Hong W K, Endicott J, Itri L M, Doos W, Batsakis J G, Bell R, Fofonoff S, Byers R, Atkinson E N, Vaughan C
N Engl J Med. 1986 Dec 11;315(24):1501-5. doi: 10.1056/NEJM198612113152401.
13-cis-Retinoic acid has been reported to be effective in treating oral leukoplakia. We randomly assigned 44 patients with this disease to receive 13-cis-retinoic acid (24 patients) or placebo (20), 1 to 2 mg per kilogram of body weight per day for three months, and followed them for six months. There were major decreases in the size of the lesions in 67 percent (16 patients) of those given the drug and in 10 percent (2 patients) of those given placebo (P = 0.0002); dysplasia was reversed in 54 percent (13 patients) of the drug group and in 10 percent (2 patients) of the placebo group (P = 0.01). The clinical response to the drug correlated with the histologic response in 56 percent (9 of 16) of the patients evaluated. Relapse occurred in 9 of 16 patients two to three months after treatment ended. The toxic effects of the drug were acceptable in all but two patients. Cheilitis, facial erythema, and dryness and peeling of the skin were common; conjunctivitis and hypertriglyceridemia also occurred. All adverse reactions could be reversed by reducing the dose or temporarily discontinuing the drug. We conclude that 13-cis-retinoic acid, even in short-term use, appears to be an effective treatment for oral leukoplakia and has an acceptable level of toxicity.
据报道，13 - 顺式维甲酸对治疗口腔白斑病有效。我们将44例该病患者随机分为两组，分别给予13 - 顺式维甲酸（24例）或安慰剂（20例），每日每千克体重1至2毫克，持续三个月，并对他们进行了六个月的随访。服用药物的患者中67%（16例）的病损大小有显著减小，而服用安慰剂的患者中这一比例为10%（2例）（P = 0.0002）；药物组中54%（13例）的发育异常得到逆转，安慰剂组中这一比例为10%（2例）（P = 0.01）。在接受评估的患者中，56%（16例中的9例）对药物的临床反应与组织学反应相关。16例患者中有9例在治疗结束后两到三个月复发。除两名患者外，该药的毒性作用均可接受。唇炎、面部红斑以及皮肤干燥和脱屑很常见；还出现了结膜炎和高甘油三酯血症。所有不良反应均可通过减少剂量或暂时停药得到逆转。我们得出结论，即使短期使用，13 - 顺式维甲酸似乎也是治疗口腔白斑病的有效药物，且毒性水平可接受。
