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A Systematic Review and Meta-Analysis of Retrospective Series of Regorafenib for Treatment of Metastatic Colorectal Cancer JOEY MERCIER 1 and IOANNIS A

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A Systematic Review and Meta-Analysis of Retrospective Series of Regorafenib for Treatment of Metastatic Colorectal Cancer JOEY MERCIER 1 and IOANNIS A ANTICANCER RESEARCH 37 : 5925-5934 (2017) doi:10.21873/anticanres.12039 Review A Systematic Review and Meta-analysis of Retrospective Series of Regorafenib for Treatment of Metastatic Colorectal Cancer JOEY MERCIER 1 and IOANNIS A. VOUTSADAKIS 1,2,3 1Northern Ontario School of Medicine, 2Division of Clinical Sciences, Sudbury, ON, Canada; 3Algoma District Cancer Program, Sault Area Hospital, Sault Ste Marie, ON, Canada Abstract. Background: Metastatic colorectal cancer is a IIIb studies. Most common adverse effects were also common disease encountered in oncology practice and consistent with those of the published phase III experience. treatment options beyond fluoropyrimidines, irinotecan, Conclusion: This systematic review and meta-analysis oxaliplatin and monoclonal antibodies against epidermal confirmed a moderate efficacy of regorafenib in later-stage growth factor receptor and vascular endothelium growth metastatic colorectal cancer in the everyday clinical practice factor (VEGF) are limited. Regorafenib, a new drug that setting outside of clinical trials. Future identification of targets tyrosine kinases such as VEGF receptor as well as biomarkers may aid in further tailoring of this treatment in others, has been added recently to the armamentarium for order to obtain maximum clinical benefit. metastatic colorectal cancer. This report analyzes the published experience with this drug in clinical practice Colorectal cancer is the most common gastrointestinal outside of clinical trials. Materials and Methods: A literature malignancy in the Western world and remains a prominent search of major databases was performed for the cause of cancer morbidity and mortality, despite progress in identification of studies of regorafenib in metastatic its management. It affects approximately 746,000 men and colorectal cancer. Studies retained for further analysis were 614,000 women yearly and it is the 3rd most common cancer in English or French, describing 20 or more patients treated in the former and the 2nd most common in the latter (1). with regorafenib monotherapy and not part of a phase I, II About one fourth of newly-diagnosed colorectal cancers are or III trial. Results of the pooled analysis of retrospective already in a metastatic stage and of those that are diagnosed studies were compared with results of the published phase III in a localized stage nearly 50% will go on to develop trials and a phase IIIb prospective study. Results: Twelve metastatic disease, in most cases becoming unresectable. publications including a total of 702 patients were included Metastatic colorectal cancer treatment options have been in the meta-analysis. Summary response rate was 2% [95% broadened to include regimens of oxaliplatin or irinotecan confidence interval (CI) =0.8-3.2%] and the disease control added to a fluoropyrimidine backbone, as well as targeted rate 38.14% (95% CI=32.35-43.93%). Summary survival treatments with bevacizumab and, for KRAS wild-type rates were 3.34 months (95% CI=2.71-3.97 months) for tumors, cetuximab or panitumumab (2-4). These treatments progression-free and 7.27 months (95% CI=6.23-8.3 months) have extended the median survival of patients with for overall survival. These were similar to the phase III and metastatic colorectal cancer to over 2 years. Nevertheless, the disease remains incurable in most patients and when the above drug options have been exhausted, there is a paucity of other options. This therapeutic vacuum has been partially This article is freely accessible online. filled recently with the approval of the tyrosine kinase inhibitor regorafenib, a fluorinated derivative of sorafenib, Correspondence to: Ioannis A. Voutsadakis, MD, Ph.D., Algoma for the third line treatment of metastatic colorectal cancer District Cancer Program, Sault Area Hospital, 750 Great Northern patients (5). Road, Sault Ste Marie, ON P6B 0A8, Canada. E-mail: [email protected], [email protected] Regorafenib (formerly known as BAY 73-4506; chemical formula: C 21 H17 CIF 4N4O4) is a small molecule multi-kinase Key Words: Colorectal cancer, metastatic, regorafenib, VEGFR, inhibitor of fms-related tyrosine kinase 1 (FLT1, also known inhibition, retrospective series. as vascular endothelial growth factor receptor 1, VEGFR1), 5925 ANTICANCER RESEARCH 37 : 5925-5934 (2017) kinase insert domain receptor (KDR, also known as by a second author to ensure accuracy. Discrepancies were VEGFR2), FLT4 (VEGFR3), TEK receptor tyrosine kinase, discussed and reconciled cooperatively. Demographic characteristics KIT proto-oncogene receptor tyrosine kinase, Raf-1 