Sorafenib is effective in the treatment of pulmonary veno-occlusive disease.

The present study is the first report of the effectiveness of sorafenib in the treatment of pulmonary veno-occlusive disease (PVOD). A 66-year-old woman with PVOD was started on sorafenib. After 3 months of treatment with a maximum dosage of 400 mg/day sorafenib, there was an improvement in the patient's New York Heart Association (NYHA) functional class from IV to III. However, because of severe painful eruptions as a side effect of sorafenib, the patient stopped sorafenib and was started on imatinib instead. This treatment resulted in a worsening of the patient's NYHA class from III to IV, so sorafenib was restarted at a reduced dosage of 300 mg/day. The resumption of sorafenib was associated with clinical improvement, specifically NYHA class from IV to II and hemodynamic amelioration, and tolerable eruptions. In conclusion, sorafenib may be a potential therapeutic strategy for the treatment of PVOD.