Dantonio Paul, Tavakoli Norma P, Migliore Brooke, McCown Elizabeth, Lim Timothy, Park Sunju, Caggana Michele, Kucera Katerina S, Phan Han, Street Natalie, Petritis Konstantinos, Vogt Robert F
Newborn Screening and Molecular Biology Branch, Division of Laboratory Sciences, National Center for Environmental Health, Centers for Disease Control and Prevention, Atlanta, GA 30333, USA.
Wadsworth Center, Division of Genetics, New York State Department of Health, Albany, NY 12208, USA.
Int J Neonatal Screen. 2023 Feb 28;9(1):13. doi: 10.3390/ijns9010013.
Pilot studies to detect newborns with Duchenne Muscular Dystrophy (DMD) by newborn bloodspot screening (NBS) have been conducted under the New York State Newborn Screening Program (NYS) and are currently in progress as part of the Early Check Program at Research Triangle Institute (RTI) International. The Newborn Screening Quality Assurance Program (NSQAP) at the U.S. Centers for Disease Control and Prevention (CDC) produced a set of seven prototype dried blood spot (DBS) reference materials spiked with varying levels of creatine kinase MM isoform (CK-MM). These DBS were evaluated over a 3-week period by CDC, NYS, and RTI, all using the same CK-MM isoform-specific fluoroimmunoassay. Results from each laboratory were highly correlated with the relative proportion of CK-MM added to each of the six spiked pools. Based on reference ranges established by NYS and RTI for their pilot studies, these contrived DBS collectively spanned the CK-MM ranges found in typical newborns and the elevated ranges associated with DMD. This set allows quality assessment over the wide range of fluctuating CK-MM levels in typical and DMD-affected newborns.
纽约州新生儿筛查项目(NYS)已开展了通过新生儿血斑筛查(NBS)检测杜氏肌营养不良症(DMD)新生儿的试点研究,目前作为研究三角研究所(RTI)国际部早期检查项目的一部分正在进行。美国疾病控制与预防中心(CDC)的新生儿筛查质量保证项目(NSQAP)制备了一组七份添加了不同水平肌酸激酶MM同工酶(CK-MM)的干血斑(DBS)参考物质。CDC、NYS和RTI在3周时间内对这些DBS进行了评估,均使用相同的CK-MM同工酶特异性荧光免疫测定法。每个实验室的结果与添加到六个加标样本库中每个样本库的CK-MM相对比例高度相关。根据NYS和RTI在其试点研究中确定的参考范围,这些人工合成的DBS共同涵盖了典型新生儿中发现的CK-MM范围以及与DMD相关的升高范围。这组样本能够对典型新生儿和受DMD影响的新生儿中广泛波动的CK-MM水平进行质量评估。