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

被引：11
作者：
Brener, Avivit [1 ,2 ,3 ]
Lebenthal, Yael [1 ,2 ,3 ]
Interator, Hagar [2 ,3 ,4 ]
Horesh, Orit [1 ]
Leshem, Avital [5 ]
Weintrob, Naomi [2 ,3 ]
Loewenthal, Neta [6 ,7 ]
Shalitin, Shlomit [1 ,3 ]
Rachmiel, Marianna [3 ,5 ]
机构：
[1] Schneider Childrens Med Ctr, Natl Ctr Childhood Diabet, Jesse Z & Sara Lea Shafer Inst Endocrinol & Diabe, IL-49202 Petah Tiqwa, Israel
[2] Tel Aviv Sourasky Med Ctr, Dana Dwek Childrens Hosp, Pediat Endocrinol & Diabet Unit, IL-64239 Tel Aviv, Israel
[3] Tel Aviv Univ, Sackler Fac Med, IL-69978 Tel Aviv, Israel
[4] Tel Aviv Sourasky Med Ctr, Nutr & Dietet Unit, IL-64239 Tel Aviv, Israel
[5] Assaf Harofeh Med Ctr, Pediat Diabet Serv, IL-70300 Zerifin, Israel
[6] Soroka Med Ctr, Pediat Diabet Unit, IL-84101 Beer Sheva, Israel
[7] Ben Gurion Univ Negev, Fac Hlth Sci, IL-84105 Beer Sheva, Israel
来源：
IMMUNOTHERAPY | 2018年 / 10卷 / 13期
关键词：
alpha-1; antitrypsin; beta cell preservation; children and adolescents; extension study; immunomodulation; safety; Type; 1; diabetes; BETA-CELL FUNCTION; GLYCEMIC CONTROL; ONSET; DISEASE; TRIAL; ALPHA(1)-ANTITRYPSIN; TRANSPLANTATION; HYPOGLYCEMIA; RESPONSES; MELLITUS;
D O I：
10.2217/imt-2018-0047
中图分类号：
R392 [医学免疫学]; Q939.91 [免疫学];
学科分类号：
100102 ;
摘要：
Promising findings of alpha-1 antitrypsin (AAT) intervention in mice models of Type 1 diabetes (T1D) led researchers to investigate AAT as a therapeutic modality for beta-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.
引用
收藏
页码：1137 / 1148
页数：12
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