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Novel treatment (new drug/intervention; established drug/procedure in new situation)
Treatment of polyglandular autoimmune syndrome type 3 using co-transplantation of insulin-secreting mesenchymal stem cells and haematopoietic stem cells
  1. Hargovind L Trivedi1,
  2. Umang G Thakkar1,
  3. Aruna V Vanikar2,
  4. Shruti D Dave2
  1. 1Department of Nephrology and Transplantation Medicine, G. R. Doshi and K. M. Mehta Institute of Kidney Diseases and Research Centre (IKDRC)- Dr H. L. Trivedi Institute of Transplantation Sciences (ITS), Ahmedabad, Gujarat, India
  2. 2Department of Pathology, Laboratory Medicine, Transfusion Services and Immunohematology, G. R. Doshi and K. M. Mehta Institute of Kidney Diseases and Research Centre (IKDRC)- Dr H. L. Trivedi Institute of Transplantation Sciences (ITS), Ahmedabad, Gujarat, India
  1. Correspondence to Professor Hargovind L Trivedi, ikdrcad1{at}sancharnet.in

Summary

The authors report a 17-year-female and a 19-year-male with uncontrolled insulin-dependent diabetes mellitus (IDDM) for ≥10 years, treated with insulin-secreting human adipose tissue derived mesenchymal stem cells (IS-h-ADMSC). Both had hypothyroidism and were diagnosed as polyglandular autoimmune syndrome type-3 (PGAS-3). PGAS are rare polyendocrinopathies with ≥2 endocrine disorders mediated by autoimmune mechanisms leading to hypo-function and organ failure. Therapeutic options are hormone replacement, immunosuppression and avoiding infection. The authors administered autologous H-AD-IS-MSC+bone marrow-derived haematopoietic stem cells (HSC) into portal circulation with conditioning of cyclophosphamide, bortezomib, rituximab and rabbit-antithymoglobulin. Over follow-up of 38 and 16 months, respectively, both are doing well with sustained fall of glycosylated haemoglobin (Hb1Ac) from 8.1 to 6.4% and 14.2 to 8.6%, respectively and C-peptide raised from 0.01 to 0.23 ng/ml and 0.1 to 0.34 ng/ml, respectively with sustained 40% decreased insulin requirement. Thus long-term control of IDDM in PGAS-3 with co-transplantation of H-AD-IS-MSC+HSC can be achieved safely and effectively.

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Footnotes

  • Competing interests None.

  • Patient consent Obtained.

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