Sickle-cell Disease—New Method of Treatment

[摘要] This is a preliminary report of a new approach to treatment of sickle-cell disease based on the knowledge that the sickling phenomenon resides in a defect in the hemoglobin which, when deoxygenated, undergoes molecular rearrangement with consequent alteration of the shape of the erythrocyte. The sickle-cell phenomenon is not seen in fully oxygenated blood. It seemed reasonable to the authors to employ carbonic anhydrase inhibitor to control reduction of hemoglobin in sickle-cell disease to such an extent as to prevent the occurrence of sickling. The carbonic anhydrase inhibitor employed was acetazolamide (Diamox®). Experiments were conducted in vitro and also in vivo in a single Negro baby 8 months of age. Before treatment the patient's blood contained 18.7% sickled cells. One hour after 2 mg of acetazolamide per kilogram the percentage of sickled cells was reduced to 11.8%; 1 hour after 7 mg of acetazolamide per kilogram the percentage of sickled cells was reduced to 7.5%. At the time of this preliminary report the patient had been treated with acetazolamide for 29 days with an increase in the hemoglobin from 7.1 to 9.4 gm/100 ml. In a preliminary period of observation of 41 days before treatment the hemoglobin had declined during two separate episodes of hemolysis. The theory of the suggested mode of treatment is discussed. A detailed account of the clinical observations is to be the subject of a further report.

[学科分类] 儿科学