title: Cabazitaxel in patients with metastatic castration-resistant prostate cancer: results of a compassionate use program in the Netherlands.
abstract: BACKGROUND
  Cabazitaxel has been reimbursed as a second-line therapy for patients with metastatic castrate-resistant prostate cancer (mCRPC) in the Netherlands since 2011. Before reimbursement was available, cabazitaxel was provided through a Compassionate Use Program (CUP). We report the results of the Dutch CUP, detailing the safety and efficacy of cabazitaxel in a routine clinical practice setting.
  
  
  METHODS
  Safety and efficacy data of all 5 Dutch centers participating in the cabazitaxel CUP were collected. Safety data were collected prospectively using the National Cancer Institute Common Toxicity Criteria for Adverse Events, version 3.0. Overall survival (OS) and progression-free survival (PFS), time to PSA progression (TTPP), and best clinical response were evaluated retrospectively.
  
  
  RESULTS
  Fifty-one patients were registered in the CUP; 49 received cabazitaxel. Forty-two of 49 patients [85.7%], 42 patients had ≥ 2 metastatic sites. Patients received on average 6 cabazitaxel cycles (range, 1-21). A dose reduction or dose delay occurred in 13 and 20 patients [26.5% and 40.9%] respectively. Prophylactic granulocyte colony-stimulating factor (G-CSF) was used in 8 patients [16.3%]. Grade ≥ 3 adverse events were observed in 25 patients [51.0%]; 16 patients [32.7%] discontinued treatment because of treatment-emergent adverse events (TEAEs). Serious adverse events (SAEs) occurred in 16 (32.7%) patients; the most frequent SAEs were hematuria (4 patients [8.3%]) and urosepsis (3 patients [6.3%]). Febrile neutropenia occurred twice; no patient had grade ≥ 3 neuropathy. No toxicity-related mortality occurred. Median follow-up was 24.1 months. Median OS was 8.7 months (interquartile range [IQR], 6.0-15.9 months); median TTPP was 2.8 months (IQR, 1.7-5.9 months).
  
  
  CONCLUSIONS
  In the Dutch CUP, patients with advanced mCRPC had delayed tumor progression with acceptable toxicities using cabazitaxel treatment.
authors: Department of Medical Oncology, Leiden University Medical Center, Leiden, the Netherlands.
authors: Michel D
authors: MD
authors: Wissing
authors: Inge M
authors: IM
authors: van Oort
authors: Winald R
authors: WR
authors: Gerritsen
authors: Alfons J M
authors: AJ
authors: van den Eertwegh
authors: Jules L L M
authors: JL
authors: Coenen
authors: Andries M
authors: AM
authors: Bergman
authors: Hans
authors: H
authors: Gelderblom
mesh_terms: D000368:Aged; D057176:Compassionate Use Trials; D018572:Disease-Free Survival; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D006801:Humans; D008297:Male; D008875:Middle Aged; D009362:Neoplasm Metastasis / Q000188:drug therapy; D009426:Netherlands; D017430:Prostate-Specific Antigen / Q000097:blood; D064129:Prostatic Neoplasms, Castration-Resistant / Q000188:drug therapy* / Q000401:mortality*; D012189:Retrospective Studies; D013534:Survival; D043823:Taxoids / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D043823:Taxoids; D016179:Granulocyte Colony-Stimulating Factor; C552428:cabazitaxel; D017430:Prostate-Specific Antigen
keywords: Cabazitaxel; Compassionate Use Program; Metastatic castrate-resistant prostate cancer; Taxane; The Netherlands