Reinhold-Keller E, Kekow J, Schnabel A, Schmitt W H, Heller M, Beigel A, Duncker G, Gross W L
Medizinische Universität Lübeck, Abteilung Klinische Rheumatologie, Bad Bramstedt, Germany.
Arthritis Rheum. 1994 Jun;37(6):919-24. doi: 10.1002/art.1780370622.
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.
The prospective study included 43 patients with biopsy-proven WG. Clinical, radiographic, laboratory, and immunologic data were evaluated for predictive values regarding the outcome of pulse CYC therapy.
Only 42% of the patients showed complete or partial remission that lasted at least 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 group; P < 0.05). In the 58% of patients who did not respond to pulse CYC treatment, there was both systemic disease involving more than 4 organ systems (mainly, the heart, nervous system, eye, and skin) and constitutional symptoms. Serious side effects induced by pulse CYC occurred in only 1 patient.
Based on these findings, pulse CYC therapy appears to be effective in WG patients with moderate disease activity and low titers of cANCA, but of little benefit in patients with severe WG. Pulse CYC should therefore not be used as first-line therapy in patients with severe and rapidly progressing forms of WG associated with high titers of cANCA.
评估脉冲环磷酰胺（CYC）治疗韦格纳肉芽肿（WG）的有效性，并确定对该治疗有反应的患者。
这项前瞻性研究纳入了43例经活检证实为WG的患者。对临床、影像学、实验室和免疫学数据进行评估，以确定其对脉冲CYC治疗结果的预测价值。
仅42%的患者在脉冲CYC治疗停止后显示完全或部分缓解，且持续至少6个月。这些有反应者疾病活动局限于上、下呼吸道的频率更高（39%，无反应组为8%；P<0.05），且治疗前经典抗中性粒细胞胞浆抗体（cANCA）滴度较低（<1:64，42%，无反应组为6%；P<0.05）。在58%对脉冲CYC治疗无反应的患者中，存在累及4个以上器官系统的全身性疾病（主要为心脏、神经系统、眼睛和皮肤）以及全身症状。脉冲CYC引起的严重副作用仅发生在1例患者中。
基于这些发现，脉冲CYC疗法似乎对疾病活动度中等且cANCA滴度低的WG患者有效，但对重症WG患者益处不大。因此，对于伴有高滴度cANCA的重症且快速进展型WG患者，脉冲CYC不应作为一线治疗方法。
