Can we define difficult-to-treat systemic sclerosis?

ABSTRACT

Introduction

Systemic sclerosis (SSc) is a chronic autoimmune rheumatic disease characterized by microvascular alterations, immunopathology, and widespread fibrosis involving various organs. It is considered difficult to treat due to several reasons: complex pathogenesis, heterogeneity, late diagnosis, limited treatment options for certain organ manifestations, lack of personalized medicine.

Areas covered

This review presents the heterogeneity, survival and organ manifestations with their risk factors of systemic sclerosis and their current treatment options, while drawing attention to difficult-to-treat forms of the disease, based on literature indexed in PubMed.

Expert opinion

Despite recent advances in the management of SSc over the last decades, the disease presents significant morbidity and mortality. Although available treatment protocols brought significant advancements in terms of survival in SSc-associated interstitial lung disease and pulmonary arterial hypertension, less success has been achieved in the treatment of Raynaud’s phenomenon and digital ulcers and the results are modest in case of heart, gastrointestinal, and renal manifestations. There are patients who do not respond to treatment and deteriorate even with adequate therapy. They can be considered difficult-to treat (D2T) cases. We have created a possible score system based on the individual organ manifestations and highlighted treatment options for the D2T SSc category.

KEYWORDS:

• Autoimmune diseases
• connective tissue diseases
• difficult-to-treat
• systemic sclerosis
• treatment

Article highlights

• Systemic sclerosis is a heterogeneous disease affecting multiple-organ systems and this complexity makes it challenging to target all aspects of the disease with a single treatment approach.

• Currently, there is no cure for systemic sclerosis, and treatment primarily focuses on managing symptoms, preventing complications or irreversible organ damage, and slowing disease progression. Medications such as immunosuppressants, vasodilators, and antifibrotic agents may be used to target specific aspects of the disease, but their effectiveness varies among individuals and different organ manifestations respond differently to the combination of the three treatment targets.

• A group of patients have progressive disease with organ damage despite increasing therapeutic potential. For this difficult-to-treat category, we have created a possible score system based on the individual organ manifestations.

• This review highlights the treatment options for D2T SSc.

Declaration of interest

G Szucs has received lecture fees from Actelion, Boehringer Ingelheim and MSD and travel expenses from Actelion and Boehringer Ingelheim for activities related to SSc.

S Szamosi has received lecture fees and travel expenses for activities related to SSc from Boehringer Ingelheim and Actelion.

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.