Mistry P K, Davies S, Corfield A, Dixon A K, Cox T M
Department of Medicine, University of Cambridge School of Clinical Medicine, Addenbrooke's Hospital.
Q J Med. 1992 Jul;83(303):541-6.
We report the beneficial effects of enzyme replacement therapy with mannose-terminated human glucocerebrosidase ('Ceredase') in a patient suffering from transfusion-dependent bone marrow failure due to Gaucher's disease. Treatment with low-dose enzyme infusions, given twice weekly, rapidly reversed the haematopoietic failure and incapacitating skeletal disease. It appears likely that prior splenectomy favourably influenced the response to this therapy.
我们报告了用甘露糖末端的人葡萄糖脑苷脂酶（“Ceredase”）进行酶替代疗法对一名因戈谢病而依赖输血的骨髓衰竭患者的有益效果。每周两次给予低剂量酶输注治疗，迅速逆转了造血功能衰竭和使人丧失能力的骨骼疾病。脾切除术似乎对这种治疗的反应产生了有利影响。
