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Autologous hematopoietic stem cell transplantation for autoimmune diseases

2005; Springer Nature; Volume: 35; Issue: 9 Linguagem: Inglês

10.1038/sj.bmt.1704892

1476-5365

Aloïs Gratwohl, Jakob Passweg, Chiara Bocelli‐Tyndall, Αthanasios Fassas, Jacob M. van Laar, Dominique Farge, M Andolina, R. Arnold, Enric Carreras, Jürgen Finke, Ina Kötter, Tomáš Kozák, Igor Lisukov, Bob Löwenberg, A Marmont, John J. Moore, Riccardo Saccardi, John A. Snowden, F.H.J. van den Hoogen, Nico Wulffraat, Xiaodong Zhao, Alan Tyndall,

Autoimmune Bullous Skin Diseases

Experimental data and early phase I/II studies suggest that high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation (HSCT) can arrest progression of severe autoimmune diseases. We have evaluated the toxicity and disease response in 473 patients with severe autoimmune disease treated with autologous HSCT between 1995 and 2003, from 110 centers participating in the European Group for Blood and Marrow Transplantation (EBMT) autoimmune disease working party database. Survival, transplant-related mortality, treatment response and disease progression were assessed. In all, 420 patients (89%; 86±4% at 3 years, median follow-up 20 months) were alive, 53 (11%) had died from transplant-related mortality (N=31; 7±3% at 3 years) or disease progression (N=22; 9±4% at 3 years). Of 370 patients, 299 evaluable for response (81%) showed a treatment response, which was sustained in 213 (71% of responders). Response was associated with disease (P 40 years, RR0.29 (0.11–0.82)). Disease progression was associated with disease (P<0.001) and conditioning intensity (high intensity, RR1; intermediate intensity, RR1.81 (0.96–3.42)); low intensity, RR2.34 (1.074–5.11)). These data from the collective EBMT experience support the hypothesis that autologous HSCT can alter disease progression in severe autoimmune disease.