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Anti-CD52 Antibody-Mediated Immune Ablation with Autologous Immune Recovery for the Treatment of Refractory Juvenile Polymyositis

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Abstract

Autologous hematopoietic stem cell transplantation (HSCT) has been used for the treatment of both adult and pediatric autoimmune diseases. However, HSCT has significant side effects (neutropenia, thrombocytopenia, infertility, cardiotoxicity) and costs (HSC collection/harvesting, blood product support). In an attempt to avoid the toxicities and costs associated with HSCT, we investigated whether immune ablation similar to that achieved following myeloablative HSCT could be achieved by the intensive administration of an anti-CD52 antibody (Campath-1H antibody). The first patient treated with the treatment regime, who had refractory juvenile polymyositis, achieved immune ablation (the elimination of pre-therapy antigen-specific T lymphocyte immunity) and has had stable clinical improvement for more than 6 years.

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Acknowledgments

This research was supported by the National Institutes of Health NCRR General Clinical Research Center Grant M01 RR-43.

The assistance of Ms. Manuela Alvarez-Wilson in the preparation of the manuscript and Dr. Fred Dorey and Choo Phei Wee in the preparation of the figures is gratefully acknowledged.

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Authors and Affiliations

  1. Division of Rheumatology, Childrens Hospital Los Angeles (CHLA), 4650 Sunset Boulevard, Mail Stop 60, Los Angeles, CA, 90027, USA

    Andreas Reiff & Bracha Shaham

  2. Division of Research Immunology/Bone Marrow Transplantation, Childrens Hospital Los Angeles (CHLA), 4650 Sunset Boulevard, Mail Stop 62, Los Angeles, CA, 90027, USA

    Kenneth I. Weinberg, Gay M. Crooks & Robertson Parkman

  3. Saban Research Institute, 4650 Sunset Boulevard, Mail Stop 62, Los Angeles, CA, 90027, USA

    Andreas Reiff, Kenneth I. Weinberg, Gay M. Crooks & Robertson Parkman

  4. Keck School of Medicine, University of Southern California, Los Angeles, CA, USA

    Andreas Reiff, Bracha Shaham, Kenneth I. Weinberg, Gay M. Crooks & Robertson Parkman

  5. Stanford University, 259 Campus Drive, Room G3035, Stanford, CA, 94305, USA

    Kenneth I. Weinberg

  6. Department of Pathology & Laboratory Medicine, David Geffen School of Medicine, University of California, Los Angeles, A7-149 Center for the Health Sciences, 10833 Le Conte Ave., Los Angeles, CA, 90095-1732, USA

    Gay M. Crooks

Authors
  1. Andreas Reiff
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  2. Bracha Shaham
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  3. Kenneth I. Weinberg
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  4. Gay M. Crooks
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  5. Robertson Parkman
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Correspondence to Robertson Parkman.

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Reiff, A., Shaham, B., Weinberg, K.I. et al. Anti-CD52 Antibody-Mediated Immune Ablation with Autologous Immune Recovery for the Treatment of Refractory Juvenile Polymyositis. J Clin Immunol 31, 615–622 (2011). https://doi.org/10.1007/s10875-011-9533-7

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  • DOI: https://doi.org/10.1007/s10875-011-9533-7

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