Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in New-Onset Type 1 Diabetes: A Multicenter Analysis
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
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| Veröffentlicht in: | Diabetes (New York, N.Y.) N.Y.), 2014-09, Vol.63 (9), p.3041-3046 |
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| container_title | Diabetes (New York, N.Y.) |
| container_volume | 63 |
| creator | D'ADDIO, Francesca VASQUEZ, Alessandro Valderrama BEN NASR, Moufida FRANEK, Edward DALONG ZHU LIRONG LI GUANG NING SNARSKI, Emilian FIORINA, Paolo |
| description | 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 |
| doi_str_mv | 10.2337/db14-0295 |
| format | Article |
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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 HbA1c 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.</description><identifier>ISSN: 0012-1797</identifier><identifier>EISSN: 1939-327X</identifier><identifier>DOI: 10.2337/db14-0295</identifier><identifier>PMID: 24947362</identifier><identifier>CODEN: DIAEAZ</identifier><language>eng</language><publisher>Alexandria, VA: American Diabetes Association</publisher><subject>Adolescent ; Adult ; Autoimmune diseases ; Biological and medical sciences ; C-Peptide - metabolism ; Child ; Children & youth ; Cyclophosphamide - therapeutic use ; Diabetes Mellitus, Type 1 - therapy ; Diabetes. Impaired glucose tolerance ; Endocrine pancreas. Apud cells (diseases) ; Endocrinopathies ; Etiopathogenesis. Screening. Investigations. Target tissue resistance ; Female ; Hematopoietic Stem Cell Transplantation - adverse effects ; Humans ; Immune system ; Immunosuppression ; Immunotherapy ; Insulin - therapeutic use ; Male ; Medical sciences ; Peptides ; Remission Induction ; Stem cells ; Transplantation, Autologous ; Transplants & implants</subject><ispartof>Diabetes (New York, N.Y.), 2014-09, Vol.63 (9), p.3041-3046</ispartof><rights>2015 INIST-CNRS</rights><rights>2014 by the American Diabetes Association. Readers may use this article as long as the work is properly cited, the use is educational and not for profit, and the work is not altered.</rights><rights>Copyright American Diabetes Association Sep 2014</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c444t-d9cef63e03c1af73404a64bdb643b6a903a3c113cc47e87d11ca006827557e953</citedby><cites>FETCH-LOGICAL-c444t-d9cef63e03c1af73404a64bdb643b6a903a3c113cc47e87d11ca006827557e953</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>314,776,780,27901,27902</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=28807564$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/24947362$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>D'ADDIO, Francesca</creatorcontrib><creatorcontrib>VASQUEZ, Alessandro Valderrama</creatorcontrib><creatorcontrib>BEN NASR, Moufida</creatorcontrib><creatorcontrib>FRANEK, Edward</creatorcontrib><creatorcontrib>DALONG ZHU</creatorcontrib><creatorcontrib>LIRONG LI</creatorcontrib><creatorcontrib>GUANG NING</creatorcontrib><creatorcontrib>SNARSKI, Emilian</creatorcontrib><creatorcontrib>FIORINA, Paolo</creatorcontrib><title>Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in New-Onset Type 1 Diabetes: A Multicenter Analysis</title><title>Diabetes (New York, N.Y.)</title><addtitle>Diabetes</addtitle><description>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 HbA1c 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.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Autoimmune diseases</subject><subject>Biological and medical sciences</subject><subject>C-Peptide - metabolism</subject><subject>Child</subject><subject>Children & youth</subject><subject>Cyclophosphamide - therapeutic use</subject><subject>Diabetes Mellitus, Type 1 - therapy</subject><subject>Diabetes. Impaired glucose tolerance</subject><subject>Endocrine pancreas. Apud cells (diseases)</subject><subject>Endocrinopathies</subject><subject>Etiopathogenesis. Screening. Investigations. Target tissue resistance</subject><subject>Female</subject><subject>Hematopoietic Stem Cell Transplantation - adverse effects</subject><subject>Humans</subject><subject>Immune system</subject><subject>Immunosuppression</subject><subject>Immunotherapy</subject><subject>Insulin - therapeutic use</subject><subject>Male</subject><subject>Medical sciences</subject><subject>Peptides</subject><subject>Remission Induction</subject><subject>Stem cells</subject><subject>Transplantation, Autologous</subject><subject>Transplants & implants</subject><issn>0012-1797</issn><issn>1939-327X</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2014</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNpdkUFv1DAQhS0EotuFA38AWUJI5RCwYyeOua22QJFKe2CRuEUTZ4JcOXYaO6X77_GqS5GQD3Pwpzfz3iPkFWfvSyHUh77jsmClrp6QFddCF6JUP5-SFWO8LLjS6oScxnjDGKvze05OSqmlEnW5IvebJQUXfoUl0qvgxz26AJ2DZO-QXuAIKUzBYrKGfk840i06R3cz-Dg58ClzwVPr6RX-Lq59xER3-wkpp-cWOkwYP9IN_ba4LIA-4Uw3Htw-2viCPBvARXx5nGvy4_On3faiuLz-8nW7uSyMlDIVvTY41AKZMBwGJSSTUMuu72opuho0E5B_uDBGKmxUz7mBbLMpVVUp1JVYk7MH3WkOtwvG1I42muwCPGbTLa-qqi4bpZqMvvkPvQnLnO_NVC1yjJrlfWvy7oEyc4hxxqGdZjvCvG85aw91tIc62kMdmX19VFy6EftH8m_-GXh7BCAacEMO1tj4j2sapqps9Q-ZUZIN</recordid><startdate>20140901</startdate><enddate>20140901</enddate><creator>D'ADDIO, Francesca</creator><creator>VASQUEZ, Alessandro Valderrama</creator><creator>BEN NASR, Moufida</creator><creator>FRANEK, Edward</creator><creator>DALONG ZHU</creator><creator>LIRONG LI</creator><creator>GUANG NING</creator><creator>SNARSKI, Emilian</creator><creator>FIORINA, Paolo</creator><general>American Diabetes Association</general><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>K9.