Materiały źródłowe

• #1 Prognostic profile of systemic sclerosis: analysis of the clinical EUSTAR cohort in China

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6235213/

  Among the cohort, 40 patients with SSc died (Table 2): 27 deaths (67.5%) were related to SSc. PAH was the leading cause of death, accounting for 55% of all deaths. The mortality rate in patients who had PAH during the disease course was 28.2% (22/78 patients). […] Systemic sclerosis is a disease that has significant clinical heterogeneity. Some patients progress rapidly and even die, whereas others remain stable and have limited symptoms such as finger sclerosis and minimal visceral involvement. Therefore, treatment strategies for SSc would benefit from the ability to predict prognosis based on clinical manifestations or laboratory parameters in the early stage of the disease.

• #2 Survival and prognosis factors in systemic sclerosis: data of a French multicenter cohort, systematic review, and meta-analysis of the literature | Arthritis Research & Therapy | Full Text

  https://arthritis-research.biomedcentral.com/articles/10.1186/s13075-019-1867-1

  Data on survival and prognosis factors in incident cohorts are scarce in systemic sclerosis (SSc). […] Overall survival rates at 1, 3, 5, and 10 years from diagnosis were 98.0%, 92.5%, 85.9%, and 71.7% respectively in the French cohort. Overall SMR was 5.73 (95% CI 4.68-6.94). […] Age at diagnosis 60 years, diffuse cutaneous SSc, scleroderma renal crisis, dyspnea, 6-min walking distance (6MWD), forced vital capacity 70%, diffusing capacity of the lungs for carbon monoxide 70%, pulmonary hypertension (PH), telangiectasia, valvular disease, malignancy, anemia, and CRP 8 mg/l were associated with a poorer survival after adjustment. […] Eighteen studies (11,719 patients) were included in the SMR meta-analysis and 36 studies (26,187 patients) in the prognosis factor analysis. […] Age at disease onset, male sex, African origin, diffuse cutaneous SSc, anti-Scl70 antibodies, cardiac and renal involvement, interstitial lung disease, PH, and malignancy were significantly associated with a worse prognosis.

• #3 Survival and prognosis factors in systemic sclerosis: data of a French multicenter cohort, systematic review, and meta-analysis of the literature | Arthritis Research & Therapy | Full Text

  https://arthritis-research.biomedcentral.com/articles/10.1186/s13075-019-1867-1

  Overall, our study highlights a high mortality rate in SSc patients and confirms previously described prognosis factors related to skin extension and organ involvement while identifying additional prognosis factors such as autoantibody status, telangiectasia, 6MWD, and valvular disease. […] The main results of our study are (i) a high risk of mortality in our cohort of incident patients, as shown by a high SMR of 5.73; (ii) the identification of age 60 years, dcSSc, dyspnea, PH, low FVC, low DLCO, kidney involvement, valvular disease, cancer, telangiectasia, shorter 6MWD, anemia, and inflammation as prognosis factors in our cohort; (iii) a high pooled SMR of 3.45 in the meta-analysis of the literature, including our new cohort; and (iv) the additional identification of male sex, African origin, ILD, cardiac involvement, and anti-Scl-70 antibodies as associated with worse prognosis in our meta-analysis, while ACA were associated with better prognosis.

• #4 Development and validation of machine learning for early mortality in systemic sclerosis | Scientific Reports

  https://www.nature.com/articles/s41598-022-22161-9

  The mortality in SSc is higher than in the general population. […] Elderly age at the onset, male sex, extensive skin involvement, and internal organ involvement are well known predictors of death among SSc. […] Our study developed a simplified machine learning for mortality prediction for SSc patients based primarily on clinicals, which could be easily accessed, low cost, time-saving (unnecessary waiting for further investigation), and available for general physicians who are not experts in SSc. […] A model using predictors using high WHO functional class and mRSS appears to be optimal for predicting mortality risk among SSc patients.

