Thalidomide for Hereditary Hemorrhagic Telangiectasia With Pulmonary Arterial Hypertension.

initiate thalidomide, which increases platelet-derived growth factor-B expression and downregulates vascular endothelial growth factor in endothelial cells, stimulating mural cell coverage and leading to normal vascular maturation.1,2 After the initiation of thalidomide (50 mg daily), the anemia was dramatically improved without blood transfusion (Figure H) and telangiectatic lesions in the tongue were no longer notable (Figure A-2). Although intensive therapy with pulmonary vasodilators might induce bleeding, we were able to add tadalafil 10 mg daily and increase ambrisentan to 7.5 mg daily without any bleeding side-effects. Twelve months after thalidomide treatment, right heart failure developed with deteriorating pulmonary hemodynamics (mean PAP, 90 mmHg; cardiac index, 1.88 L/min/m2; Table, January 2014). Wedged distal pulmonary angiography showed markedly decreased peripheral vessels (Figure I-2) as compared with that before thalidomide therapy (Figure I-1). After the discontinuation of thalidomide, the bleeding recurred and PAH did not improve, as reflected by serial changes in B-type natriuretic peptide and tricuspid regurgitation pressure gradient (Figure G,H). Finally, the patient died due to right heart failure. Thalidomide was beneficial against mucocutaneous bleeding,1–7 but careful consideration is required with regard to its initiation in HHT patients with PAH.