共 12 条
Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome
被引:87
作者:

Rao, VK
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机构: NIAID, LCID, NIH, Bethesda, MD 20892 USA

Dugan, F
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机构: NIAID, LCID, NIH, Bethesda, MD 20892 USA

Dale, JK
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机构: NIAID, LCID, NIH, Bethesda, MD 20892 USA

Davis, J
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h-index: 0
机构: NIAID, LCID, NIH, Bethesda, MD 20892 USA

Tretler, J
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机构: NIAID, LCID, NIH, Bethesda, MD 20892 USA

Hurley, JK
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机构: NIAID, LCID, NIH, Bethesda, MD 20892 USA

Fleisher, T
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机构: NIAID, LCID, NIH, Bethesda, MD 20892 USA

Puck, J
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机构: NIAID, LCID, NIH, Bethesda, MD 20892 USA

Straus, SE
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h-index: 0
机构: NIAID, LCID, NIH, Bethesda, MD 20892 USA
机构:
[1] NIAID, LCID, NIH, Bethesda, MD 20892 USA
[2] NHGRI, NIH, Bethesda, MD 20892 USA
[3] NIH, Warren Grant Magnuson Clin Ctr, Bethesda, MD 20892 USA
关键词:
autoimmune lymphoproliferative syndrome;
mycophenolate mofetil;
autoimmune neutropenia;
autoimmune haemolytic anaemia;
immune-mediated thrombocytopenia;
D O I:
10.1111/j.1365-2141.2005.05496.x
中图分类号:
R5 [内科学];
学科分类号:
1002 ;
100201 ;
摘要:
Autoimmune lymphoproliferative syndrome (ALPS) is a disorder of apoptosis associated most often with heritable FAS mutations leading to lymphadenopathy, hypersplenism and chronic refractory autoimmune cytopenias. Mycophenolate mofetil (MMF) was used to treat cytopenias in 13 ALPS patients aged 9 months to 17 years from a cohort of 118 children (aged < 18 years) and 82 adults. Twelve responded for a median follow-up of 49 weeks (range 38-240 weeks), defined by maintenance of adequate blood counts and reduction in dosage or cessation of other immunosuppressive agents. This preliminary experience suggests that MMF may spare steroid usage in patients with ALPS-associated cytopenias.
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页码:534 / 538
页数:5
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