Flores-Pulido Andrey Arturo, Jiménez-Pérez Víctor Manuel, García-Chong Néstor Rodolfo
Secretaría de Salud, Centro Regional de Alta Especialidad de Chiapas, Hospital de Especialidades Pediátricas, Chiapas, México.
Universidad Autónoma de Nuevo León, Facultad de Ciencias Químicas, Departamento de Materiales, Nuevo León, México.
Gac Med Mex. 2019;155(2):191-195. doi: 10.24875/GMM.18004310.
Menkes disease is a neurodegenerative and lethal pathology caused by gene mutations of the copper-transporting ATP-7A enzyme; it manifests itself by neurological symptoms and connective tissue changes of varying severity. Timely subcutaneous use of copper histidinate (Cu-His) is determinant for quality of life. We report the first experiences in Mexico on Cu-His synthesis and its safe use in 3 cases where hypocupremia and hypoceruloplasminemia were corroborated. With advice of the Hospital for Sick Children of Toronto Canada, we prepared a 500 µg/mL solution. In all three cases were 250 µg of Cu-His applied without relevant undesirable effects for 30 days. Serum copper (Cu, expressed in µg/L) and ceruloplasmin (Cp, in mg/dL) were determined: case 1, Cu days 0 and 30, 8 and 504 µg/L; Cp days 0 and 30, 4 and 10.75 mg/dL; case 2, Cu days 0 and 30, <50 and 502 µg/L; Cp days 0 and 30, 2 and 15 mg/dL; case 3, Cu days 0 and 30, 3 and 84.2 µg/L; Cp days 0 and 30, 4 and 10.7 mg/dL. In Mexico, it is possible to safely synthesize Cu-His and treat MD, which must be intentionally sought.
门克斯病是一种由铜转运ATP - 7A酶基因突变引起的神经退行性致命疾病;其表现为不同严重程度的神经症状和结缔组织变化。及时皮下使用组氨酸铜(Cu - His)对生活质量起决定性作用。我们报告了墨西哥首例关于组氨酸铜合成及其在3例确诊为低铜血症和低铜蓝蛋白血症病例中的安全使用情况。在加拿大多伦多病童医院的建议下,我们配制了500 µg/mL的溶液。在所有3例病例中,均应用了250 µg组氨酸铜,持续30天,未出现相关不良影响。测定了血清铜(Cu,单位为µg/L)和铜蓝蛋白(Cp,单位为mg/dL):病例1,第0天和第30天的血清铜分别为8和504 µg/L;第0天和第30天的铜蓝蛋白分别为4和10.75 mg/dL;病例2,第0天和第30天的血清铜分别<50和502 µg/L;第0天和第30天的铜蓝蛋白分别为2和15 mg/dL;病例3,第0天和第30天的血清铜分别为3和84.2 µg/L;第0天和第30天的铜蓝蛋白分别为4和10.7 mg/dL。在墨西哥,可以安全地合成组氨酸铜并治疗门克斯病,必须有意寻找此类病例。