Stem Cell Treatment Improves Quality of Life in Systemic Sclerosis

September 28, 2020

Autologous hematopoietic stem cell transplants (AHSCT) improve the quality of life in patients with systemic sclerosis (SSc), researchers say.

Autologous hematopoietic stem cell transplants (AHSCT) improve the quality of life in patients with systemic sclerosis (SSc), researchers say.

The finding provides reassurance that the side effects of the treatment don’t outweigh its benefits, according to Nancy Maltez, M.D., of the Ottawa Hospital in Ottawa, Canada and colleagues.

“This collaborative study adds considerable complementary data to traditional biomedical outcome measures to support the role of AHSCT in SSc,” researchers wrote in Arthritis & Rheumatology.

Systemic sclerosis is an autoimmune disease that causes fibrosis of the skin and internal organs. In addition to killing many of those who suffer from it, it can make their day to day lives miserable.

Randomized controlled trials have shown that stem cell transplants slow the disease and save lives, making it the best treatment for the early severe stage of the disease. But the transplants themselves can be toxic and even deadly.

So far, only small, short-term studies have measured health-related quality of life in patients transplanted with stem cells. They lacked detail and were limited to the context of clinical trials, Dr. Maltez and her colleagues reported.

To fill that gap, they compared 41 patients who had undergone the transplants with 65 who had instead been treated with cyclophosphamide, azathioprine, methotrexate or mycophenolate mofetil.

On the Short Form Health Survey-36 (SF-36), patients rate their health across several domains on a scale from 0 to 100, where a higher score means better health.

At baseline, mean SF-36 scores were similar between the 2 groups. But after a year of treatment, the scores had diverged. By the seventh year, patients who got stem cells scored themselves a mean of 14.40 points higher than patients in the control group. (The scale goes from 0 to 100 and higher scores mean better health.) The difference was statistically significant (P = 0.001).

There were no differences in the mental component of the SF-36, a result the researchers found surprising.

On the Health Assessment Questionnaire-Disability Index (HAQ), patients rate their ability to carry out tasks of daily living from 0 (without difficult) to 3 (unable). After seven years, the mean HAQ scores were 0.94 points lower for the patients who had received stem cells.

Analyzing components of the scores, the researchers found that skin and lung function were both closely related to tasks of daily living and quality of life.

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REFERENCE

Maltez, N., Puyade, M., Wang, M., et al. Autologous hematopoietic stem cell transplant in systemic sclerosis is associated with marked improvement in health‐related quality of life. Arthritis Rheumatol. Accepted Author Manuscript. doi:10.1002/art.41519