Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy.

IF 2.4 Q1 PEDIATRICS Molecular and cellular pediatrics Pub Date : 2020-09-03 DOI:10.1186/s40348-020-00103-7
Judith Beschle, Michaela Döring, Christiane Kehrer, Christa Raabe, Ute Bayha, Manuel Strölin, Judith Böhringer, Andrea Bevot, Nadja Kaiser, Benjamin Bender, Alexander Grimm, Peter Lang, Ingo Müller, Ingeborg Krägeloh-Mann, Samuel Groeschel
{"title":"Early clinical course after hematopoietic stem cell transplantation in children with juvenile metachromatic leukodystrophy.","authors":"Judith Beschle,&nbsp;Michaela Döring,&nbsp;Christiane Kehrer,&nbsp;Christa Raabe,&nbsp;Ute Bayha,&nbsp;Manuel Strölin,&nbsp;Judith Böhringer,&nbsp;Andrea Bevot,&nbsp;Nadja Kaiser,&nbsp;Benjamin Bender,&nbsp;Alexander Grimm,&nbsp;Peter Lang,&nbsp;Ingo Müller,&nbsp;Ingeborg Krägeloh-Mann,&nbsp;Samuel Groeschel","doi":"10.1186/s40348-020-00103-7","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Long-term outcomes of hematopoietic stem cell transplantation (HSCT) in children with juvenile metachromatic leukodystrophy (MLD) have been investigated systematically, while short-term effects of HSCT on the course of the disease remain to be elucidated.</p><p><strong>Results: </strong>In this study, the clinical course was evaluated over the first 24 months following HSCT, conducted at our center in 12 children with juvenile MLD (mean follow-up 6.75 years, range 3-13.5) and compared with 35 non-transplanted children with juvenile MLD. Motor function (GMFM-88 and GMFC-MLD), cognitive function (FSIQ), peripheral neuropathy (tibial nerve conduction velocity), and cerebral changes (MLD-MR severity score) were tested prospectively. Seven children remained neurologically stable over a long period, five exhibited rapid disease progression over the first 12 to 18 months after transplantation. In the latter, time from first gross motor symptoms to loss of independent walking was significantly shorter compared with non-transplanted patients at the same stage of disease (p < 0.02). Positive prognostic factors were good motor function (GMFM = 100%, GMFC-MLD = 0) and a low MR severity score (≤ 17) at the time of HSCT.</p><p><strong>Conclusions: </strong>Our results show that if disease progression occurs, this happens early on after HSCT and proceeds faster than in non-transplanted children with juvenile MLD, indicating that HSCT may trigger disease progression.</p>","PeriodicalId":74215,"journal":{"name":"Molecular and cellular pediatrics","volume":null,"pages":null},"PeriodicalIF":2.4000,"publicationDate":"2020-09-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1186/s40348-020-00103-7","citationCount":"17","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular and cellular pediatrics","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1186/s40348-020-00103-7","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 17

Abstract

Background: Long-term outcomes of hematopoietic stem cell transplantation (HSCT) in children with juvenile metachromatic leukodystrophy (MLD) have been investigated systematically, while short-term effects of HSCT on the course of the disease remain to be elucidated.

Results: In this study, the clinical course was evaluated over the first 24 months following HSCT, conducted at our center in 12 children with juvenile MLD (mean follow-up 6.75 years, range 3-13.5) and compared with 35 non-transplanted children with juvenile MLD. Motor function (GMFM-88 and GMFC-MLD), cognitive function (FSIQ), peripheral neuropathy (tibial nerve conduction velocity), and cerebral changes (MLD-MR severity score) were tested prospectively. Seven children remained neurologically stable over a long period, five exhibited rapid disease progression over the first 12 to 18 months after transplantation. In the latter, time from first gross motor symptoms to loss of independent walking was significantly shorter compared with non-transplanted patients at the same stage of disease (p < 0.02). Positive prognostic factors were good motor function (GMFM = 100%, GMFC-MLD = 0) and a low MR severity score (≤ 17) at the time of HSCT.

Conclusions: Our results show that if disease progression occurs, this happens early on after HSCT and proceeds faster than in non-transplanted children with juvenile MLD, indicating that HSCT may trigger disease progression.

Abstract Image

Abstract Image

Abstract Image

查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
儿童偏色差性脑白质营养不良患者造血干细胞移植后的早期临床过程。
背景:造血干细胞移植(HSCT)治疗青少年色差性脑白质营养不良(MLD)的长期结果已经被系统地研究过,而HSCT对该疾病病程的短期影响仍有待阐明。结果:在本研究中,我们对12例青少年MLD儿童(平均随访6.75年,范围3-13.5年)进行了HSCT后的前24个月的临床病程进行了评估,并与35例未移植的青少年MLD儿童进行了比较。前瞻性检测运动功能(GMFM-88和GMFC-MLD)、认知功能(FSIQ)、周围神经病变(胫神经传导速度)和大脑变化(MLD-MR严重程度评分)。7名儿童长期保持神经系统稳定,5名儿童在移植后的前12至18个月内表现出疾病的快速进展。后者从出现大运动症状到丧失独立行走的时间明显短于同阶段未移植患者(p < 0.02)。阳性预后因素为HSCT时良好的运动功能(GMFM = 100%, GMFC-MLD = 0)和低MR严重程度评分(≤17)。结论:我们的研究结果表明,如果发生疾病进展,这种进展发生在HSCT后早期,并且比未移植的少年MLD儿童更快,这表明HSCT可能引发疾病进展。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 去求助
来源期刊
CiteScore
2.20
自引率
0.00%
发文量
0
期刊最新文献
Early metabolic and hemodynamic indicators of kidney dysfunction in mice offspring from parental low protein diet. Fatty acids from nutrition sources for preterm infants and their effect on plasma fatty acid profiles. Description of bone health in adolescents and young persons with Klinefelter syndrome – results from a pilot study Monogenic lupus – from gene to targeted therapy B cell academy of the gut: an update on gut associated germinal centre B cell dynamics.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1