A novel iloprost administration method with portable syringe pump for the treatment of acral ulcers and Raynaud's phenomenon in systemic sclerosis patients. A pilot study (ILOPORTA).

OBJECTIVES: The objective of the study is to evaluate the feasibility, safety and efficacy of home infusion of iloprost with the new portable syringe pump Infonde®, for the treatment of scleroderma-related Raynaud's phenomenon and digital ulcers. METHODS: 12 scleroderma patients were treated with iloprost at home, using the pump, with infusion cycles of 2 days per month (24 hours a day), for 6 months. RESULTS: The home treatment proved feasible since ten patients (83%) completed the entire infusion cycle, thus satisfying the feasibility target imposed by the protocol (75%). Side effects related to the device or venous access occurred in 3 out of 65 total 48-hour infusions (4.6%). They mostly consisted in phlebitis. No adverse events related to the device management were reported. Among the ten patients who completed the infusions, three showed a reduction in the number of ulcers, three maintained the same number, and four had no ulcers throughout the observation period. Patient's perception of their quality of life and wellness during home infusions, expressed with the Visual Analogue Scale (VAS) improved from 79/100 at the first infusion to 91/100 at the end of the study. All patients expressed a positive global judgment regarding this innovative method of iloprost infusion. CONCLUSIONS: The infusion of iloprost at home with Infonde® is feasible, safe and effective. Moreover, this approach presents potential advantages from the economic and organisational point of view. Because of the pilot design of our study, these results need to be confirmed in larger randomised trials.

Idiopathic hypereosinophilic syndrome presenting with severe vasculitis successfully treated with imatinib.

Idiopathic hypereosinophilic syndrome (HES) is a rare disorder characterized by peripheral eosinophilia exceeding 1500/mm3, a chronic course, absence of secondary causes, and signs and symptoms of eosinophil-mediated tissue injury. One of the best-characterized forms of HES is the one associated with FIP1L1-PDGFRA gene rearrangement, which was recently demonstrated as responsive to treatment with the small molecule kinase inhibitor drug, imatinib mesylate. Here, we describe the case of a 51-year-old male, whose symptoms satisfied the clinical criteria for HES with cutaneous and cardiac involvement and who also presented with vasculitic brain lesions and retroperitoneal bleeding. Molecular testing, including fluorescence in situ hybridization, of bone marrow and peripheral blood showed no evidence of PDGFR rearrangements. The patient was initially treated with high-dose steroid therapy and then with hydroxyurea, but proved unresponsive to both. Upon subsequent initiation of imatinib mesilate, the patient showed a dramatic improvement in eosinophil count and progressed rapidly through clinical recovery. Long-term follow-up confirmed the efficacy of treatment with low-dose imatinib and with no need of supplemental steroid treatment, notwithstanding the absence of PDGFR rearrangement.