Health-related quality of life in systemic sclerosis before and after autologous haematopoietic stem cell transplant—a systematic review

Abstract

Objectives In severe rapidly progressive SSc, autologous haematopoietic stem cell transplantation (AHSCT) allows significant improvements in overall and event-free survival. We undertook this study to identify, appraise and synthesize the evidence on health-related quality of life (HRQoL) before and after AHSCT for SSc. Methods We performed a systematic review of the literature, following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines, in PubMed and ScienceDirect from database inception to 1 February 2019. All articles with original HRQoL data were selected. Results The search identified 1080 articles, of which 8 were selected: 3 unblinded randomized controlled trials [American Scleroderma Stem Cell versus Immune Suppression Trial (ASSIST), Autologous Stem Cell Transplantation International Scleroderma, Scleroderma: Cyclophosphamide or Transplantation), 3 uncontrolled phase I or II trials and 2 cohort studies. HRQoL data from 289 SSc patients treated with AHSCT and 125 treated with intravenous CYC as a comparator with median 1.25–4.5 years follow-up were included. HRQoL was evaluated with the HAQ Disability Index (HAQ-DI; 275 patients), the 36-item Short Form Health Survey (SF-36; 249 patients) and the European Quality of Life 5-Dimensions questionnaire (EQ-5D; 138 patients). The quality of the studies was moderate to low. AHSCT was associated with significant improvement in the HAQ-DI (P = 0.02–<0.001), SF-36 Physical Component Summary score (P = 0.02–<0.0001) and EQ-5D index-based utility score (P < 0.001). The SF-36 Mental Component Summary score improved in the ASSIST (n = 19) and one small retrospective cohort (n = 30 patients, P = 0.005) but did not improve significantly in 2 randomized controlled trials (n = 200 patients, P = 0.1–0.91). Conclusion AHSCT in severe SSc patients is associated with significant and durable improvement in physical HRQoL.

Association of Autologous Hematopoietic Stem Cell Transplantation in Systemic Sclerosis With Marked Improvement in Health‐Related Quality of Life

“…These results are consistent with the purported positive impact of autologous HSCT on physical HRQoL in patients with SSc, which was reported in a recent systematic review of the literature (18). In that review, the most robust evidence was found to be from the ASTIS and SCOT trials.…”

“…Our group recently undertook a systematic review of the literature to assess current knowledge of the impacts of autologous HSCT on HRQoL in SSc. We demonstrated a promising improvement in physical HRQoL post–autologous HSCT (18). However, the evidence was limited by the characteristics of the studies, including relatively small sample sizes, lack of details on individual HRQoL domains, and short duration of follow‐up (median 1.25–4.5 years).…”

“…However, the evidence was limited by the characteristics of the studies, including relatively small sample sizes, lack of details on individual HRQoL domains, and short duration of follow‐up (median 1.25–4.5 years). Moreover, most patients were treated in the setting of clinical trials, limiting the generalizability of results to real‐world clinical practice (18).…”

Autologous Hematopoietic Stem Cell Transplantation for Behçet’s Disease: A Retrospective Survey of Patients Treated in Europe, on Behalf of the Autoimmune Diseases Working Party of the European Society for Blood and Marrow Transplantation

“…Over the past 20 years, aHSCT has become a standard of care for early rapidly progressive Systemic Sclerosis (6)(7)(8)(9)(10)(11) and has emerged as a promising treatment modality for severe Multiple Sclerosis (12)(13)(14) and Crohn's Disease (12,15). But patients with rarer diseases, such as BD, have undergone aHSCT based on compassionate use.…”

The patient knows best: a new self‐reported outcome measure for systemic sclerosis

“…However, SHAQ is heavily dependent on a tool developed for arthritis and is often improperly scored, making interpretation of results difficult. It correlates with long-term outcome in SSc 8 and may show improvement after treatments such as autologous stem cell transplantation, 9 but it is not disease specific. A feasible global measure is required, and, once fully validated, the Cochin 17-item Scleroderma Functional scale has real potential as a new PROM for clinical trials and practice.…”