Systemic Sclerosis Therapy with Iloprost: A Prospective Observational Study of 30 Patients Treated for a Median of 3 Years

Abstract:

Iloprost is useful in the short-term treatment of severe Raynaud’s phenomenon and ischaemic ulcers in patients with systemic sclerosis (SSc), but its long-term effects are largely unknown. The aim of this study was to report long-term outcome (median follow-up 36 months) in a prospective observational study of a cohort of 30 consecutive patients with SSc who received iloprost therapy with maintenance infusions every 3 weeks after an initial cycle of 5 consecutive days. At the end of the observation, compared to the pretreatment point, we observed complete healing of digital ulcers in 19/21 patients (90%), a decrease of the Raynaud’s phenomenon visual analogue score from 10/10 (25th–75th percentile 7–10) to 5/10 (4–6.75) (P <0.001) and, in patients with diffuse cutaneous involvement, of the modified Rodnan skin thickness score from 25.5 (16.5–31.5) to 16 (13.5–20) (P= 0.02), minimal improvement of the Health Assessment Questionnaire from 0.87 (0.68–1.37) to 0.75 (0.62–1.25), which was neither statistically nor clinically significant. The forced vital capacity was not significantly changed, but the diffusion capacity corrected for the alveolar volume decreased from 71% (54–76.7) of the expected value to 62% (51.5–71) (P= 0.02). In one patient with limited SSc a positive effect on pulmonary hypertension was observed. Six patients, after a median of 25 months of treatment and healing of digital ulcers, discontinued the therapy; after a median of 10 months ulcers did not recur in five of these six. Other reasons for discontinuation were: tolerability (1), disease progression (normotensive renal crisis: 1), and death due to intracranial haemorrhage (1). This same patient had previously suffered a central retinal vein thrombosis. In conclusion, long-term therapy with iloprost in patients with SSc has a durable effectiveness on ischaemic ulcers and Raynaud’s phenomenon, but it is not possible to conclude that the natural history of the disease was modified.