Balcerzak S P, Grever M R, Sing D E, Bishop J N, Segal M L
J Lab Clin Med. 1982 Sep;100(3):345-55.
The lack of effective therapy of sickle cell anemia has prompted investigation of a large number of antisickling agents. The most promising drug, cyanate, was found in previous studies to be toxic when given systemically. The present study examined a semiautomated system for extracorporeal treatment with cyanate of the blood of four patients with sickle cell anemia. All patients achieved hemoglobin carbamylation levels greater than 1 mode of cyanate per mode of Hb4, with minimal systemic exposure to cyanate and no serious toxicity. Hematologic response occurred in two patients and possibly a third. Further study will be required to determine the efficacy of continuous extracorporeal carbamylation in the treatment of sickle cell anemia.
镰状细胞贫血缺乏有效的治疗方法,这促使人们对大量抗镰变剂进行研究。最有前景的药物氰酸盐,在先前的研究中发现全身给药时具有毒性。本研究检测了一种半自动化系统,用于对4例镰状细胞贫血患者的血液进行体外氰酸盐治疗。所有患者的血红蛋白氨甲酰化水平均达到每摩尔Hb4大于1摩尔氰酸盐,全身接触氰酸盐极少,且无严重毒性。两名患者出现血液学反应,第三名患者可能也有反应。需要进一步研究以确定持续体外氨甲酰化治疗镰状细胞贫血的疗效。