proto- of the patients treated with regorafenib recorded for the current pooled analysis consisted of the age of the patients, Eastern oncogene, serine/threonine kinase, v-RAF murine sarcoma Cooperative Oncology Group performance status (ECOG PS), viral oncogene homolog B (BRAF) and the commonly number and type of previous lines of treatment for metastatic V600E mutated BRAF variant, platelet-derived growth factor disease, number and site of organs involved by metastatic cancer, receptor (PDGFR) and fibroblast growth factor receptor location of the primary site (colon or rectum) and mutation status (FGFR) (6). It is orally available given at a dose of 160 mg of KRAS . Response rate (RR), defined as the sum of complete (CR) daily in an intermittent schedule of 3 weeks out of 4. and partial (PR) responses, disease control rate (DCR), defined as Regorafenib was approved in 2012 by the Food and Drug the sum of RR and stable disease (SD), median PFS and median OS were efficacy outcomes of interest and were extracted from the Administration as monotherapy for the treatment of included publications. Toxicity of all grades, as well as grade 3 and metastatic colorectal cancer refractory to fluoropyrimidine, 4, were also outcomes of interest for this pooled analysis and where oxaliplatin and irinotecan-based chemotherapy, anti-VEGF thus recorded from the published articles when available. A therapy, and, if KRAS wild-type, anti-epidermal growth comparison was made with the respective pooled data of the factor receptor (EGFR) therapy (7). The approval was based regorafenib arms from the two published randomized phase III on its efficacy compared to best supportive care in a phase studies of the drug in metastatic colorectal cancer (8, 9) to III study that showed prolongation of progression-free determine whether the populations treated in phase III trials were similar to those reported off trial and whether the drug had a similar survival (PFS) by 0.2 months and prolongation of overall efficacy and toxicity profile when used in a trial setting to that in survival (OS) by 1.4 months (8). An additional phase III trial everyday practice. An additional comparison was made with the confirmed these results, showing improvement of PFS by 1.5 population and outcomes of a published prospective registry of months and of OS by 2.5 months (9). Moreover, a regorafenib treatment (10). prospective series analyzing patients participating in a Descriptive statistics were computed for both the characteristics compassionate program showed a median PFS of 2.7 months of interest and the outcome measures. Some studies included in the and median OS of 5.6 months (10). Several small series have pooled analysis did not provide complete population data, and, in those instances, the means and confidence intervals (CIs) were now been published describing the experience of individual calculated using only the number of patients from the studies that centers or national programs with the drug in every day included the data of interest. The number of series from which each practice, all of which are off study. This current report sought outcome of interest was derived was determined and presented on to pool these studies and analyze the efficacy and toxicity of each occasion. Heterogeneity amongst studies was evaluated with regorafenib in daily practice, outside of clinical trials and to Cochran’s Q and I 2 tests. The fixed-effect model was used when compare the efficacy and toxicity observed in this setting between-study heterogeneity was low. Alternatively, when with the trial experience. heterogeneity was moderate or high, a random-effect model was used for calculation of the pooled summary statistic (13). Materials and Methods Calculations were performed in Excel (Microsoft Corp., Redmond, WA, USA) based on a previously described method with modifications as needed (14). Medline/PubMed and Embase databases were searched in order to identify published articles on regorafenib treatment for metastatic colorectal cancer. A search of the grey literature, in accordance with Results Cochrane’s MECIR standards, was also performed to locate any unpublished studies on regorafenib (11). The search used the terms Two hundred and thirty-eight publications were initially “regorafenib” and “metastatic colorectal cancer”. Inclusion criteria retrieved (Figure 1). Thirty-three studies were preclinical and were publication in English or French language, describing clinical were excluded. An additional 27 studies were excluded experience in series of 20 or more patients evaluable for efficacy or because they were not in English (none would have been toxicity, not being part of a phase I, II or III trial, and receiving included even if in English based on the abstract). From the regorafenib as a monotherapy. Publications in other languages or available only in abstract form were excluded. Also excluded were remaining 178 clinical reports,
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