title: Immunoablation and autologous hematopoietic stem cell transplantation in the treatment of new-onset type 1 diabetes mellitus: long-term observations.
abstract: The activity of the autoimmune mechanism underlying type 1 diabetes mellitus (T1DM) can be suppressed when immunoablation and autologous hematopoietic stem cell transplantation (AHSCT) are applied early in the course of the disease. We report here a single centre experience with this treatment modality. Twenty-four patients underwent a AHSCT preceded by immunoablative conditioning with high-dose cyclophosphamide and anti-thymocyte globulin. During the 52-month median time of follow-up 20 out of 23 patients (87%) remained for at least 9.5 months without the use of exogenous insulin. The median time of T1DM remission for these patients was 31 months (range of 9.5-80 months). Among the patients available for follow-up (n=20), four remain insulin free (for 80, 61, 42 and 34 months). The average glycated hemoglobin (HbA1c) concentrations were 10.9% at diagnosis, 5.9% at 1 year, 6.4% at 2 years, 6.8% at 3 years and 7.1% at 4 years after AHSCT. No severe complications of diabetes were seen, however one of the patients died of pseudomonas sepsis in the course of neutropenia after AHSCT. AHSCT leads to a remission of T1DM with good glycemic control in the vast majority of patients, with the period of remission lasting over 5 years in some patients.
authors.affiliation: Department of Hematology, Oncology and Internal Medicine, Medical University of Warsaw, Warszawa, Poland.
authors.forename: E
authors.identifier: http://orcid.org/0000-0003-2700-7571
authors.initials: E
authors.lastname: Snarski
authors.affiliation: Department of Internal Medicine, Diabetology and Endocrinology, Central Hospital, Ministry of Internal Affairs and Administration, Warszawa, Poland.
authors.forename: A
authors.initials: A
authors.lastname: Milczarczyk
authors.affiliation: Department of Hematology, Oncology and Internal Medicine, Medical University of Warsaw, Warszawa, Poland.
authors.forename: K
authors.initials: K
authors.lastname: Hałaburda
authors.affiliation: Department of Hematology, Oncology and Internal Medicine, Medical University of Warsaw, Warszawa, Poland.
authors.forename: T
authors.initials: T
authors.lastname: Torosian
authors.affiliation: Department of Hematology, Oncology and Internal Medicine, Medical University of Warsaw, Warszawa, Poland.
authors.forename: M
authors.initials: M
authors.lastname: Paluszewska
authors.affiliation: Department of Hematology, Oncology and Internal Medicine, Medical University of Warsaw, Warszawa, Poland.
authors.forename: E
authors.initials: E
authors.lastname: Urbanowska
authors.affiliation: Department of Hematology, Oncology and Internal Medicine, Medical University of Warsaw, Warszawa, Poland.
authors.forename: M
authors.initials: M
authors.lastname: Król
authors.affiliation: Department of Hematology, Oncology and Internal Medicine, Medical University of Warsaw, Warszawa, Poland.
authors.forename: P
authors.initials: P
authors.lastname: Boguradzki
authors.affiliation: Department of Internal Medicine, Diabetology and Endocrinology, Central Hospital, Ministry of Internal Affairs and Administration, Warszawa, Poland.
authors.forename: K
authors.initials: K
authors.lastname: Jedynasty
authors.affiliation: Department of Internal Medicine, Diabetology and Endocrinology, Central Hospital, Ministry of Internal Affairs and Administration, Warszawa, Poland.|Mossakowski Medical Research Centre, Polish Academy of Sciences, Warszawa, Poland.
authors.forename: E
authors.initials: E
authors.lastname: Franek
authors.affiliation: Department of Hematology, Oncology and Internal Medicine, Medical University of Warsaw, Warszawa, Poland.
authors.forename: W
authors.initials: W
authors.lastname: Wiktor-Jedrzejczak
mesh_terms: D000293:Adolescent; D000328:Adult; D064592:Autografts; D003922:Diabetes Mellitus, Type 1 / Q000097:blood / Q000276:immunology / Q000401:mortality / Q000628:therapy; D005260:Female; D005500:Follow-Up Studies; D006442:Glycated Hemoglobin / Q000276:immunology / Q000378:metabolism; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D012074:Remission Induction; D013997:Time Factors; D019172:Transplantation Conditioning
chemical_list: D006442:Glycated Hemoglobin; C517652:hemoglobin A1c protein, human