Long-term insulin independence in type 1 diabetes mellitus using a simplified autologous stem cell transplant
Abstract
CONTEXT: Efforts to find a cure for type 1 diabetes have focused on the removal of the autoimmune pathophysiologic substrate, with the use of immunosuppressive regimens including autologous hematopoietic stem cell transplantation (AHSCT). OBJECTIVE: The main objective of determining long-term insulin independence as well as changes in glycated hemoglobin (HbA1c). Secondary outcomes were procedure morbidity and the need for hospital management. DESIGN: We enrolled patients with type 1 diabetes between 2012 and 2014. Median follow-up was 34 months (range, 25-56 mo). SETTING: Ambulatory care. INTERVENTIONS: We decided to carry out an AHSCT protocol using a less toxic and affordable simplified method based on fludarabine in an outpatient setting. PATIENTS: Patients were of both sexes, age 8-25 years, with positive levels of anti-GAD antibodies, a C-peptide level >1.0 ng/mL, and <3 months since diagnosis. MAIN OUTCOME MEASURE(S): Insulin independence. RESULTS: Sixteen patients were included. Overall response was 81% with seven patients achieving insulin independence (44%); six were partial responders (37%) whereas three were nonresponders (19%). The HbA1c level showed a mean decrease of -2.3% at 6 months in the Insulin Independence group. Median age was 12 years old (range, 8-17 years old). A mean of 11.5 × 10(6) CD34+ cells (SD ± 8.2) was obtained. Related mortality at 100 days was 0% as well as during follow-up. Outpatient setting was 100%. CONCLUSIONS: Simplified AHSCT in an outpatient setting is a feasible, safe and potentially therapeutic intervention for early-onset type 1 diabetes.
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Autologous haemopoietic stem-cell transplantation and autologous T-regulatory cell administration are other novel approaches.
“Other novel approaches include autologous haemopoietic stem-cell transplantation165,166 and autologous T-regulatory cell administration.167–169”
From Type 1 diabetes (2018) · cites this paper as [165]
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- Authors
- Cantú-Rodríguez, O. G., Lavalle-González, F., Herrera-Rojas, M. Á., Jaime-Pérez, J. C., Hawing-Zárate, J. Á., Gutiérrez-Aguirre, C. H., Mancias-Guerra, C., González-Llano, O., Zapata-Garrido, A., Villarreal-Pérez, J. Z., & Gómez-Almaguer, D.
- Affiliations
- Universidad Autónoma de Nuevo León (MX)
- Venue
- The Journal of Clinical Endocrinology & Metabolism
- Publisher
- The Endocrine Society
- Published
- 2016-05
- Volume
- 101
- Issue
- 5
- Pages
- 2141-2148
- ISSN
- 0021-972X (ISSN-L)
- 1945-7197
- OpenAlex
- W2264672021
- Cited by
- 39
- References
- 34
- Citations by year
- 2026: 3 · 2025: 3 · 2024: 5 · 2023: 4 · 2022: 7 · 2021: 4 · 2019: 2 · 2018: 5 · 2017: 2 · 2016: 3 · 2014: 1
- FWCI
- 3.22
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- The Journal of Clinical Endocrinology & Metabolism (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Diabetes and associated disorders
- Health Sciences › Medicine › Surgery › Pancreatic function and diabetes
- Health Sciences › Medicine › Endocrinology, Diabetes and Metabolism › Diabetes Management and Research
- Keywords
- type 1 diabetes, autologous stem cell transplantation, insulin independence, fludarabine, outpatient transplantation, HbA1c, early-onset diabetes, glutamic acid decarboxylase antibodies, C-peptide, CD34+ cells, autoimmune disease pathogenesis
- MeSH terms
- Adolescent, Adult, Blood Glucose, Child, Diabetes Mellitus, Type 1, Humans, Immunosuppressive Agents, Transplantation, Autologous, Treatment Outcome, Hematopoietic Stem Cell Transplantation, Young Adult
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