Hydroxychloroquine versus phlebotomy in the treatment of porphyria cutanea tarda

Cainelli, T.; Padova, C. Di; Marchesi, L.; Gori, Giulia; Rovagnati, P; Podenzani, S; Bessone, E; Cantoni, L

doi:10.1111/j.1365-2133.1983.tb01062.x

Cited by 32 publications

(17 citation statements)

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“…Clinical improvement of skin fragility with antimalarial treatment typically takes 4–6 months, and complete remission can be achieved in 10–12 months, similarly to phlebotomy . Remission typically lasts 17–24 months, although complete remission is possible .…”

Section: Porphyria Cutanea Tardamentioning

confidence: 61%

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Cutaneous porphyrias part II: treatment strategies

et al. 2013

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The porphyrias are diverse in pathophysiology, clinical presentation, severity, and prognosis, presenting a diagnostic and therapeutic challenge. Although not easily curable, the dermatological manifestations of these diseases, photosensitivity and associated cutaneous pathology, can be effectively prevented and managed. Sun avoidance is essential, and patient education regarding the irreversibility of photocutaneous damage is a necessary corollary. Beyond preventative measures, the care of fragile, vulnerable skin, and pain management, each of the porphyrias has a limited number of unique additional therapeutic options. Many of the treatments have been published only in small case series or anecdotal reports and do not have well-understood nor proven mechanisms of action. This article presents a comprehensive review of available therapeutic options and long-term management recommendations for the cutaneous porphyrias.

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Section: Porphyria Cutanea Tardamentioning

confidence: 61%

“…Remission typically lasts 17–24 months, although complete remission is possible . Clinically, the two medications achieve similar results except that hydroxychloroquine (given at 200 mg twice weekly in these studies) may have provided a shorter remission period than chloroquine …”

Section: Porphyria Cutanea Tardamentioning

confidence: 99%

Cutaneous porphyrias part II: treatment strategies

et al. 2013

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“…Therefore, further studies assessing short and long term effects of hydroxychloroquine treatment even at this dose level in PCT are needed. Whether relapse rates are different after discontinuing hydroxychloroquine when plasma porphyrin levels are normal for at least one month, as in this study, or after an arbitrary treatment period such as one year 26 is also not known.…”

Section: Discussionmentioning

confidence: 93%

“…Few studies have compared the safety and effectiveness of these treatments prospectively, 19, 26 and none have compared compliance, to our knowledge. We designed an unblinded pragmatic trial comparing these two therapies in achieving the clear biochemical endpoint of a normal plasma porphyrin concentration.…”

Section: Introductionmentioning

confidence: 99%

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

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Kormos-Hallberg

Lee

et al. 2012

Clinical Gastroenterology and Hepatology

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Background & Aims Porphyria cutanea tarda (PCT) is an iron-related disorder caused by reduced activity of hepatic uroporphyrinogen decarboxylase (UROD); 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 that received phlebotomy and 6.1 months for patients treated with hydroxychloroquine treatment (6.7 and 6.5 months 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 [HR], 2.19; 95% confidence interval [CI], 0.95–5.06) for all patients. Patients that 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.

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“…Chlumskä et al (19) fanden nach drei-bis achtmonatiger CQ-Therapie keine wesentliche Änderung des Schadensbildes. Andere Autoren (20) beobachteten nach einjähriger CQ-Therapie von Alkoholikern fast ausschließlich mit chronischen Hepatiteden und Zirrhosen trotz weitgehender biochemischer Remission keine Änderung oder eine Zunahme der nach Nekrose, entzündlichen Infiltraten und Fibrösen beurteilten Aktivität der Lebererkrankungen. Wir haben dagegen bei ca.…”

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1992

LaboratoriumsMedizin

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Tumor markers (TM) are only tumor-associated probes reflecting tumor development. Being of protein, lipid and/or carbohydrate nature, they are expressed in or on tumor cells or induced in their environment. Most of them are circulating antigens appearing in serum or other body fluids, whose concentration results as sum of tumor marker expression, synthesis, release, catabolism, excretion as well as of tumor blood supply. TM concentration correlates in a high manner with the development, extention and therapy-induced tumor reduction. TM determination is performed by highly sensitive radio-or enzyme immunoassays, fluorescence and luminescence assays by means of poly and/or monoclonal antibodies. An appropriate interpretation of individual serial TM follow-up curves demands knowledge about intra-and inter-assay variation of TM test, the catabolism and excretion mode of TM and about pitfalls of transitory levels changes by acute treatment and unspecific influence of interfering human antibodies; in addition statistical parameters of the target TM (sensitivity, specificity, prevalence, positive/negative predictive value) should be known. According to TM indication, circulating TM are useless for screening and tumor localization, conditionally useful for surveillance of risk groups, staging and prognosis, but essentially useful as adjuncts in the follow-up care of tumor patients after operation or during radio-, chemo-or hormone therapy, where their level fluctation may anticipate the clinically detectable course of disease by a lead-time from 1 to 6 months. The indication for TM determination depends on therapeutic consequences and the frequency of serial measurements on different follow-up care recommendations, e. g. following extensive treatment, on change of therapy, on new staging and on an unclear alteration in the course of disease. Keywords: Tumor marker -tumor marker test -tumor marker statistic -cancer follow-up care Lab.med. 16: 13 (1992) 13 Brought to you by | University of Arizona Authenticated Download Date | 6/7/15 6:41 AM Spezifität RN RN + FP Effizienz Prävalenz RP + RN RP+FP+RN+FN Krank Krank+ nicht Krank Youden-lndex J = (Se+Sp-1) Abb. 1: Einfache statistische Parameter zur Beurteilung von Tumormarker-Tests. Brought to you by | University of Arizona Authenticated Download Date | 6/7/15 6:41 AM Lab.med. 16: 15 (1992) 15 Brought to you by | University of Arizona Authenticated Download Date | 6/7/15 6:41 AM 10 12 Abb. 6: Diagramm der positiv und negativ prädiktiven Werte von Serum-CEA bei Patienten mit Bronchialkarzinom (n = 38) versus Mesotheliom (n = 53) in Abhängigkeit vom Cutoff (Prävalenz *--16 Lab.med. 16: 16 (1992) Brought to you by | University of Arizona Authenticated Download Date | 6/7/15 6:41 AM Abb. 3: Verlaufsuntersuchungen bei Patientinnen mit Ovarialkarzinom (NED = no evidence for disease), Rem. '= Remission, NC -no change, Rez. = Rezidiv und Prog. -Progression nach GTMG (3). Lab.med. 16: 23 (1992) 23 Brought to you by |

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Hydroxychloroquine versus phlebotomy in the treatment of porphyria cutanea tarda

Cited by 32 publications

References 23 publications

Cutaneous porphyrias part II: treatment strategies

Cutaneous porphyrias part II: treatment strategies

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

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