INFLUENCE OF DISEASE MANIFESTATION AND ANTINEUTROPHIL CYTOPLASMIC ANTIBODY TITER ON THE RESPONSE TO PULSE CYCLOPHOSPHAMIDE THERAPY IN PATIENTS WITH WEGENERS GRANULOMATOSIS

Objective. To assess the effectiveness of pulse cyclophosphamide (CYC) in the treatment of Wegener's granulomatosis (WG) and to identify the patients who are responsive to the treatment. Methods. The prospectiv e study included 43 patients with biopsy-proven WG. Clinical, radiogra phic, laboratory, and immunologic data were evaluated for predicitive values regarding the outcome of pulse CYC therapy. Results. Only 42% o f the patients showed complete or partial remission that lasted at lea st 6 months after cessation of pulse CYC therapy. These responders had a higher frequency of disease activity limited to the upper and lower respiratory tract (39%, versus 8% in the nonresponder group; P < 0.05 ) and had lower titers of classic antineutrophil cytoplasmic antibody (cANCA) prior to treatment (<1:64 42%, versus 6% in the nonresponder g roup; P < 0.05). In the 58% of patients who did not respond to pulse C YC treatment, there was both systemic disease involving more than 4 or gan systems (mainly, the heart, nervous system, eye, and skin) and con stitutional symptoms. Serious side effects induced by pulse CYC occurr ed in only 1 patient. Conclusion. Based on these findings, pulse CYC t herapy appears to be effective in WG patients with moderate disease ac tivity and low titers of cANCA, but of little benefit in patients with severe WG. Pulse CYC should therefore not be used as first-line thera py ill patients with severe and rapidly progressing forms of WG associ ated with high titers of cANCA.