Tanaka Masato, Kobayashi Akira, Kuwabara Haruhiro, Nirei Jun, Ozawa Junichi, Sawano Kentaro, Shibata Nao, Nagasaki Keisuke, Saitoh Akihiko
Department of Pediatrics, Niigata University Medical and Dental Hospital, Niigata 951-8514, Japan.
Department of Pediatrics, Niigata University Graduate School of Medical and Dental Sciences, Niigata 951-8514, Japan.
Children (Basel). 2024 Sep 27;11(10):1176. doi: 10.3390/children11101176.
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.
婴儿期全身性动脉钙化(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会导致严重高血压和心力衰竭,约50%的患者在出生后6个月内死亡。特别是患有GACI的早产儿常因心功能不成熟而死亡。双膦酸盐对治疗GACI有效;然而,目前尚无标准化的治疗方案。我们遇到一例孕30周出生的患有GACI的早产儿。超声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,从而诊断为GACI。我们给予静脉注射帕米膦酸盐,她的心脏收缩功能得到改善。最初预定的给药间隔为2个月。然而,在帕米膦酸盐给药1个月后心脏收缩功能恶化。因此,我们缩短了给药间隔并给予第二疗程的帕米膦酸盐,这改善了她的心脏功能。然后我们改用口服依替膦酸盐。为了提高患有GACI的早产儿的发病率和死亡率,通过检查动脉中的高强度病变并尽早给予适当类型的双膦酸盐来早期诊断GACI非常重要。