小剂量羟氯喹治疗迟发性皮肤卟啉病与放血疗法同样有效。
Low-dose hydroxychloroquine is as effective as phlebotomy in treatment of patients with porphyria cutanea tarda.
机构信息
Department of Preventive Medicine and Community Health, University of Texas Medical Branch, Galveston, TX, USA.
出版信息
Clin Gastroenterol Hepatol. 2012 Dec;10(12):1402-9. doi: 10.1016/j.cgh.2012.08.038. Epub 2012 Sep 14.
BACKGROUND & AIMS: Porphyria cutanea tarda (PCT) is an iron-related disorder caused by reduced activity of hepatic uroporphyrinogen decarboxylase; it can be treated by phlebotomy or low doses of hydroxychloroquine. We performed a prospective pilot study to compare the efficacy and safety of these therapies.
METHODS
We analyzed data from 48 consecutive patients with well-documented PCT to characterize susceptibility factors; patients were treated with phlebotomy (450 mL, every 2 weeks until they had serum ferritin levels of 20 ng/mL) or low-dose hydroxychloroquine (100 mg orally, twice weekly, until at least 1 month after they had normal plasma levels of porphyrin). We compared the time required to achieve a normal plasma porphyrin concentration (remission, the primary outcome) for 17 patients treated with phlebotomy and 13 treated with hydroxychloroquine.
RESULTS
The time to remission was a median 6.9 months for patients who received phlebotomy and 6.1 months for patients treated with hydroxychloroquine treatment (6.7 and 6.5 mo for randomized patients), a difference that was not significant (log-rank, P = .06 and P = .95, respectively). The sample size was insufficient to confirm noninferiority of hydroxychloroquine treatment (hazard ratio, 2.19; 95% confidence interval, 0.95-5.06) for all patients. Patients who received hydroxychloroquine had substantially better compliance. There were no significant side effects of either treatment.
CONCLUSIONS
Hydroxychloroquine, 100 mg twice weekly, is as effective and safe as phlebotomy in patients with PCT, although noninferiority was not established. Given these results, higher-dose regimens of hydroxychloroquine, which have more side effects, do not seem justified. Compliance was better and projected costs were lower for hydroxychloroquine than phlebotomy treatment. Long-term studies are needed to compare durability of response. ClinicalTrials.gov number, NCT01573754.
背景与目的
迟发性皮肤卟啉病（PCT）是一种与铁相关的疾病，由肝尿卟啉原脱羧酶活性降低引起；该病可以通过放血或低剂量羟氯喹进行治疗。我们进行了一项前瞻性试点研究，比较了这两种治疗方法的疗效和安全性。
方法
我们分析了 48 例经证实的 PCT 患者的数据，以确定易感性因素；患者接受放血治疗（450mL，每两周一次，直到血清铁蛋白水平达到 20ng/mL）或低剂量羟氯喹治疗（100mg 口服，每周两次，至少在血浆卟啉水平正常后 1 个月）。我们比较了 17 例接受放血治疗和 13 例接受羟氯喹治疗的患者达到正常血浆卟啉浓度（缓解，主要结局）所需的时间。
结果
接受放血治疗的患者缓解时间中位数为 6.9 个月，接受羟氯喹治疗的患者缓解时间中位数为 6.1 个月（随机分组患者为 6.7 和 6.5 个月），差异无统计学意义（对数秩检验，P=0.06 和 P=0.95）。由于样本量不足，无法确认羟氯喹治疗（危险比，2.19；95%置信区间，0.95-5.06）对所有患者均具有非劣效性。接受羟氯喹治疗的患者依从性显著提高。两种治疗方法均无明显副作用。
结论
每周两次 100mg 羟氯喹与放血治疗一样有效且安全，尽管未证实羟氯喹非劣效性。鉴于这些结果，更高剂量的羟氯喹方案（副作用更多）似乎没有理由。与放血治疗相比，羟氯喹的依从性更好，预计成本更低。需要进行长期研究以比较两种治疗方案缓解的持久性。临床试验注册号：NCT01573754。
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