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Review

 

Autologous stem cell transplantation in systemic sclerosis: a systematic review

1, 2, 3, 4, 5

 

  1. The Department of Rheumatology, Fiona Stanley Hospital, Murdoch, Western Australia, Australia.
  2. The Departments of Medicine and Rheumatology, The University of Melbourne at St. Vincent’s Hospital Melbourne, Fitzroy, Victoria, Australia.
  3. The Department of Rheumatology, St. Vincent’s Hospital Melbourne, Fitzroy, Victoria, Australia.
  4. The Department of Haematology, Fiona Stanley Hospital, Murdoch, Western Australia, Australia.
  5. The Department of Rheumatology, Fiona Stanley Hospital, Murdoch, Western Australia, Australia. janet.roddy@health.wa.gov.au

CER10242
2017 Vol.35, N°4 ,Suppl.106
PI 0198, PF 0207
Review

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PMID: 28869416 [PubMed]

Received: 09/01/2017
Accepted : 29/05/2017
In Press: 30/08/2017
Published: 12/10/2017

Abstract

OBJECTIVES:
Haematopoetic autologous stem cell transplantation (ASCT) has emerged as a treatment option for patients with refractory, severe autoimmune disease. This is a systematic review of the current literature on ASCT in adult patients with systemic sclerosis (SSc).
METHODS:
Original articles published between 2005 and 2016 that evaluated the use of ASCT in patients with SSc were reviewed with respect to the primary outcomes of overall and transplant related mortality (TRM) rates, and secondary outcomes of changes in modified Rodnan Skin Score (mRSS), forced vital capacity (FVC), progression/event free survival (P/EFS) and quality of life measures. We also focussed on patient characteristics, the ASCT conditioning and mobilisation regimens used, and their relationship to patient outcome in each study.
RESULTS:
Of the 155 articles found, only 9 articles were suitable for review. There were 2 placebo-controlled trials (RCTs), ASTIS and ASSIST, and 7 observational and cohort studies. In general, patients undergoing ASCT had diffuse SSc with mRSS >14, and interstitial lung disease. The 2 RCTs showed a benefit in P/EFS (80-81%), FVC and quality of life measures in ASCT compared to monthly cyclophosphamide. All the studies showed an improvement in mRSS. TRM rates varied among studies, from 0 to 23%, with a trend to higher mortality rates in studies using higher doses of cyclophosphamide or myeloablative conditioning regimens.
CONCLUSIONS:
We conclude that ASCT is beneficial in some patients with SSc and that patient selection and conditioning regimens are critical determinants of prognosis and mortality post-ASCT.

Rheumatology Article