Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in New-Onset Type 1 Diabetes: A Multicenter Analysis
PBN-AR
Instytucja
Instytut Medycyny Doświadczalnej i Klinicznej im. Mirosława Mossakowskiego Polskiej Akademii Nauk
Informacje podstawowe
Główny język publikacji
en
Czasopismo
DIABETES
ISSN
0012-1797
EISSN
Wydawca
AMER DIABETES ASSOC
DOI
URL
Rok publikacji
2014
Numer zeszytu
9
Strony od-do
3041-3046
Numer tomu
63
Identyfikator DOI
Liczba arkuszy
0,73
Słowa kluczowe
en
PLACEBO-CONTROLLED TRIAL
TOLERANCE
MELLITUS
Streszczenia
Język
en
Treść
Type 1 diabetes (T1D) is one of the major autoimmune diseases affecting children and young adults worldwide. To date, the different immunotherapies tested have achieved insulin independence in <5\% of treated individuals. Recently, a novel hematopoietic stem cell (HSC)-based strategy has been tested in individuals with new-onset T1D. The aim of this study was to determine the effects of autologous nonmyeloablative HSC transplantation in 65 individuals with new-onset T1D who were enrolled in two Chinese centers and one Polish center, pooled, and followed up for 48 months. A total of 59\% of individuals with T1D achieved insulin independence within the first 6 months after receiving conditioning immunosuppression therapy (with antithymocyte globulin and cyclophosphamide) and a single infusion of autologous HSCs, and 32\% remained insulin independent at the last time point of their follow-up. All treated subjects showed a decrease in HbA(1c) levels and an increase in C-peptide levels compared with pretreatment. Despite a complete immune system recovery (i.e., leukocyte count) after treatment, 52\% of treated individuals experienced adverse effects. Our study suggests the following: 1) that remission of T1D is possible by combining HSC transplantation and immunosuppression; 2) that autologous nonmyeloablative HSC transplantation represents an effective treatment for selected individuals with T1D; and 3) that safer HSC-based therapeutic options are required.
Cechy publikacji
ORIGINAL_ARTICLE
Inne
System-identifier
590676
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