• #5 Sine scleroderma, limited cutaneous, and diffused cutaneous systemic sclerosis survival and predictors of mortality | Arthritis Research & Therapy | Full Text

  https://arthritis-research.biomedcentral.com/articles/10.1186/s13075-021-02672-y

  Systemic sclerosis (SSc) is associated with a variability of mortality rates in the literature. […] To determine the mortality and its predictors in a long-term follow-up of a bi-centric cohort of SSc patients. […] The overall survival rates were 97.6% at 1 year, 93.4% at 3 years, 87.1% at 5 years, 77.9% at 10 years, and 61.3% at 15 years. […] Long-term data confirmed high mortality of SSc. Male sex, DLCO 70%, cardiac involvement, and CRP 5mg/l were identified as independent predictors of mortality. […] Male sex, cardiac involvement, DLCO 70%, and CRP 5 mg/l are strong predictors of mortality in systemic sclerosis. […] Sine scleroderma subtype has better survival than diffuse or limited cutaneous subtypes. […] The majority of deaths (53.6%) was not attributed to SSc directly, especially cardiovascular mortality (37.8%). […] Our study identifies strong predictors of mortality male sex, cardiac involvement, DLCO 70%, and CRP 5 mg/l. Non-SSc-related death is more frequent than SSc-related deaths, of whom cardiovascular disease is the most common.

• #6 Scleroderma: Practice Essentials, Background, Pathophysiology

  https://emedicine.medscape.com/article/331864-overview

  Survival in patients with diffuse cutaneous disease has improved significantly; currently, the 5-year survival is estimated to be about 80%. Five-year survival in patients with limited cutaneous disease is approximately 90%. […] Factors associated with a more severe prognosis are as follows: Younger age, African descent, Rapid progression of skin symptoms, Greater extent of skin involvement, Anemia, Elevated erythrocyte sedimentation rate (ESR), Pulmonary, renal, and cardiac involvement. […] Systemic sclerosis has the highest case-specific mortality among the systemic autoimmune diseases. Pulmonary hypertension, pulmonary fibrosis (interstitial lung disease), and scleroderma renal crisis are the most frequent causes of death. […] Survival has improved in recent decades and correlates best with the clinical disease subtype (diffuse cutaneous vs limited cutaneous) and with the extent of organ involvement. Five-year survival among patients with diffuse cutaneous systemic sclerosis has improved significantly, from 69% in the 1990-1993 cohort to 84% in the 2000-2003 cohort. Five-year survival among the patients with limited cutaneous systemic sclerosis remained very high and unchanged for the same periods (93% and 91%, respectively).

• #7

  https://link.springer.com/article/10.1007/s10067-024-07039-7

  The clinical manifestations of systemic sclerosis (SSc) are highly variable, resulting in varied outcomes and complications. Diverse fibrosis of the skin and internal organs, vasculopathy, and dysregulated immune system lead to poor and varied prognoses in patients with SSc subtypes. Therefore, this study aimed to develop a personalized tool for predicting the prognosis of patients with SSc. […] The results of Cox-based multivariate regression analysis suggested that pulmonary arterial hypertension, digital ulcer, and Modified Rodnan Skin Score (mRSS) were independent risk factors for poor prognosis in patients with SSc and significant risk factors in random survival forest (RSF) surveys. […] Machine-learning models can help us better understand the prognosis of patients with SSc and comprehensively evaluate the clinical characteristics of each individual. The early identification of the characteristics of high-risk patients can improve the prognosis of those with SSc.

