Hematopoietic Stem Cell Transplantation Improves Functional Outcomes of Systemic Sclerosis Patients

Abstract

Background/Objective We sought to evaluate if autologous hematopoietic stem cell transplantation (AHSCT) influences the functional status of systemic sclerosis (SSc) patients. Methods From 2014 to 2018, a cohort of 27 SSc patients was assessed before, and at 6 and 12 months after AHSCT for modified Rodnan’s skin score (mRSS), mouth opening, hand grip strength, range of motion (ROM), functional ability of upper limbs (DASH questionnaire and Cochin hand function scale—CHFS), 6-minute walk test (6MWT), and quality of life (SF-36 questionnaire). Linear regression models with random effects and Spearman’s test were used for statistical analysis. Results At 6 and 12 months after AHSCT, respectively, we observed significant improvement of mRSS (p < 0.01 and p < 0.01), mouth opening (p = 0.02 and p < 0.01), hand function (DASH, p < 0.01 and p < 0.01; CHFS, p < 0.01 and p < 0.01; strength, p < 0.01 and p < 0.01), physical capacity (6MWT, p = 0.02 and p = 0.03) and physical (p < 0.01 and p < 0.01) and mental (ns and p = 0.02) component scores of SF-36. At 12 months after AHSCT, ROM measurements improved (p < 0.05) in five out of six evaluated joints in both hands, compared to baseline. Correlation was significant between physical capacity and quality of life (R = 0.62; p < 0.01), between DASH and quality of life (R = −0.48; p = 0.03), and between skin involvement and wrist ROM measures (dominant hand, R = −0.65, p < 0.01; non-dominant hand, R = −0.59; p < 0.01). Conclusions AHSCT enhances the functional status of SSc patients in the first year of follow-up, significantly improving hand function, physical capacity and quality of life. These results are interpreted as positive outcomes of AHSCT for SSc.

Hematopoietic stem cell transplantation for systemic sclerosis: Brazilian experience

“…Skin involvement in SSc has a negative impact on survival and on quality of life [36]. A previous study from our group, which included a subset of patients also enrolled in this present study, has shown that autologous HSCT improves functional outcomes of SSc patients, including functional capacity measured by the 6-min walking distance, inspiratory and expiratory lung pressures, joint range-of-motion, hand function and mouth opening, as well as physical and mental aspects of quality of life [37]. We consider important to evaluate such outcomes, as they reflect the positive impact of HSCT on the daily life of patients.…”

Autologous Hematopoietic Stem Cell Therapy of the Subjects with Systemic Sclerosis: Electromyographic Results of the Masticatory Muscles

“…AHSCT in patients with systemic sclerosis induces the production of new cells from the immune and hematopoietic systems, for the purpose of eliminating autoreactivity and, as consequence, recovering the patient's general health (Lee et al, 2016). Skeletal muscles are involved in this autoimmune disease (Costa-Pereira et al, 2019), however, little is known about how masticatory muscles behave in the context of systemic sclerosis.…”

Life after Autologous Hematopoietic Stem Cell Transplantation for Systemic Sclerosis

“…A recent study showed improvements of these functional parameters at 6 and 12 months after AHSCT, when compared to baseline. 53 Although the mRSS is the universally used method to quantify skin thickening in SSc, it bears intrinsic limitations and depends on evaluator expertise and opinion that affect reproducibility and consistency of the method. 54 Therefore, the reliability of mRSS to evaluate skin outcomes after AHSCT has been questioned.…”

“…Physical capacity, assessed by the 6-minute walk test (6MWT), improved after AHSCT, as an indirect evidence of better pulmonary function, although the cardiovascular and musculoskeletal systems may have participated. 53 Another method to evaluate how AHSCT affects interstitial lung disease is high-resolution quantitative computed tomography. Studies have shown improvement of lung volumes and/or pulmonary tissue quality (evaluated by density), after transplantation, associated with pulmonary function outcomes.…”