Patient-reported outcome measures in systemic sclerosis–related interstitial lung disease for clinical practice and clinical trials

Saketkoo, Lesley Ann; Scholand, Mary Beth; Lammi, Matthew R.; Russell, Anne-Marie

doi:10.1177/2397198320904178

Cited by 19 publications

(13 citation statements)

References 99 publications

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“…Clearly, there is a need for guidance in the new treatment regimens, in particular regarding the use of upfront or add-on combination treatment with immunosuppressants/antifibrotic, which could be the possible second/third level option in case of treatment failure. Hopefully, our understanding of the pathogenesis of SSc-ILD will evolve, along with the development of specific therapies for the organ systems affected by this disease, thus improving patients' survival, function, and quality of life [92,93]. Funding: The authors received no financial support for the research, authorship, and/or publication of this article.…”

Section: Discussionmentioning

confidence: 99%

The Treatment of Lung Involvement in Systemic Sclerosis

et al. 2021

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Systemic sclerosis (SSc) patients are often affected by interstitial lung disease (ILD) and, although there have been recent treatment advances, it remains the leading cause of death among SSc, with a 10-year mortality up to 40%. African Americans and subjects with diffuse cutaneous SSc or anti-topoisomerase 1 antibodies are most commonly affected. Currently, early ILD diagnosis can be made, and it is pivotal to improve the prognosis. The diagnostic mainstay test for SSc-ILD is high-resolution computed tomography for the morphology and pulmonary function tests for the functional aspects. Treatment planning and intensity are guided by the disease severity and risk of progression. Traditionally, therapy has depended on combinations of immunosuppressants, particularly cyclophosphamide and mycophenolate mofetil, which can be supplemented by targeted biological and antifibrotic therapies. Benefits have been observed in trials on hematopoietic autologous stem cell transplantation for patients with progressive SSc, whilst lung transplantation is reserved for refractory SSc-ILD cases. Herein, recent advances in SSc-ILD treatment will be explored.

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Section: Discussionmentioning

confidence: 99%

The Treatment of Lung Involvement in Systemic Sclerosis

et al. 2021

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“…Value-based health care, patient-reported outcomes (PRO) and treatment satisfaction are core values for new reference standards and quality metrics in patient management and drug approval by regulatory agencies (39,40). The creation of a new assessment tool for a complex rheumatological disease, like lcSSc, requires the incorporation of the patients' perspective, especially for the identification of the most important domains for this CRI.…”

Section: Challenges Ahead For a Combined Index Dedicated To Lcsscmentioning

confidence: 99%

“…Value-based healthcare, patient-reported outcomes (PROs), and treatment satisfaction are core values for new reference standards and therefore quality metrics in patient management and drug approval by regulatory agencies. 50,51 The creation of a new assessment tool for a complex rheumatological disease, like lcSSc, requires the incorporation of the patients’ perspective, especially for the identification of the most important domains for this combined response index. Comprehensive identification of outcome measures, including PRO, will therefore require highlighting the most relevant items within the domains that are considered the most bothersome by patients with lcSSc.…”

Section: Unmet Needs For a Combined Index Dedicated To Lcssc: The Casmentioning

confidence: 99%

Considerations for a combined index for limited cutaneous systemic sclerosis to support drug development and improve outcomes

