Christensen Robert D, Agarwal Archana M, Yaish Hassan M, Reading N Scott, O'Brien Elizabeth A, Prchal Josef T
1 University of Utah, Salt Lake City, UT, USA.
2 Intermountain Healthcare, Salt Lake City, UT, USA.
Clin Pediatr (Phila). 2018 Jan;57(1):19-26. doi: 10.1177/0009922816687326. Epub 2017 Jan 15.
Various mutations in the genes encoding alpha spectrin (SPTA1) or beta spectrin (SPTB) are known to cause erythrocyte membrane disorders, sometimes associated with severe neonatal jaundice and anemia. We used a next-generation sequencing panel to evaluate 3 unrelated neonates who had puzzling cases of nonimmune hemolytic jaundice. In each case, we identified novel mutations in either SPTA1 or SPTB. Correlating erythrocyte morphology, clinical course, and computational analysis, we submit that each of the 3 variants is a probable pathogenic cause of the hereditary hemolytic conditions in these patients. We hope other pediatric practitioners caring for neonates with what appears to be idiopathic severe neonatal hyperbilirubinemia will look for spectrin variants as a possible cause, because additional cases with these specific variants along with this clinical phenotype are needed to confirm our postulate that these 3 cases are indeed pathogenic mutations.
已知编码α-血影蛋白(SPTA1)或β-血影蛋白(SPTB)的基因发生的各种突变会导致红细胞膜紊乱,有时还会伴有严重的新生儿黄疸和贫血。我们使用新一代测序平台评估了3例患有非免疫性溶血性黄疸疑难病例的无亲缘关系的新生儿。在每例病例中,我们在SPTA1或SPTB中发现了新的突变。通过将红细胞形态、临床病程和计算分析相关联,我们认为这3种变体中的每一种都是这些患者遗传性溶血性疾病的可能致病原因。我们希望其他照顾看似患有特发性严重新生儿高胆红素血症新生儿的儿科医生将寻找血影蛋白变体作为可能的病因,因为需要更多具有这些特定变体以及这种临床表型的病例来证实我们的假设,即这3例病例确实是致病突变。