Autologous stem cell transplantation for refractory opsoclonus myoclonus ataxia syndrome. Issue 8 (25th April 2018)

• Record Type: Journal Article
• Authors: Johnston, Donna L.; Murray, Sean; Irwin, Meredith S.; Doyle, John; Schechter, Tal
• Is Part Of: Pediatric blood & cancer. Volume 65:Issue 8(2018)
• Page Start: n/a
• Page End: n/a
• Publication Date: 2018-04-25
• Subjects: ataxia -- myoclonus -- opsoclonus -- transplant; Tumors in children -- Periodicals; Blood -- Diseases -- Periodicals; Cancer in children -- Periodicals; 618.92
• DOI: 10.1002/pbc.27110
• Languages: English
• ISSNs: 1545-5009

Abstract

Abstract: Opsoclonus, myoclonus, ataxia syndrome (OMA) is a severe neurologic disorder often associated with neuroblastoma. It is challenging to treat and can have long‐term neurologic sequelae. Current recommended therapies include intravenous immunoglobulin, corticosteroids, rituximab, and chemotherapy (cyclophosphamide). We present two cases who were refractory to conventional therapy and underwent autologous stem cell transplantation (ASCT). One patient had complete resolution of symptoms following ASCT and the other patient had minimal change in symptoms with this therapy. These findings support consideration of ASCT as a therapeutic option for patients with refractory OMA after failure of known effective therapies.