TY - JOUR
T1 - Rituximab, IVIg, and Tetracosactide (ACTH1-24) Combination Immunotherapy (RITE-CI) for Pediatric Opsoclonus-Myoclonus Syndrome
T2 - Immunomarkers and Clinical Observations
AU - Pranzatelli, Michael R.
AU - Tate, Elizabeth D.
AU - Alber, Michael
AU - Awadalla, Maha
AU - Blumkin, Lubov
AU - Lina, Elena S.
AU - Leiz, Steffen
AU - Móser, Judit
N1 - Publisher Copyright:
© 2018 Georg Thieme Verlag KG Stuttgart New York.
PY - 2018/4/1
Y1 - 2018/4/1
N2 - Opsoclonus-myoclonus syndrome (OMS) is a neuroinflammatory disorder with pervasive morbidity that warrants better treatments. Twelve children with moderate/severe OMS (total score 23 ± 6) who did not remit to multiple immunotherapies were evaluated for neuroinflammation in a case-control study using cerebrospinal fluid (CSF) lymphocyte subset analysis by flow cytometry, chemokine/cytokine analysis by enzyme-linked immunoadsorption assay (ELISA), and oligoclonal bands by immunofixation with isoelectric focusing. Observations made on empirical treatment with rituximab, IVIg, and tetracosactide combination immunotherapy (coined RITE-CI) were analyzed. All of the patients tested for multiple inflammatory markers were positive; 75% had ≥3 CSF markers. Fifty percent had CSF oligoclonal bands; 58%, B cell expansion; and 50 to 100%, elevated concentrations of multiple chemokines and neuronal/axonal marker neurofilament light chain. After RITE-CI, total score dropped significantly in the group (-85%, p < 0.0001) from moderate to trace, and by 2 to 4 severity categories in each patient. The 24-week schedule was well tolerated and clinically effective for moderate or severe OMS, as were other schedules. RITE-CI is feasible and effective as rescue therapy and presents an initial option for children with moderate/severe OMS. Though preliminary, the schedule can be adjusted to patient severity, propensity for relapse, and other factors.
AB - Opsoclonus-myoclonus syndrome (OMS) is a neuroinflammatory disorder with pervasive morbidity that warrants better treatments. Twelve children with moderate/severe OMS (total score 23 ± 6) who did not remit to multiple immunotherapies were evaluated for neuroinflammation in a case-control study using cerebrospinal fluid (CSF) lymphocyte subset analysis by flow cytometry, chemokine/cytokine analysis by enzyme-linked immunoadsorption assay (ELISA), and oligoclonal bands by immunofixation with isoelectric focusing. Observations made on empirical treatment with rituximab, IVIg, and tetracosactide combination immunotherapy (coined RITE-CI) were analyzed. All of the patients tested for multiple inflammatory markers were positive; 75% had ≥3 CSF markers. Fifty percent had CSF oligoclonal bands; 58%, B cell expansion; and 50 to 100%, elevated concentrations of multiple chemokines and neuronal/axonal marker neurofilament light chain. After RITE-CI, total score dropped significantly in the group (-85%, p < 0.0001) from moderate to trace, and by 2 to 4 severity categories in each patient. The 24-week schedule was well tolerated and clinically effective for moderate or severe OMS, as were other schedules. RITE-CI is feasible and effective as rescue therapy and presents an initial option for children with moderate/severe OMS. Though preliminary, the schedule can be adjusted to patient severity, propensity for relapse, and other factors.
KW - chronic-relapsing OMS
KW - cosyntropin
KW - neuroblastoma
KW - neuroimmunology
KW - paraneoplastic syndrome
KW - pediatric neuroinflammation
UR - http://www.scopus.com/inward/record.url?scp=85038629208&partnerID=8YFLogxK
U2 - 10.1055/s-0037-1609038
DO - 10.1055/s-0037-1609038
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C2 - 29258131
AN - SCOPUS:85038629208
SN - 0174-304X
VL - 49
SP - 123
EP - 134
JO - Neuropediatrics
JF - Neuropediatrics
IS - 2
ER -