Aussie rheumas accept stem cell treatment for systemic sclerosis

3 minute read

But the national survey reveals concerns over treatment-related morbidity and access to the treatment remain.

What are the barriers to, and concerns about, using haemopoietic stem cell transplants in systemic sclerosis patients?

Autologous haemopoietic stem cell transplants are an effective treatment for systemic sclerosis – potentially the most severe connective tissue disease – but clinicians are still hesitant to embrace it as an acceptable treatment due to concerns over treatment-related mortality.

New Australian research, published in the Internal Medicine Journal, shows although two-thirds of local rheumatologists agreed the risk of mortality was a significant barrier in prescribing AHSCT for systemic sclerosis, only 15% felt this risk was too great to warrant not using it.

“Our results indicate that rheumatologists would consider AHSCT in patients with early disease, particularly with progressive lung and skin involvement. [This study] indicates rheumatologists have a good understanding of the patients deemed to have a high risk of poor disease outcomes,” the researchers wrote. 

Researchers collected anonymous survey responses from 63 rheumatologists and trainees, estimated to account for 20% of all rheumatologists in the country at the time of the survey. Forty percent of respondents had previously recommended AHSCT for a patient with systemic sclerosis.

When asked about their perceptions of AHSCT for systemic sclerosis, 78% of respondents felt it was an acceptable treatment for the condition, with only 5% disagreeing. Rheumatologists who were aware of the phase III trial evidence supporting the use of AHSCT were more likely to view AHSCT as an acceptable treatment.

More than 75% of respondents felt AHSCT should be considered in the early, inflammatory stages of the disease, with over 80% suggesting progressive lung involvement was the most important trigger in referring for treatment, followed by rapidly progressing lung disease (~70%) and a failure to respond to other therapies (~45%).

Sixty-five percent of respondents acknowledged the threat of treatment-related mortality was a significant barrier to referring AHSCT, but only 15% felt the treatment posed an unacceptable risk to patients. Patient comorbidities were also seen as a significant barrier to referral.

“There was [also] a clear indication that a reduction in treatment toxicity would increase the likelihood of future patient referral for AHSCT,” the researchers wrote.

Despite the support the AHSCT, access to the treatment remains an issue for Australian rheumatologists. Only 59% of respondents indicated AHSCT was available in their state, and 32% of respondents indicating the nearest centre that could perform AHSCT for systemic sclerosis was located more than 100km away from their principal place of practice.

“To our knowledge, AHSCT for systemic sclerosis was only available at centres in New South Wales and Queensland at the time of the survey,” the researchers noted.

The results of the survey may be biased by the small number of respondents being made up of rheumatologists with a specific interest in systemic sclerosis, and the fact data were collected back in 2019 but only submitted for publication in December 2023.

EULAR updated its recommendations for the treatment of systemic sclerosis to include AHSCT for selected patients with a rapidly progressing form of the disease back in 2016.

Internal Medicine Journal 2024, online 31 May

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