Autologous haematopoietic stem cell transplantation for systemic lupus erythematosus: Data from the European Group for Blood and Marrow Transplantation registry

B. Alchi, D. Jayne, M. Labopin, O. Kotova, V. Sergeevicheva, T. Alexander, F. Gualandi, B. Gruhn, J. Ouyang, P. Rzepecki, G. Held, A. Sampol, J. Voswinkel, P. Ljungman, A. Fassas, M. Badoglio, R. Saccardi, D. Farge

Research output: Contribution to journalArticle

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Abstract

Objectives: Patients with systemic lupus erythematosus (SLE) refractory to conventional immunosuppression suffer substantial morbidity and mortality due to active disease and treatment toxicity. Immunoablation followed by autologous stem cell transplantation (ASCT) is a novel therapeutic strategy that potentially offers new hope to these patients. Methods: This retrospective survey reviews the efficacy and safety of ASCT in 28 SLE patients from eight centres reported to the European Group for Blood and Marrow Transplantation (EBMT) registry between 2001 and 2008. Results: Median disease duration before ASCT was 52 (nine to 396) months, 25/28 SLE patients (89%) were female, age 29 (1648) years. At the time of ASCT, eight (one to 11) American College of Rheumatology (ACR) diagnostic criteria for SLE were present and 17 (60%) patients had nephritis. Peripheral blood stem cells were mobilized with cyclophosphamide and granulocyte-colony stimulating factor in 93% of patients, and ex vivo CD34 stem cell selection was performed in 36%. Conditioning regimens were employed with either low (n=10) or intermediate (18) intensities. With a median follow-up of 38 (one to 110) months after ASCT, the five-year overall survival was 81±8%, disease-free survival was 29±9%, relapse incidence (RI) was 56±11% and nonrelapse mortality was 15±7%. Graft manipulation by CD34+ selection was associated with a lower RI (p=0.001) on univariate analysis. There were five deaths within two years after ASCT: three caused by infection, one by secondary autoimmune disease and one by progressive SLE. Conclusions: Our data further support the concept of immunoablation and ASCT to re-induce long-term clinical and serologic remissions in refractory SLE patients even in the absence of maintenance therapy. This study also suggests a beneficial effect of ex vivo graft manipulation on prevention of relapses post-transplantation in SLE. Lupus (2013) 22, 245253.

Original languageEnglish
Pages (from-to)245-253
Number of pages9
JournalLupus
Volume22
Issue number3
DOIs
Publication statusPublished - Mar 2013

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Hematopoietic Stem Cell Transplantation
Stem Cell Transplantation
Systemic Lupus Erythematosus
Registries
Transplants
Recurrence
Mortality
Nephritis
Incidence
Granulocyte Colony-Stimulating Factor
Secondary Prevention
Coinfection
Cyclophosphamide
Immunosuppression
Autoimmune Diseases
Disease-Free Survival
Stem Cells
Therapeutics
Transplantation
Morbidity

Keywords

  • Anti-DNA antibodies
  • Anticardiolipin Antibodies
  • Cardiovascular Disease
  • Haematologic changes
  • Nephritis
  • Systemic Lupus Erythematosus

ASJC Scopus subject areas

  • Rheumatology

Cite this

Autologous haematopoietic stem cell transplantation for systemic lupus erythematosus : Data from the European Group for Blood and Marrow Transplantation registry. / Alchi, B.; Jayne, D.; Labopin, M.; Kotova, O.; Sergeevicheva, V.; Alexander, T.; Gualandi, F.; Gruhn, B.; Ouyang, J.; Rzepecki, P.; Held, G.; Sampol, A.; Voswinkel, J.; Ljungman, P.; Fassas, A.; Badoglio, M.; Saccardi, R.; Farge, D.

In: Lupus, Vol. 22, No. 3, 03.2013, p. 245-253.

Research output: Contribution to journalArticle

Alchi, B, Jayne, D, Labopin, M, Kotova, O, Sergeevicheva, V, Alexander, T, Gualandi, F, Gruhn, B, Ouyang, J, Rzepecki, P, Held, G, Sampol, A, Voswinkel, J, Ljungman, P, Fassas, A, Badoglio, M, Saccardi, R & Farge, D 2013, 'Autologous haematopoietic stem cell transplantation for systemic lupus erythematosus: Data from the European Group for Blood and Marrow Transplantation registry', Lupus, vol. 22, no. 3, pp. 245-253. https://doi.org/10.1177/0961203312470729
Alchi, B. ; Jayne, D. ; Labopin, M. ; Kotova, O. ; Sergeevicheva, V. ; Alexander, T. ; Gualandi, F. ; Gruhn, B. ; Ouyang, J. ; Rzepecki, P. ; Held, G. ; Sampol, A. ; Voswinkel, J. ; Ljungman, P. ; Fassas, A. ; Badoglio, M. ; Saccardi, R. ; Farge, D. / Autologous haematopoietic stem cell transplantation for systemic lupus erythematosus : Data from the European Group for Blood and Marrow Transplantation registry. In: Lupus. 2013 ; Vol. 22, No. 3. pp. 245-253.
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T2 - Data from the European Group for Blood and Marrow Transplantation registry

AU - Alchi, B.

AU - Jayne, D.

AU - Labopin, M.

AU - Kotova, O.

AU - Sergeevicheva, V.

AU - Alexander, T.

AU - Gualandi, F.

AU - Gruhn, B.

AU - Ouyang, J.

AU - Rzepecki, P.

AU - Held, G.

AU - Sampol, A.

AU - Voswinkel, J.

AU - Ljungman, P.

AU - Fassas, A.

AU - Badoglio, M.

AU - Saccardi, R.

AU - Farge, D.

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N2 - Objectives: Patients with systemic lupus erythematosus (SLE) refractory to conventional immunosuppression suffer substantial morbidity and mortality due to active disease and treatment toxicity. Immunoablation followed by autologous stem cell transplantation (ASCT) is a novel therapeutic strategy that potentially offers new hope to these patients. Methods: This retrospective survey reviews the efficacy and safety of ASCT in 28 SLE patients from eight centres reported to the European Group for Blood and Marrow Transplantation (EBMT) registry between 2001 and 2008. Results: Median disease duration before ASCT was 52 (nine to 396) months, 25/28 SLE patients (89%) were female, age 29 (1648) years. At the time of ASCT, eight (one to 11) American College of Rheumatology (ACR) diagnostic criteria for SLE were present and 17 (60%) patients had nephritis. Peripheral blood stem cells were mobilized with cyclophosphamide and granulocyte-colony stimulating factor in 93% of patients, and ex vivo CD34 stem cell selection was performed in 36%. Conditioning regimens were employed with either low (n=10) or intermediate (18) intensities. With a median follow-up of 38 (one to 110) months after ASCT, the five-year overall survival was 81±8%, disease-free survival was 29±9%, relapse incidence (RI) was 56±11% and nonrelapse mortality was 15±7%. Graft manipulation by CD34+ selection was associated with a lower RI (p=0.001) on univariate analysis. There were five deaths within two years after ASCT: three caused by infection, one by secondary autoimmune disease and one by progressive SLE. Conclusions: Our data further support the concept of immunoablation and ASCT to re-induce long-term clinical and serologic remissions in refractory SLE patients even in the absence of maintenance therapy. This study also suggests a beneficial effect of ex vivo graft manipulation on prevention of relapses post-transplantation in SLE. Lupus (2013) 22, 245253.

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KW - Haematologic changes

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