CUFENCE (trientine dichlorhydrate)

• Key points

Favourable opinion for reimbursement in the treatment of Wilson's disease in adults, adolescents and children ≥ 5 years intolerant to D-penicillamine therapy.

• What therapeutic improvement?

No clinical added value in the management of Wilson's disease.

• Role in the care pathway?

If left untreated, Wilson’s disease is fatal. The prognosis depends on the severity of liver and neurological damage. The treatment of Wilson’s disease includes a diet low in certain foods high in copper (chocolate, hazelnuts, shellfish, mushrooms, liver), zinc acetate or sulfate, which reduce the intestinal absorption of copper and, above all and primarily, D-penicillamine or trientine, which are copper chelating agents. The objective of treatment is to maintain serum free copper levels within acceptable limits. Zinc salts and/or D-penicillamine are recommended as first-line treatments, whereas trientine is recommended as a second-line treatment. Several adverse effects of D-penicillamine may lead to treatment discontinuation. These are mucocutaneous (early: rash, pruritus; late: gingivitis, stomatitis, ulcerous lesions, drug eruption, pemphigus), renal (proteinuria), respiratory (interstitial lung disease and bronchiolitis obliterans), haematological (thrombocytopenia, agranulocytosis and bone marrow failure) and autoimmune disturbances (myasthenia, polymyositis and drug-induced lupus). In this case, patients are treated with trientine.

Role of the medicinal product in the care pathway

CUFENCE (trientine dihydrochloride) is a second-line treatment in patients intolerant to D-penicillamine.

• Special recommendations

The Committee recommends that initial prescription of CUFENCE- trientine dihydrochloride (and CUPRIOR, another proprietary medicinal product containing trientine) along with any treatment changes be performed by a centre specialising in the management of Wilson’s disease patients, to guarantee optimal clinical and biological monitoring and to include all these patients on the national Wilson’s disease registry.