Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome.
We hypothesized that sirolimus, an mTOR inhibitor, may be effective in patients with autoimmune lymphoproliferative syndrome (ALPS) and treated patients who were intolerant to or failed other therapies. Four patients were treated for autoimmune cytopenias; all had a rapid complete or near complete response. Two patients were treated for autoimmune arthritis and colitis, demonstrating marked improvement. Three patients had complete resolution of lymphadenopathy and splenomegaly and all patients had a reduction in double negative T cells, a population hallmark of the disease. Based on these significant responses, we recommend that sirolimus be considered as second-line therapy for patients with steroid-refractory disease.
Teachey
David T
DT
Pediatric Hematology and Oncology, Children's Hospital of Philadelphia, University of Pennsylvania School of Medicine, Philadelphia PA 19104, USA. teacheyd@email.chop.edu
Greiner
Robert
R
Seif
Alix
A
Attiyeh
Edward
E
Bleesing
Jack
J
Choi
John
J
Manno
Catherine
C
Rappaport
Eric
E
Schwabe
Dirk
D
Sheen
Cecilia
C
Sullivan
Kathleen E
KE
Zhuang
Hongming
H
Wechsler
Daniel S
DS
Grupp
Stephan A
SA
D000744:Anemia, Hemolytic, Autoimmune / Q000000981:diagnostic imaging / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D004334:Drug Administration Schedule; D005500:Follow-Up Studies; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008297:Male; D049268:Positron-Emission Tomography; D011859:Radiography; D020123:Sirolimus / Q000627:therapeutic use*
D007166:Immunosuppressive Agents; D020123:Sirolimus