Sorafenib in patients with progressive epithelioid hemangioendothelioma: a phase 2 study by the French Sarcoma Group (GSF/GETO).

Abstract

BACKGROUND:

There is no standard treatment for progressive epithelioid hemangioendothelioma (EHE). To investigate the significant vascularization of EHE, the activity/toxicity of sorafenib in patients with progressive EHE was explored.

METHODS:

In this multicenter, 1-stage, phase 2 trial of sorafenib (800 mg daily), the primary endpoint, which was chosen by default, was the 9-month progression-free rate. All patients had documented progressive disease at the time of study entry.

RESULTS:

Fifteen patients were enrolled between June 2009 and February 2011. The median age was 57 years (range, 31-76 years), and the ratio of men to women was 9:6. The performance status was zero in 10 patients and 1 in 5 patients. Twelve patients had metastases, mainly in the lung (12 patients), liver (5 patients), and bone (3 patients). Five patients had received prior chemotherapy (doxorubicin in 5 patients and taxane in 3 patients). The median sorafenib treatment duration was 124 days (range, from 27 to >271 days). Seven patients required dose reductions or transient treatment discontinuation. The 9-month progression-free rate was 30.7% (4 of 13 patients). The 2-month, 4-month, and 6-month progression-free rate was 84.6% (11 of 13 patients), 46.4% (6 of 13 patients), and 38.4% (5 of 13 patients), respectively. Two partial responses were observed that lasted 2 months and 9 months.

CONCLUSIONS:

Further clinical trials exploring sorafenib as treatment of progressive EHE are needed.

• Authors: Christine Chevreau, Axel Le Cesne, Isabelle Ray-Coquard, Antoine Italiano, Angela Cioffi, Nicolas Isambert, Yves Marie Robin, Charles Fournier, Stéphanie Clisant, Loic Chaigneau, Jacques-Olivier Bay, Emmanuelle Bompas, Eric Gauthier, Jean Y Blay, Nicolas Penel
• Journal: Cancer (Cancer) Vol. 119 Issue 14 Pg. 2639-44 (Jul 15 2013) ISSN: 1097-0142 [Electronic] United States
• PMID: 23589078 (Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
• Copyright: © 2013 American Cancer Society.

Chemical References

• Antineoplastic Agents
• Phenylurea Compounds
• Protein Kinase Inhibitors
• Niacinamide
• Sorafenib

Topics

• Adult
• Aged
• Antineoplastic Agents (administration & dosage, adverse effects, therapeutic use)
• Disease Progression
• Disease-Free Survival
• Drug Administration Schedule
• Female
• France
• Head and Neck Neoplasms (drug therapy)
• Hemangioendothelioma, Epithelioid (drug therapy, secondary)
• Humans
• Liver Neoplasms (drug therapy)
• Male
• Middle Aged
• Niacinamide (administration & dosage, adverse effects, analogs & derivatives, therapeutic use)
• Phenylurea Compounds (administration & dosage, adverse effects, therapeutic use)
• Protein Kinase Inhibitors (therapeutic use)
• Rare Diseases
• Sorafenib
• Time Factors
• Treatment Outcome