Abstract | OBJECTIVES: CASE SUMMARY: DISCUSSION: CONCLUSION: In this, we highlight a case of discontinuation of penicillamine in a patient with Wilson's disease without substitution with alternative regimen. This was caused by unavailability of the alternative agents such as trientine in our country. Consequently, the patient progressed to decompensated liver cirrhosis with encephalopathy and eventually passed-away within 5 months. One recent study supports a combination of trientine and zinc in treating patient with decompensated liver cirrhosis. This combination is capable of reversing liver failure and prevents the need of liver transplantation. Both trientine and zinc are not registered in Malaysia. Therefore, liver transplantation was probably the only treatment option for this patient. Hence, non-availability of orphan drugs in clinical practice is certainly a subject of serious concern. Systems for better management of patients with rare diseases need to be instituted by all the institutions concerned.
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Authors | C C Ping, Y Hassan, N A Aziz, R Ghazali, A Awaisu |
Journal | Journal of clinical pharmacy and therapeutics
(J Clin Pharm Ther)
Vol. 32
Issue 1
Pg. 101-7
(Feb 2007)
ISSN: 0269-4727 [Print] England |
PMID | 17286794
(Publication Type: Case Reports, Journal Article)
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Chemical References |
- Chelating Agents
- Trace Elements
- Penicillamine
- Zinc
- Trientine
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Topics |
- Adult
- Chelating Agents
(adverse effects, therapeutic use)
- Fatal Outcome
- Female
- Hepatolenticular Degeneration
(drug therapy)
- Humans
- Liver Cirrhosis
(etiology)
- Malaysia
- Orphan Drug Production
- Penicillamine
(adverse effects, therapeutic use)
- Recurrence
- Trace Elements
(supply & distribution)
- Trientine
(supply & distribution)
- Zinc
(supply & distribution)
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