Zaffanello Marco, Zamboni Giorgio, Fontana Elena, Zoccante Leonardo, Tatò Luciano
Regional Center for Neonatal Congenital Errors of Metabolism, Department of Pediatrics, University of Verona, Verona, Italy.
Child Neuropsychol. 2003 Sep;9(3):184-8. doi: 10.1076/chin.9.3.184.16457.
We report the case of a child with partial biotinidase deficiency and autistic developmental disorder. We arrived at the diagnosis of biotinidase deficiency when the child was almost 4 years of age. Consequently, he began cofactor biotin treatment (10 mg daily) which did not resolve his autistic behavior. His younger brother was affected by partial biotinidase deficiency diagnosed at birth through our neonatal screening program. He was precociously treated with cofactor biotin therapy (10 mg daily) and did not show any behavioral abnormality or developmental delay. Since the brain is quite vulnerable to biotin deficiency, delayed biotin therapy could result in neurological damage. Our patient is the first case of partial biotinidase deficiency associated with autism. We hypothesize that the low biotinidase activity could have caused biotin deficiency in his brain and cerebrospinal fluids and consequently serious neurological problems, such as stereotyped and autistic behaviors, which were irreversible in spite of biotin supplementation.
我们报告了一例患有部分生物素酶缺乏症和自闭症谱系障碍的儿童病例。该患儿在快4岁时被诊断出生物素酶缺乏症。因此,他开始接受生物素辅助因子治疗(每日10毫克),但这并未改善他的自闭症行为。他的弟弟通过我们的新生儿筛查项目在出生时被诊断为部分生物素酶缺乏症。他早早地接受了生物素辅助因子治疗(每日10毫克),未表现出任何行为异常或发育迟缓。由于大脑对生物素缺乏非常敏感,生物素治疗延迟可能会导致神经损伤。我们的患者是首例与自闭症相关的部分生物素酶缺乏症病例。我们推测,低生物素酶活性可能导致其大脑和脑脊液中生物素缺乏,进而引发严重的神经问题,如刻板行为和自闭症行为,尽管补充了生物素,这些问题仍不可逆。