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A recently published paper shows that skeletal response to low-dose enzyme replacement therapy is comparable to that seen on high-dose therapy. The paper entitled 'Effect of Low-dose Enzyme Replacement Therapy on Bones in Gaucher Disease Patients with Severe Skeletal Involvement' by Dr Deborah Elstein and colleagues is published in Blood Cells, Molecules, and Diseases, 15 June 1996. A short summary of the paper is given below.
The paper reviews 14 adult patients who have undergone enzyme replacement therapy with either Ceredase or Cerezyme for two to four years. All had objective signs of severe skeletal involvement prior to therapy and more than half were splenectomised. Their age ranges from 18 to 51 years. Ten are women and four men. Another group of 11 untreated Gauchers patients were used as a control to compare results.
All treated patients reported significant improvement in bone pains, with reduction in frequency and/or intensity of pain episodes (six patients), or complete disappearance of skeletal pain (eight patients) often within the first six months of treatment. However one patient developed a new avascular necrosis (death in a bone) after two years of skeletal improvement and disappearance of bone pains. No untreated patient reported changes in bone pain and one patient developed avascular necrosis during the period of the study. All but one treated patient showed improvement in cortical bone thickness.
'Our results confirm earlier reports by Beutler et al of bone improvement with enzyme replacement therapy, and give credence to the hypothesis that the low-dose protocol is comparable to high-dose regimens vis-a-vis skeletal response as in other parameters and in approximately the same time frame. Although this conclusion cannot be verified without a well-controlled, prospective, double-blind clinical study (which is probably unrealistic), it puts the burden of proof on those advocating a protocol which is four times more expensive and not absolutely without additional risk.'
Elstein's original article
Source: Gauchers News September 1996
© Copyright Gauchers Association 1996