Table 2. Clinical trials of average risk medulloblastoma.
| Study (reference) | No. of patients | Cohort criteria | Study outcome | Radiation dose (Gy) | Chemotherapy | Survival | |
|---|---|---|---|---|---|---|---|
| CCG 9892 [37] | 65 | 3–10 yr, M0, R0 | Non-randomized trial, feasibility of reduced dose CSI (23.4 Gy) and adjuvant CT | CSI 23.4/PF 55.8 | VCR during RT, CCNU/CDDP/VCR | 5-yr PFS 79% | |
| SIOP PNET3 [35] | 179 (pre-RT CT 90, RT alone 89) | 3–16 yr, M0 | Randomized trial, pre-RT vs. RT alone (outcome of CT after surgery and before RT) | CSI 35/PF 55 | None vs. VCR, VP, alternating CBP and CPM | 5-yr EFS 67.0% (pre-RT CT 74.2% vs. RT alone 59.7%) | |
| SJMB-96 [45] (average risk arm) | 86 | 3–21 yr M0, R0 | Non-randomized trial, 5-yr EFS | CSI 23.4/PF 36/TB 55.8 | CPM, CDDP, VCR+SCR (4 cycles) | 5-yr EFS 83% | |
| HIT-SIOP | 169 | 4–21 yr, except | Randomized trial evaluating STRT vs. HFRT | Conventional RT CSI 23.4/PF 54 HFRT CSI 36/PF 60 |
VCR during RT; adjuvant CDDP, CCNU, VCR 8 cycles | 5-yr EFS 77% for STRT, 78% for conventional RT group 78% | |
| PNET4 [39] | 169 | LC/A histology | |||||
| COG A9961 [36] | A 193 B 186 |
3–21 yr, M0 or R0 | Randomized trial evaluating adjuvant CPM vs. CCNU based chemo |
CSI 23.4/PF 55.8 | A: CCNU, CDDP, VCR B: CPM, CDDP, VCR |
A: 5-yr EFS 81% B: 5-yr EFS 86% |
|
| SJMB-03 [40] | 227 | 3–21 yr, M0, GTR or NTR (R0) | Non-randomized trial, evaluating dose-intensive chemotherapy | CSI 23.4/PF 55.8 | VCR, CDDP, CPM with autologous SCR 4 cycles | 5-yr PFS 83.2% | |
| ACNS 0331 [38] | 549 | 3–21 yr, M0, ≤1.5 cm2 | Randomized trial, 1) evaluating PFRT vs. IFRT, 2) standard dose CSI (24 Gy) vs. low dose CSI (18 Gy) for young children (3–7 yrs) | CSI 23.4 or 18/PF or IFRT 54 | A: CDDP, CCNU, VCR B: CPM,VCR (AABAABAAB) |
5-yr EFS 81.4 (82.5% for IFRT, 80.5% for PFRT; 82.9% for SDCSI vs. 71.4% for LDCSI) | |
CSI, craniospinal irradiation; PF, posterior fossa; VCR, vincristine; CCNU, lomustine; CDDP, cisplatin; PFS, progression-free survival; RT, radiotherapy; CT, chemotherapy; VP, etoposide; CBP, carboplatin; CPM, cyclophosphamide; EFS, event free survival; TB, tumor bed; SCR, stem cell rescue; LC/A, large cell/anaplastic; HFRT, hyperfractionated radiation therapy; GTR, gross total resection; NTR, near total resection; PFRT, posterior fossa radiation therapy; IFRT, involved field radiation therapy; SDCSI, standard dose CSI; LDCSI, low dose CSI