Low-dose hydroxychloroquine is as effective as phlebotomy in treatment of patients with porphyria cutanea tarda.

Abstract

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.

• Authors: Ashwani K Singal, Csilla Kormos-Hallberg, Chul Lee, Vaithamanithi M Sadagoparamanujam, James J Grady, Daniel H Freeman Jr, Karl E Anderson
• Journal: Clinical gastroenterology and hepatology : the official clinical practice journal of the American Gastroenterological Association (Clin Gastroenterol Hepatol) Vol. 10 Issue 12 Pg. 1402-9 (Dec 2012) ISSN: 1542-7714 [Electronic] United States
• PMID: 22985607 (Publication Type: Comparative Study, Journal Article, Randomized Controlled Trial, Research Support, N.I.H., Extramural, Research Support, U.S. Gov't, P.H.S.)
• Copyright: Copyright © 2012 AGA Institute. Published by Elsevier Inc. All rights reserved.

Chemical References

• Enzyme Inhibitors
• Porphyrins
• Hydroxychloroquine

Topics

• Adult
• Aged
• Enzyme Inhibitors (administration & dosage, adverse effects)
• Female
• Humans
• Hydroxychloroquine (administration & dosage, adverse effects)
• Male
• Middle Aged
• Phlebotomy (adverse effects, methods)
• Plasma (chemistry)
• Porphyria Cutanea Tarda (drug therapy, surgery)
• Porphyrins (blood)
• Prospective Studies
• Treatment Outcome