Low Dose - what does it mean?

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If Cerezyme is taken more frequently than once every two weeks, the overall dose can be lower. This cuts the cost and may be safer.

Low dose originally meant Dr Ernest Beutler's regime of 2.3 units of Ceredase per kilogram of body weight three times a week, the equivalent of 13.8 units/kg/bw every two weeks but fractionated (which means divided into smaller amounts and given at more frequent intervals). This is the equivalent of 30 units per calendar month. One of the confusions arise if you talk about every 4 weeks or calendar month. Here we shall talk about every two weeks.

This low dose compares with high dose of 60 units/kg/bw every two weeks and the so-called moderate dose of 30 units/kg/bw every two weeks. It is thought many US patients are now on the 30 units/kg/bw every two weeks regime.

However in the UK in some cases, the 'low' dose has been adapted to 5 units kg/bw twice a week (making 20 units every two weeks, fractionated). Some patients have the same overall dose of 20 units given only once a week i.e. 10 units kg/bw once a week.

In the Netherlands, on the whole, the starting dose is half-Beutler, that is 1.15 units kg/bw three times a week (making 6.9 units every two weeks, fractionated). This you could call this 'low, low' dose. If it doesn't work, the dose is doubled until it does.

In South Africa a protocol of 10 units per kilogram of body weight every two weeks is being used.

This follows a trial in Israel reported in 1996 which showed that for infusions of Cerezyme of 15 units/kg/bw every two weeks, the response was not significantly different if given once in every two weeks or in a fractionated form three times a week.

To sum up:

It should be noted that many patients don't receive exactly these amounts. Because Cerezyme comes in 200 unit or 400 unit bottles, these figures are either rounded up or down so that all the powder from the bottle gets used up. The size of the bottle makes it more difficult to prescribe smaller variations in dose than with Ceredase which used to have a 50 unit bottle.

Beneficial results, including bone results, are being reported on low dose. Dr Ari Zimran in Israel has compared results for low and high doses and found no differences. Paul Guyt from Holland has also reported benefit to himself and others.

Many patients in the States have been lowered from 60 units /kg/bw every two weeks to 30 units /kg/bw every two weeks. New patients at the largest treatment centre at Mount Sinai in New York now generally start at 30 units/kg/bw once every two weeks with the same results as those who started at double that dose. A few patients have their dose fractionated to once a week as it is thought to be more effective in their cases.

Having infusions more than twice a week can be a drag. Twice a week for most people is tolerable although maybe not for children. Having said that, Gill infuses her son through a vein three times a week over two hours. Some children (and adults) have had a portacath fitted to enable easier access to veins but in some cases this has produced its own problems.

Many who have twice/three times a week infusions, or even once a week or less, infuse themselves or their children or partners at home where there is far less hassle and less risk of infection. Our UK survey showed that hardly anyone found home infusion a problem and a paper written by Dr Zimran reported easy home infusions in Israel as well. Most Dutch patients also infuse at home.

We hope this is of help. It seems that there has to be some flexibility between the two extremes of dosages and frequency.

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Source: Gauchers News . Updated September 2001. Original 1996.
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