Maeda Akiko, Sakai Daiki, Yokota Satoshi, Yamamoto Midori, Sugita Sunao, Hirami Yasuhiko, Mandai Michiko, Maeda Tadao, Takahashi Masayo, Kurimoto Yasuo
Kobe City Eye Hospital, Japan.
Kobe City General Hospital, Japan.
Am J Ophthalmol Case Rep. 2025 Jul 9;39:102383. doi: 10.1016/j.ajoc.2025.102383. eCollection 2025 Sep.
To evaluate the safety and therapeutic effects of induced pluripotent stem (iPS) cell-derived retinal pigment epithelium (RPE) transplantation for -associated Leber congenital amaurosis (5-LCA).
A 46-year-old male patient with 5-LCA underwent allogeneic iPS cell-derived RPE transplantation. The patient's best-corrected visual acuity (VA) prior to treatment was 2.0 (logMAR). A cell suspension of iPS cell-derived RPE was transplanted into the subretinal space. On day 15 post-transplantation, intraocular pressure (IOP) increased to 46 mmHg due to local steroid treatment, resulting in a decrease in VA to light perception (LP). Retinal imaging on day 71 revealed that most transplanted cells had migrated and formed an epiretinal membrane (ERM). The ERM was surgically removed on day 112. Two years post-transplantation, the patient reported improved vision, with VA improving to 1.4 (logMAR) from LP. Full-field stimulus testing (FST) and microperimetry demonstrated increased retinal sensitivity. These improvements have been maintained for up to 4 years post-treatment.
Although this case raised safety concerns regarding the use of cell suspension for RPE transplantation, RPE transplantation may still serve as a potential therapeutic option for patients with -associated retinopathy, particularly those who are not eligible or older age for gene therapy.
评估诱导多能干细胞（iPS）来源的视网膜色素上皮（RPE）移植治疗5型莱伯先天性黑蒙（5-LCA）的安全性和治疗效果。
一名患有5-LCA的46岁男性患者接受了同种异体iPS细胞来源的RPE移植。治疗前患者的最佳矫正视力（VA）为2.0（logMAR）。将iPS细胞来源的RPE细胞悬液移植到视网膜下间隙。移植后第15天，由于局部类固醇治疗，眼压（IOP）升至46 mmHg，导致VA下降至光感（LP）。移植后第71天的视网膜成像显示，大多数移植细胞已迁移并形成视网膜前膜（ERM）。在第112天通过手术切除了ERM。移植后两年，患者报告视力有所改善，VA从LP提高到1.4（logMAR）。全视野刺激测试（FST）和微视野检查显示视网膜敏感性增加。这些改善在治疗后长达4年一直保持。
尽管该病例引发了对使用细胞悬液进行RPE移植的安全性担忧，但RPE移植对于5型相关视网膜病变患者，尤其是那些不符合基因治疗条件或年龄较大的患者，仍可能是一种潜在的治疗选择。
