Systemic sclerosis (SS)
Transplant advances and outcomes
Systemic sclerosis (SS) or scleroderma is a rare autoimmune disease affecting approximately 50 per 100,000 people in the United States. [1] It is characterized by debilitating effects on the connective tissue and various internal organs, caused by genetic and environmental predispositions. [2]
The use of autologous or allogeneic HCT is being considered for the treatment of SS. Preclinical and clinical studies suggest that high-dose immunotherapy alongside allogeneic HCT may improve outcomes. [3]
Risks related to the treatment regimen, graft-versus-host disease, relapse and overall transplant eligibility must be weighed when choosing the best option and stem cell source for transplant. [3]
Recommended timing for transplant consultation
- At diagnosis or with diffuse disease
- With increasing skin tightness score (modified Rodnan skin score, [mRSS])
- Evidence of decrease ( <80%) in % predicted pulmonary function tests: forced vital capacity (FVC) and/or diffusion capacity (DLCO)
References
- Zhong L, Pope M, Shen Y, Hernandez JJ, Wu L. Prevalence and incidence of systemic sclerosis: A systematic review and meta-analysis. Int J Rheum Dis. 2019;22(12):2096-2107. doi:10.1111/1756-185X.13716. Access
- Asano Y. Systemic sclerosis. The Journal of Dermatology. 2018;45(2):128-138. doi:10.1111/1346-8138.14153. Access
- Sullivan KM, Sarantopoulos S. Allogeneic HSCT for autoimmune disease: a shared decision. Nature reviews Rheumatology. 2019;15(12):701-702. doi:10.1038/s41584-019-0306-7. Access