</scope><scope>NAPCQ</scope><scope>7X8</scope></search><sort><creationdate>20140901</creationdate><title>Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in New-Onset Type 1 Diabetes: A Multicenter Analysis</title><author>D'ADDIO, Francesca ; VASQUEZ, Alessandro Valderrama ; BEN NASR, Moufida ; FRANEK, Edward ; DALONG ZHU ; LIRONG LI ; GUANG NING ; SNARSKI, Emilian ; FIORINA, Paolo</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c444t-d9cef63e03c1af73404a64bdb643b6a903a3c113cc47e87d11ca006827557e953</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2014</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Autoimmune diseases</topic><topic>Biological and medical sciences</topic><topic>C-Peptide - metabolism</topic><topic>Child</topic><topic>Children & youth</topic><topic>Cyclophosphamide - therapeutic use</topic><topic>Diabetes Mellitus, Type 1 - therapy</topic><topic>Diabetes. Impaired glucose tolerance</topic><topic>Endocrine pancreas. Apud cells (diseases)</topic><topic>Endocrinopathies</topic><topic>Etiopathogenesis. Screening. Investigations. Target tissue resistance</topic><topic>Female</topic><topic>Hematopoietic Stem Cell Transplantation - adverse effects</topic><topic>Humans</topic><topic>Immune system</topic><topic>Immunosuppression</topic><topic>Immunotherapy</topic><topic>Insulin - therapeutic use</topic><topic>Male</topic><topic>Medical sciences</topic><topic>Peptides</topic><topic>Remission Induction</topic><topic>Stem cells</topic><topic>Transplantation, Autologous</topic><topic>Transplants & implants</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>D'ADDIO, Francesca</creatorcontrib><creatorcontrib>VASQUEZ, Alessandro Valderrama</creatorcontrib><creatorcontrib>BEN NASR, Moufida</creatorcontrib><creatorcontrib>FRANEK, Edward</creatorcontrib><creatorcontrib>DALONG ZHU</creatorcontrib><creatorcontrib>LIRONG LI</creatorcontrib><creatorcontrib>GUANG NING</creatorcontrib><creatorcontrib>SNARSKI, Emilian</creatorcontrib><creatorcontrib>FIORINA, Paolo</creatorcontrib><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>ProQuest Health & Medical Complete (Alumni)</collection><collection>Nursing & Allied Health Premium</collection><collection>MEDLINE - Academic</collection><jtitle>Diabetes (New York, N.Y.)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>D'ADDIO, Francesca</au><au>VASQUEZ, Alessandro Valderrama</au><au>BEN NASR, Moufida</au><au>FRANEK, Edward</au><au>DALONG ZHU</au><au>LIRONG LI</au><au>GUANG NING</au><au>SNARSKI, Emilian</au><au>FIORINA, Paolo</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in New-Onset Type 1 Diabetes: A Multicenter Analysis</atitle><jtitle>Diabetes (New York, N.Y.)</jtitle><addtitle>Diabetes</addtitle><date>2014-09-01</date><risdate>2014</risdate><volume>63</volume><issue>9</issue><spage>3041</spage><epage>3046</epage><pages>3041-3046</pages><issn>0012-1797</issn><eissn>1939-327X</eissn><coden>DIAEAZ</coden><abstract>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 HbA1c 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.</abstract><cop>Alexandria, VA</cop><pub>American Diabetes Association</pub><pmid>24947362</pmid><doi>10.2337/db14-0295</doi><tpages>6</tpages><oa>free_for_read</oa></addata></record> |
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| source | MEDLINE; Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals; PubMed Central; Journals@Ovid Complete |
| subjects | Adolescent Adult Autoimmune diseases Biological and medical sciences C-Peptide - metabolism Child Children & youth Cyclophosphamide - therapeutic use Diabetes Mellitus, Type 1 - therapy Diabetes. Impaired glucose tolerance Endocrine pancreas. Apud cells (diseases) Endocrinopathies Etiopathogenesis. Screening. Investigations. Target tissue resistance Female Hematopoietic Stem Cell Transplantation - adverse effects Humans Immune system Immunosuppression Immunotherapy Insulin - therapeutic use Male Medical sciences Peptides Remission Induction Stem cells Transplantation, Autologous Transplants & implants |
| title | Autologous Nonmyeloablative Hematopoietic Stem Cell Transplantation in New-Onset Type 1 Diabetes: A Multicenter Analysis |
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