Blair Marcia A, Riddle Megan E, Wells Jennifer F, Breviu Brian A, Hedera Peter
Department of Neurology; Vanderbilt University, Nashville, TN 37232-8552, USA.
Pediatr Neurol. 2007 Jun;36(6):382-6. doi: 10.1016/j.pediatrneurol.2007.02.003.
Age of symptom onset of hereditary spastic paraplegia varies from infancy to the eighth decade. Infantile onset of hereditary spastic paraplegia without a positive family history may cause difficulties in reaching the correct diagnosis and misdiagnosis as a diplegic form of cerebral palsy is particularly common. Infantile onset of hereditary spastic paraplegia caused by mutations in the spastin gene (SPAST) is very rare and previously was mostly associated with codominant mutations in this gene. We present a kindred with infantile onset of spastic paraplegia in three successive generations caused by confirmed de novo novel mutation 1537G>A (G471D) in SPAST. Several family members were previously diagnosed as having cerebral palsy. Infantile onset of hereditary spastic paraplegia may be caused by mutations in multiple genes, and this phenotype does not reliably predict the genotype. Pediatric neurologists need to be aware of relatively frequent de novo mutations in hereditary spastic paraplegia genes and a possibility that this condition presents in infancy without a positive family history.
遗传性痉挛性截瘫的症状出现年龄从婴儿期到八十岁不等。无阳性家族史的婴儿期遗传性痉挛性截瘫发病可能导致难以做出正确诊断,误诊为双瘫型脑瘫的情况尤为常见。由痉挛蛋白基因(SPAST)突变引起的婴儿期遗传性痉挛性截瘫非常罕见,以前大多与该基因的共显性突变有关。我们报告了一个家系,其中连续三代出现婴儿期痉挛性截瘫,由SPAST基因中确认的新发突变1537G>A(G471D)引起。此前有几名家庭成员被诊断为患有脑瘫。婴儿期遗传性痉挛性截瘫可能由多个基因突变引起,这种表型并不能可靠地预测基因型。儿科神经科医生需要意识到遗传性痉挛性截瘫基因中相对频繁的新发突变,以及这种疾病在婴儿期无阳性家族史时出现的可能性。