Intravenous immunoglobulin with prednisone and risk-adapted chemotherapy for children with opsoclonus myoclonus ataxia syndrome associated with neuroblastoma.

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Intravenous immunoglobulin with prednisone and risk-adapted chemotherapy for children with opsoclonus myoclonus ataxia syndrome associated with neuroblastoma (ANBL00P3): a randomised, open-label, phase 3 trial Dr Pedro A de Alarcon, MD, Katherine K Matthay, MD, Wendy B London, PhD, Arlene Naranjo, PhD, Sheena C Tenney, MSPH, Jessica A Panzer, MD, Michael D Hogarty, MD, Julie R Park, MD, John M Maris, MD, Susan L Cohn, MD The Lancet Child & Adolescent Health Volume 2, Issue 1, Pages 25-34 (January 2018) DOI: 10.1016/S2352-4642(17)30130-X Copyright © 2018 Elsevier Ltd Terms and Conditions

Figure 1 Study design IVIG=intravenous immunoglobulin. ACTH=adrenocorticotropic hormone. OMA=opsoclonus myoclonus ataxia syndrome. *OMA evaluation timepoints include: at diagnosis, monthly for 6 months, then every other month until 1 year, 18 months, 2 years, and once per year thereafter up to 10 years. The Lancet Child & Adolescent Health 2018 2, 25-34DOI: (10.1016/S2352-4642(17)30130-X) Copyright © 2018 Elsevier Ltd Terms and Conditions

Figure 2 Trial profile IVIG=intravenous immunoglobulin. ACTH=adrenocorticotropic hormone. OMA=opsoclonus myoclonus ataxia syndrome. *Parent refused further treatment. The Lancet Child & Adolescent Health 2018 2, 25-34DOI: (10.1016/S2352-4642(17)30130-X) Copyright © 2018 Elsevier Ltd Terms and Conditions

Figure 3 Neuroblastoma event-free, OMA progression-free, and overall survival of patient cohort NBL=neuroblastoma. EFS=event free survival. OMA=opsoclonus myoclonus ataxia syndrome. PFS=progression free survival. OS=overall survival. The Lancet Child & Adolescent Health 2018 2, 25-34DOI: (10.1016/S2352-4642(17)30130-X) Copyright © 2018 Elsevier Ltd Terms and Conditions

Figure 4 OMA progression-free survival for IVIG and no IVIG groups OMA=opsoclonus myoclonus ataxia syndrome. IVIG=intravenous immunoglobulin. The Lancet Child & Adolescent Health 2018 2, 25-34DOI: (10.1016/S2352-4642(17)30130-X) Copyright © 2018 Elsevier Ltd Terms and Conditions