• #8 Development and validation of machine learning for early mortality in systemic sclerosis | Scientific Reports

  https://www.nature.com/articles/s41598-022-22161-9

  The degree or severity of fibrosis is associated with poor prognosis in Thai SSc as well as other populations. […] Mortality risk escalates the more numerous the involvements: 1 internal organ involvement had a 5 times greater risk than no involvements, while 2 internal organ involvements had a 10 times greater mortality risk. […] According to the literature review, the various clinical predictors of mortality in SSc include older age at onset, dcSSc, positive for anti-Scl70 antibody, internal organ involvement (pulmonary fibrosis (PF), PAH, renal crisis, cardiac involvement), and high mRSS. […] Our study aimed to develop and validate a simplified predictive model from machine learning for mortality prediction among Thai SSc patients, making it a more precise referral tool for general practitioners, a guide for close monitoring, modification of the predictors of death, treatment planning, and minimizing mortality risk.

• #9 Survival and prognosis factors in systemic sclerosis: data of a French multicenter cohort, systematic review, and meta-analysis of the literature | Arthritis Research & Therapy | Full Text

  https://arthritis-research.biomedcentral.com/articles/10.1186/s13075-019-1867-1

  Our results show that mortality is still high in SSc. Strong prognosis factors identified at baseline are age at diagnosis 60 years, dcSSc subtype, scleroderma renal crisis, severe dyspnea, FVC and DLCO 70%, PH, anemia, and CRP 8 mg/l. Our study also suggests the prognosis value of telangiectasia, 6MWD, valvular disease, cancer, and autoantibody status.

• #10 Predictors of disease worsening defined by progression of organ damage in diffuse systemic sclerosis: a European Scleroderma Trials and Research (EUSTAR) analysis | Annals of the Rheumatic Diseases

  https://ard.bmj.com/content/78/9/1242

  Objectives Mortality and worsening of organ function are desirable endpoints for clinical trials in systemic sclerosis (SSc). The aim of this study was to identify factors that allow enrichment of patients with these endpoints, in a population of patients from the European Scleroderma Trials and Research group database. […] Of 1451 patients meeting the inclusion criteria, 706 had complete data on outcome parameters and were included in the analysis. Of the 42 outcome predictors, eight remained in the final regression model. There was substantial evidence for a strong association between disease progression and age, active digital ulcer (DU), lung fibrosis, muscle weakness and elevated C-reactive protein (CRP) level. Active DU, CRP elevation, lung fibrosis and muscle weakness were also associated with a significantly shorter time to disease progression.

• #11 Predictors of disease worsening defined by progression of organ damage in diffuse systemic sclerosis: a European Scleroderma Trials and Research (EUSTAR) analysis | Annals of the Rheumatic Diseases

  https://ard.bmj.com/content/78/9/1242

  The use of the predictive factors presented here could enable cohort enrichment with patients at risk for overall disease worsening in SSc clinical trials. […] By using a novel statistical approach to analyse data from a clinical registry, we successfully identified predictors of severe disease worsening defined as organ failure within a period of 123 months in patients with dcSSc. Based on our logistic regression model, we showed that the probability of a 60-year-old patient with lung fibrosis, DU, muscle weakness and CRP elevation developing disease worsening within the observation period increases to 74.5% compared with 32.2% for the whole study population. […] The predictive factors of age, presence of DU, lung fibrosis, CRP elevation and muscle weakness represent important aspects of the disease and also correspond to the key characteristic features of vasculopathy (DU), autoimmunity/inflammation (CRP elevation) and tissue fibrosis (lung fibrosis).

• #12 Prognostic profile of systemic sclerosis: analysis of the clinical EUSTAR cohort in China

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6235213/

  In china, although there is a high prevalence of ILD in patients with SSc (85.5%), most patients have reserved pulmonary function, which means that ILD is not the most important factor in the death of patients with SSc and is also not a risk factor for poor prognosis. Only ILD with pulmonary dysfunction is associated with poor outcome. The 10-year cumulative rate (87.8%) in patients with SSc in China is slightly lower than in Europe, and PAH and arrhythmia at baseline are independent prognostic factors, whereas PAH instead of ILD is the leading cause of death in patients with SSc. […] Univariate survival analysis suggested that SRC, PAH, arrhythmia, pericardial effusion, skin score15, FVC%70% and DLCO%60% on pulmonary function tests (PFT), and respiratory symptoms at baseline were associated with poor prognosis. Further Cox multivariate analysis confirmed that PAH (hazard ratio (HR) 6.248, 95% CI 2.855, 13.674) and arrhythmia (HR 4.729, 95% CI 1.588, 14.082) at baseline are independent risk factors for poor outcome in patients with SSc.

