Safety and feasibility of umbilical cord blood transplantation in children with neuronal ceroid lipofuscinosis: a retrospective study.

IF 5.4 2区 医学 Q1 CELL & TISSUE ENGINEERING Stem Cells Translational Medicine Pub Date : 2024-10-29 DOI:10.1093/stcltm/szae080
Andrea Bauchat, Veronika Polishchuk, Vanessa A Fabrizio, Jennifer E Brondon, Kristin M Page, Timothy A Driscoll, Paul L Martin, Kris M Mahadeo, Joanne Kurtzberg, Vinod K Prasad
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Abstract

Ceroid lipofuscinosis neuronal (CLN) encompasses rare inherited neurodegenerative disorders that present in childhood with clinical features including epilepsy, psychomotor delay, progressive vision loss, and premature death. Published experience utilizing umbilical cord blood transplant (UCBT) for these disorders is limited. This retrospective analysis includes patients with CLN (2, 3, and 5) who underwent UCBT from 2012 to 2020. All subjects (n = 8) received standard-of-care myeloablative conditioning. Four also enrolled in clinical trial NCT02254863 and received intrathecal DUOC-01 cells posttransplant. Median age at UCBT was 5.9 years. All subjects achieved neutrophil engraftment with >95% donor chimerism at a median of 28.5 days. Sinusoidal obstructive syndrome was not observed. Severe acute graft-versus-host disease occurred in 12.5%. Other complications included autoimmune hemolytic anemia (25%) and viral reactivation/infection (62.5%). No transplant-related mortality was observed. Two CLN2 patients died, 1 from progressive disease and 1 from unknown cause at days +362 and +937, respectively. With median follow-up of 8 years, overall survival at 100 days and 24 months was 100% and 88%, respectively. Three of 4 CLN3 subjects stabilized Hamburg motor and language scores. While UCBT appears safe and feasible in these patients, given the variable expression and natural history, extended follow-up and further studies are needed to elucidate the potential impact of UCBT on clinical outcomes.

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神经细胞类脂膜炎患儿脐带血移植的安全性和可行性:一项回顾性研究。
类脂质神经病(CLN)是一种罕见的遗传性神经退行性疾病,在儿童期发病,临床特征包括癫痫、精神运动发育迟缓、进行性视力丧失和过早死亡。已发表的利用脐带血移植(UCBT)治疗这些疾病的经验非常有限。本回顾性分析包括2012年至2020年期间接受脐带血移植的CLN(2、3和5)患者。所有受试者(n = 8)均接受了标准护理的骨髓溶解调理。其中四人还参加了 NCT02254863 临床试验,并在移植后接受了鞘内 DUOC-01 细胞治疗。UCBT时的中位年龄为5.9岁。所有受试者在中位28.5天时都实现了中性粒细胞移植,供体嵌合率大于95%。未观察到窦道阻塞综合征。12.5%的受试者出现了严重的急性移植物抗宿主疾病。其他并发症包括自身免疫性溶血性贫血(25%)和病毒再激活/感染(62.5%)。未观察到与移植相关的死亡率。两名CLN2患者分别在+362天和+937天因疾病进展和不明原因死亡。中位随访时间为 8 年,100 天和 24 个月的总存活率分别为 100% 和 88%。4 名 CLN3 受试者中有 3 人的汉堡运动和语言评分趋于稳定。虽然 UCBT 在这些患者中似乎是安全可行的,但鉴于其表现和自然病史的多变性,还需要延长随访时间并开展进一步研究,以阐明 UCBT 对临床结果的潜在影响。
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来源期刊
Stem Cells Translational Medicine
Stem Cells Translational Medicine CELL & TISSUE ENGINEERING-
CiteScore
12.90
自引率
3.30%
发文量
140
审稿时长
6-12 weeks
期刊介绍: STEM CELLS Translational Medicine is a monthly, peer-reviewed, largely online, open access journal. STEM CELLS Translational Medicine works to advance the utilization of cells for clinical therapy. By bridging stem cell molecular and biological research and helping speed translations of emerging lab discoveries into clinical trials, STEM CELLS Translational Medicine will help move applications of these critical investigations closer to accepted best patient practices and ultimately improve outcomes. The journal encourages original research articles and concise reviews describing laboratory investigations of stem cells, including their characterization and manipulation, and the translation of their clinical aspects of from the bench to patient care. STEM CELLS Translational Medicine covers all aspects of translational cell studies, including bench research, first-in-human case studies, and relevant clinical trials.
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