Department of Gastroenterology, Children's Hospital of Fudan University, Shanghai, China.
Translational Gastroenterology Unit, University of Oxford, Oxford, UK.
J Pediatr Gastroenterol Nutr. 2021 Feb 1;72(2):276-281. doi: 10.1097/MPG.0000000000002937.
Advances in genetic technologies provide opportunities for patient care and ethical challenges. Clinical care of patients with rare Mendelian disorders is often at the forefront of those developments. Whereas in classical polygenic inflammatory bowel disease (IBD), the predictive value of genetic variants is very low, predictive prenatal genetic diagnosis can inform families at high risk of severe genetic disorders. Patients with IL-10 signalling defects because of pathogenic variants in IL10RA, Il10RB, and IL10 develop severe infantile onset inflammatory bowel disease that is completely penetrant and has a high morbidity and substantial mortality despite treatment.
We performed a survey among tertiary specialist paediatric centers of 10 countries on the utilization of predictive prenatal genetic diagnosis in IL-10 signalling defects. We retrospectively report prenatal genetics in a series of 8 families.
International variation in legislation, guidelines, expert opinion, as well as cultural and religious background of families and clinicians results in variable utilization of preimplantation and prenatal genetic testing for IL-10 signalling defects. Eleven referrals for prenatal diagnosis for IL-10 signalling defects were identified across 4 countries. We report on 8 families who underwent prenatal preimplantation monogenic testing after in vitro fertilization (n = 2) and/or by amniocentesis/chorion villus sampling (n = 6). A genetic diagnosis was established in 1 foetus and excluded in 7 foetuses (all IL10RA variants).
Prenatal genetic testing for IL10R-defects is feasible, yet the legal and ethical considerations are complex and controversial. In some countries, predictive genetics for IL-10-related signalling defects is entering clinical practice.
遗传技术的进步为患者护理和伦理挑战提供了机会。对患有罕见孟德尔疾病患者的临床护理通常处于这些发展的前沿。虽然在经典的多基因炎症性肠病(IBD)中,遗传变异的预测价值非常低,但预测性产前遗传诊断可以为患有严重遗传疾病高风险的家庭提供信息。由于 IL10RA、Il10RB 和 IL10 中的致病性变异,IL-10 信号缺陷的患者会发展为严重的婴儿期起病炎症性肠病,该病完全外显,尽管进行了治疗,但发病率和死亡率仍很高。
我们对 10 个国家的三级专科儿科中心进行了一项关于 IL-10 信号缺陷预测性产前遗传诊断利用情况的调查。我们回顾性报告了一系列 8 个家庭的产前遗传学情况。
立法、指南、专家意见、家庭和临床医生的文化和宗教背景的国际差异导致 IL-10 信号缺陷的植入前和产前遗传检测的利用情况存在差异。在 4 个国家共发现了 11 例 IL-10 信号缺陷的产前诊断转诊。我们报告了 8 个家庭,他们在体外受精后(n=2)和/或通过羊膜穿刺术/绒毛膜绒毛取样(n=6)进行了产前植入前单基因检测。在 1 个胎儿中建立了遗传诊断,在 7 个胎儿中排除了遗传诊断(均为 IL10RA 变异)。
IL10R 缺陷的产前基因检测是可行的,但法律和伦理考虑因素复杂且存在争议。在一些国家,针对 IL-10 相关信号缺陷的预测遗传学已进入临床实践。