Limited utility of rapamycin in severe, refractory Wegener's granulomatosis.
OBJECTIVE
We report our experience using rapamycin in patients with Wegener's granulomatosis (WG) who failed to achieve remission with conventional treatment.


METHODS
Eight patients received rapamycin for severe, refractory WG. Clinical outcomes were reviewed retrospectively.


RESULTS
Four patients treated with rapamycin sustained remissions of at least 6 months' duration while receiving prednisone<or=10 mg/day; 2 relapsed during followup. Five patients discontinued rapamycin due to continued disease activity, cancer, or adverse events.


CONCLUSIONS
Toxicities due to rapamycin were numerous; consistent proof of efficacy in this pilot experience with WG was not seen.
Koening
Curry L
CL
Cleveland Clinic, Center for Vasculitis Care and Research, Department of Rheumatic and Immunologic Diseases, Cleveland, OH, USA. Curry.Koening@hsc.utah.edu
Hernández-Rodríguez
José
J
Molloy
Eamonn S
ES
Clark
Tiffany M
TM
Hoffman
Gary S
GS
D000328:Adult; D005260:Female; D005500:Follow-Up Studies; D014890:Granulomatosis with Polyangiitis / Q000188:drug therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage* / Q000009:adverse effects; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D012720:Severity of Illness Index; D020123:Sirolimus / Q000008:administration & dosage* / Q000009:adverse effects; D017211:Treatment Failure; D055815:Young Adult
D007166:Immunosuppressive Agents; D020123:Sirolimus