Cholestyramine and Porphyrin-Binding

Lischner, HaroldW.

doi:10.1016/s0140-6736(66)92064-2

Cited by 11 publications

(3 citation statements)

References 11 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Many therapeutic regimens have been proposed for liver disease like vitamin E [36], cholestyramine [37], chenodeoxycholic acid [38], blood transfusion [39] and hematin infusion [40], Their efficacy in the prevention of liver failure has not been clear ly established so far. These therapies can produce serious side effects or discomfort for the patient.…”

Section: Avoidance O F Possible Risk Factors For Liver Disease In Eppmentioning

confidence: 99%

Hepatic Disease in Erythropoietic Protoporphyria

Mooyaart¹,

Jong²,

Veen³

et al. 1986

Dermatology

View full text Add to dashboard Cite

A standardized hepatological investigation was performed in 9 unselected patients with erythropoietic protoporphyria (EPP). The aim of this study was to detect early liver involvement due to EPP and to determine the significance of several diagnostic procedures. Scintigraphy revealed slight enlargement of liver and spleen in all cases. Light microscopic examination of liver tissue in 7 patients showed protoporphyrin deposition in 4 and signs of fibrosis in 3 cases. Cirrhosis was not found. Electron microscopical examination (EM) of all 7 cases was negative and needle-shaped crystals were not found. Therefore we regard EM of little diagnostic value in the detection of early liver involvement. The literature on fatal and asymptomatic cases of liver involvement in EPP is discussed with emphasis on possible predictive and provocative factors. A proposal for EPP patients intending to reduce possible risk factors is made.

show abstract

Section: Avoidance O F Possible Risk Factors For Liver Disease In Eppmentioning

confidence: 99%

Hepatic Disease in Erythropoietic Protoporphyria

Mooyaart¹,

Jong²,

Veen³

et al. 1986

Dermatology

View full text Add to dashboard Cite

show abstract

“…Therapeutic interventions in the past have included protection from sunlight (which is essential until definitive treatment is implemented), beta-carotene to reduce oxidative damage, 1,2 splenectomy to decrease anemia and thrombocytopenia, 1-12 transfusion regimens 13 and i.v. hematin (oxidized free heme) therapy 8,[14][15][16] to suppress hemoglobin production, adsorbents to bind to porphyrins and increase their excretion, 17,[20][21][22] hydroxyurea, 13,23,24 and steroids. 13,[17][18][19][20] The most effective therapy would be one that increases uroporphyrinogen III cosynthase activity into the normal range, as it is reduced to 0 to 40% of normal in erythrocytes and fibroblasts of patients with CEP (approximately 50% of normal in carriers).…”

Section: Discussionmentioning

confidence: 99%

Treatment of congenital erythropoietic porphyria in children by allogeneic stem cell transplantation: a case report and review of the literature

Shaw

Mancini

McConnell

et al. 2001

Bone Marrow Transplant

View full text Add to dashboard Cite

Summary:Congenital erythropoietic porphyria (CEP) is a rare autosomal recessive disorder of porphyrin metabolism in which the genetic defect is the deficiency of uroporphyrinogen III cosynthase (UIIIC). Deficiency of this enzyme results in an accumulation of high amounts of uroporphyrin I in all tissues leading to hemolytic anemia, splenomegaly, erythrodontia, bone fragility, exquisite photosensitivity and mutilating skin lesions. We describe the case of a 23-month-old boy who was cured of his CEP by a matched-sibling allogeneic bone marrow transplant, and review the published clinical experience regarding transplantation in this disease. He is alive and disease-free 15 months post transplant. All of his disease manifestations except for the erythrodontia have resolved. His UIIIC level and stool and erythrocyte porphyrin metabolites have almost completely corrected. He is the sixth child reported to be cured of this disease by stem cell transplantation, five cases being long-term survivors. If patients with this disease have an HLA-matched sibling, then stem cell transplantation should be strongly considered because this is currently the only known curative therapy. Bone Marrow Transplantation (2001) 27, 101-105. Keywords: congenital erythropoietic porphyria; porphyria; stem cell transplantation; bone marrow transplantation Congenital erythropietic porphyria (CEP), or Gunther's disease, is one of the rarest of the porphyrias, a group of metabolic disorders in which excessive quantities of porphyrins or their precursors are produced. This autosomal recessive disorder is caused by a defect of the enzyme uroporphyrinogen III cosynthase (UIIIC) that leads to an accumulation of uroporphyrinogen I and coproporphyrinogen I in all tissues. The primary clinical manifestations of this accumulation include erythrodontia, bone fragility, hypertrichosis,

show abstract

“…The liver must be able to excrete the excess protoporphyrin it has accumulated. Previous studies indicate that the liver can mobilize protoporphyrin deposits even though they are in crystalline form (4, 29,30), presumably because the crystals are in equilibrium with soluble protoporphyrin.…”

Section: Discussionmentioning

confidence: 99%

Effect of Hematin Administration to Patients With Protoporphyria and Liver Disease

Bloomer¹,

Pierach²

1982

Hepatology

View full text Add to dashboard Cite

Hepatic damage in protoporphyria appears to be caused by a toxic effect of excess protoporphyrin. Therapy which reduces the formation of excess protoporphyrin may, therefore, be helpful. We examined the effects of hematin administered i.v. to two patients with protoporphyria and decompensated cirrhosis. Neither patient had side effects from the compound or manifested signs of toxicity. The vascular disappearance of hematin in one patient was similar to that in patients with porphyria who do not have structural liver disease. In both patients, biochemical changes occurred that were compatible with a reduced rate of protoporphyrin formation. Thus, hematin administration may be useful in treating patients with protoporphyria who develop liver disease.

show abstract

Cholestyramine and Porphyrin-Binding

Cited by 11 publications

References 11 publications

Hepatic Disease in Erythropoietic Protoporphyria

Hepatic Disease in Erythropoietic Protoporphyria

Treatment of congenital erythropoietic porphyria in children by allogeneic stem cell transplantation: a case report and review of the literature

Effect of Hematin Administration to Patients With Protoporphyria and Liver Disease

Contact Info

Product

Resources

About