Skip to main content
. 2023 Jan 3;24(1):856. doi: 10.3390/ijms24010856

Table 5.

Published evidence of second-line systemic therapy in rhabdomyosarcoma.

Regimen Type of Study N Setting RR PFS (Months) Toxicity Ref
Vinorelbine Phase II 33 (13 RMS) Pediatric sarcoma patients 50% 3.5 (all the series) 63% G3-4 neutropenia [29]
Vinorelbine-Cyclophosphamide Phase II 117 (50 RMS) Pediatric and <25 and adults relapsed tumors 36% NR 38% G3-4 neutropenia [26]
Cyclophosphamide-Topotecan Retrospective 15 (6 RMS) Adult relapsed sarcoma patients 33% (2/6) 2.5 (all the series) 47% hematologic toxicity [30]
Cyclophosphamide-Topotecan Phase II window trial 61 Pediatric metastatic RMS (1st line) 47% NR (3-y DFS: 10%) G3-4: leucopenia 52%, anemia 37% [31]
Cyclophosphamide-Topotecan Phase II 91 (15 RMS) Pediatric relapsed tumors 66% (10/15 RMS) NR G3-4: leucopenia 53%, anemia 27% [32]
Vincristine-Irinotecan Phase II window trial (2 regimens) 92 Pediatric relapsed RMS 26–37% NR (1y-FFS 37 and 38%) ≥G3: 50–66% [33]
Vincristine-temozolomide-irinotecan Retrospective 19 Pediatric relapsed RMS 0 PFS-3 months: 23% NR [34]
Topotecan-carboplatin Phase II 38 Pediatric relapsed RMS 28% 5-y PFS 14% 63% G4 hematologic [13]
Cisplatin-etoposide Phase II 27 (21 RMS) Pediatric relapsed tumors 33% (7/21) NR NR [12]
Ifosfamide-carboplatin-etoposide (ICE) Phase I and II trials analysis 97 (27 RMS) Pediatric and adolescent relapse sarcoma 66% (RMS) NR G3-4 hematologic: 100% [36]
Vincristine, ifosfamide and doxorubicin (VIA)/etoposide, ifosfamide and cisplatin (VIP) Retrospective 6 Adult patients (advance disease) 100% NR NR [28]
Several regimenes Retrospective 49 Pediatric relapsed RMS NR NR [10]
Carboplatin-epirubicin-
vincristine ad ifosfamide-vincristine-
etoposide (CEV/IVE)
15 73.3%
Vincristine/irinotecan ± temozolomide (VI[T]) 7 42.9%
Gemcitabine-docetaxel Retrospective 19 (5 RMS) Pediatric relapsed sarcoma 40% (2/5) 2 (all series) G3-4 toxicity 74% [35]

RR: response rate; PFS: progression-free survival; NR: not reported; DFS: disease-free survival; FFS: failure-free survival; RMS: rhabdomyosarcoma.