Type 1 diabetes mellitus (DM) results from a cell-mediated autoimmune attack against pancreatic beta cells. At the time of clinical diagnosis, approximately 60 percent to 80 percent of the beta-cell mass has been destroyed, according to background information in the article. Beta-cell preservation has been shown to be an important target in the management of type 1 DM and in the prevention of its related complications.
Julio C. Voltarelli, M.D., Ph.D., of the University of São Paulo, Brazil; and colleagues conducted a study to examine the effect of high-dose immunosuppression followed by autologous nonmyeloablative hematopoietic stem cell transplantation (AHST) to preserve beta-cell function in 15 newly diagnosed patients with type 1 DM. AHST, which uses a patient's own blood stem cells, involves the removal and treatment of the stem cells, and their return to the patient by intravenous injection.
During a 7 to 36-month follow-up, 14 patients became insulin-free (one for 35 months, four for at least 21 months, seven for at least six months; and two with late response were insulin-free for one and five months, respectively). Among those, one patient resumed insulin use one year after AHST. The only severe adverse effects were pneumonia in one patient and endocrine dysfunction in two others.
"Very encouraging results were obtained in a small number of patients with early-onset disease,” the authors summarise. “Ninety-three percent of patients achieved different periods of insulin independence and treatment-related toxicity was low, with no mortality.” However, more randomised controlled trials and further biological studies are necessary to confirm the role of this treatment, the authors say.
MEDICA.de; Source: American Medical Association (AMA)