McMurray J Scott, Connor Nadine, Ford Charles N
Division of Otolaryngology-Head and Neck Surgery, Department of Surgery, University of Wisconsin School of Medicine and Public Health, Madison, Wisconsin 53792, USA.
Ann Otol Rhinol Laryngol. 2008 Jul;117(7):477-83. doi: 10.1177/000348940811700702.
We performed a prospective, double-blind, placebo-controlled, longitudinal adjuvant therapy trial to determine the efficacy of cidofovir in the treatment of severe recurrent respiratory papillomatosis (RRP). Although results of case series suggest that cidofovir may decrease the frequency and rapidity of papilloma regrowth, no blinded placebo-controlled studies have demonstrated efficacy.
Adults and children (n = 19) with aggressive RRP received either active drug (cidofovir) or placebo. When surgical intervention was needed, drug or placebo was injected into affected areas after surgical removal of disease. The following measures were made at baseline and at 2-month intervals for the course of 12 months: Derkay papilloma severity grading scale, Voice Handicap Index, Health-Related Quality of Life, and total number of procedures performed over 12 months.
At 2- and 12-month follow-ups, there was a significant (p < .05) improvement in the Derkay Severity Score within the cidofovir and placebo groups, but no difference between groups, and no difference between groups in the number of procedures performed. Significant improvement was found in Voice Handicap Index scores in the cidofovir group at the 12-month follow-up. No differences were seen in Health-Related Quality of Life.
A randomized, blinded, placebo-controlled trial is necessary in the study of RRP, because the natural history of the disease can include remissions and reactivations. We found a significant improvement in the Derkay Severity Score 12 months after the baseline assessment in patients treated with cidofovir. This effect, however, was also seen in the placebo group. Accordingly, we were unable to provide proof of efficacy of cidofovir in the treatment of RRP.
我们进行了一项前瞻性、双盲、安慰剂对照的纵向辅助治疗试验，以确定西多福韦治疗重度复发性呼吸道乳头状瘤病（RRP）的疗效。尽管病例系列研究结果表明西多福韦可能会降低乳头状瘤再生的频率和速度，但尚无双盲安慰剂对照研究证明其疗效。
19例患有侵袭性RRP的成人和儿童接受活性药物（西多福韦）或安慰剂治疗。当需要手术干预时，在手术切除病变后将药物或安慰剂注入受影响区域。在基线时以及在12个月的疗程中每隔2个月进行以下测量：Derkay乳头状瘤严重程度分级量表、嗓音障碍指数、健康相关生活质量以及12个月内进行的手术总数。
在2个月和12个月的随访中，西多福韦组和安慰剂组的Derkay严重程度评分均有显著改善（p <.05），但两组之间无差异，手术次数也无差异。在12个月的随访中，西多福韦组的嗓音障碍指数评分有显著改善。健康相关生活质量方面未见差异。
在RRP的研究中进行随机、双盲、安慰剂对照试验是必要的，因为该疾病的自然病程可能包括缓解和复发。我们发现接受西多福韦治疗的患者在基线评估12个月后Derkay严重程度评分有显著改善。然而，安慰剂组也出现了这种效果。因此，我们无法提供西多福韦治疗RRP疗效的证据。
