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Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)
Ariane L Herrick1,2, Xiaoyan Pan3, Sébastien Peytrignet3
1Centre for Musculoskeletal Research, The University of Manchester, Salford Royal NHS Foundation Trust, Manchester Academic Health Science Centre, Manchester, UK.
Annals of the Rheumatic Diseases
|February 12, 2017
Summary
This observational study compared early diffuse cutaneous systemic sclerosis treatments. Immunosuppressants showed modest benefits over 12 months, indicating a need for improved therapies for dcSSc.
Area of Science:
- Rheumatology
- Immunology
- Clinical Trials
Background:
- Randomized controlled trials for early diffuse cutaneous systemic sclerosis (dcSSc) are challenging due to patient rarity.
- An observational approach was employed to evaluate current treatment strategies for early dcSSc.
Purpose of the Study:
- To compare the effectiveness of methotrexate, mycophenolate mofetil (MMF), cyclophosphamide, and no immunosuppressant in early dcSSc patients.
- To assess changes in modified Rodnan skin score (mRSS) and survival rates across different treatment protocols.
Main Methods:
- Prospective observational cohort study of early dcSSc patients (within 3 years of symptom onset).
- Patients received one of four treatment protocols: methotrexate, MMF, cyclophosphamide, or no immunosuppressant.
- Inverse probability of treatment (IPT) weights were used to adjust for baseline confounding; mRSS and survival were primary and secondary outcomes, respectively.
Main Results:
- Statistically significant reductions in mRSS were observed at 12 months across all treatment groups, including the 'no immunosuppressant' group.
- No significant differences in survival were found between the treatment protocols after IPT weighting.
- Survival at 24 months was poorest in the group that received no immunosuppressant (84.0%).
Conclusions:
- Findings suggest that immunosuppressants may be beneficial for early dcSSc.
- The overall treatment benefit observed over 12 months appears modest.
- There is a clear need for the development of more effective treatments for early dcSSc.
