Arthritis & Rheumatism, Volume 62,
November 2010 Abstract Supplement

Abstracts of the American College of
Rheumatology/Association of Rheumatology Health Professionals
Annual Scientific Meeting
Atlanta, Georgia November 6-11, 2010.

Long-Term Outcome of Autologous Hematopoietic Stem Cell Transplantation (AutoHSCT) Using Lymphoablative Conditioning in Treatment-Resistant Systemic Lupus Erythematosus.

Illei11,  Gabor G., Hasni7,  Sarfaraz A., Nikolov1,  Nikolay P., Hakim2,  Francis, Leitman2,  Susan, Balow9,  James, Austin4,  Howard

Bldg. 22, Room 3335, HFD-570, Silver Spring, MD
National Institute of Neurological Disroders and Stroke
NIDCR, NIH #10 1N114, Bethesda, MD
National Cancer Institute
National Institue of Arthritis, Musculoskelatal and Skin Diseases
National Institue of Dibetes, Digestive and Kidney DIseases
National Institue of Neurological Diseases and Stroke
National Institute of Arthritis, Musculoskelatal and Skin Diseases
National Institute of Arthritis, Musculoskelatal and Skin Diseases, Bethesda, MD
National Institute of Arthritis, Musculoskeletal and Skin Diseases
National Institute of Diabetes, Digestive and Kidney Diseases


Despite improvements in the outcomes of SLE patients with major organ involvement, treatment failure and relapse continue to affect significant proportion of patients. In this pilot study we tested if intensive lymphoablation followed by autoHSCT can lead to sustained, complete, treatment-free remission in severe, recalcitrant SLE and whether this approach fundamentally changes abnormal immune response.


Patients were enrolled with active SLE despite prior treatment with IV cyclophosphamide (CYC). Of the 8 patients treated, 2 had transverse myelitis, 1 retinal vasculitis and 5 WHO Class IV nephritis. Stem cell mobilization regimen consisted of 2,000 mg/m2 CYC, 750 mg/m2 rituximab (RTX) and G-CSF. Conditioning regimen consisted of 750 mg/m2 RTX, 4.8 g/m2 CYC and 120 mg/m2 fludarabine, followed by CD34+ selected stem cell infusion and G-CSF.

All immunosuppressive medications and hydroxychloroquine (HCQ) were stopped at the start of mobilization and steroids were rapidly tapered off after the transplant. Clinical response was evaluated by organ specific response criteria. Disease activity indices (SLEDAI and SLAM) were used to assess overall lupus activity. The primary endpoint was complete response (CR) at 24 months defined as no lupus activity and no treatment for lupus (including HCQ and steroids).


Among the 8 patients, there were 2 early deaths (one from diffuse alveolar damage, one from mycobacterial meningoencephalitis). One patient had lupus flare (retinal vasculitis responding to corticosteroids) 6 months post-transplant. Five patients were tapered off corticosteroids, achieved CR criteria within 6 months of transplant and SLEDAI scores of 0. One of these patients had SLE relapse 18 months post-transplant which responded to standard treatments, whereas 4 continue to be in CR beyond 4 (n=2) to 5 years (n=2). The reconstituted immune system of long-term responders showed a significant shift from a phenotype dominated by memory and activated effector T and B cells at baseline to a predominantly naïve phenotype post-transplant. In contrast, the patient who flared had an early recurrence of memory B cells and circulating plasma cells preceeding the flare.


Our data indicate that lymphoablative autoHSCT leads to sustained (over 5 years) clinical and serologic remission, without the use of any maintenance therapy in a subset of otherwise recalcitrant SLE patients. This clinical benefit is associated with marked normalization of the immune repertoire. Reducing the intensity of conditioning and/or exclusion of patients with multiple organ dysfunction may decrease short term toxicity and would make this approach an acceptable alternative for the treatment of severe SLE.

To cite this abstract, please use the following information:
Illei, Gabor G., Hasni, Sarfaraz A., Nikolov, Nikolay P., Hakim, Francis, Leitman, Susan, Balow, James, et al; Long-Term Outcome of Autologous Hematopoietic Stem Cell Transplantation (AutoHSCT) Using Lymphoablative Conditioning in Treatment-Resistant Systemic Lupus Erythematosus. [abstract]. Arthritis Rheum 2010;62 Suppl 10 :1161
DOI: 10.1002/art.28927

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