Lescoat

Murphy

Roofeh

et al. 2020

Journal of Scleroderma and Related Disorders

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Systemic sclerosis (systemic scleroderma) is characterized by a heterogeneous range of clinical manifestations. Systemic sclerosis is classified into limited cutaneous systemic sclerosis and diffuse cutaneous systemic sclerosis subgroups based on the extent of skin involvement. Randomized controlled trials in scleroderma have mainly focused on diffuse cutaneous systemic sclerosis partly because the measurement of skin involvement, critical for evaluating a therapeutic intervention, is more dynamic in this subset. Nonetheless, limited cutaneous systemic sclerosis, the most common cutaneous subset (about two-third), is also associated with significant morbidity and detrimental impact on health-related quality of life. The lack of interventional studies in limited cutaneous systemic sclerosis is partly due to a lack of relevant outcome measures to evaluate this subgroup. Combining several clinically meaningful outcomes selected specifically for limited cutaneous systemic sclerosis may improve representativeness in clinical trials and responsiveness of outcomes measured in randomized controlled trials. A composite index dedicated to limited cutaneous systemic sclerosis combining such relevant outcomes could advance clinical trial development for limited cutaneous systemic sclerosis by providing the opportunity to test and select among candidate drugs that could act as disease-modifying treatments for this neglected subgroup of systemic sclerosis. This proposed index would include items selected by expert physicians and patients with limited cutaneous systemic sclerosis across domains grounded in the lived experience of limited cutaneous systemic sclerosis. This article reviews the reasons behind the relative neglect of limited cutaneous systemic sclerosis, discusses the current state of outcome measures for limited cutaneous systemic sclerosis, identifies challenges, and proposes a roadmap for a combined limited cutaneous systemic sclerosis-specific treatment response index.

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“…Studies have shown that cough, dyspnoea, changes in physical function and fatigue are the lung-related symptoms of most concern to patients with connective tissue disease (CTD)-associated ILD, including SSc [ 10 , 11 ]. Thus, PRO measures are usually assessed in randomised clinical trials in SSc-ILD as secondary endpoints [ 12 ].…”

Section: Introductionmentioning

confidence: 99%

Impact of lung function decline on time to hospitalisation events in systemic sclerosis-associated interstitial lung disease (SSc-ILD): a joint model analysis

et al. 2022

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Background Interstitial lung disease (ILD) is a common organ manifestation in systemic sclerosis (SSc) and is the leading cause of death in patients with SSc. A decline in forced vital capacity (FVC) is an indicator of ILD progression and is associated with mortality in patients with SSc-associated ILD (SSc-ILD). However, the relationship between FVC decline and hospitalisation events in patients with SSc-ILD is largely unknown. The objective of this post hoc analysis was to investigate the relationship between FVC decline and clinically important hospitalisation endpoints. Methods We used data from SENSCIS®, a phase III trial investigating the efficacy and safety of nintedanib in patients with SSc-ILD. Joint models for longitudinal and time-to-event data were used to assess the association between rate of decline in FVC% predicted and hospitalisation-related endpoints (including time to first all-cause hospitalisation or death; time to first SSc-related hospitalisation or death; and time to first admission to an emergency room [ER] or admission to hospital followed by admission to intensive care unit [ICU] or death) during the treatment period, over 52 weeks in patients with SSc-ILD. Results There was a statistically significant association between FVC decline and the risk of all-cause (n = 78) and SSc-related (n = 42) hospitalisations or death (both P < 0.0001). A decrease of 3% in FVC corresponded to a 1.43-fold increase in risk of all-cause hospitalisation or death (95% confidence interval [CI] 1.24, 1.65) and a 1.48-fold increase in risk of SSc-related hospitalisation or death (95% CI 1.23, 1.77). No statistically significant association was observed between FVC decline and admission to ER or to hospital followed by admission to ICU or death (n = 75; P = 0.15). The estimated slope difference for nintedanib versus placebo in the longitudinal sub-model was consistent with the primary analysis in SENSCIS®. Conclusions The association of lung function decline with an increased risk of hospitalisation suggests that slowing FVC decline in patients with SSc-ILD may prevent hospitalisations. Our findings also provide evidence that FVC decline may serve as a surrogate endpoint for clinically relevant hospitalisation-associated endpoints. Trial registration ClinicalTrials.govNCT02597933. Registered on 8 October 2015.

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Patient-reported outcome measures in systemic sclerosis–related interstitial lung disease for clinical practice and clinical trials

Cited by 19 publications

References 99 publications

The Treatment of Lung Involvement in Systemic Sclerosis

The Treatment of Lung Involvement in Systemic Sclerosis

Considerations for a combined index for limited cutaneous systemic sclerosis to support drug development and improve outcomes

Impact of lung function decline on time to hospitalisation events in systemic sclerosis-associated interstitial lung disease (SSc-ILD): a joint model analysis

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