Table 1.
Summary of studies included in review
| Study | Disease | Time period | HSCT (n) | Chemotherapy (n) | Findings | Comments |
|---|---|---|---|---|---|---|
| Pui (2002)6 | Infant t(4;11) ALL | 1983–1995 | 28 | 103 | Chemotherapy EFS 23%, OS 33% | Retrospective study of 11 cooperative groups and single institutions |
| Pui (2003)7 | HSCT EFS 18%, OS 25% | |||||
| Marco8 | Infant ALL and AML | 1990–1998 | 22 in CR1 | None | 5-y DFS for HSCT in CR1 71% for all infants, 50% for MLL-R infants | Retrospective, multi-institution; 5-y DFS for ALL 56%; 5-y DFS for AML 73% |
| Sanders9 | Infant ALL | 1982–2003 | 40 | None | 3-y DFS overall 42%; 3-y DFS for HSCT in CR1 76% | Retrospective, single institution; 62.5% of patients MLL-R; 43% received HSCT in CR1 |
| Murray10 | Infant ALL | 1986–2005 | 4 | 5 | HSCT OS 75%; chemotherapy OS 60% | Retrospective, single institution, all age < 6 mo; 44% MLL-R; 3 HSCT in CR1 |
| Jacobsohn11 | Infant ALL | 1992–2005 | 16 in CR1 | None | 4-y EFS 75% | Retrospective, single institution; 38% age ≤ 6 mo; all had MLL-R |
| Woolfrey12 | AML/MDS age < 2 y | 1974–1995 | 34 AML | None | 5-y DFS in CR1 38%; 5-y OS in CR1 54% | Retrospective, single institution; 38% HSCT in CR1 |
| Eapen13 | ALL and AML age < 18 mo | 1990–2001 | 146 ALL 121 AML |
None | 3-y OS for matched sibling HSCT in CR1 54% 3-y OS for unrelated HSCT in CR1 62% |
Retrospective, registry study; 60% HSCT in CR1 |
| Chessels14 | Infant ALL | 1992–1999 | 12 in CR1 | 57 | 3-y EFS ≈35% for both HSCT and chemotherapy | Infant 92 trial; MLL-R 67%; HSCT offered for MLL-R with matched sibling in CR1 |
| Isoyama15 | Infant MLL-R ALL | 1995–1998 | 19 in CR1 | 8 in CR1 | EFS HSCT 58%; EFS chemotherapy 63% | MLL96 trial; HSCT offered for MLL-R with HLA-matched donor in CR1 |
| Kosaka16 | Infant MLL-R ALL | 1998–2002 | 29 in CR1 | 15 in CR1 | 3-y EFS HSCT in CR1 64%; 3-y EFS overall 44% | MLL98 trial; HSCT offered for MLL-R in CR1; chemotherapy determined by CD10 status |
| Mann17 | Infant MLL-R ALL | 1999–2006 | 37 in CR1 | 240 in CR1 | 4-y DFS HSCT 60%; 4-y DFS chemotherapy 47% | Interfant-99 trial; HSCT offered for poor response to prophase; DFS advantage limited to high-risk group |
| Dreyer18 | Infant MLL-R ALL | 1996–2000 | 53 | 47 | 5-y EFS HSCT 49%; 5-y EFS chemotherapy 49% | CCG 1953 and POG 9407 trials; HSCT offered if matched or single antigen mismatched donor; 47% HSCT in CR1 |
| Kawasaki19 | Infant AML | 1995–1998 | 6 in CR1 | 26 in CR1 | EFS HSCT 100%; EFS chemotherapy 77% | Japan Infant Leukemia Study Group study; HSCT offered if HLA-matched donor in CR1; 50% MLL-R |
| Klusmann20 | AML age < 2 y | 1998–2004 | 15 | 69 | 5-y DFS HSCT 53%; 5-y DFS chemotherapy 46% | AML-BFM-98 trial; HSCT offered for high-risk features with HLA-matched sibling in CR1; DFS and OS advantage of HSCT in MLL-R |
| Creutzig5 | Infant AML | 1998–2010 | 14 in CR1 | Not compared | OS HSCT 93% | AML-BFM-98 and AML-BFM- 2004 trials; HSCT offered for high-risk features with HLA-matched sibling in CR1; 56% MLL-R |
BFM indicates Berlin-Frankfurt-Münster.