Abstract:
BACKGROUND:Although hematopoietic stem cell transplantation (HSCT) is offered as a curative therapy for pediatric myelodysplastic syndrome (MDS), it may cause severe complications and mortality. Several reports have shown the efficacy of immunosuppressive therapy (IST) in adult patients with refractory anemia (RA), but its safety and efficacy remains to be fully elucidated in childhood RA. PROCEDURE:Eleven children diagnosed with RA and enrolled on a prospective multicenter trial conducted by the Japanese Childhood MDS Study Group were eligible for analysis. If patients showed transfusion dependent or suffered from infection due to neutropenia, they received IST consisting of antithymocyte globulin (ATG), cyclosporine (CyA), and methylprednisolone (mPSL). RESULTS:Eight children received IST, 2 received only supportive therapy, and one underwent HSCT without IST. Five (63%) of eight children who received IST showed hematological response. Of note, one patient showed the disappearance of monosomy 7 after IST. Responders were significantly younger than non-responders (29 months vs. 140 months; P = 0.03). No severe adverse events related to IST were reported in this study. Of 6 children with chromosomal abnormalities who received IST, four showed hematological response. The probability of failure-free and overall survival at 5 years was 63 +/- 17% and 90 +/- 9% respectively. CONCLUSION:IST is likely to be a safe and effective modality for childhood RA.
journal_name
Pediatr Blood Cancerjournal_title
Pediatric blood & cancerauthors
Hasegawa D,Manabe A,Yagasaki H,Ohtsuka Y,Inoue M,Kikuchi A,Ohara A,Tsuchida M,Kojima S,Nakahata T,Japanese Childhood MDS Study Group.doi
10.1002/pbc.22121subject
Has Abstractpub_date
2009-12-01 00:00:00pages
1011-5issue
6eissn
1545-5009issn
1545-5017journal_volume
53pub_type
临床试验,杂志文章,多中心研究abstract:BACKGROUND:Alvespimycin (17-DMAG, KOS-1022), a potent small-molecule inhibitor of the protein chaperone Hsp90, is being developed as an anticancer agent because of the multiple Hsp90 client proteins involved in cancer cell growth and survival. PROCEDURES:Alvespimycin was tested against the in vitro panel of the Pediat...
journal_title:Pediatric blood & cancer
pub_type: 杂志文章
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
pub_type: 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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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
pub_type: 杂志文章
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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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journal_title:Pediatric blood & cancer
pub_type: 临床试验,杂志文章
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更新日期:2007-09-01 00:00:00
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pub_type: 杂志文章,多中心研究,随机对照试验
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journal_title:Pediatric blood & cancer
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journal_title:Pediatric blood & cancer
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pub_type: 杂志文章
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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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更新日期:2009-08-01 00:00:00
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journal_title:Pediatric blood & cancer
pub_type: 杂志文章,多中心研究,随机对照试验
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更新日期:2016-08-01 00:00:00