Sporadic porphyria cutanea tarda: Treatment with chloroquine decreases hyperglycemia and reduces development of metabolic syndrome

González-Estrada, Aurora; Gómez-Morales, Luis; Gonzalez‐Estrada, Alexei; García-Morillo, Salvador

doi:10.1016/j.ejim.2014.02.009

Cited by 5 publications

(4 citation statements)

References 11 publications

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“…Finally, another genetic predisposition to liver damage could be hypothesized, as described in erythropoietic protoporphyria, through the impairment of the expression of ATP-binding cassette transporter G2 [26]. In the future, massive accumulation of porphyrins in the liver could justify a preventive approach to protect the liver, as has been suggested, for example, with chloroquine for its anti-inflammatory action in porphyria cutanea tarda [27], or more recently by an induced iron deficiency in CEP [22].…”

Section: Discussionmentioning

confidence: 99%

Bone Marrow Transplantation in Congenital Erythropoietic Porphyria: Sustained Efficacy but Unexpected Liver Dysfunction

Besnard

Galmiche-Rolland

Debray

et al. 2020

Biology of Blood and Marrow Transplantation

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Congenital erythropoietic porphyria (CEP) is a rare disease characterized by erosive photosensitivity and chronic hemolysis due to a defect of the enzyme uroporphyrinogen-III-synthase (UROS). To date, hematopoietic stem cell transplantation (HSCT) is the only curative therapy for the devastating early and severe form of the disease. We describe 6 patients with CEP treated with HSCT (3 of them twice after failure of a first graft) between 1994 and 2016 in our center, including 2 of the very first living patients treated more than 20 years ago. Four patients are doing well at 6 to 25 years post-HSCT, with near-normal biochemical parameters of porphyrin metabolism without the cutaneous or hematologic features of CEP. One patient died within the first year after HSCT from severe graft-versus-host disease (GVHD), and 1 child died of unexplained acute hepatic failure at 1 year after HSCT, despite full donor chimerism. Retrospectively, it appears that all but 1 child had increased transaminase activity with onset from the early postnatal period, which was significantly more marked in the child who died of liver failure. In contrast, liver function values progressively normalized after engraftment in all other children. Liver pathology before HSCT for 3 patients revealed varying degrees of portal, centrilobular, and perisinusoidal fibrosis; clarification of hepatocytes; and cytosolic porphyrin deposits. The liver porphyrin content in biopsy specimens was >60 times the normal values. Despite difficult engraftment, the long-term efficacy of HSCT in CEP appears to be favorable and reinforces its benefits for the severe form of CEP. Hepatic involvement requires careful evaluation before and after HSCT and further investigation into its pathophysiology and care.

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Section: Discussionmentioning

confidence: 99%

Bone Marrow Transplantation in Congenital Erythropoietic Porphyria: Sustained Efficacy but Unexpected Liver Dysfunction

Besnard

Galmiche-Rolland

Debray

et al. 2020

Biology of Blood and Marrow Transplantation

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“…In these cases, a low-dose regimen of chloroquine analogues gives favourable results without untoward reactions. 37 , 38 …”

Section: Indications For Chloroquine Analoguesmentioning

confidence: 99%

Chloroquine analogues in drug discovery: new directions of uses, mechanisms of actions and toxic manifestations from malaria to multifarious diseases

Al‐Bari

2015

Journal of Antimicrobial Chemotherapy

359

306

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Antimalarial drugs (e.g. chloroquine and its close structural analogues) were developed primarily to treat malaria; however, they are beneficial for many dermatological, immunological, rheumatological and severe infectious diseases, for which they are used mostly today. Chloroquine and hydroxychloroquine, two of the most fascinating drugs developed in the last 50 years, are increasingly recognized for their effectiveness in myriad non-malarial diseases. In advanced research, chloroquine and hydroxychloroquine have been shown to have various immunomodulatory and immunosuppressive effects, and currently have established roles in the management of rheumatic diseases, lupus erythematosus (different forms) and skin diseases, and in the treatment of different forms of cancer. Recently, chloroquine analogues have also been found to have metabolic, cardiovascular, antithrombotic and antineoplastic effects. This review is concerned with the lysosomotropic, anti-inflammatory and immunomodulatory mechanisms of chloroquine, hydroxychloroquine, quinacrine and related analogues, and the current evidence for both their beneficial effects and potential adverse manifestations in various diseases.

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“…The roles of CHQ, HCQ, and their analogues has been examined in many dermatological disorders; however, the outcomes have not been consistently positive. While FDA has granted approval for the treatment of discoid lupus97 and small clinical studies have shown positive benefits for porphyria cutanea tarda,98,99 in a large randomized clinical trial of dermatomyositis, HCQ treatment for 24 weeks did not show significant improvements in mouth and eye dryness compared with that in the placebo group 58. The immunological properties of CHQ analogues have also been tested in many bacterial, viral, and parasitic infections such as Q fever, chikungunya fever, giardiasis, amoebiasis, Ebola virus, and HIV-1 infections 2.…”

Section: Chq In Dermatology and Infectionsmentioning

confidence: 99%

Expanding horizons for clinical applications of chloroquine, hydroxychloroquine, and related structural analogues

Shukla¹,

Shukla²

2019

DIC

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Several experimental and clinical studies have transformed the traditional antimalarial role of chloroquine (CHQ) and related structural analogues to potent therapeutic agents for a host of nonmalarial indications. The expanding clinical applicability for these drugs includes rheumatological and cardiovascular disorders (CVD), chronic kidney disease (CKD), oncology, and a variety of nonmalarial infections. These clinical advancements are primarily related to pleiotropic pharmacological actions of these drugs, including immunomodulation, anti-inflammatory properties, and capabilities of inducing autophagy and apoptosis at a cellular level. Historically, many clinical benefits in nonmalarial indications were first recognized through serendipitous observations; however, with numerous ongoing systematic clinical studies, the clinical horizons of these drugs have a promising future.

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Sporadic porphyria cutanea tarda: Treatment with chloroquine decreases hyperglycemia and reduces development of metabolic syndrome

Cited by 5 publications

References 11 publications

Bone Marrow Transplantation in Congenital Erythropoietic Porphyria: Sustained Efficacy but Unexpected Liver Dysfunction

Bone Marrow Transplantation in Congenital Erythropoietic Porphyria: Sustained Efficacy but Unexpected Liver Dysfunction

Chloroquine analogues in drug discovery: new directions of uses, mechanisms of actions and toxic manifestations from malaria to multifarious diseases

Expanding horizons for clinical applications of chloroquine, hydroxychloroquine, and related structural analogues

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