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The following is issued on behalf of the Hospital Authority:
The Hospital Authority (HA) Expert Panel on Rare Metabolic Diseases met today (December 8) to discuss and deliberate on the treatment modality of two brothers with Pompe Disease. The meeting arrived at a unanimous decision that both patients are suitable for the use of Enzyme Replacement Therapy (ERT).
The doctor in charge has been informed to convey the Expert Panel's decision to the patients and their family. Both of them are currently receiving specialist treatment at the outpatient clinic of Princess Margaret Hospital.
Dr Luk Che-chung, Expert Panel Chairman and Cluster Chief Executive of Hong Kong West Cluster, said after the meeting that, "Expert Panel members have made the decision with reference to established clinical indicators and with due consideration given to the latest evidence-based criteria as published in the New England Journal of Medicine in April this year. While exercising their professional judgement, the experts have carefully assessed the patients' clinical condition and a balance of the risks and benefits of the treatment. I have to thank all the experts for their valuable inputs to help us assist the patients in need."
Dr Luk pointed out that the doctor in charge will explain in detail the treatment process to the two patients including the clinical risk and expected outcome. The procedure will need to be performed in the Intensive Care Unit. The clinical effectiveness and outcome will be regularly reviewed to assess the risk factor and benefits of the treatment. Adjustment of the treatment plan will be made on a need basis.
According to the experts, replacement therapy will need to be conducted together with other supportive treatment as a total package to achieve the best possible outcome, including appropriate level of exercise and dietetic therapy.
Members of the Expert Panel include four senior paediatricians, a senior clinical oncologist and a senior clinical pharmacologist. Experts from other specialties, such as Medicine and Orthopaedics, will be co-opted as necessary in the discussion and assessment of applications.
HA has been given additional funding on drugs by the Government this year (2010-11), part of which will be allocated to the spending on ERT for six types of rare metabolic diseases including Mucopolysaccharidoses I, II and VI, Pompe Disease, Fabry Disease, and Gaucher Disease.
Ends/Wednesday, December 8, 2010
Issued at HKT 20:06
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