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Autologous non-myeloablative hematopoietic stem cell transplantation for refractory systemic vasculitis
  1. Laisvyde Statkute (l-statkute{at}northwestern.edu)
  1. Northwestern University Feinberg School of Medicine, United States
    1. Yu Oyama (y-oyama{at}northwestern.edu)
    1. Northwestern University Feinberg School of Medicine, United States
      1. Walter G Barr (w-barr{at}northwestern.edu)
      1. Northwestern University Feinberg School of Medicine, United States
        1. Robert Sufit (r-sufit{at}northwestern.edu)
        1. Northwestern University Feinberg School of Medicine, United States
          1. Sam Ho (s-ho{at}northwestern.edu)
          1. Northwestern University Feinberg School of Medicine, United States
            1. Larissa Verda (larissaverda{at}yahoo.com)
            1. Northwestern University Feinberg School of Medicine, United States
              1. Yvonne Loh (y-loh{at}northwestern.edu)
              1. Northwestern University Feinberg School of Medicine, United States
                1. Kimberly Yaung (kyaung{at}nmff.org)
                1. Northwestern University Feinberg School of Medicine, United States
                  1. Kathleen Quigley (k-quigley{at}northwestern.edu)
                  1. Northwestern University Feinberg School of Medicine, United States
                    1. Richard K Burt (rburt{at}northwestern.edu)
                    1. Northwestern University Feinberg School of Medicine, United States

                      Abstract

                      Objective: For patients with systemic vasculitis (SV) refractory to conventional therapy, new treatment strategies aimed at aggressive induction of remission and relapse prevention are being sought. We herein report our single center experience in treating 4 patients with refractory SV employing non-myeloablative autologous hematopoietic stem cell transplantation (HSCT).

                      Methods: Four patients with refractory SV (2 – with neurovascular Behcet’s disease, 1 – with neurovascular Sjogren’s syndrome, and 1 – with Wegener’s granulomatosis) were involved in an IRB and FDA approved phase I clinical trial of high dose chemotherapy and autologous HSCT. Peripheral blood stem cells were mobilized with cyclophosphamide (Cy) and granulocyte-colony stimulating factor. Conditioning regimen consisted of Cy 200 mg/kg and rabbit anti-thymocyte globulin 5.5 mg/kg IV.

                      Results: All 4 patients tolerated HSCT well without transplant related mortality or any significant toxicity. At median follow up of 28 (range 22-36) months all patients are alive. Three patients (1 each with Behcet’s, Sjogren’s, and Wegener’s) entered a sustained remission at 6, 6 and 24 months, respectively, after the transplant. They had significant decrease in both, disease activity and disease or treatment related damage, measures (Birmingham Vasculitis Activity Score and Vasculitis Damage Index, respectively). All 3 patients who achieved remission discontinued immunosuppressive therapy at the time of transplant and have not required treatment since. One patient with Behcet’s disease and positive for HLA-B51 has not improved after HSCT.

                      Conclusion: We suggest non-myeloablative autologous HSCT is safe and effective treatment for select patients with SV refractory to conventional immunosuppressive therapies.

                      • Behcet's disease
                      • Sjögren's syndrome
                      • Wegener's granulomatosis
                      • stem cell transplantation
                      • systemic vasculitis

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