Abstract:
:Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal stem cell disorder. Eculizumab and bone marrow transplantation are disease-modifying treatments for PNH but may not be readily available in resource-constrained settings. Of 52 pediatric patients with PNH, 20 had classical PNH and 32 had PNH/aplastic anemia (PNH/AA). Median time to diagnosis was 30 months in classical PNH patients. Renal failure was present in four patients (20%). Six (30%) achieved complete response, 10 (50%) achieved partial response with androgens in classical PNH. Two underwent allogenic stem cell transplantation. In the PNH/AA group, 16 (50%) were in CR and seven (21%) were in PR with anti-thymocyte globulin ± cyclosporine.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Halder R,Mishra P,Aggarwal M,Mannivanan P,Dhawan R,Seth T,Tyagi S,Mahapatra M,Pati HP,Saxena Rdoi
10.1002/pbc.27712subject
Has Abstractpub_date
2020-04-01 00:00:00pages
e27712issue
4eissn
1545-5009issn
1545-5017journal_volume
67pub_type
杂志文章abstract:BACKGROUND:The identification of hemophagocytosis (HPC) in tissue or bone marrow (BM) represents only one of 5/8 criteria needed for the diagnosis of hemophagocytic lymphohistiocytosis (HLH). Yet, confirmation of HPC in bone marrow aspirates (BMA) is often relied upon to make therapeutic decisions. There is no standard...
journal_title:Pediatric blood & cancer
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更新日期:2008-09-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2012-05-01 00:00:00
abstract::Congenital amegakaryocytic thrombocytopenia (CAMT) is a rare autosomal recessive disorder characterized by thrombocytopenia from failure of megakaryopoiesis. CAMT is one of the bone marrow failure syndromes, and the disease progression may involve other lineages leading to pancytopenia. The genetic background of CAMT ...
journal_title:Pediatric blood & cancer
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更新日期:2011-02-01 00:00:00
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journal_title:Pediatric blood & cancer
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doi:10.1002/pbc.27499
更新日期:2019-01-01 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:2018-01-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2011-10-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章,评审
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journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
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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:2009-12-01 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:2015-12-01 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:2009-08-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2017-05-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.24330
更新日期:2013-03-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究
doi:10.1002/pbc.26013
更新日期:2016-09-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章
doi:10.1002/pbc.26295
更新日期:2017-04-01 00:00:00
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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:2008-05-01 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:2010-08-01 00:00:00
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更新日期:2016-01-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2014-03-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2013-12-01 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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更新日期:2017-08-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2016-03-01 00:00:00
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journal_title:Pediatric blood & cancer
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更新日期:2009-08-01 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
pub_type: 更正并重新发布的文章,杂志文章
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更新日期:2019-08-01 00:00:00