Use of Sirolimus (Rapamycin) for Treatment of Cytopenias and Lymphoproliferation Linked to Autoimmune Lymphoproliferative Syndrome (ALPS). Two Case Reports

被引:7
作者
Cayrol, Julie [1 ,2 ]
Colino, Carmen Garrido [1 ]
机构
[1] Gregorio Maranon Childrens Hosp, Dept Pediat Hematol Oncol, Biomed Res Inst, Madrid, Spain
[2] Royal Childrens Hosp, Childrens Canc Ctr, Melbourne, Vic, Australia
关键词
autoimmune lymphoproliferative syndrome; lymphoproliferation; cytopenias; sirolimus; SYNDROME ALPS;
D O I
10.1097/MPH.0000000000000785
中图分类号
R73 [肿瘤学];
学科分类号
100214 ;
摘要
Autoimmune lymphoproliferative syndrome (ALPS) is a disorder of lymphocyte apoptosis. Children present with chronic nonmalignant lymphadenopathy, hepatosplenomegaly, and autoimmune cytopenias. Recent advances show efficacy of treatment with immunosuppressive drugs. Sirolimus, an mammalian target of rapamycin inhibitor, improves autoimmune cytopenias and lymphoproliferation, with a safe profile. We present 2 patients, a 5-year-old girl and 15-year-old boy, diagnosed with ALPS with initial partial response to steroid treatment. Autoimmune cytopenias and lymphoproliferation then became refractory to treatment, with recurrence of symptoms. In both cases, treatment with sirolimus was started, with a rapid response, complete remission of cytopenias, and resolution of lymphoproliferation, with no significant adverse effects. Conclusion: sirolimus is an effective and safe drug for controlling children with cytopenias and lymphoproliferation linked to ALPS.
引用
收藏
页码:E187 / E190
页数:4
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