Low-Dose Hydroxychloroquine Is as Effective as Phlebotomy in Treatment of Patients With Porphyria Cutanea Tarda

Ashwani K. Singal, Csilla Kormos-Hallberg, Chul Lee, V-M Ramanujam, James J. Grady, Daniel H. Freeman, Karl Anderson

Research output: Contribution to journalArticle

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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 andP= .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. number,NCT01573754

Original languageEnglish (US)
Pages (from-to)1402-1409
Number of pages8
JournalClinical Gastroenterology and Hepatology
Volume10
Issue number12
DOIs
StatePublished - Dec 2012

Fingerprint

Porphyria Cutanea Tarda
Hydroxychloroquine
Phlebotomy
Therapeutics
Porphyrins
Compliance
Uroporphyrinogen Decarboxylase
Ferritins
Sample Size
Iron
Prospective Studies
Confidence Intervals
Safety
Costs and Cost Analysis

Keywords

  • 4-Aminoquinolines
  • Clinical Trial
  • Drug
  • Liver Disease

ASJC Scopus subject areas

  • Gastroenterology
  • Hepatology

Cite this

Low-Dose Hydroxychloroquine Is as Effective as Phlebotomy in Treatment of Patients With Porphyria Cutanea Tarda. / Singal, Ashwani K.; Kormos-Hallberg, Csilla; Lee, Chul; Ramanujam, V-M; Grady, James J.; Freeman, Daniel H.; Anderson, Karl.

In: Clinical Gastroenterology and Hepatology, Vol. 10, No. 12, 12.2012, p. 1402-1409.

Research output: Contribution to journalArticle

Singal, Ashwani K. ; Kormos-Hallberg, Csilla ; Lee, Chul ; Ramanujam, V-M ; Grady, James J. ; Freeman, Daniel H. ; Anderson, Karl. / Low-Dose Hydroxychloroquine Is as Effective as Phlebotomy in Treatment of Patients With Porphyria Cutanea Tarda. In: Clinical Gastroenterology and Hepatology. 2012 ; Vol. 10, No. 12. pp. 1402-1409.
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