Stem Cell Transplant Improves Quality of Life in Scleroderma Patients, Study Indicates

Autologous hematopoietic stem cell transplant (AHSCT) — replacing patients’ damaged bone marrow with their own healthy blood stem cells —  improves health-related quality of life among people with scleroderma to a greater extent than conventional treatment, a study shows.

The study, “Autologous hematopoietic stem cell transplant in systemic sclerosis is associated with marked improvement in health‐related quality of life,” was published in the journal Arthritis & Rheumatology.

AHSCT is a treatment that functions as a “reset” of the immune response. Typically, the procedure involves using chemotherapy or similar medications to wipe out the immune system, then repopulating it with cells previously collected from the same patient.

AHSCT has shown efficacy in clinical trials of systemic scleroderma, demonstrating superiority over standard treatment with Cytoxan (cyclophosphamide) in terms of limiting disease progression and prolonging survival. Although effective, AHSCT is associated with considerable treatment-related toxicity, which can limit a person’s health-related quality of life (HRQoL).

Understanding the effect of treatments on quality of life — especially in a real-world setting — is important for making informed choices.

“The purpose of this study was to generate HRQoL data to supplement standard biomedical variables, including disease progression and survival, that are usually emphasized in the medical literature,” the scientists from France and Canada wrote.

Here, they analyzed information from both countries. As AHSCT is rarely performed in Canada, the team said, data from scleroderma patients treated with AHSCT in France was compared to Canadian patients who met transplant criteria but did not undergo this procedure.

Specifically, 41 people with scleroderma who had undergone AHSCT were compared to 65 patients who met all criteria for AHSCT, but were instead given other treatments, namely cyclophosphamide, azathioprine, methotrexate and/or CellCept (mycophenolate mofetil).

Both groups were majority female. Relative to the controls, the AHSCT group had a longer disease duration (mean 2.6 vs. 1.5 years), was younger when meeting the criteria for AHSCT (mean 44.7 vs. 53.1 years), and had a greater proportion of patients with interstitial lung disease (92.7% vs. 49.2%).

HRQoL was measured with the Short Form Health Survey-36 (SF-36), which uses a scale from 0 to 100, with higher scores representing better HRQoL.

Prior to treatment, SF-36 scores were generally similar between the two groups, although physical function was better in the AHSCT group. However, these patients outperformed the comparator group at all subsequent annual visits (up to seven visits) in physical function, bodily pain, general health and social function, but not in vitality, emotional skills, and mental health.

Detailed analyses indicated that the difference was largest in physical function.

“Further research is needed to understand trajectories of mental HRQoL in [scleroderma],” the researchers wrote.

Notably, the differences in the physical component of SF-36 that favored AHSCT were greater than five points, meaning that they were considered clinically meaningful.

Another assessment used was the Health Assessment Questionnaire-Disability Index (HAQ-DI), which measures disability on a scale from zero (no disability) to three (severe disability).

Mirroring the SF-36 results, HAQ-DI scores were significantly lower at all subsequent annual assessments, indicating eased symptoms in the AHSCT group relative to the comparator group.

“In fact, differences appeared to increase in favour of AHSCT over time,” the scientists wrote.

The changes were all more than 0.5 points in magnitude, which is above the 0.14 points threshold for clinically meaningful change in the HAQ-DI.

Results of a related measurement, the Scleroderma HAQ (S-HAQ), which assesses how much symptoms such as pain, breathlessness and digital ulcers interfere with daily activities, also showed superiority of AHSCT over comparators, in terms of improving HRQoL.

“We found that AHSCT was associated with early marked improvement, well beyond minimal clinically important differences, in physical HRQoL and function as compared to conventional care and that this effect was sustained over time,” the researchers concluded.

From these results, they calculated that — on average — for every four people with scleroderma who undergo AHSCT, one will experience clinically meaningful improvement in physical HRQoL that is greater than what would be achieved with conventional care.

“This collaborative study adds considerable complementary data to traditional biomedical outcome measures to support the role of AHSCT in SSc,” the investigators added.