Aussie rheumatologists’ positive perceptions of AHSCT for SSc


By Siobhan Calafiore

24 Jun 2024

Autologous haemopoietic stem cell transplantation has gained acceptance as a therapy for systemic sclerosis among Australian rheumatologists but treatment toxicity remains a barrier to patient referral, a survey suggests.

Responses from 68 rheumatologists identified key areas of need to be access, studies to refine patient selection criteria and development of autologous haemopoietic stem cell transplantation (AHSCT) protocols that improve safety.

The Australian Rheumatology Association distributed a national survey on perceptions of AHSCT for systemic sclerosis (SSc) via emailed newsletter, receiving responses from one in five rheumatologists and trainees.

Most rheumatologists had at least 11 years of experience and saw 2-5 patients with SSc per month. About 40% of participants had previously referred a patient for AHSCT and just over half (54%) had an AHSCT centre within 100km of them.

Survey findings revealed a high acceptability of AHSCT as a treatment for SSc, with 77.8% of rheumatologists either agreeing or strongly agreeing with its role as an appropriate treatment option and only 4.8% disagreeing with the statement.

Most respondents believed that AHSCT should be considered in early, inflammatory disease and disagreed with its use for late, severe disease.

Writing in the Internal Medicine Journal [link here], the researchers, which included rheumatologists and haematologists from St Vincent’s Hospital in Sydney, said progressive lung disease, followed by rapidly progressive skin disease and a lack of response to other therapies, was considered the main referral criteria for SSc.

The majority of respondents agreed that the treatment improved outcomes for skin disease and SSc-associated interstitial lung disease.

“This largely aligns with the indications for AHSCT in previous studies and indicates rheumatologists have a good understanding of the patients deemed to have a high risk of poor disease outcomes,” the authors wrote.

About 60% of participants correctly identified phase III trial evidence as the level of evidence that was currently available for AHSCT in SSc, as opposed to case reports only or phase II trials only. Rheumatologists who understood the evidence base were more likely to consider AHSCT as an acceptable treatment for SSc.

Adverse effects

Almost two-thirds of participants (65.1%) agreed or strongly agreed that treatment-associated mortality was a significant barrier to referral for AHSCT, and only 15.2% of participants agreed or strongly agreed that this risk was unacceptable.

Other barriers were patient comorbidities and treatment-associated morbidity.

About 38% of rheumatologists agreed or strongly agreed that AHSCT should only be considered after all other therapeutic options had been attempted.

Nearly all respondents (92%) agreed or strongly agreed that reduction of treatment toxicity would increase their likelihood to refer patients for AHSCT. Improved access to treatment and further positive data on treatment outcomes were the other factors listed that would positively influence their referral pattern.

They authors concluded that having a specialist HSCT centre for SSc in each state – with only NSW and Queensland having centres available for SSc at the time of survey in 2019 – would provide both equity of access and quality of care.

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