NONMYELOABLATIVE AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR REFRACTORY CIDP

Abstract

Most patients with chronic inflammatory demyelinating polyneuropathy (CIDP) initially respond to corticosteroids, immunosuppressive drugs, IV immune globulin (IVIg), and plasma exchange (PE). However, more than half relapse, with some developing a recurrent or persistent clinical course resulting in severe disability and even death.1,2 Nonmyeloablative autologous hematopoietic stem cell transplantation (HSCT) for severe autoimmune diseases has demonstrated promising results, with reports of durable remissions and lower toxicity compared to myeloablative HSCT.3 Herein, we report the first patient treated in a phase I trial of autologous HSCT utilizing a nonmyeloablative regimen for refractory CIDP. ### Methods. Eligibility criteria include age 65 years or less; definite or probable CIDP according to the criteria of the Ad Hoc Subcommittee of the American Academy of Neurology AIDS Task Force; failure of standard therapy, i.e., incomplete response or relapse after corticosteroids, IVIg, or PE; and failure to respond to at least one second-line immunosuppressive drug (methotrexate, azathioprine, cyclosporine, tacrolimus, or mycophenolate mofetil [MMF]). CD34-enriched peripheral blood stem cells …