |||||||
| MRD <0.01%: 66.3% ± 7.9% vs 60.3 ± 9.2%; P = .77 | ||||||||
| MRD ≥0.01%: 29.4% ± 14.3% vs 16.7% ± 10.8%; P = .67 | ||||||||
| 5-y OS: 58.9% ± 4.8% | ||||||||
| HSCT vs no HSCT: 65.6% ± 6.9% vs 53.8% ± 7.8%; P = .32 |
ch, chromosomes; CI, confidence interval; CIBMTR, Center for International Blood and Marrow Transplant Research; COG, Children’s Oncology Group; DFS, disease-free survival; HR, high risk (age ≥10 y or white blood cell count ≥50,000/µL); LFS, leukemia-free survival; NCI, National Cancer Institute; NR, not reported; SR, standard risk (age 1 to <10 y and white blood cell count <50 x 103/µL).
*
HSCT analyses limited to patients with <44 chromosomes.
†
Age range eligible for study (actual age range not reported).