Table 5.
Summary of previous clinical series using HDCT/auto-SCT for children with ATRT
| Source | Type of study | No of patients | Age, median (range, mo)a) | Pre-HDCT chemotherapyb) | HDCT regimenb) | RTb) | Final survival outcome |
|---|---|---|---|---|---|---|---|
| Hilden et al. (2004) [1] | Retrospective review | 42a) (13)b) | 24 (1-118) | Multiple regimens | CT×3 (5), others (8) | None (8), L-RT (3), CSI (1), CSI+L-RT (1) | NED (6), DOD (6), TD (1)b) |
| Tekautz et al. (2005) [2] | SJMB96 | 9 (7) | All > 36 | - | CpCyV4 | CSI+L-RT before HDCT (7) | NED (5), AWD (1), DOD (1)b) |
| Gardner et al. (2008) [11] | Head Start-I | 6 (2) | 35.5 (4-45) | 0, 3 cycles | CTE (2) | None (2) | DOD (2)b), 3-yr EFS 0%b) |
| Head Start-II | 7 (5) | 28 (5-52) | 5 cycles | CTE (5) | None (3), CSI+L-RT after HDCT (1), L-RT after relapse (1) | NED (3), DOD (1), TD (1)b), 3-yr EFS 43±19%b) | |
| Finkelstein-Shechter et al. (2010) [17] | Retrospective review | 8 (6) | 35 (11-43) | 3 cycles (5), IRS-III (1) | CT×3 | None (4), L-RT before HDCT (1), L-RT after HDCT (1) | NED (4), DOD (2)b) |
| Lafay-Cousin et al. (2012) [6] | Retrospective review | 50 (18) | 17 (1-188) | Multiple regimens | CTE (4), CT3 (14) | NA | Alive (9), median survival 40.8 mob); 2-yr OS 48±12%b) |
| Park et al. (2012) [18] | KSPNO S-082 | 9 (6) | 12 (4-28) | 6 cycles (5), 4 cycles (1) | First: CTE, Second: CM | None (1), L-RT before HDCT (1), CSI+L-RT after HDCT (4) | NED (4), AWD (1), DOD (1)b); 3-yr EFS 0%, 3-yr OS 53±17%b) |
| Zaky et al. (2014) [19] | Head Start-III | 19 (5) | 13 (7-27) | 5 cycles | CTE | None (4), CSI+L-RT after relapse (1) | NED (3), AWD (1), DOD (1)b); 3-yr EFS 21±9%, 3-yr OS 26±10%b) |
HDCT/autoSCT, high-dose chemotherapy and autologous stem cell transplantation; ATRT, atypical teratoid/rhabdoid tumor; RT, radiotherapy; CT, carboplatin+thiotepa; L-RT, local RT; CSI, craniospinal irradiation; NED, no evidence of disease; DOD, died of disease; TD, toxic death; CpCyV, cisplatin+cyclophosphamide vincristine; AWD, alive with disease; IRS-III, Intergroup Rhabdomyosarcoma III; CTE, carboplatin+thiotepa+etoposide; EFS, event-free survival; NA, not available; OS, overall survival; CM, cyclophosphamide+melphalan.
a)
Data of all patients,
b)
Data of patients who underwent HDCT.