Oral Treprostinil Improves Peripheral Vascular Function in Scleroderma

Shah AA, Schiopu E, Hummers LK, et al., Open label study of escalating doses of oral treprostinil diethanolamine in patients with systemic sclerosis and digital ischemia: pharmacokinetics and correlation with digital perfusion. Arthritis Research & Therapy (2013) 15(2):R54    [Epub ahead of print]

A phase I pharmacokinetic clinical trial shows that escalating doses of oral treprostinil diethanolamine temporarily improve cutaneous perfusion and temperature in scleroderma patients with peripheral vascular disease and digital ulcers.

The dual-center, open-label trial among 19 scleroderma patients with digital ulcers (mostly Caucasian women, mean age 48, half with limited cutaneous scleroderma), demonstrates that gradually increasing doses of treprostinil diethanolamine improve skin perfusion over 8 weeks of treatment.

The drug under investigation is a new salt form of the prostacyclin analogue treprostinil sodium in an oral sustained release (SR) osmotic tablet, meant to be taken with a meal. An injectable form of treprostinil is approved for treatment of pulmonary arterial hypertension. IV prostacyclin and ischemic pain, and promote healing of digital ulcers.

This is the first study to show that cutaneous perfusion (as seen on laser Doppler imaging) may improve with an oral prostacyclin formulation, with a safety profile similar to that of the infusion.