• #13 Scleroderma: Practice Essentials, Background, Pathophysiology

  https://emedicine.medscape.com/article/331864-overview

  For patients with scleroderma renal crisis, 1-year mortality is 20-30% and 5-year mortality is 30-50%; for normotensive scleroderma renal crisis, which accounts for about 10% of all cases, 1-year mortality is 60%. […] Mortality associated with scleroderma renal crisis has declined significantly in recent decades. In contrast, pulmonary involvement (interstitial lung disease and/or pulmonary arterial hypertension) has become the most common cause of death in patients with systemic sclerosis.

• #14 Study Finds that Prognosis for Systemic Sclerosis can be Predicted with this Simple Test – Patient Worthy

  https://patientworthy.com/2018/07/19/study-finds-prognosis-sclerosis-predicted/

  According to a story from Scleroderma News, the results from a recent study suggest that the six minute walk test can accurately predict outcomes for patients with systemic sclerosis. A low score is associated with more severe disease and an increased risk of death. According to the data, every meter a patient was able to walk correlated to a single percentage point in survival chances. […] While the six minute walk test has not been commonly employed in systemic sclerosis, it appears to have the ability to play a valuable role in predicting outcomes for patients.

• #15 A prediction model for progressive disease in systemic sclerosis | RMD Open

  https://rmdopen.bmj.com/content/1/1/e000113

  Our study shows that individualised management in patients with SSc is a reasonable possibility. Using the developed prediction model, the chance for progressive disease could be increased from 39 to 67-89%, advocating annual stringent follow-up at least in patients with friction rubs, proximal muscle weakness and low maximum oxygen uptake at baseline. […] Maximum oxygen uptake as measured by CPET was identified as a possible new biomarker for progressive disease in SSc.

• #16 Development and validation of machine learning for early mortality in systemic sclerosis | Scientific Reports

  https://www.nature.com/articles/s41598-022-22161-9

  Clinical predictors of mortality in systemic sclerosis (SSc) are diversely reported due to different healthcare conditions and populations. A simple predictive model for early mortality among patients with SSc is needed as a precise referral tool for general practitioners. […] Early mortality was defined as dying within 5 years after the onset of SSc. […] The final 2 models with the highest predictive performance comprise the modified Rodnan skin score (mRSS) and the WHO-FCII for Model 1 and mRSS and WHO-FCIII for Model 2. Model 1 provided the highest predictive performance, followed by Model 2. […] This simplified machine learning model for predicting early mortality among patients with SSc could guide early referrals to specialists and help rheumatologists with close monitoring and management planning.

• #17 Scleroderma Lung Involvement, Autoantibodies, and Outcome Prediction: The Confounding Effect of Time | The Journal of Rheumatology

  https://www.jrheum.org/content/44/4/404

  Systemic sclerosis (SSc) remains a poorly understood disease and so far none of the routinely used immunosuppressive treatments has been definitively shown to benefit longterm disease outcome. […] Early detection and monitoring of PF and PH may benefit outcome by permitting earlier intervention in severe or progressive cases. […] Multiple attempts have been made to develop prediction models, both for development and for outcome, in already present SSc-related lung disease. […] An algorithm combining results from HRCT and PFT was developed by Goh, et al and seems to have a very good prognostic value, allowing for an easy and quick staging of lung disease and assessment of the need for immunosuppressive treatment. […] This highlights the importance of accounting for disease duration in any research into outcome prediction in patients with SSc.

