Chelation of elevated copper levels

Clinical criteria:

• Patient must have a diagnosis of Wilson disease,

AND

• Patient must be intolerant to penicillamine.

Treatment criteria:

• Must be treated by a specialist medical practitioner, where this authority application is to initiate treatment with this drug, of the following type: (i) gastroenterologist, (ii) hepatologist, (iii) neurologist; the authority prescription must be completed by the specialist prescriber; OR

• Must be treated by a medical practitioner (of any type), where this authority application is continuing established trientine treatment (of any specified salt) initiated by one of the above mentioned specialist types; OR

• Must be treated by a nurse practitioner where this authority application is continuing established trientine treatment (of any specified salt) initiated by one of the above mentioned specialist types.

Prior to seeking the initial authority approval, establish evidence of excess copper levels based on at least one of: (i) clinical symptoms, (ii) measured serum copper levels, (iii) measured urinary copper levels.

Document what these findings were in the patient's medical records. Do not supply them in this authority application.

Refer to the following definitions if in doubt over what constitutes an acceptable intolerance to penicillamine:

Side effects of penicillamine occurring soon after initiation (within first few weeks/months):

(i) fever, (ii) rash, (iii) enlarged lymph nodes, (iv) neutropenia, (v) thrombocytopenia, (vi) proteinuria, (vii) severe, persistent nausea.

Side effects of penicillamine developing later:

(i) nephrotic syndrome, (ii) glomerulonephritis, (iii) total bone marrow aplasia, (iv) skin changes (cutis laxa, elastosis perforans serpiginosa, pemphigus), (v) myasthenia gravis, (vi) polymyositis, (vii) Goodpasture syndrome, (viii) optic neuritis, (ix) proteinuria (1-2 grams/day or equivalent in children, depending on specialist Wilson disease and renal review), (x) haematuria (if cause unknown), (xi) thrombocytopenia/leukopenia, (xii) bleeding related to thromobocytopenia/leukopenia, (xiii) lupus-like syndrome (haematuria, proteinuria, positive antinuclear antibody), (xiv) arthralgia.

At the time of the first authority application for this drug, document the details (date of reaction, severity of reaction, dose of penicillamine, etc) of the penicillamine intolerance, if not already done, in the patient's medical records. Do not supply these details in this authority application.