TGFβ level in healthy and children with Marfan syndrome—effective reduction under sartan therapy
IntroductionTransforming growth factor β (TGFβ) metabolism plays an important role in the pathogenesis of Marfan syndrome (MFS). Accordingly, drug therapy uses TGFβ receptor blockade to slow down the cardiovascular manifestations, above all aortic root dilatation. Angiotensin II type 1 receptor bloc...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2024-02-01
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| Series: | Frontiers in Pediatrics |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fped.2024.1276215/full |
| _version_ | 1797324298227023872 |
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| author | Veronika C. Stark Jakob Olfe Daniel Diaz-Gil Daniel Diaz-Gil Yskert von Kodolitsch Rainer Kozlik-Feldmann Johannes Reincke Maria Stark Peter Wiegand Tanja Zeller Tanja Zeller Thomas S. Mir |
| author_facet | Veronika C. Stark Jakob Olfe Daniel Diaz-Gil Daniel Diaz-Gil Yskert von Kodolitsch Rainer Kozlik-Feldmann Johannes Reincke Maria Stark Peter Wiegand Tanja Zeller Tanja Zeller Thomas S. Mir |
| author_sort | Veronika C. Stark |
| collection | DOAJ |
| description | IntroductionTransforming growth factor β (TGFβ) metabolism plays an important role in the pathogenesis of Marfan syndrome (MFS). Accordingly, drug therapy uses TGFβ receptor blockade to slow down the cardiovascular manifestations, above all aortic root dilatation. Angiotensin II type 1 receptor blockers (ARBs) have been shown to reduce TGFβ levels in adults. Data on childhood are lacking and are now being investigated in the TiGer For Kids study presented here.MethodsWe examined 125 children without chronic disease and 31 pediatric Marfan patients with a proven FBN1 variant with regard to TGFβ levels. In addition, we measured TGFβ levels during the initiation of ARB therapy in pediatric Marfan patients.ResultsIn children without chronic disease, TGFβ levels were found to decrease from childhood to adolescence (p < 0.0125). We could not measure a relevantly increased TGFβ level in pediatric Marfan patients. However, we showed a significant suppression of the TGFβ level after treatment with ARBs (p < 0.0125) and a renewed increase shortly before the next dose.DiscussionThe TGFβ level in childhood changes in an age-dependent manner and decreases with age. The TGFβ level drops significantly after taking ARBs. Based on our experience and data, a TGFβ receptor blockade in childhood seems reasonable. So far, TGFβ level cannot be used as an MFS screening biomarker. |
| first_indexed | 2024-03-08T05:54:03Z |
| format | Article |
| id | doaj.art-dcd0c6f19cb2415fbf4f173e5b957b85 |
| institution | Directory Open Access Journal |
| issn | 2296-2360 |
| language | English |
| last_indexed | 2024-03-08T05:54:03Z |
| publishDate | 2024-02-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pediatrics |
| spelling | doaj.art-dcd0c6f19cb2415fbf4f173e5b957b852024-02-05T04:58:28ZengFrontiers Media S.A.Frontiers in Pediatrics2296-23602024-02-011210.3389/fped.2024.12762151276215TGFβ level in healthy and children with Marfan syndrome—effective reduction under sartan therapyVeronika C. Stark0Jakob Olfe1Daniel Diaz-Gil2Daniel Diaz-Gil3Yskert von Kodolitsch4Rainer Kozlik-Feldmann5Johannes Reincke6Maria Stark7Peter Wiegand8Tanja Zeller9Tanja Zeller10Thomas S. Mir11Clinic for Pediatric Heart Medicine and Adults with Congenital Heart Disease, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyClinic for Pediatric Heart Medicine and Adults with Congenital Heart Disease, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyClinic for Pediatric Heart Medicine and Adults with Congenital Heart Disease, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyDepartment of Cardiac Surgery & Department of Pediatric, Boston Children’s Hospital/ Harvard Medical School, Boston, MA, United StatesDepartment of Cardiology, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyClinic for Pediatric Heart Medicine and Adults with Congenital Heart Disease, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyClinic for Pediatric Heart Medicine and Adults with Congenital Heart Disease, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyInstitute of Medical Biometry and Epidemiology, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyClinic for Pediatric Heart Medicine and Adults with Congenital Heart Disease, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyDepartment of General and Interventional Cardiology, University Center of Cardiovascular Science, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyDeutsches Zentrum für Herzkreislaufforschung, Hamburg, GermanyClinic for Pediatric Heart Medicine and Adults with Congenital Heart Disease, University Heart and Vascular Center, University Medical Center Hamburg-Eppendorf, Hamburg, GermanyIntroductionTransforming growth factor β (TGFβ) metabolism plays an important role in the pathogenesis of Marfan syndrome (MFS). Accordingly, drug therapy uses TGFβ receptor blockade to slow down the cardiovascular manifestations, above all aortic root dilatation. Angiotensin II type 1 receptor blockers (ARBs) have been shown to reduce TGFβ levels in adults. Data on childhood are lacking and are now being investigated in the TiGer For Kids study presented here.MethodsWe examined 125 children without chronic disease and 31 pediatric Marfan patients with a proven FBN1 variant with regard to TGFβ levels. In addition, we measured TGFβ levels during the initiation of ARB therapy in pediatric Marfan patients.ResultsIn children without chronic disease, TGFβ levels were found to decrease from childhood to adolescence (p < 0.0125). We could not measure a relevantly increased TGFβ level in pediatric Marfan patients. However, we showed a significant suppression of the TGFβ level after treatment with ARBs (p < 0.0125) and a renewed increase shortly before the next dose.DiscussionThe TGFβ level in childhood changes in an age-dependent manner and decreases with age. The TGFβ level drops significantly after taking ARBs. Based on our experience and data, a TGFβ receptor blockade in childhood seems reasonable. So far, TGFβ level cannot be used as an MFS screening biomarker.https://www.frontiersin.org/articles/10.3389/fped.2024.1276215/fullMarfanTGFβsartanconnective tissue disordergenetic aortic diseaseaortic dilatation |
| spellingShingle | Veronika C. Stark Jakob Olfe Daniel Diaz-Gil Daniel Diaz-Gil Yskert von Kodolitsch Rainer Kozlik-Feldmann Johannes Reincke Maria Stark Peter Wiegand Tanja Zeller Tanja Zeller Thomas S. Mir TGFβ level in healthy and children with Marfan syndrome—effective reduction under sartan therapy Frontiers in Pediatrics Marfan TGFβ sartan connective tissue disorder genetic aortic disease aortic dilatation |
| title | TGFβ level in healthy and children with Marfan syndrome—effective reduction under sartan therapy |
| title_full | TGFβ level in healthy and children with Marfan syndrome—effective reduction under sartan therapy |
| title_fullStr | TGFβ level in healthy and children with Marfan syndrome—effective reduction under sartan therapy |
| title_full_unstemmed | TGFβ level in healthy and children with Marfan syndrome—effective reduction under sartan therapy |
| title_short | TGFβ level in healthy and children with Marfan syndrome—effective reduction under sartan therapy |
| title_sort | tgfβ level in healthy and children with marfan syndrome effective reduction under sartan therapy |
| topic | Marfan TGFβ sartan connective tissue disorder genetic aortic disease aortic dilatation |
| url | https://www.frontiersin.org/articles/10.3389/fped.2024.1276215/full |
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