患有全身动脉钙化的早产儿因早期诊断和适当的双膦酸盐治疗而存活下来:病例报告。

IF 2 4区 医学 Q2 PEDIATRICS Children-Basel Pub Date : 2024-09-27 DOI:10.3390/children11101176
Masato Tanaka, Akira Kobayashi, Haruhiro Kuwabara, Jun Nirei, Junichi Ozawa, Kentaro Sawano, Nao Shibata, Keisuke Nagasaki, Akihiko Saitoh
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引用次数: 0

摘要

婴儿全身动脉钙化症(GACI)是一种以动脉钙化为特征的罕见疾病。GACI 由 ENPP1 或 ABCC6 基因突变引起。GACI 会导致严重的高血压和心力衰竭,大约 50% 的患者会在最初 6 个月内死亡。特别是,患有 GACI 的早产儿往往因心脏功能不成熟而死亡。双膦酸盐可有效治疗 GACI,但目前尚无标准化的治疗方案。我们接诊了一例妊娠 30 周的早产儿,患有 GACI。超声波检查显示动脉有高强度病变,计算机断层扫描显示全身动脉钙化,从而确诊为 GACI。我们为她静脉注射了帕米膦酸钠,她的心脏收缩情况有所改善。最初计划的用药间隔为 2 个月。然而,在服用帕米膦酸钠 1 个月后,她的心脏收缩情况恶化。因此,我们缩短了给药间隔,并服用了第二个疗程的帕米膦酸钠,这改善了她的心功能。随后,我们改用口服依替膦酸钠。为了改善患有 GACI 的早产儿的发病率和死亡率,通过检查动脉的高强度病变并及早使用适当类型的双膦酸盐来获得 GACI 的早期诊断非常重要。
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Preterm Infant with Generalized Arterial Calcification of Infancy Who Survived Due to Early Diagnosis and Appropriate Treatment with Bisphosphonates: A Case Report.

Generalized arterial calcification of infancy (GACI) is a rare disease characterized by arterial calcification. GACI is caused by a mutation in the ENPP1 or ABCC6 genes. GACI causes severe hypertension and heart failure, and approximately 50% of patients die within the first 6 months. In particular, preterm infants with GACI often die due to immature cardiac function. Bisphosphonates are effective in treating GACI; however, no standardized treatment regimen is available. We experienced a case of a preterm infant with GACI born at 30 weeks gestation. Ultrasonography showed high-intensity lesions in the arteries, and computed tomography revealed calcification of the arteries throughout the body, leading to the diagnosis of GACI. We administered intravenous pamidronate, and her cardiac contraction improved. The initial scheduled interval between drug administrations was 2 months. However, the cardiac contraction worsened 1 month after the pamidronate administration. Therefore, we decreased the dosing interval and administered a second course of pamidronate, which improved her cardiac function. We then switched to oral etidronate. To improve the morbidity and mortality rates of preterm infants with GACI, it is important to obtain an early diagnosis of GACI by investigating high-intensity lesions in the arteries and performing early administration of an appropriate type of bisphosphonate.

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来源期刊
Children-Basel
Children-Basel PEDIATRICS-
CiteScore
2.70
自引率
16.70%
发文量
1735
审稿时长
6 weeks
期刊介绍: Children is an international, open access journal dedicated to a streamlined, yet scientifically rigorous, dissemination of peer-reviewed science related to childhood health and disease in developed and developing countries. The publication focuses on sharing clinical, epidemiological and translational science relevant to children’s health. Moreover, the primary goals of the publication are to highlight under‑represented pediatric disciplines, to emphasize interdisciplinary research and to disseminate advances in knowledge in global child health. In addition to original research, the journal publishes expert editorials and commentaries, clinical case reports, and insightful communications reflecting the latest developments in pediatric medicine. By publishing meritorious articles as soon as the editorial review process is completed, rather than at predefined intervals, Children also permits rapid open access sharing of new information, allowing us to reach the broadest audience in the most expedient fashion.
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