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.
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.
Authors: José Carlos Jaime-Pérez; Mariana González-Treviño; Jesús D Meléndez-Flores; Eugenia M Ramos-Dávila; Olga G Cantú-Rodriguez; César H Gutiérrez-Aguirre; Dionicio A Galarza-Delgado; David Gómez-Almaguer Journal: Clin Rheumatol Date: 2021-09-29 Impact factor: 2.980
Authors: Howard R Seay; Amy L Putnam; Judit Cserny; Amanda L Posgai; Emma H Rosenau; John R Wingard; Kate F Girard; Morey Kraus; Angela P Lares; Heather L Brown; Katherine S Brown; Kristi T Balavage; Leeana D Peters; Ashley N Bushdorf; Mark A Atkinson; Jeffrey A Bluestone; Michael J Haller; Todd M Brusko Journal: Mol Ther Methods Clin Dev Date: 2016-12-24 Impact factor: 6.698
Authors: Wen-I Yeh; Howard R Seay; Brittney Newby; Amanda L Posgai; Filipa Botelho Moniz; Aaron Michels; Clayton E Mathews; Jeffrey A Bluestone; Todd M Brusko Journal: Front Immunol Date: 2017-10-26 Impact factor: 7.561
Authors: Lacy E Lowry; Maryanne C Herzig; Barbara A Christy; Richard Schäfer; Shibani Pati; Andrew P Cap; James A Bynum Journal: Stem Cell Rev Rep Date: 2021-01-08 Impact factor: 6.692
Authors: Ida Pastore; Emma Assi; Moufida Ben Nasr; Andrea Mario Bolla; Anna Maestroni; Vera Usuelli; Cristian Loretelli; Andy Joe Seelam; Ahmed Abdelsalam; Gian Vincenzo Zuccotti; Francesca D'Addio; Paolo Fiorina Journal: Front Immunol Date: 2021-07-09 Impact factor: 7.561
Authors: Michael J Haller; Stephen E Gitelman; Peter A Gottlieb; Aaron W Michels; Daniel J Perry; Andrew R Schultz; Maigan A Hulme; Jonathan J Shuster; Baiming Zou; Clive H Wasserfall; Amanda L Posgai; Clayton E Mathews; Todd M Brusko; Mark A Atkinson; Desmond A Schatz Journal: Diabetes Date: 2016-09-26 Impact factor: 9.461