Rheumatology 1999; 38: 767-772
© 1999 British Society for Rheumatology
Paediatric Rheumatology: Autologous Stem Cell Transplantation in Rheumatic Diseases of Childhood |
Haematopoietic stem cell transplantation for active systemic lupus erythematosus
Series Editor: P. Woo
Northwestern University School of Medicine, Division of Hematology Oncology and the Robert H. Lurie Cancer Center, 250 East Superior, Wesley Pavilion, Room 1456, Chicago, IL 60611, USA
Correspondence to:
A. Traynor.
Abstract
Objective. For patients with systemic lupus erythematosus (SLE) who are at risk of disease-related mortality, we have initiated a protocol of intensive immunosuppression and haematopoietic stem cell support. The first patient enrolled in this study was in the midst of a lupus flare manifest by nephritis and rapidly declining renal function, uncontrolled hypertension, immune-mediated cytopenias, and serositis characterized by a large pericardial effusion and abdominal pain. Antinuclear antibody (ANA), anti-double-stranded (ds) DNA and complement were abnormal. This patient is now more than 1 yr post-stem cell transplant and is taking no immunosuppressive medication. Her serologies are normal, effusions have resolved, blood pressure is normal and renal function is markedly improved. The clinical and serological course of this patient is summarized here.
Methods. Autologous haematopoietic stem cells (HSC) were mobilized with cyclophosphamide (2.0 g/m2 ) and granulocyte colony-stimulating factor (G-CSF) (10 µg/kg/day). Stem cells were enriched ex vivo using CD34-positive immunoselection and reinfused after immunosuppression with cyclophosphamide (200 mg/kg) and antithymocyte globulin (ATG) (90 mg/kg).
Results. White blood cell engraftment with an absolute neutrophil count (ANC) of >500/µl (0.5x109 /l) and platelet engraftment with a non-transfused platelet count of >20000/µl (20x109 /l) occurred on day 10 and 14, respectively. Therapy was complicated by a cell lysis-like effect with hyperphosphataemia, hyperuricaemia, normal anion gap metabolic acidosis and transient exacerbation of renal insufficiency.
Conclusion. This is the first autologous T-cell-depleted haematopoietic stem cell transplantation performed to treat lupus in an active flare. This patient has, for the first time since disease onset (13 yr ago), entered a complete clinical and serological remission which persists at >1 yr of follow-up. The durability of this remission is unknown.
KEY WORDS: Haematopoietic stem cell transplantation, Autologous, Systemic lupus erythematosus.
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