Use of defibrotide in the treatment of Raynaud's phenomenon associated with progressive systemic sclerosis or essential mixed cryoglobulinemia
Ranieri, G.; De Mitrio, V.; Petronelli, M.; Ancona, A.; Dammacco, F.
La Clinica Terapeutica 126(5): 335-343
1988
ISSN/ISBN: 0009-9074 PMID: 2973963 Document Number: 318204
Defibrotide, a polydeoxyribonucleotide extracted from mammalian organs with enhancing properties on natural fibrinolysis and prostacyclin production from vascular wall, was evaluated in 17 patients with Raynaud's phenomenon, 8 of whom with progressive systemic sclerosis and 9 with essential mixed cryoglobulinemia. Defibrotide was administered as follows: 1200 mg daily were infused i.v. in 2 hours for 7 days and then 200 mg b.i.d. were injected i.m. for 20 days. The frequency and severity of Raynaud's attacks were monitored, during the four weeks of treatment, by the use of in-clinic questionnaires and patients' daily diaries. A 'cold stress test' was performed, before and after treatment, in all patients whereas thermographic studies of the hands were carried out in three subjects only. Moreover, many routine, blood coagulation and fibronolysis tests were performed in all patients to evaluate the drugs mechanism of action or any biological side effects. Altogether clinical subjective and objective evaluation demonstrated a response in 12 out of 17 patients. One possible mechanism of action might be a remarkable activation of the blood fibrinolytic system, as demonstrated by the improvement of dilute clot lysis time (TLCD) and of euglobulin lysis time (TLE). An alternative explanation might be the selective stimulation of vascular prostacyclin. We conclude that defibrotide might represent a new and interesting approach to the pharmacologic management of Raynaud's phenomenon.