The task force consisted of 32 SSc clinical experts from Europe and the USA, 2 patients nominated by the pan-European patient association for SSc (Federation of European Scleroderma Associations (FESCA)), a clinical epidemiologist and 2 research fellows.
All centres from the EULAR Scleroderma Trials and Research (EUSTAR) group were invited to submit and select clinical questions concerning SSc treatment using a Delphi approach.
These recommendations also give directions for future clinical research in SSc.
Based on the published evidence and expert opinion, 16 updated recommendations regarding pharmacological treatment of SSc-specific organ involvement were formulated.
Management of SSc also includes (early) diagnosis of the disease, early diagnosis of internal organ involvement, identification of patients at risk of development of new organ complications and deterioration of the disease, as well as non-pharmacological treatments, of which most of are beyond the scope of this project.
In view of risk of side effects and route of administration usually requiring hospitalisation, intravenous iloprost should be considered in particular in patients with SSc with DUs not responding to oral therapy.
In severe cases, combination therapy with oral vasodilator and intravenous iloprost can be used. However, the increased risk of side effects should be taken into account.
Side effects of PDE-5 inhibitors are discussed in the previous paragraph regarding PDE-5 inhibitors in the treatment of RP.
Based on these data, the experts concluded that PDE-5 inhibitors can be efficacious in treating SSc-related DUs.
There are two major concerns related to the use of bosentan and other ERA: potential liver injury and teratogenicity.
This has been underlined by the publication of the recently published new guidelines of the pulmonology and cardiology societies.
This approach is in line with recently published guidelines regarding management of PAH in general, and seems particularly important in patients with SSc-PAH known to have more progressive disease than patients with other forms of PAH.
Potential risks of bone marrow suppression, teratogenicity, gonadal failure and haemorrhagic cystitis must be always considered.
In view of the high risk of treatment-related side effects and of early treatment-related mortality, careful selection of patients with SSc for this kind of treatment and the experience of the medical team are of key importance.
Careful evaluation of the benefit to risk ratio in individual patients with SSc selected for HSCT should be done by experts.
Further studies should help to identify subgroups of patients with SSc in whom HSCT would be most beneficial.
Recommendation: Experts recommend immediate use of ACE inhibitors in the treatment of SRC
The greatest changes have been made in treatments of vascular complications of SSc and mirror the progress which had been made in this field during the last several years.
These include the introduction of PDE-5 inhibitors for SSc-related RP and DUs, riociguat and new aspects for ERAs, prostacyclin analogues and PDE-5 inhibitors for SSc-PAH.
The new recommendation regarding the use of fluoxetine for SSc-related RP was also added.
Similar to the 2009 recommendations, the present recommendations address only pharmacological treatments which were considered most relevant and received consensus from the expert panel.
As SSc is an uncommon and clinically heterogeneous disease, appropriate testing of therapies is difficult.
Treatment with tocilizumab (subcutaneous 162 mg/week) was associated with a favourable trends in skin score improvement as compared with placebo after 24 weeks (p=0.09) and 48 weeks (p=0.06).
In addition, encouraging changes in FVC were noted.
In view of promising effects of tocilizumab on skin and lung involvement, it is concluded that further studies are warranted before definitive conclusions can be made about its risks and benefits in SSc.
The preliminary results of this study, recently published as an abstract of the 2015 ACR annual congress, indicate that mycophenolate mofetil (up to 3 g/day orally for 2 years) was comparable with oral CYC (2 mg/kg/day for 1 year followed by matching placebo for the second year) with regard to FVC course at 24th month.
Other therapies, considered promising by the experts, were addressed in the research agenda.
To read my articles:
Rare Disease Day 2018, Research - Taking Part in Clinical Trials, Click here
March Autoimmune Disease Awareness Month 2018, Click here
Global patient video, Click here
Becoming a Patient Research Ambassador for the NIHR, Click here
If we only had more RESEARCH investment for Scleroderma, Raynaud's, Autoimmune Rare Disease, Click here
The Importance of Medical Research and Awareness to the Scleroderma, Raynaud's, Autoimmune Rare Disease patient, Click here
Expert Specialist Centres, Click here
World Scleroderma Day 29th June 2016, Click here
World Scleroderma Day 29th June 2017, Click here
Why Global Collaboration is important to the Rare Disease Patient, Click here
2016 Rare Disease Day Patient Voice