However there was little uptake in the lungs at the extremely low doses given to these patients. More Ceredase and Cerezyme was taken up in the livers of patients who had previously had splenectomy. The livers of splenectomised patients demonstrated greater reduction in size after treatment than in those with the spleen intact. After Ceredase and Cerezyme reached the liver and the spleen, about half was destroyed within two hours but the remainder was present for more than 43 hours. A small amount of enzyme appeared to be lost in the urine.
Most patients have a good response to enzyme treatment.
However some have an extraordinary response while a small number of patients respond poorly. Patients who have a malignancy are also slow to respond to enzyme treatment. Patients with advanced cirrhosis of the liver also do not respond well to enzyme treatment. Liver Transplant Dr Mistry then described a 33 year old man with Gauchers disease who was brought as an emergency to the Royal Free Hospital Casualty at 5am about a year ago. The young man came in with massive life-threatening bleeding due to advanced liver cirrhosis.