Table 1.
Literature documenting response, by drug, for patients with relapsed or refractory HB or HCC.
| Treatment | Number of patients | Reported response | Reference | Quality rating |
|---|---|---|---|---|
| Reponses | ||||
| Doxorubicin-containing | RECIST or Descriptive response | |||
| Doxorubicin (often administered with cisplatin) | 36 HB | 13 PR | Malogolowkin, 200814 | 3 |
| Irinotecan-containing | ||||
| Irinotecan | 1 HB | 1 Prolonged SD in HB; 1 decreased AFP in HB, 1 in HCC | Blaney, 200115 | 2 |
| Irinotecan | 3 HB | 3 decreased AFP; 1 of the 3 with decreased tumor size | Katzenstein, 200216 | 4 |
| Irinotecan | 1 HB | Normalization of AFP | Palmer, 200317 | 5 |
| Irinotecan | 5 HB | 1 SD | Vassal, 200318 | 2 |
| Irinotecan | 1 HB | 1 Decreased AFP, decreased tumor size | Ijichi, 200619 | 5 |
| Irinotecan | 8 HB, 3 HCC | 1 CR (HB) | Bomgaars, 200720 | 2 |
| VCR/irinotecan/tem | 4 HB, 1 HCC | 3 SD (HB); 2 PD (1 HB, 1 HCC) | McKnall-Knapp, 201021 | 2 |
| Irinotecan | 1 HB | 1 CR | Qayed, 201022 | 4 |
| VCR/irinotecan | 1 HB | 1 PR | Qayed, 201022 | 4 |
| VCR/irinotecan/tem | 3 HB | 1 SD then PD | Wagner, 201023 | 2 |
| Irinotecan | 23 HB | 6 PR; 11 SD; 6 PD | Zsiros, 201224 | 2 |
| Temsirolimus, irinotecan/tem | 2 HB | 1 “objective response” | Bagatell, 201425 | 2 |
| VCR/irinotecan | 7 HB | 4 PR; 2 SD; 1 PD | Zhang, 201526 | 2 |
| VCR/irinotecan | 1 HB | 1 PR | Powers, 201927 | 5 |
| Irinotecan/sorafenib | 4 HB, 2 HCC | 1 PR and 3 with PD (HB); 1 PR and 1 SD (HCC); all with previous receipt of irinotecan | Keino, 202028 | 2 |
| Platinum/etoposide-containing | ||||
| Carbo/etop | 1 HB | Decreased size of primary; pulm clearance | Lockwood, 199329 | 5 |
| Ifos/cisplatin/etop | 1 HB | 1 CR | Van Hoff, 199530 | 2 |
| Carbo/etop | 12 HB | 1 CR; 5 PR; 1 SD; 5 PD | Fuchs, 199931 | 2 |
| Ifos/carbo/etop | 1 HB | Decreased AFP | Katzenstein, 200216 | 4 |
| Ifos/carbo/etop | 1 HB | 1 NR | Matsunaga, 200332 | 4 |
| CTX/etop/cisplatin/doxo | 1 HB | 1 NR | Matsunaga, 200332 | 4 |
| Melphalan/etop/carbo | 2 HB | 2 NR | Matsunaga, 200332 | 4 |
| Carbo/doxo/etop | 1 HB | 1 NR | Matsunaga, 200332 | 4 |
| Ifos/carbo/etop | 2 HB | 1 SD; 1 PD | Loss, 200433 | 2 |
| Ifos/carbo/etop | 1 HB | Transient decrease in AFP | Qayed, 201022 | 4 |
| Carbo/ifos/doxo/etop | 1 HB | 1 NR | Miyamura, 201134 | 5 |
| Ifos/carbo/etop | 1 HB | 1 CR | Natarajan, 202035 | 5 |
| Ifos/carbo/etop | 1 HB | Not well documented; DOD | Hou, 202136 | 4 |
| VCR/carbo/5FU (1 with carbo/etop, 2 with ifos/carbo/etop, 1 with VCR/CTX/5FU) | 4 HB | Not well documented; ultimately NED | Hou, 202136 | 4 |
| Targeted therapy | ||||
| GPC-3 vaccine | 7 HB (2 with image evaluable disease) | Not well documented; 2 NE | Tsuchiya, 201737 | 2 |
| Other | ||||
| Thiotepa/melphalan (with autologous stem cell rescue) | 4 | 3 CR, 1 PR | Hara, 199838 | 4 |
Quality rating key: 1) Properly powered and conducted randomized clinical trial or scoping review with meta-analysis; 2) Well-designed controlled trial without randomization or prospective comparative cohort trial; 3) Case-control studies or retrospective cohort study; 4) Case series with or without intervention or cross-sectional study; 5) Opinion of respected authorities or case reports.
AFP, alpha-fetoprotein; ANED, alive no evidence of disease; CR, complete response; DOD, died of disease; HB, hepatoblastoma; NE, non-evaluable; NED, no evidence of disease; NR, no response; PD, progressive disease; PR, partial response; RT, radiation therapy; SD, stable disease; Ifos, ifosfamide; carbo, carboplatin; etop, etoposide; VCR, vincristine; tem, temozolomide; 5FU, 5-fluorouracil; CTX, cyclophosphamide; doxo, doxorubicin.