Outcomes in progressive systemic sclerosis treated with autologous hematopoietic stem cell transplantation compared with combination therapy

Shiri Keret, Israel Henig, Tsila Zuckerman, Lisa Kaly, Aniela Shouval, Abid Awisat, Itzhak Rosner, Michael Rozenbaum, Nina Boulman, Ariela Dortort Lazar, Yair Molad, Firas Sabbah, Mohammad E. Naffaa, Emilia Hardak, Gleb Slobodin, Doron Rimar

Research output: Contribution to journalArticlepeer-review

3 Scopus citations

Abstract

Objectives: Autologous hematopoietic stem cell transplantation (AHSCT) has been shown to improve long-term survival for early diffuse progressive SSc compared with CYC. CYC, however, does not provide a long-term benefit in SSc. The combination of MMF and rituximab is a potent alternative regimen. We aimed to retrospectively compare the outcomes of SSc patients who underwent AHSCT to patients who met the eligibility criteria for AHSCT but received upfront combination therapy with MMF and rituximab. Methods: Repeated assessments of modified Rodnan Skin Score (mRSS), forced vital capacity (FVC), and diffusing capacity (DLCO) values were conducted. Clinical improvement was defined as an mRSS decrease >25% or an FVC increase >10%. Event-free survival (EFS) was defined in the absence of persistent major organ failure or death. Results: Twenty-one SSc patients in the combination therapy group were compared with 16 in the AHSCT group. Age, sex and disease duration were similar between the two groups. Clinical improvement at 12 months was seen in 18 (86%) patients in the combination group compared with 13 (81%) in the AHSCT group (P = 0.7). The hazard ratio for EFS at 24 months favoured the combination group (HR = 0.09, P = 0.04). During follow-up, both groups exhibited a significant and comparable reduction in mRSS and an increase in FVC values at each time interval up to 24 months. Conclusion: MMF and rituximab compared with AHSCT in SSc patients eligible for AHSCT resulted in similar skin and lung clinical improvement with a better safety profile at 24 months.

Original languageEnglish
Pages (from-to)1534-1538
Number of pages5
JournalRheumatology
Volume63
Issue number6
DOIs
StatePublished - 3 May 2024

Bibliographical note

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Keywords

  • autologous stem cell transplantation
  • biologic treatment
  • event-free survival
  • interstitial lung disease
  • SSc
  • treatment-related mortality

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