Treatment of Gaucher's Disease

Abstract

We are pleased that Figueroa et al. (Dec. 3 issue)¹ have reaffirmed the effectiveness of alglucerase (Ceredase) in reducing the manifestations of Gaucher's disease with a previously published,^2,3 relatively low-dose regimen. However, we believe that the data presented are not adequate to conclude that a lower-dose regimen with frequent administration is equivalent to a higher-dose regimen given every two weeks. The comparison of the data obtained in this study with those in other studies,^4,5 though suggestive, is less than rigorous in providing convincing evidence of comparable responses to therapy. The variability in the progression and manifestations of Gaucher's disease makes comparisons between studies difficult, particularly when such small numbers of patients are included. Admittedly, the rarity of this disease does not encourage large-scale comparative trials. Thus, questions remain whether this lower-dose regimen provides an equivalent frequency, rate, or extent of response in patients with Gaucher's disease. Although some patients may respond to this regimen, others may require higher doses to achieve meaningful responses within reasonable time frames. In view of these difficulties it would be lamentable if the study by Figueroa et al. was used to limit access to effective doses of alglucerase for those patients who require it.