medwireNews: Autologous hematopoietic stem cell transplantation (AHSCT) leads to early and sustained clinically relevant improvements in health-related quality of life (HRQoL) among patients with systemic sclerosis (SSc), study findings indicate.
Nancy Maltez (The Ottawa Hospital, Ontario, Canada) and colleagues believe their results provide “robust complementary data to standard biomedical variables, including overall and event‐free survival, to support the physical benefits of AHSCT in SSc.”
Their retrospective study analyzed HRQoL data among 41 SSc patients who underwent AHSCT and 65 SSc patients who met transplant criteria but were treated with conventional care.
They found that the mean physical component summary scores of the Short Form Health Survey‐36 (SF‐36) were a significant 7.02 points higher at 1 year in patients treated with AHSCT than in those who received conventional care, after adjustment for baseline scores and other potential confounders.
After 7 years, the difference had increased to a significant 14.40 points, with the effect sizes at both timepoints “well beyond minimal clinically important differences” of at least 5.00 points, the researchers write in Arthritis & Rheumatology.
Among the individual domains of the SF-36, the greatest differences between the two groups were observed for physical function.
The researchers also looked at data from the Health Assessment Questionnaire (HAQ) and found that, at 1 year, the overall mean scores were a significant 0.57 points lower – indicating improvement – among the patients who received AHSCT versus those who did not.
The improvements in HAQ increased with time, to 0.94 points at 7 years, and while they remained clinically significant, exceeding the minimal clinically important difference of 0.14 points, statistical significance was lost.
For the HAQ, the greatest differences over 7 years were observed in the domains that focused on pain and Raynaud’s syndrome.
Maltez and co-authors also note that the improvements they observed in HRQoL were reflected in greater improvements in skin scores and lung function among the patients who received AHSCT compared with those who did not.
Finally, they calculated that approximately four patients would need to be treated with AHSCT for one to experience a clinically meaningful improvement in physical HRQoL and function greater than that observed with conventional care.
Of note, there were no significant differences between the two groups in the mental component summary scores of the SF-36 and the team therefore concludes that “further research is needed to understand trajectories of mental HRQoL in SSc.”
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Arthritis Rheumatol 2020; doi:10.1002/art.41519