Efficacy of Cord Blood Cell Therapy for Hutchinson–Gilford Progeria Syndrome—A Case Report

Hutchinson–Gilford progeria syndrome (HGPS) is an extremely rare premature aging disorder characterized by short stature and atherosclerosis-induced death within teenage years. A 13-year-old male diagnosed with HGPS was administered three intravenous infusions of allogeneic cord blood (CB) cells fro...

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Autores principales:	Mi Ri Suh, Ikhyun Lim, Jongwook Kim, Pil-Sung Yang, Jin Seung Choung, Hye Ryeong Sim, Sung Chan Ha, MinYoung Kim
Formato:	article
Lenguaje:	EN
Publicado:	MDPI AG 2021
Materias:	cord blood cell therapy Hutchinson–Gilford progeria syndrome inflammation atherosclerosis Biology (General) QH301-705.5 Chemistry QD1-999
Acceso en línea:	https://doaj.org/article/94927f4d29bf4d7c9871f508d0d1e0b6
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spelling	oai:doaj.org-article:94927f4d29bf4d7c9871f508d0d1e0b62021-11-25T17:55:20ZEfficacy of Cord Blood Cell Therapy for Hutchinson–Gilford Progeria Syndrome—A Case Report10.3390/ijms2222123161422-00671661-6596https://doaj.org/article/94927f4d29bf4d7c9871f508d0d1e0b62021-11-01T00:00:00Zhttps://www.mdpi.com/1422-0067/22/22/12316https://doaj.org/toc/1661-6596https://doaj.org/toc/1422-0067Hutchinson–Gilford progeria syndrome (HGPS) is an extremely rare premature aging disorder characterized by short stature and atherosclerosis-induced death within teenage years. A 13-year-old male diagnosed with HGPS was administered three intravenous infusions of allogeneic cord blood (CB) cells from unrelated donors at four-month intervals to evaluate the safety and its therapeutic efficacy. Adverse events were monitored in addition to height, weight, laboratory blood tests, joint range of motion (ROM), and carotid Doppler. Cytokine and receptor assays were also performed. The patient exhibited an increase in growth rate for both height and weight. One year after therapy initiation, evident amelioration in pulse wave velocity, bilateral maximal intima-media thickness, and dyslipidemic status were observed, which were in abrupt aggravation prior to treatment. Further, an increase in flexibility occurred in some joints of the upper extremities. No serious adverse events were observed throughout the study period and one year beyond. A molecular assay revealed downregulation of proinflammatory and atherosclerosis, representing cytokine expressions following the administration of CB cells. This is the first reported case of an allogeneic CB trial in a patient with HGPS showing therapeutic effects of CB with improvements in anthropometric measures, joint ROM with amelioration of atherosclerosis, and dyslipidemia induced by anti-inflammatory and anti-atherosclerotic responses.Mi Ri SuhIkhyun LimJongwook KimPil-Sung YangJin Seung ChoungHye Ryeong SimSung Chan HaMinYoung KimMDPI AGarticlecord blood cell therapyHutchinson–Gilford progeria syndromeinflammationatherosclerosisBiology (General)QH301-705.5ChemistryQD1-999ENInternational Journal of Molecular Sciences, Vol 22, Iss 12316, p 12316 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	cord blood cell therapy Hutchinson–Gilford progeria syndrome inflammation atherosclerosis Biology (General) QH301-705.5 Chemistry QD1-999
spellingShingle	cord blood cell therapy Hutchinson–Gilford progeria syndrome inflammation atherosclerosis Biology (General) QH301-705.5 Chemistry QD1-999 Mi Ri Suh Ikhyun Lim Jongwook Kim Pil-Sung Yang Jin Seung Choung Hye Ryeong Sim Sung Chan Ha MinYoung Kim Efficacy of Cord Blood Cell Therapy for Hutchinson–Gilford Progeria Syndrome—A Case Report
description	Hutchinson–Gilford progeria syndrome (HGPS) is an extremely rare premature aging disorder characterized by short stature and atherosclerosis-induced death within teenage years. A 13-year-old male diagnosed with HGPS was administered three intravenous infusions of allogeneic cord blood (CB) cells from unrelated donors at four-month intervals to evaluate the safety and its therapeutic efficacy. Adverse events were monitored in addition to height, weight, laboratory blood tests, joint range of motion (ROM), and carotid Doppler. Cytokine and receptor assays were also performed. The patient exhibited an increase in growth rate for both height and weight. One year after therapy initiation, evident amelioration in pulse wave velocity, bilateral maximal intima-media thickness, and dyslipidemic status were observed, which were in abrupt aggravation prior to treatment. Further, an increase in flexibility occurred in some joints of the upper extremities. No serious adverse events were observed throughout the study period and one year beyond. A molecular assay revealed downregulation of proinflammatory and atherosclerosis, representing cytokine expressions following the administration of CB cells. This is the first reported case of an allogeneic CB trial in a patient with HGPS showing therapeutic effects of CB with improvements in anthropometric measures, joint ROM with amelioration of atherosclerosis, and dyslipidemia induced by anti-inflammatory and anti-atherosclerotic responses.
format	article
author	Mi Ri Suh Ikhyun Lim Jongwook Kim Pil-Sung Yang Jin Seung Choung Hye Ryeong Sim Sung Chan Ha MinYoung Kim
author_facet	Mi Ri Suh Ikhyun Lim Jongwook Kim Pil-Sung Yang Jin Seung Choung Hye Ryeong Sim Sung Chan Ha MinYoung Kim
author_sort	Mi Ri Suh
title	Efficacy of Cord Blood Cell Therapy for Hutchinson–Gilford Progeria Syndrome—A Case Report
title_short	Efficacy of Cord Blood Cell Therapy for Hutchinson–Gilford Progeria Syndrome—A Case Report
title_full	Efficacy of Cord Blood Cell Therapy for Hutchinson–Gilford Progeria Syndrome—A Case Report
title_fullStr	Efficacy of Cord Blood Cell Therapy for Hutchinson–Gilford Progeria Syndrome—A Case Report
title_full_unstemmed	Efficacy of Cord Blood Cell Therapy for Hutchinson–Gilford Progeria Syndrome—A Case Report
title_sort	efficacy of cord blood cell therapy for hutchinson–gilford progeria syndrome—a case report
publisher	MDPI AG
publishDate	2021
url	https://doaj.org/article/94927f4d29bf4d7c9871f508d0d1e0b6
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Efficacy of Cord Blood Cell Therapy for Hutchinson–Gilford Progeria Syndrome—A Case Report

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