• #18 A prediction model for progressive disease in systemic sclerosis | RMD Open

  https://rmdopen.bmj.com/content/1/1/e000113

  63 patients had progressive disease, including 8 patients who died 18 months after first evaluation. […] Using the prediction model, the predicted chance for progressive disease increased from a pretest chance of 37% to 67-89%. […] Using the prediction model, the chance for progressive disease for individual patients could be doubled. […] A prediction model assessing the chance for progressive disease for individual patients at short term was currently developed. […] The present study aimed to develop a model that predicts progressive disease in the short term, defined by either deterioration of organ functions, or mortality, in patients with SSc. […] The predicted probability of progressive disease was calculated for every patient. […] The prediction model showed a reliable calibration, predicting progressive disease in agreement with the observed progressive disease.

• #19

  https://link.springer.com/article/10.1007/s10067-024-07039-7

  Regarding predictive performance, the random survival forest model was more effective than the Cox model and had unique advantages in analyzing nonlinear effects and variable importance. […] Machine learning using the simple clinical features of patients with systemic sclerosis (SSc) to predict mortality can guide attending physicians, and the early identification of high-risk patients with SSc and referral to experts will assist rheumatologists in monitoring and management planning.

• #20 Machine Learning Assisted Prediction of Progression in Systemic Sclerosis Patients: An Approach to Concise, Tailored Model Construction Using Outpatient Clinical Data – ACR Meeting Abstracts

  https://acrabstracts.org/abstract/machine-learning-assisted-prediction-of-progression-in-systemic-sclerosis-patients-an-approach-to-concise-tailored-model-construction-using-outpatient-clinical-data/

  Machine Learning Assisted Prediction of Progression in Systemic Sclerosis Patients: An Approach to Concise, Tailored Model Construction Using Outpatient Clinical Data […] Systemic Sclerosis (SSc) disease course can range from mild, to very severe with progressive organ involvement within months. […] In this study we aim to develop a tailormade model to guide annual assessment in individual SSc patients. […] In 492 SSc patients (range of follow-up 2-10 years), disease progression during total follow-up was observed in 52% after median 4 years (range 1-8), including myocardial progression in 29%, lung progression in 23%, skin progression in 16%, and death in 12%. […] Our data confirm the severe nature of SSc with cumulative progression in 52%. Although precise risk stratification for the individual patient is difficult, a machine learning approach enabled us to classify 29% of patients as low risk. In this group annual assessment programs might be less extensive.

• #21 Scleroderma Lung Involvement, Autoantibodies, and Outcome Prediction: The Confounding Effect of Time | The Journal of Rheumatology

  https://www.jrheum.org/content/44/4/404

  As a result, any study investigating predictors of organ complications in SSc should take into account the timing of those complications and the disease duration in the study subjects. […] One inevitable conclusion from this article is that there is a real need for even more detailed and larger studies, assessing the effect of autoantibody specificities on the outcome of lung complications in patients with SSc.

• #22 14.00 Immune System Disorders – Adult | Disability | SSA

  https://www.ssa.gov/disability/professionals/bluebook/14.00-Immune-Adult.htm

  Systemic sclerosis (scleroderma) constitutes a spectrum of disease in which thickening of the skin is the clinical hallmark. […] In diffuse cutaneous systemic sclerosis (also known as diffuse scleroderma), major organ or systemic involvement can include the gastrointestinal tract, lungs, heart, kidneys, and muscle in addition to skin or blood vessels. […] Documentation involves differentiating the clinical features of systemic sclerosis (scleroderma) from other autoimmune disorders. However, there may be an overlap. […] Repeated manifestations of systemic sclerosis (scleroderma), with at least two of the constitutional symptoms or signs (severe fatigue, fever, malaise, or involuntary weight loss) and one of the following at the marked level: Limitation of activities of daily living. […] Limitation in maintaining social functioning. […] Limitation in completing tasks in a timely manner due to deficiencies in concentration, persistence, or pace.

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