Long-term safety of α-1 antitrypsin therapy in children and adolescents with Type 1 diabetes

Avivit Brener; Yael Lebenthal; Hagar Interator; Orit Horesh; Avital Leshem; Naomi Weintrob; Neta Loewenthal; Shlomit Shalitin; Marianna Rachmiel

doi:10.2217/imt-2018-0047

Long-term safety of α-1 antitrypsin therapy in children and adolescents with Type 1 diabetes

Avivit Brener, Yael Lebenthal^*, Hagar Interator, Orit Horesh, Avital Leshem, Naomi Weintrob, Neta Loewenthal, Shlomit Shalitin, Marianna Rachmiel

^*Corresponding author for this work

Research output: Contribution to journal › Article › peer-review

13 Scopus citations

Abstract

Promising findings of α-1 antitrypsin (AAT) intervention in mice models of Type 1 diabetes (T1D) led researchers to investigate AAT as a therapeutic modality for β-cell preservation in recent-onset T1D patients. Our prospective, open-label Phase I/II extension study demonstrated that the administration of multiple repeated AAT infusions (up to 36) to AAT-sufficient pediatric T1D patients is safe and well-tolerated. Long-term surveillance of participants (up to 5 years) from diabetes onset revealed normal growth and pubertal progression through adolescence to attainment of full puberty and near adult height. No serious adverse events, clinical or laboratory abnormalities were reported. Given its safety profile, AAT may be an individualized-tailored innovative immunotherapy in AAT-sufficient pediatric patients with diverse immune-related medical conditions. ClinicalTrials.gov Identifier: NCT0166119.

Original language	English
Pages (from-to)	1137-1148
Number of pages	12
Journal	Immunotherapy
Volume	10
Issue number	13
DOIs	https://doi.org/10.2217/imt-2018-0047
State	Published - Sep 2018

Keywords

Type 1 diabetes
children and adolescents
extension study
immunomodulation
safety
α-1 antitrypsin
β cell preservation

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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title = "Long-term safety of α-1 antitrypsin therapy in children and adolescents with Type 1 diabetes",

abstract = "Promising findings of α-1 antitrypsin (AAT) intervention in mice models of Type 1 diabetes (T1D) led researchers to investigate AAT as a therapeutic modality for β-cell preservation in recent-onset T1D patients. Our prospective, open-label Phase I/II extension study demonstrated that the administration of multiple repeated AAT infusions (up to 36) to AAT-sufficient pediatric T1D patients is safe and well-tolerated. Long-term surveillance of participants (up to 5 years) from diabetes onset revealed normal growth and pubertal progression through adolescence to attainment of full puberty and near adult height. No serious adverse events, clinical or laboratory abnormalities were reported. Given its safety profile, AAT may be an individualized-tailored innovative immunotherapy in AAT-sufficient pediatric patients with diverse immune-related medical conditions. ClinicalTrials.gov Identifier: NCT0166119.",

keywords = "Type 1 diabetes, children and adolescents, extension study, immunomodulation, safety, α-1 antitrypsin, β cell preservation",

author = "Avivit Brener and Yael Lebenthal and Hagar Interator and Orit Horesh and Avital Leshem and Naomi Weintrob and Neta Loewenthal and Shlomit Shalitin and Marianna Rachmiel",

note = "Publisher Copyright: {\textcopyright} 2018 Future Medicine Ltd.",

year = "2018",

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doi = "10.2217/imt-2018-0047",

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AU - Brener, Avivit

AU - Lebenthal, Yael

AU - Interator, Hagar

AU - Horesh, Orit

AU - Leshem, Avital

AU - Weintrob, Naomi

AU - Loewenthal, Neta

AU - Shalitin, Shlomit

AU - Rachmiel, Marianna

PY - 2018/9

Y1 - 2018/9

N2 - Promising findings of α-1 antitrypsin (AAT) intervention in mice models of Type 1 diabetes (T1D) led researchers to investigate AAT as a therapeutic modality for β-cell preservation in recent-onset T1D patients. Our prospective, open-label Phase I/II extension study demonstrated that the administration of multiple repeated AAT infusions (up to 36) to AAT-sufficient pediatric T1D patients is safe and well-tolerated. Long-term surveillance of participants (up to 5 years) from diabetes onset revealed normal growth and pubertal progression through adolescence to attainment of full puberty and near adult height. No serious adverse events, clinical or laboratory abnormalities were reported. Given its safety profile, AAT may be an individualized-tailored innovative immunotherapy in AAT-sufficient pediatric patients with diverse immune-related medical conditions. ClinicalTrials.gov Identifier: NCT0166119.

AB - Promising findings of α-1 antitrypsin (AAT) intervention in mice models of Type 1 diabetes (T1D) led researchers to investigate AAT as a therapeutic modality for β-cell preservation in recent-onset T1D patients. Our prospective, open-label Phase I/II extension study demonstrated that the administration of multiple repeated AAT infusions (up to 36) to AAT-sufficient pediatric T1D patients is safe and well-tolerated. Long-term surveillance of participants (up to 5 years) from diabetes onset revealed normal growth and pubertal progression through adolescence to attainment of full puberty and near adult height. No serious adverse events, clinical or laboratory abnormalities were reported. Given its safety profile, AAT may be an individualized-tailored innovative immunotherapy in AAT-sufficient pediatric patients with diverse immune-related medical conditions. ClinicalTrials.gov Identifier: NCT0166119.

KW - Type 1 diabetes

KW - children and adolescents

KW - extension study

KW - immunomodulation

KW - safety

KW - α-1 antitrypsin

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Long-term safety of α-1 antitrypsin therapy in children and adolescents with Type 1 diabetes

Abstract

Keywords

UN SDGs

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