Rizzo Manuela, Amicone Maria, Sellitti Maria Luigia, Marino Antonella, Sannino Anna, Capuano Ivana, Pisani Antonio
Dipartimento di Sanità Pubblica, Università degli Studi di Napoli "Federico II", Napoli, Italia.
G Ital Nefrol. 2023 Dec 22;40(6):2023-vol6.
Hyperprolinemia is a rare genetic condition due to mutations in proline metabolic pathway. Type I Hyperprolinemia (HPI) typically causes neuropsychiatric disorders, and diagnosis is usually confirmed in pediatric population with suggestive neuropsychiatric involvement by elevated serum proline levels and elevated urinary proline, hydroxyproline, and glycine levels. The possible coexistence of nephropathy in patients with HPI, often specified as malformative urinary disease, is often mentioned. However, reports of HPI diagnosis due to kidney impairment do not exist in scientific literature yet. Here we present the case of a patient presenting with chronic kidney disease secondary to obstructive nephropathy who received a HPI diagnosis in adulthood. Interestingly, the family study showed the same 22q11.21 deletion and elevated blood proline levels in the father, who had no clinical anomalies. We therefore suggest, in light of the high frequency of mutations involving 22q11 and PRODH in the general population, to consider these rare alterations in patients with congenital urinary malformations, even in the presence of nuanced neurological symptoms and negative family history.
高脯氨酸血症是一种由于脯氨酸代谢途径突变引起的罕见遗传病。I型高脯氨酸血症(HPI)通常会导致神经精神障碍,通常在血清脯氨酸水平升高以及尿脯氨酸、羟脯氨酸和甘氨酸水平升高提示有神经精神受累的儿科人群中确诊。HPI患者中常被称为畸形性泌尿系统疾病的肾病可能并存,这一点常被提及。然而,科学文献中尚未有因肾功能损害而诊断为HPI的报道。在此,我们报告一例成年患者,因梗阻性肾病继发慢性肾脏病而被诊断为HPI。有趣的是,家族研究显示,父亲存在相同的22q11.21缺失且血脯氨酸水平升高,但他没有临床异常。因此,鉴于普通人群中涉及22q11和脯氨酸脱氢酶(PRODH)的突变频率较高,我们建议,对于先天性泌尿系统畸形患者,即使存在细微的神经症状且家族史阴性,也应考虑这些罕见的变异。