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.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Johnston DL,Murray S,Irwin MS,Doyle J,Schechter Tdoi
10.1002/pbc.27110subject
Has Abstractpub_date
2018-08-01 00:00:00pages
e27110issue
8eissn
1545-5009issn
1545-5017journal_volume
65pub_type
杂志文章abstract::We describe a child with dyserythropoietic anemia, thrombocytosis, functional platelet defect, and megakaryocyte dysplasia. We show that (i) this constellation of hematopoietic abnormalities was due to a germline mutation within the 5' untranslated region (5'UTR) of globin transcription factor 1 (GATA1); (ii) the muta...
journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
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更新日期:2015-06-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2020-12-01 00:00:00
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
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更新日期:2009-02-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章
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更新日期:2011-08-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2010-10-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章
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更新日期:2013-04-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章
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更新日期:2011-07-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
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更新日期:2014-01-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
doi:10.1002/pbc.26853
更新日期:2018-02-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.25900
更新日期:2016-05-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2019-06-01 00:00:00
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
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更新日期:2012-09-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2020-05-01 00:00:00
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
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更新日期:2018-08-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章
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更新日期:2018-03-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章,实务指引
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更新日期:2013-03-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2013-04-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
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更新日期:2018-09-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章
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更新日期:2016-03-01 00:00:00