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Safety and efficacy of HSCT for systemic sclerosis across clinical trials

Nature Reviews Rheumatology volume 16, page 661 (2020)Cite this article

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Haematopoietic stem cell transplantation was superior to cyclophosphamide for the treatment of systemic sclerosis in three randomized clinical trials: ASSIST1, ASTIS2 and SCOT3. Although all three trials infused autologous haematopoietic cells, they employed different preparative conditioning regimens, for which the relative safety and efficacy are unknown. We read with interest the News & Views commentary by Burt and Farge (Systemic sclerosis: autologous HSCT is efficacious, but can we make it safer? Nat. Rev. Rheumatol. 14, 189–191 (2018))4 comparing the results of these trials plus an uncontrolled retrospective study of the ASSIST regimen5. However, misapplied cross-study comparisons led to several faulty conclusions.

Burt and Farge asserted “the SCOT trial did not improve pulmonary function”4. However, in a longitudinal analysis of forced vital capacity (FVC) trends, transplant was superior to cyclophosphamide6. The supplemental analysis they cited did not evaluate the treatment effect on FVC, as the event-free survival advantage of transplant is removed.

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References

  1. Burt, R. K. et al. Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial. Lancet 378, 498–506 (2011).

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  2. van Laar, J. M. et al. Autologous hematopoietic stem cell transplantation versus intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. JAMA 311, 2490–2498 (2014).

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  3. Sullivan, K. M. et al. Myeloablative autologous stem-cell transplantation for severe scleroderma. N. Engl. J. Med. 378, 35–47 (2018).

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  4. Burt, R. K. & Farge, D. Systemic sclerosis: autologous HSCT is efficacious, but can we make it safer? Nat. Rev. Rheumatol. 14, 189–191 (2018).

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  5. Burt, R. K. et al. Cardiac involvement and treatment-related mortality after non-myeloablative haemopoietic stem-cell transplantation with unselected autologous peripheral blood for patients with systemic sclerosis: a retrospective analysis. Lancet 381, 1116–1124 (2013).

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  6. Keyes-Elstein, L. et al. Longitudinal trends in clinical disease features after myeloablative autologous stem-cell transplantation or cyclophosphamide in severe scleroderma [abstract]. Arthritis Rheum. 70 (suppl. 10), 902 (2018).

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  7. Sullivan, K. M. & Keyes-Elstein, L. Cross trial comparisons in clinical practice reviews: proceed with caution. Nat. Rev. Rheumatol. https://doi.org/10.1038/s41584-020-0492-3 (2020).

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Authors and Affiliations

  1. Rho Federal Systems, Durham, NC, USA

    Lynette Keyes-Elstein & Ashley Pinckney

  2. National Institute of Allergy and Infectious Diseases, Bethesda, MD, USA

    Erica Brittain & Ellen A. Goldmuntz

  3. Duke University Medical Center, Durham, NC, USA

    Keith M. Sullivan

Authors
  1. Lynette Keyes-Elstein
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  2. Erica Brittain
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  3. Ashley Pinckney
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  4. Ellen A. Goldmuntz
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  5. Keith M. Sullivan
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Corresponding author

Correspondence to Keith M. Sullivan.

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The authors declare no competing interests.

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Keyes-Elstein, L., Brittain, E., Pinckney, A. et al. Safety and efficacy of HSCT for systemic sclerosis across clinical trials. Nat Rev Rheumatol 16, 661 (2020). https://doi.org/10.1038/s41584-020-0493-2

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