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Simplified autologous hematopoietic stem cell transplantation shows promise in type 1 diabetes

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February 23, 2016

In patients with type 1 diabetes, simplified autologous hematopoietic stem cell transplantation in an outpatient setting appears to be a safe and effective intervention, according to study data.

Fernando Lavalle-González, MD, an endocrinologist at the Universidad Autónoma de Nuevo León in Mexico, and colleagues evaluated 16 children (median age, 12 years) with type 1 diabetes to determine the effect of simplified autologous hematopoietic stem cell transplantation in the outpatient setting on long-term insulin independence, changes in HbA1c and the safety of the procedure. The median follow-up was 34 months.

All patients underwent the procedure on a 100% outpatient basis without severe complications, and there was no mortality at 100 days of follow-up.

The overall response was 81%, with a reduction in overall daily insulin requirements from 0.41 U/kg to 0.32 U/kg by the third month (P = .46). Insulin dependence was achieved by seven participants (44%); five were insulin-independent by the third month and one attained freedom from insulin within the first week after transplantation. Two participants who achieved partial insulin independence at 6 months postprocedure achieved complete insulin independence at 11 months and 1 year posttransplant. Three patients were deemed nonresponders, and the remaining six showed partial insulin independence. In all groups, HbA1c levels demonstrated a total mean decrease of 0.87% during the 3 months of study, a 1% decrease at 6 months and 1.6% decrease at the last follow-up. At 6 months, the insulin-independent group exhibited a mean HbA1c reduction of –2.3%.

“The transplant was fully accomplished on an outpatient basis, thereby reducing costs, limiting exposure to nosocomial infections and avoiding the inconvenience of hospitalization,” the researchers wrote. “This method should be further studied in a larger cohort including an appropriate contemporary control group as it appears to be capable of changing the natural history of type 1 diabetes mellitus.” – by Jennifer Byrne

Disclosure: The researchers report no relevant financial disclosures.

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Michael Haller

Michael Haller

As we search for a means to safely and effectively prevent and reverse type 1 diabetes, a recently published paper by Cantu-Rodriguez and colleagues, demonstrating the efficacy of a simplified autologous stem cell transplant for patients with new-onset type 1 diabetes, has once again forced diabetologists to reexamine notions of clinical equipoise. In this paper, the authors describe the use of an entirely outpatient regimen consisting of (1) stem cell mobilization via cyclophosphamide and granulocyte-colony stimulating factor, (2) apheresis of CD34 cells, (3) immune conditioning with cyclophosphamide and fludarabine and (4) intravenous infusion of the previously collected (noncryopreserved) CD34 cells. The authors reported insulin independence in six patients nearly 1 year after transplantation and partial insulin independence in eight subjects. Only two subjects were classified as nonresponders. 

While conceptually similar to efforts initiated by Julio Voltarelli (JAMA. 2007;297:1568-1576.), this effort takes the approach one step further by promoting an entirely outpatient-based regimen through the use of fludarabine in place of thymoglobulin. While concerns related to risks and side effects have largely rendered similar immuno-ablative approaches untenable in the United States, several groups, including my own, have sought to emulate the concept of combination immunotherapy through the use of lower-dose or less-risky immunotherapeutic combinations. Specifically, the Type 1 Diabetes TrialNet is currently performing a study of low-dose anti-thymocyte globulin plus G-CSF in new-onset type 1 patients (

Michael Haller, MD
Chief and Fellowship Director
Associate Professor
Pediatric Endocrinology
University of Florida

Disclosure: Haller reports no relevant financial disclosures.