Use of mycophenolate mofetil for chronic, refractory immune cytopenias in children with autoimmune lymphoproliferative syndrome

被引:87
作者
Rao, VK
Dugan, F
Dale, JK
Davis, J
Tretler, J
Hurley, JK
Fleisher, T
Puck, J
Straus, SE
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.
引用
收藏
页码:534 / 538
页数:5
相关论文
共 12 条
[1]   Is posttransplant lymphoproliferative disorder (PTLD) caused by any specific immunosuppressive drug or by the transplantation per se? [J].
Birkeland, SA ;
Hamilton-Dutoit, S .
TRANSPLANTATION, 2003, 76 (06) :984-988
[2]   A prospective randomized trial comparing cyclosporine and short course methotrexate with cyclosporine and mycophenolate mofetil for GVHD prophylaxis in myeloablative allogeneic bone marrow transplantation [J].
Bolwell, B ;
Sobecks, R ;
Pohlman, B ;
Andresen, S ;
Rybicki, L ;
Kuczkowski, E ;
Kalaycio, M .
BONE MARROW TRANSPLANTATION, 2004, 34 (07) :621-625
[3]   The efficacy and safety of B-cell depletion with anti-CD20 monoclonal antibody in adults with chronic immune thrombocytopenic purpura [J].
Cooper, N ;
Stasi, R ;
Cunningham-Rundles, SS ;
Feuerstein, MA ;
Leonard, JP ;
Amadori, S ;
Bussel, JB .
BRITISH JOURNAL OF HAEMATOLOGY, 2004, 125 (02) :232-239
[4]   Autoimmune lymphoproliferative syndrome with somatic Fas mutations [J].
Holzelova, E ;
Vonarbourg, C ;
Stolzenberg, MC ;
Arkwright, PD ;
Selz, F ;
Prieur, AM ;
Blanche, S ;
Bartunkova, J ;
Vilmer, E ;
Fischer, A ;
Le Deist, F ;
Rieux-Laucat, F .
NEW ENGLAND JOURNAL OF MEDICINE, 2004, 351 (14) :1409-1418
[5]   Mycophenolate mofetil (MMF) for the treatment of steroid-resistant idiopathic thrombocytopenic purpura [J].
Hou, M ;
Peng, J ;
Shi, Y ;
Zhang, CQ ;
Qin, P ;
Zhao, CL ;
Ji, XB ;
Wang, XY ;
Zhang, MH .
EUROPEAN JOURNAL OF HAEMATOLOGY, 2003, 70 (06) :353-357
[6]   Mycophenolate mofetil for the treatment of refractory auto-immune haemolytic anaemia and auto-immune thrombocytopenia purpura [J].
Howard, J ;
Hoffbrand, AV ;
Prentice, HG ;
Mehta, A .
BRITISH JOURNAL OF HAEMATOLOGY, 2002, 117 (03) :712-715
[7]   Pharmacokinetics and tolerance of mycophenolate mofetil in renal transplant children [J].
Jacqz-Aigrain, E ;
Shaghaghi, EK ;
Baudouin, V ;
Popon, M ;
Zhang, DL ;
Maisin, A ;
Loirat, C .
PEDIATRIC NEPHROLOGY, 2000, 14 (02) :95-99
[8]   A blinded, long-term, randomized multicenter study of mycophenolate mofetil in cadaveric renal transplantation - Results at three years [J].
Mathew, TH .
TRANSPLANTATION, 1998, 65 (11) :1450-1454
[9]   Long-term outcomes in adults with chronic ITP after splenectomy failure [J].
McMillan, R ;
Durette, C .
BLOOD, 2004, 104 (04) :956-960
[10]   Somatic mutations - Not just for cancer anymore [J].
Puck, JM ;
Straus, SE .
NEW ENGLAND JOURNAL OF MEDICINE, 2004, 351 (14) :1388-1390