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Walker UA, Saketkoo LA, Distler O. Haematopoietic stem cell transplantation in systemic sclerosis. RMD Open. 2018;4(1):e000533doi: 10.1136/rmdopen-2017-000533.
Walker, U. A., Saketkoo, L. A., & Distler, O. (2018). Haematopoietic stem cell transplantation in systemic sclerosis. RMD open, 4(1), e000533. https://doi.org/10.1136/rmdopen-2017-000533
Walker, Ulrich A, et al. "Haematopoietic stem cell transplantation in systemic sclerosis." RMD open vol. 4,1 (2018): e000533. doi: https://doi.org/10.1136/rmdopen-2017-000533
Walker UA, Saketkoo LA, Distler O. Haematopoietic stem cell transplantation in systemic sclerosis. RMD Open. 2018 Jun 17;4(1):e000533. doi: 10.1136/rmdopen-2017-000533. eCollection 2018. PMID: 30018796; PMCID: PMC6045702.
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RMD Open. 2018 Jun 17;4(1):e000533. doi: 10.1136/rmdopen-2017-000533. eCollection 2018.

Haematopoietic stem cell transplantation in systemic sclerosis.

RMD open

Ulrich A Walker, Lesley Ann Saketkoo, Oliver Distler

Affiliations

  1. Department of Rheumatology, University Hospital Basel, Basel, Switzerland.
  2. Tulane University School of Medicine Lung Center, New Orleans Scleroderma and Sarcoidosis Patient Care and Research Center, University Medical Center - Comprehensive Pulmonary Hypertension Center, New Orleans, Louisiana, USA.
  3. Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland.

PMID: 30018796 PMCID: PMC6045702 DOI: 10.1136/rmdopen-2017-000533

Abstract

Three randomised controlled trials of haematopoietic stem cell transplantation (HSCT) in systemic sclerosis (SSc) demonstrated long-term survival benefits, induction of clinically meaningful, sustained improvement of forced vital capacity with improvements in skin thickening, vasculopathy and health-related quality of life, in contrast to a clinical decline in standard of care control groups. These benefits, however, must be weighed against the increased risk of transplant-related mortality. Further, with disease progression, severe extensive internal organ involvement and damage ensues, constituting an exclusion criterion for safety reasons, leaving a limited window whereby patients with SSc are eligible for HSCT. Although autologous HSCT offers the possibility of drug-free remission, relapse can occur, requiring re-initiation of disease modifying antirheumatic drugs. HSCT is also associated with secondary autoimmune diseases and gonadal failure. HSCT should be proposed for carefully selected patients with early rapidly progressive diffuse SSc whose clinical picture portends a poor prognosis for survival, but yet lacks advanced organ involvement.

Keywords: pulmonary fibrosis; systemic sclerosis; treatment

Conflict of interest statement

Competing interests: None declared.

References

  1. Bone Marrow Transplant. 1999 Apr;23(7):637-46 - PubMed
  2. Lancet. 2016 Jun 25;387(10038):2630-2640 - PubMed
  3. N Engl J Med. 1997 Mar 27;336(13):897-904 - PubMed
  4. Ann Rheum Dis. 2017 Aug;76(8):1327-1339 - PubMed
  5. Rheumatology (Oxford). 2018 Mar 1;57(3):441-450 - PubMed
  6. Br J Rheumatol. 1996 Apr;35(4):364-72 - PubMed
  7. J Rheumatol. 2012 Feb;39(2):269-75 - PubMed
  8. Blood. 2006 Aug 1;108(3):1100-5 - PubMed
  9. Ann Rheum Dis. 2010 Oct;69(10):1809-15 - PubMed
  10. Bone Marrow Transplant. 2016 Apr;51(4):501-5 - PubMed
  11. Ann Rheum Dis. 2004 Aug;63(8):974-81 - PubMed
  12. Rheumatol Int. 2016 Nov;36(11):1563-1568 - PubMed
  13. Bone Marrow Transplant. 2017 Nov;52(11):1495-1503 - PubMed
  14. Ann Rheum Dis. 2001 Jun;60(6):577-84 - PubMed
  15. Ann Rheum Dis. 2009 Jan;68(1):94-8 - PubMed
  16. Ann Rheum Dis. 2011 Oct;70(10):1788-92 - PubMed
  17. Rheumatology (Oxford). 2015 Dec;54(12):2126-33 - PubMed
  18. Haematologica. 2010 Feb;95(2):284-92 - PubMed
  19. Nat Rev Rheumatol. 2017 Apr;13(4):244-256 - PubMed
  20. N Engl J Med. 2018 Jan 4;378(1):35-47 - PubMed
  21. Bone Marrow Transplant. 2007 Apr;39(7):435-7 - PubMed
  22. Nature. 2005 Jun 2;435(7042):620-7 - PubMed
  23. Semin Arthritis Rheum. 2014 Apr;43(5):666-72 - PubMed
  24. Blood. 2008 Feb 1;111(3):989-96 - PubMed
  25. Blood. 2007 Aug 15;110(4):1388-96 - PubMed
  26. Bone Marrow Transplant. 2017 Jan;52(1):53-58 - PubMed
  27. J Rheumatol. 2009 Jul;36(7):1460-3 - PubMed
  28. Rheumatology (Oxford). 2014 May;53(5):919-22 - PubMed
  29. Blood. 2002 Sep 1;100(5):1602-10 - PubMed
  30. JAMA. 2014 Jun 25;311(24):2490-8 - PubMed
  31. PLoS One. 2008 Jan 16;3(1):e1452 - PubMed
  32. Br J Haematol. 2012 May;157(3):281-90 - PubMed
  33. Lancet. 2011 Aug 6;378(9790):498-506 - PubMed
  34. Lancet. 2013 Mar 30;381(9872):1116-24 - PubMed
  35. Arthritis Rheum. 2006 Jun;54(6):1982-6 - PubMed
  36. Bone Marrow Transplant. 2012 Jun;47(6):770-90 - PubMed
  37. Blood. 2007 Mar 15;109(6):2643-548 - PubMed
  38. Ann Rheum Dis. 2017 Nov;76(11):1897-1905 - PubMed
  39. Ann Rheum Dis. 2008 Jul;67(7):1057-9 - PubMed
  40. Ann Rheum Dis. 2008 Jan;67(1):98-104 - PubMed
  41. Rheumatology (Oxford). 1999 Aug;38(8):757-61 - PubMed
  42. Blood. 2011 Aug 11;118(6):1693-8 - PubMed
  43. Arthritis Res Ther. 2008;10(5):R124 - PubMed
  44. Arthritis Rheum. 2001 Jun;44(6):1351-8 - PubMed
  45. Med Decis Making. 2008 Nov-Dec;28(6):926-37 - PubMed
  46. Bone Marrow Transplant. 2007 Sep;40(6):549-55 - PubMed

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