Stem cell transplantation may be an option for patients whose lives are threatened by medically refractory vasculitis, Dr. Thomas Daikeler and his colleagues have reported.
Up to 20% of vasculitis patients don't respond to conventionally dosed immunosuppression, Dr. Daikeler said in an interview. Options for these patients are very limited, he said. However, his review of 20 patients who received the transplants showed that 93% responded positively, with 7 achieving sustained complete remission (Ann. Rheum. Dis. 2006 Sept. 1 [Epub doi:10.1136/ard.2006.056630]).
Dr. Daikeler, of the University of Basel, Switzerland, reviewed outcomes in 15 patients included in the European League Against Rheumatism (EULAR) database or the PROMISE international medical database, and 5 additional patients identified through a Medline search.
The report focused on the details of the 15 EULAR/PROMISE patients. Their median age was 37 years (age range 10–57 years). Four had cryoglobulinemia, three had Behçet's syndrome, three had Wegener's granulomatosis, and the others had Churg-Strauss angiitis, Takayasu's arteritis, polychondritis, and a polyarteritis nodosa. All patients had active disease and had failed intensive immunosuppressive therapy, including cytostatic drugs. Most of them (14) had an autologous stem cell transplant first; 1 had an allogeneic transplant first.
At the time of the analysis, the median follow-up for all patients was 44 months (range 16–84 months). The response rate was 93%. Overall, seven responded partially and needed maintenance immunosuppression for minor disease, seven patients were in complete remission, and one patient showed stable disease.
Three patients died. One patient with partial response relapsed 24 months after allogenic transplant and died of died of graft-versus-host disease; a second patient who achieved complete response of his underlying polyarteritis nodosa died of lung cancer 2 years after autologous transplant; the third patient showed no response to therapy and died of right ventricular failure due to severe preexisting pulmonary hypertension 26 months after the initial transplant.
Among the 14 patients who received an autologous transplant, there were 2 relapses—one at 2 months and one at 24 months post transplant. One of these patients then received an allogeneic stem cell transplant and relapsed again 2 years later with headache and aphthous disease. However, the patient was successfully treated with four cycles of rituximab and cyclophosphamide.
The second relapsed patient received another autologous transplant 24 months after the first one. This was followed by another relapse 4 months later, and then an allogeneic transplant, which led to partial remission lasting for 24 months. Six patients in the group experienced neutropenic fever, and two others had reactivation of cytomegalic and Epstein-Barr virus. One patient experienced transient pancytopenia and transient cardiotoxicity.
Among the five patients identified in the Medline search, the following outcomes occurred: complete remission 18 months after transplant in a patient with polyarteritis nodosa; cessation of all disease activity, no medication necessary, for a 4-year-old with intestinal Behçet's syndrome; cessation of all immunosuppressive therapy 18 months after transplant for a patient with Takayasu's arteritis; cessation of disease activity, no medication necessary, for a child with small-vessel vasculitis; complete remission 16 months after transplant for patient with Wegener's granulomatosis.
Since the graft-versus-host disease is an issue only with allogeneic transplants, Dr. Daikeler suggested that autologous transplants for these patients be evaluated in larger clinical trials.