Drug-induced lysosomal storage disease (DILSD) caused by cationic amphiphilic drugs (CADs), which exhibits toxic manifestations and pathological findings mimicking Fabry disease (α-galactosidase A deficiency), has attracted the interests of clinicians and pathologists. Although the affected region is lysosomes in both the diseases, DILSD is characterized by intralysosomal accumulation of phospholipids and Fabry disease that of globotriaosylceramide (Gb3) and globotriaosylsphingosine (Lyso-Gb3). However, it is unknown whether administration of CADs affects the catabolism of Gb3 and Lyso-Gb3 in Fabry disease. In this study, we independently administered hydroxychloroquine/amiodarone to wild-type and Fabry mice and examined the effects of the drugs on the enzyme activity and substrates accumulated in organs and tissues. The results revealed that the administration of the drugs induced accumulation of phosphatidylcholine in both the wild-type and Fabry mice. However, reduction of α-galactosidase A activity in the organs and tissues of the wild-type mice was not found, and the storage of Gb3 and Lyso-Gb3 was not accelerated by these drugs in the Fabry mice. This suggests that hydroxychloroquine/amiodarone do not have any significant impact on the catabolism of Gb3 and Lyso-Gb3 in organs and tissues of both wild-type and Fabry mice.

由阳离子两亲性药物（CADs）引起的药物性溶酶体贮积病（DILSD），其毒性表现和病理表现类似于法布里病（α-半乳糖苷酶A缺乏症），引起了临床医生和病理学家的兴趣。尽管在这两种疾病中受影响的区域都是溶酶体，但 DILSD 的特征是磷脂在溶酶体内积累，而法布里病则是球三糖神经酰胺 (Gb3) 和球三糖神经鞘氨醇 (Lyso-Gb3)。然而，尚不清楚给予 CAD 是否会影响 Fabry 病中 Gb3 和 Lyso-Gb3 的分解代谢。在这项研究中，我们独立地向野生型和法布里小鼠给药羟氯喹/胺碘酮，并检查了药物对酶活性和器官和组织中积累的底物的影响。结果表明，在野生型和法布里小鼠中，给药这些药物均诱导了磷脂酰胆碱的积累。然而，未发现野生型小鼠器官和组织中 α-半乳糖苷酶 A 活性的降低，并且这些药物在 Fabry 小鼠中没有加速 Gb3 和 Lyso-Gb3 的储存。这表明羟氯喹/胺碘酮对野生型和法布里小鼠的器官和组织中 Gb3 和 Lyso-Gb3 的分解代谢没有任何显着影响。