Allogeneic hematopoietic SCT for patients with autoimmune diseases

Abstract

Allogeneic hematopoietic SCT (HSCT) has been used as treatment for single patients with autoimmune diseases (AD). To summarise currently available information, we analyzed all patients who underwent allogeneic HSCT for AD and who reported to the European Group for Blood and Marrow Transplantation (EBMT) database. Thirty-five patients receiving 38 allogeneic transplantations for various hematological and non-hematological AD were identified. Four patients had had an allogeneic HSCT for a conventional hematological indication in the past. Fifty-five per cent of the transplantation procedures led to a complete clinical response of the refractory AD and 23% to at least a partial response. The median duration of response at the last followup was 70.7 (15.2-130) months. Three patients relapsed at a median of 12.3 months after HSCT. Treatment-related mortality at 2 years was 22.1% (95% CI: 7.3-36.9%). Two deaths were caused by progression of AD. The probability of survival at 2 years was 70%. No single factor predicting the outcome could be identified. The retrospective nature of this study and the heterogeneous, partly incomplete data are its limitations. However, allogeneic HSCT can induce remission in patients suffering from refractory AD. These data provide the basis for carefully conducted prospective trials.

Successful allogeneic cord blood transplantation in a patient with Evans syndrome leads to correction of hereditary angioedema type I as secondary effect

“…Treatment-related mortality at 2 years follow-up was 22.1% (95% confidence interval: 7.3 --36.9%). 6 We report the case of a 7-year-old boy with ES and concomitant HAE, both cured by allogeneic cord blood SCT. Only one similar case was published in 2005 correcting myelofibrosis and recurrent acquired angioedema following nonmyeloablative allo-SCT.…”

Long-term remission in pediatric Wegener granulomatosis following allo-SCT after reduced-intensity conditioning

“…1,2 Kidney or lung involvement at diagnosis is associated with a threefold increased mortality, and up to 30% of patients with WG requiring intensive care die within the first month. 3,6 Immunoablative chemotherapy with subsequent auto-SCT has become an accepted therapy for severe autoimmune diseases, 7 but disease recurrence remains a major obstacle. 4 Auto-SCT for WG has been associated with persistence of ANCAs and new autoimmune phenomena.…”

Significant improvement of Takayasu arteritis after cord blood transplantation in a patient with myelodysplastic syndrome

“…Some studies have demonstrated that cord blood stem cells have the potential to induce vasculogenesis. 11 There have been reports of CBT for AID, such as Evans syndrome, especially in pediatric settings, 3,8 but only anecdotal data are available. To our knowledge, this is the first report indicating the effectiveness of CBT for TA.…”

“…1-3, 8 Daikeler et al 8 performed a retrospective analysis of 35 patients receiving allogeneic HSCT for AID, and its response rate and TRM at 2 years were 79% (CR 55% and PR 24%) and 22.1%, respectively. They concluded that allogeneic HSCT can induce remission in patients with refractory AID.…”