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Nonmyeloablative allogeneic bone marrow transplantation for treatment of childhood overlap syndrome and small vessel vasculitis

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A 13-year-old Caucasian female with a systemic connective tissue disease (overlap syndrome with pulmonary vasculitis) underwent nonmyeloablative allogeneic BMT after failure of prolonged combination immunosuppressives to induce remission. The procedure also included cotransplantation of donor bone chips as a source of stromal cells. The unique protocol allowed good engraftment of hematopoietic (>95%) and bone core stromal cells (>60%). The patient was clinically improved, stable, and off all immunosuppressive medications 36 months post-transplant. To our knowledge, this is the first pediatric nonmyeloablative BMT with cotransplantation of stromal cells solely for treatment of an autoimmune disease.

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References

  1. Marmont AM . Stem cell transplantation for severe autoimmune diseases: progress and problems. Haematologica 1998; 83: 733–743.

    CAS  PubMed  Google Scholar 

  2. Burt RK, Slavin S, Burns WH, Marmont AM . Induction of tolerance in autoimmune diseases by hematopoietic stem cell transplantation: getting closer to a cure? Blood 2002; 99: 768–784.

    Article  CAS  PubMed  Google Scholar 

  3. Tyndall A, Koike T . High-dose immunoablative therapy with hematopoietic stem cell support in the treatment of severe autoimmune disease: current status and future direction. Intern Med 2002; 41: 608–612.

    Article  PubMed  Google Scholar 

  4. Eisenberg RA, Izui S, McConahey PJ et al. Male determined accelerated autoimmune disease in BXSB mice: transfer by bone marrow and spleen cells. J Immunol 1980; 125: 1032–1036.

    CAS  PubMed  Google Scholar 

  5. Ikehara S, Kawamura M, Takao F et al. Organ-specific and systemic autoimmune diseases originate from defects in hematopoietic stem cells. Proc Natl Acad Sci USA 1990; 87: 8341–8344.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  6. Good RA, Verjee T . Historical and current perspectives on bone marrow transplantation for prevention and treatment of immunodeficiencies and autoimmunities. Biol Blood Marrow Transplant 2001; 7: 123–135.

    Article  CAS  PubMed  Google Scholar 

  7. Hinterberger W, Hinterberger-Fischer M, Marmont A . Clinically demonstrable anti-autoimmunity mediated by allogeneic immune cells favorably affects outcome after stem cell transplantation in human autoimmune diseases. Bone Marrow Transplant 2002; 30: 753–759.

    Article  CAS  PubMed  Google Scholar 

  8. Slavin S, Nagler A, Naparstek E et al. Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood 1998; 91: 756–763.

    CAS  PubMed  Google Scholar 

  9. Slavin S, Nagler A, Varadi G, Or R . Graft vs autoimmunity following allogeneic non-myeloablative blood stem cell transplantation in a patient with chronic myelogenous leukemia and severe systemic psoriasis and psoriatic polyarthritis. Exp Hematol 2000; 28: 853–857.

    Article  CAS  PubMed  Google Scholar 

  10. Marmont AM, Gualandi F, van Lint MT, Bacigalupo A . Refractory Evans' syndrome treated with allogeneic SCT followed by DLI. Demonstration of a graft-versus-autoimmunity effect. Bone Marrow Transplant 2003; 31: 399–402.

    Article  CAS  PubMed  Google Scholar 

  11. Chakrabarti S, Handa SK, Bryon RJ et al. Will mixed chimerism cure autoimmune diseases after a nonmyeloablative stem cell transplant? Transplantation 2001; 72: 340–342.

    Article  CAS  PubMed  Google Scholar 

  12. Oyama Y, Papadopoulos EB, Miranda M et al. Allogeneic stem cell transplantation for Evans syndrome. Bone Marrow Transplant 2001; 28: 903–905.

    Article  CAS  PubMed  Google Scholar 

  13. Chamarthy LM, de la Morena M, Lacson A et al. Pulmonary vasculitis presenting as atypical asthma. Ann Allergy Asthma Immunol 2000; 84: 495–498.

    Article  CAS  PubMed  Google Scholar 

  14. El-Badri NS, Wang BY, Cherry, Good RA . Osteoblasts promote engraftment of allogeneic hematopoietic stem cells. Exp Hematol 1998; 26: 110–116.

    CAS  PubMed  Google Scholar 

  15. Ishida T, Inaba M, Hisha H et al. Requirement of donor-derived stromal cells in the bone marrow for successful allogeneic bone marrow transplantation. Complete prevention of recurrence of autoimmune diseases in MRL/MP-Ipr/Ipr mice by transplantation of bone marrow plus bones (stromal cells) from the same donor. J Immunol 1994; 152: 3119–3127.

    CAS  PubMed  Google Scholar 

  16. Imasawa T, Utsunomiya Y, Kawamura T et al. The potential of bone marrow-derived cells to differentiate to glomerular amesangial cells. J Am Soc Nephrol 2001; 12: 1401–1409.

    CAS  PubMed  Google Scholar 

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Acknowledgements

We express our sincere appreciation to Dr Allison Green, the primary care pediatrician, and all specialty physicians, including Drs Martin Klemperer, Gail Cawkwell, and Magdalen Gondor for their excellent care and collaboration. We thank Drs Noorbibi Day and Joe M Jones for critical review and Ms Tazim Verjee for help in the preparation of this manuscript.

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Correspondence to O Y Jones.

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Jones, O., Good, R. & Cahill, R. Nonmyeloablative allogeneic bone marrow transplantation for treatment of childhood overlap syndrome and small vessel vasculitis. Bone Marrow Transplant 33, 1061–1063 (2004). https://doi.org/10.1038/sj.bmt.1704482

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