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Published on: November 18, 2022
Long-Term Insulin Independence in Type 1 Diabetes Mellitus Using a Simplified Autologous Stem Cell Transplant
Olga Graciela Cantú-Rodríguez1, Fernando Lavalle-González1, Miguel Ángel Herrera-Rojas1
1Hematology Service (O.G.C.-R., M.A.H.-R., J.C.J.-P., J.A.H.-Z., C.H.G.-A., C.M.-G., O.G.-L., D.G.-A.), and Endocrinology Service (F.L.-G., A.Z.-G., J.Z.V.-P.), Hospital Universitario "Dr José Eleuterio González," Universidad Autónoma de Nuevo León, Monterrey, México 64 460.
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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