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• W130455737 abstract "Autologous non-myeloablative hematopoietic stem cell transplantation (HSCT) is a method of delivering intense immune suppression. We, therefore, evaluated the safety and clinical outcome of autologous non-myeloablative HSCT in 2 autoimmune diseases relapsing-remitting multiple sclerosis (RRMS) failing interferon therapy and new onset Type 1 Diabetes mellitus. Peripheral blood hematopoietic stem cells (HSC) were mobilized with cyclophosphamide (2.0 g/m2) and granulocyte colony stimulating factor (10 ug/kg/day). HSCT conditioning regimen was cyclophosphamide (200 mg/kg) and rabbit anti-thymocyte globulin (either 6 mg/kg or 4.5 mg/kg). Eligible patients with RRMS had, despite interferon beta, 2 corticosteroid treated relapses within the prior 12 months, or 1 such relapse and gadolinium enhancing lesion(s) on magnetic resonance imaging (MRI) demonstrated on an occasion distinct from the relapse. All patients are surviving, 17 patients (81%) improved by at least 1.0 or more EDSS points, and five patients (24%) relapsed but achieved another remission after further immunosuppression. After a mean of 3 years, progression-free survival is 100%, and relapse-free survival is 76%. Significant improvement occurred in neurologic disability determined by extended disability status scale (EDSS), neurologic rating score (NRS), paced auditory serial addition test (PASAT), and 25 foot walk, as well as quality of life as measured by the short form-36 (SF-36). Non-myeloablative autologous HSCT in RRMS appears capable of reversing neurologic deficits. Eligible patients for HSCT of T1DM were diagnosed in the previous 6 weeks by clinical findings and hyperglycemia and confirmed with positive antibodies against glutamic acid decarboxylase. 23 type 1 diabetic patients (aged between 13 and 31 years) were treated. During a 7-to 58-month follow-up (mean 29.8) 20 patients without previous ketoacidosis and not receiving steroids during the preparative regimen became insulin-free; 12 patients maintained this status for 14 to 52 months (mean 31) while 8 patients relapsed and resumed insulin use at low dose (0.1 to 0.3 IU/kg). In the continuous insulin-independent group, HbA1c levels were always <7.0%, continuous glucose monitoring was within normal levels and mean AUC of C-peptide levels increased significantly from 225.0 ng/mL pre-transplantation to 785.4 ng/mL at +24 months (P <0.001) and to 728.1 ng/mL at +36 months (P = 0.03). In the transient insulin-independent group mean AUC of C-peptide levels also increased from 148.9 ng/ml before transplantation to 546.8 ng/mL at +36 months (P = 0.001) that was sustained at + 48 months In this group, two patients regained insulin independence after treatment with sitagliptin which was associated with increase in C-peptide levels. HSCT induces insulin and drug free remission for up to 5 years post HSCT and appears to preserve or allow regeneration of islet cell mass with maximal C-peptide levels reached 2–3 years after HSCT." @default.
• W130455737 created "2016-06-24" @default.
• W130455737 creator A5016017302 @default.
• W130455737 date "2010-04-01" @default.
• W130455737 modified "2023-09-27" @default.
• W130455737 title "Non‐myeloablative autologous HSCT for relapsing remitting multiple sclerosis (RRMS) and new onset type 1 diabetes mellitus (TIDM)" @default.
• W130455737 doi "https://doi.org/10.1096/fasebj.24.1_supplement.64.3" @default.
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