More work is needed to understand the role immunosuppression may have in treating GI involvement in scleroderma. This is timely and relevant in light of the recent SCOT trial.6 In this study, scleroderma patients treated with myeloablative autologous stem-cell transplantation experienced significantly higher rates of disease-free survival at 52 months post-treatment compared with those treated with cyclophosphamide. Although treatment-related mortality in the transplantation group was 3% at 54 months and 6% at 72 months, compared with 0% in the cyclophosphamide group, this study has thrust myeloablative autologous stem cell transplantation into the spotlight. This attention has resulted in many providers and patients wondering in which scenarios it may be reasonable to pursue this course of treatment.
With much work remaining to better understand diagnosis and treatment of GI disease in patients with scleroderma, the presenters closed their remarks with hope for a future in which many questions are answered through research and clinical experience. Indeed, it is more than a gut feeling that such a future is important for the many patients dealing with the daily symptoms created by GI involvement in scleroderma.
Jason Liebowitz, MD, recently completed his fellowship in rheumatology at Johns Hopkins University, Baltimore, where he also earned his MD. He is currently in practice with Arthritis, Rheumatic, and Back Disease Associates, New Jersey.
References
- Richard N, Hudson M, Wang M, et al. Severe gastrointestinal disease in very early systemic sclerosis is associated with early mortality. Rheumatology (Oxford). 2019 Apr 1;58(4):636–644.
- Abozaid HSM, Imam HMK, Abdelaziz MM, et al. High-resolution manometry compared with the University of California, Los Angeles Scleroderma Clinical Trials Consortium GIT 2.0 in systemic sclerosis. Semin Arthritis Rheum. 2017 Dec;47(3):40–408.
- McMahan ZH, Hummers LK. Gastrointestinal involvement in systemic sclerosis: Diagnosis and management. Curr Opin Rheumatol. 2018 Nov;30(6):533–540.
- Hansi N, Thoua N, Carulli M, et al. Consensus best practice pathway of the U.K. scleroderma study group: Gastrointestinal manifestations of systemic sclerosis. Clin Exp Rheumatol. 2014 Nov-Dec;32(6 Suppl 86):S-214-21.
- Bhattacharyya A, Sahhar J, Milliken S, et al. Autologous hematopoietic stem cell transplant for systemic sclerosis improves anemia from gastricantral vascular ectasia. J Rheumatol. 2015 Mar;42(3):554-555.
- Sullivan KM, Goldmuntz EA, Keyes-Elstein L, et al. Myeloablative autologous stem-cell transplantation for severe scleroderma. N Engl J Med. 2018 Jan 4;378(1):35–47.