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Autologous stem cell transplantation for refractory opsoclonus myoclonus ataxia syndrome
Author(s) -
Johnston Donna L.,
Murray Sean,
Irwin Meredith S.,
Doyle John,
Schechter Tal
Publication year - 2018
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.27110
Subject(s) - medicine , rituximab , cyclophosphamide , refractory (planetary science) , myoclonus , ataxia , hematopoietic stem cell transplantation , autologous stem cell transplantation , transplantation , surgery , chemotherapy , oncology , pediatrics , lymphoma , anesthesia , psychiatry , physics , astrobiology
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.