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肺高血压国家转诊中心的遗传咨询。

Genetic counselling in a national referral centre for pulmonary hypertension.

机构信息

Université Paris Sud, Faculté de Médecine, Université Paris-Saclay, Le Kremlin Bicêtre, France AP-HP, Centre de Référence de l'Hypertension Pulmonaire Sévère, Département Hospitalo-Universitaire (DHU) Thorax Innovation (TORINO), Service de Pneumologie, Hôpital de Bicêtre, Le Kremlin Bicêtre, France UMR_S999, Université Paris-Sud, INSERM, Laboratoire d'Excellence (LabEx) en Recherche sur le Médicament et l'Innovation Thérapeutique (LERMIT), Centre Chirurgical Marie Lannelongue, Le Plessis Robinson, France Both authors contributed equally to this work.

Université Paris Sud, Faculté de Médecine, Université Paris-Saclay, Le Kremlin Bicêtre, France AP-HP, Centre de Référence de l'Hypertension Pulmonaire Sévère, Département Hospitalo-Universitaire (DHU) Thorax Innovation (TORINO), Service de Pneumologie, Hôpital de Bicêtre, Le Kremlin Bicêtre, France UMR_S999, Université Paris-Sud, INSERM, Laboratoire d'Excellence (LabEx) en Recherche sur le Médicament et l'Innovation Thérapeutique (LERMIT), Centre Chirurgical Marie Lannelongue, Le Plessis Robinson, France.

出版信息

Eur Respir J. 2016 Feb;47(2):541-52. doi: 10.1183/13993003.00717-2015. Epub 2015 Dec 23.

Abstract

Genetic causes of pulmonary arterial hypertension (PAH) and pulmonary veno-occlusive disease (PVOD) have been identified, leading to a growing need for genetic counselling.Between 2003 and 2014, genetic counselling was offered to 529 PAH and 100 PVOD patients at the French Referral Centre for Pulmonary Hypertension.Mutations in PAH-predisposing genes were identified in 72 patients presenting as sporadic PAH (17% of cases; 62 mutations in BMPR2, nine in ACVRL1 (ALK1) and one in ENG) and in 94 patients with a PAH family history (89% of cases; 89 mutations in BMPR2, three in ACVRL1 (ALK1) and two in KCNK3). Bi-allelic mutations in EIF2AK4 were identified in all patients with a family history of PVOD (n=19) and in seven patients (8.6%) presenting as sporadic PVOD. Pre-symptomatic genetic diagnosis was offered to 272 relatives of heritable PAH patients, identifying mutations in 36.4% of them. A screening programme is now offered to asymptomatic mutation carriers to detect PAH in an early phase and to identify predictors of outcomes in asymptomatic BMPR2 mutation carriers. BMPR2 screening allowed us to offer pre-implantation diagnosis to two couples with a BMPR2 mutation.Genetic counselling can be implemented in pulmonary hypertension centres.

摘要

遗传性肺动脉高压(PAH)和肺静脉闭塞性疾病(PVOD)的病因已被确定,这导致对遗传咨询的需求不断增长。在 2003 年至 2014 年期间,法国肺动脉高压转诊中心为 529 名 PAH 患者和 100 名 PVOD 患者提供了遗传咨询。在作为散发性 PAH 出现的 72 名患者(占病例的 17%;62 个 BMPR2 突变,9 个 ACVRL1(ALK1)突变,1 个 ENG 突变)和有 PAH 家族史的 94 名患者(占病例的 89%;89 个 BMPR2 突变,3 个 ACVRL1(ALK1)突变,2 个 KCNK3 突变)中发现了 PAH 易感基因的突变。在所有有 PVOD 家族史的患者(n=19)和作为散发性 PVOD 出现的 7 名患者(8.6%)中均发现 EIF2AK4 的双等位基因突变。为遗传性 PAH 患者的 272 名亲属提供了预症状遗传诊断,其中 36.4%的亲属发现了突变。现在为无症状突变携带者提供了一项筛查计划,以在早期阶段发现 PAH,并确定无症状 BMPR2 突变携带者的预后预测因子。BMPR2 筛查使我们能够为一对携带 BMPR2 突变的夫妇提供胚胎植入前诊断。遗传咨询可以在肺动脉高压中心实施。

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