An economic analysis of autologous hematopoietic stem cell transplantation (AHSCT) in the treatment of new onset type 1 diabetes.
OBJECTIVE
Autologous hematopoietic stem cell transplantation (AHSCT) is an emerging treatment option in new onset type 1 diabetes (T1DM), leading to a remission of the T1DM for a longer time period in up to 50 % of patients. The aim of the study was to analyze the cost-effectiveness of this treatment option compared with standard insulin therapy.


METHODS
The medical records of patients who had undergone immunoablation with AHSCT for new onset T1DM were analyzed for the cost-effectiveness of the treatment using the IMS CORE Diabetes Model.


RESULTS
The expected survival of patients with T1DM treated solely with insulin (without transplantation) was estimated to be 34.4 years, and their quality-adjusted survival was 13.8 QALY, whereas the expected survival of the patients treated with AHSCT was 34.9 years when the HbA1c benefit over standard treated patients lasted for 2, 35.4 years with 8-year benefit and even up to 40.3 years with the lifelong benefit scenario. Values under the threshold of ICER were reached after 8 years of sustained benefit in terms of HbA1c concentration. If discounting was not applied, the threshold values were reached after 3 years of HbA1c benefit over the standard group, independent of insulin use after transplantation.


CONCLUSIONS
The results of our study show that hematopoietic stem cell transplantation could be cost-effective in treating new onset T1DM, providing that the benefits of the transplantation lasted over 3-8 years, depending on application of discounting.
Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, ul. Banacha 1a, 02-097, Warsaw, Poland. emiliansnarski@gmail.com.
Emilian
E
Snarski
Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, ul. Banacha 1a, 02-097, Warsaw, Poland.
Daria
D
Szmurło
Institute of Hematology and Transfusion Medicine, Warsaw, Poland.
Kazimierz
K
Hałaburda
Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, ul. Banacha 1a, 02-097, Warsaw, Poland.
Małgorzata
M
Król
Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, ul. Banacha 1a, 02-097, Warsaw, Poland.
Elżbieta
E
Urbanowska
Department of Internal Diseases, Diabetology and Endocrinology, Central Hospital, Ministry of Internal Affairs and Administration, Warsaw, Poland.
Alicja
A
Milczarczyk
Department of Internal Diseases, Diabetology and Endocrinology, Central Hospital, Ministry of Internal Affairs and Administration, Warsaw, Poland.|Mossakowski Medical Research Centre, Polish Academy of Sciences, Warsaw, Poland.
Edward
E
Franek
Department of Hematology, Oncology and Internal Diseases, Medical University of Warsaw, ul. Banacha 1a, 02-097, Warsaw, Poland.
Wiesław
W
Wiktor-Jedrzejczak
D003362:Cost-Benefit Analysis; D003922:Diabetes Mellitus, Type 1 / Q000188:drug therapy / Q000401:mortality / Q000601:surgery*; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000191:economics*; D006801:Humans; D007328:Insulin / Q000627:therapeutic use; D008297:Male; D015996:Survival Rate; D014182:Transplantation, Autologous
D007328:Insulin
Cost-effectiveness; Diabetes; Stem cell; Transplantation