Table 1. Trials and studies investigating the effect of NAC on tumour downstaging (pCR and downstaging) and outcomes (progression free survival and overall survival) in patients with MIBC.
| Clinical study (design) | N | Original stage | Treatments | pDS rate | pCR rate | Effect of pDS on OS |
|---|---|---|---|---|---|---|
| SWOG (phase 3) (4) | 317 | T2-T4a | MVAC vs. RC | – | 38% vs. 15% | OS for patient with pCR was 85% |
| Health and Research Informatics System, Florida (retrospective) (7) | 1,113 | T1-T4/N0-3/M0-1 | dd-MVAC vs. GC | 52.2% vs. 41.3% | 41.3% vs. 24.5% | – |
| MSKCC (phase 2) (8) | 49 | T2-T4aN0M0 | dd-GC | – | 15% | pDS was associated with better RFS and OS |
| RISC and NCDB (retrospective) (9) | 181 (RISC) and 2,010 (NCDB) | T2-4N0M0 | NAC | 33% and 35% | 20% and 15% | pDS was associated with better OS |
| Nordic Trials 1 and 2 (retrospective) (10) | 449 | T2-T4aNxM0, pT4aN0-N+M0 | NAC vs. RC | – | 22.7% vs. 12.5% | pCR was associated with better OS in NAC group |
| European and American study (retrospective) (11) | 304 | cN1-3 | MVAC or GC | 48% (pN0) | – | OS was associated with pN0 and negative margins |
| Population-based study (retrospective) (12) | 659 | cN1 and cN2/3 | NAC vs. RC | 39% (cN1) and 27% (cN2/3) | – | pDS was associated with better OS |
pDS, pathologic downstaging; pCR, pathologic complete response; SWOG, Southwest Oncology Group; MVAC, Methotrexate, Vinblastine, Doxorubicin and Cisplatin; RC, radical cystectomy; dd-MAVAC, dose dense MVAC; GC, Gemcitabine Cisplatin; MSKCC, Memorial Sloan-Kettering Cancer Center; dd-GC, dese dense Gemcitabine Cisplatin; RISC, Retrospective International Study of Cancers of the Urothelial Tract; NCDB, National Cancer Database; NAC, neoadjuvant chemotherapy.