Real-World, Observational, Retrospective Study to Evaluate the Effectiveness and Safety of Treatment with Sorafenib in Patients with Advanced Hepatocellular Carcinoma
Background: Hepatocellular carcinoma (HCC) accounts for approximately 90% of liver cancer cases. Sorafenib, the first drug to demonstrate survival benefits for advanced HCC, was validated through the SHARP randomized clinical trial (RCT). While RCTs are essential for assessing new therapies, real-wo...
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MDPI AG
2024-11-01
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| Series: | Current Oncology |
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| Online Access: | https://www.mdpi.com/1718-7729/31/11/500 |
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| author | Angélica Richart Csipak Leonardo G. da Fonseca Rossana Verónica Mendoza López Maria Del Pilar Estevez-Diz |
| author_facet | Angélica Richart Csipak Leonardo G. da Fonseca Rossana Verónica Mendoza López Maria Del Pilar Estevez-Diz |
| author_sort | Angélica Richart Csipak |
| collection | DOAJ |
| description | Background: Hepatocellular carcinoma (HCC) accounts for approximately 90% of liver cancer cases. Sorafenib, the first drug to demonstrate survival benefits for advanced HCC, was validated through the SHARP randomized clinical trial (RCT). While RCTs are essential for assessing new therapies, real-world studies provide additional insights into their effectiveness in routine clinical practice. This study aimed to evaluate sorafenib’s real-world effectiveness by analyzing overall survival (OS) and the time to radiological and symptomatic progression. Methods: Data from 368 patients treated with sorafenib at a Brazilian Cancer Center between 2009 and 2020 were retrospectively reviewed. Results: The median OS was 9.6 months, and the time to radiological progression was 5.3 months, similar to the SHARP trial. However, the time to symptomatic progression was shorter (2.3 months) than the SHARP study (4.1 months). In terms of safety, 27.4% of patients presented clinically relevant toxicities, and 24.5% needed to discontinue treatment due to toxicity. Conclusions: Overall, sorafenib demonstrated effectiveness in the studied population, with OS and radiological progression times comparable to SHARP study results. The difference in symptomatic progression may be due to the study’s retrospective nature and limitations. |
| format | Article |
| id | doaj-art-fe460e021d524f749f9af28c95d9ab33 |
| institution | OA Journals |
| issn | 1198-0052 1718-7729 |
| language | English |
| publishDate | 2024-11-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Current Oncology |
| spelling | doaj-art-fe460e021d524f749f9af28c95d9ab332025-08-20T02:28:04ZengMDPI AGCurrent Oncology1198-00521718-77292024-11-0131116778679010.3390/curroncol31110500Real-World, Observational, Retrospective Study to Evaluate the Effectiveness and Safety of Treatment with Sorafenib in Patients with Advanced Hepatocellular CarcinomaAngélica Richart Csipak0Leonardo G. da Fonseca1Rossana Verónica Mendoza López2Maria Del Pilar Estevez-Diz3Oncology, Instituto do Cancer do Estado de Sao Paulo, Faculdade de Medicina da Universidade de Sao Paulo, São Paulo 01246-000, BrazilOncology, Instituto do Cancer do Estado de Sao Paulo, Faculdade de Medicina da Universidade de Sao Paulo, São Paulo 01246-000, BrazilComprehensive Center for Precision Oncology C2PO, Center for Translational Research in Oncology, Instituto do Cancer do Estado de Sao Paulo, Faculdade de Medicina da Universidade de Sao Paulo, São Paulo 01246-000, BrazilOncology, Instituto do Cancer do Estado de Sao Paulo, Faculdade de Medicina da Universidade de Sao Paulo, São Paulo 01246-000, BrazilBackground: Hepatocellular carcinoma (HCC) accounts for approximately 90% of liver cancer cases. Sorafenib, the first drug to demonstrate survival benefits for advanced HCC, was validated through the SHARP randomized clinical trial (RCT). While RCTs are essential for assessing new therapies, real-world studies provide additional insights into their effectiveness in routine clinical practice. This study aimed to evaluate sorafenib’s real-world effectiveness by analyzing overall survival (OS) and the time to radiological and symptomatic progression. Methods: Data from 368 patients treated with sorafenib at a Brazilian Cancer Center between 2009 and 2020 were retrospectively reviewed. Results: The median OS was 9.6 months, and the time to radiological progression was 5.3 months, similar to the SHARP trial. However, the time to symptomatic progression was shorter (2.3 months) than the SHARP study (4.1 months). In terms of safety, 27.4% of patients presented clinically relevant toxicities, and 24.5% needed to discontinue treatment due to toxicity. Conclusions: Overall, sorafenib demonstrated effectiveness in the studied population, with OS and radiological progression times comparable to SHARP study results. The difference in symptomatic progression may be due to the study’s retrospective nature and limitations.https://www.mdpi.com/1718-7729/31/11/500advanced hepatocellular carcinomasorafenibreal-worldsurvivaleffectiveness |
| spellingShingle | Angélica Richart Csipak Leonardo G. da Fonseca Rossana Verónica Mendoza López Maria Del Pilar Estevez-Diz Real-World, Observational, Retrospective Study to Evaluate the Effectiveness and Safety of Treatment with Sorafenib in Patients with Advanced Hepatocellular Carcinoma Current Oncology advanced hepatocellular carcinoma sorafenib real-world survival effectiveness |
| title | Real-World, Observational, Retrospective Study to Evaluate the Effectiveness and Safety of Treatment with Sorafenib in Patients with Advanced Hepatocellular Carcinoma |
| title_full | Real-World, Observational, Retrospective Study to Evaluate the Effectiveness and Safety of Treatment with Sorafenib in Patients with Advanced Hepatocellular Carcinoma |
| title_fullStr | Real-World, Observational, Retrospective Study to Evaluate the Effectiveness and Safety of Treatment with Sorafenib in Patients with Advanced Hepatocellular Carcinoma |
| title_full_unstemmed | Real-World, Observational, Retrospective Study to Evaluate the Effectiveness and Safety of Treatment with Sorafenib in Patients with Advanced Hepatocellular Carcinoma |
| title_short | Real-World, Observational, Retrospective Study to Evaluate the Effectiveness and Safety of Treatment with Sorafenib in Patients with Advanced Hepatocellular Carcinoma |
| title_sort | real world observational retrospective study to evaluate the effectiveness and safety of treatment with sorafenib in patients with advanced hepatocellular carcinoma |
| topic | advanced hepatocellular carcinoma sorafenib real-world survival effectiveness |
| url | https://www.mdpi.com/1718-7729/31/11/500 |
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