Cost-effectiveness of alginic acid in combination with proton pump inhibitor for the treatment of gastroesophageal reflux disease in systemic sclerosis patients.

BACKGROUND: Systemic sclerosis (SSc) patients often become refractory to proton pump inhibitors (PPI)-a standard treatment for gastroesophageal reflux disease (GERD)-and intolerant to PPI in combination with domperidone. PPI with alginic acid is an alternative treatment option, but alginic acid is costly. OBJECTIVES: We compared the costs and effectiveness of alginic acid plus PPI versus standard treatments (PPI with/without antacids as needed and lifestyle modifications) for GERD in SSc patients unsuitable for, or intolerant to, domperidone. METHODS: An economic evaluation using the Markov model was conducted among SSc patients aged between 40 and 65 years with GERD, having a partial or non-response to 4 weeks of standard-dose omeprazole (40 mg/day) and being unsuitable for or intolerant to domperidone. Using a societal perspective, we computed the incremental cost-effectiveness ratios (ICERs) in terms of Thai baht (THB) per quality-adjusted life-year (QALY) between a combination of alginic acid plus PPI and standard treatment for GERD. The lifetime time horizon was used. RESULTS: The ICER for alginic acid plus PPI versus standard treatments was 377 101 THB/QALY. According to the one-way sensitivity analysis, the cost of alginic acid was the most impactful parameter. If the market prices of alginic acid plus PPI were reduced by 61%, this treatment option would become cost-effective at the willingness-to-pay threshold of 160 000 THB/QALY (34.68 THB/USD data on 25 May 2023). Furthermore, if alginic acid were included in the public health insurance program, the national budget would be increased by 66 313 THB per patient, resulting in an overall budget increase of 5 106 101 to 8 885 942 THB compared with the standard treatment. CONCLUSIONS: Alginic acid plus PPI does not represent good value for money compared with the standard treatment among such SSc patients in Thailand unless its price is reduced significantly.

Clinical characteristics and mortality rate of Thai elderly-onset systemic sclerosis.

OBJECTIVES: To identify the clinical differences and mortality rate between adult and elderly onset systemic sclerosis (SSc). METHODS: We conducted a historical cohort study of SSc patients during January 2007-December 2011. The SSc patients were 60 and over classified as elderly onset SSc. Cox regression analysis was used to estimate the probability of survival and for assessing the factors associated with mortality. RESULTS: The medical records of 350 SSc patients were reviewed; 53 (15.1%) had elderly onset SSc. According to the multivariate analysis, elderly onset SSc has a higher WHO functional class, more frequent weakness, more frequent hyperCKaemia, and less pulmonary fibrosis than adult onset SSc (p=0.004, 0.02, 0.02, 0.02, respectively). The incidence of mortality was 3.8 per 100 person-year with a median survival rate of 15.9 years (95%CI 12.4-17.3). The mortality rate of elderly SSc onset was significantly higher than that of adult SSc onset (HR 5.71; 95%CI 3.54-9.20). The median survival of elderly and adult onset SSc was 4.9 years and 16.1 years, respectively. The Cox regression analysis indicated that presence of digital ulcer and tendon friction rub had a respective HR of 7.39 (95%CI 1.28-42.60) and 37.23 (95%CI 2.10-659.09) for predicting mortality of elderly onset SSc. CONCLUSIONS: Myopathy and limitation of physical activity were frequently found among elderly onset SSc over against pulmonary involvement than in adult onset SSc. Mortality of elderly onset SSc was 5.7 times higher, and median survival was 11 years shorter, than adult onset SSc.

Self-assessment of skin tightness severity by scleroderma patients.

AIMS: Skin tightness progression determines the severity and mortality of systemic sclerosis (SSc). The modified Rodnan skin score (mRSS) is a skin tightness assessment tool, albeit inter-physician variability is a limitation. Our objectives were to evaluate the correlation and agreement of skin tightness assessment between patient self-assessment and physician-assessment. METHODS: A descriptive study was conducted on the masked, self-assessments of mRSS (at week 0, 4 and 12) by 23 Thai adult SSc patients seen at Srinagarind Hospital, KhonKaen University, between March 2014 and February 2015. Correlation between the physician and patient assessments was estimated using Pearson's. The intra-class correlation coefficient (ICC) and limit of agreement by Bland-Altman were determined. RESULTS: We included 23 SSc patients (female to male ratio being 1.1:1). All had the diffuse cutaneous SSc subset. The skin thickness assessment correlation was moderate at baseline (Pearson's r = 0.68) and improved to a good correlation at week 4 and 12 (r = 0.78 and 0.86, respectively). The ICC showed good agreement (0.761) at week 0 and increased to excellent at week 4 (0.846) and 12 (0.910). The patient self-assessment score was higher than the physician assessment, which slightly decreased over time: mean difference = 4.30 (95% LOA;-9.54 to 18.14) at week 0, then 3.78 (95% LOA,-10.20 to 17.77) at week 4, and 3.16 (95% LOA,-7.78 to 14.10) at week 12. CONCLUSION: The respective modified Rodnan skin score assessment by patient versus the physician was highly correlated with a high level of agreement. Validation of generalized patient self-assessment needs to be confirmed.

Inter- and Intra-Observer Reliability of Modified Rodnan Skin Score Assessment in Thai Systemic Sclerosis Patients: A Validation for Multicenter Scleroderma Cohort Study.

BACKGROUND: Assessment of the severity of skin tightness by the modified Rodnan skin score (mRSS) for systemic sclerosis (SSc) has been found feasible, valid, and reliable. Despite being a major clinical outcome, it has not yet been validated by Scleroderma Research Group. OBJECTIVE: To (a) determine the inter-observer variability vis-à-vis mRSS assessment by members ofthe Scleroderma Research Group before and after mRSS-assessment training by an experienced rheumatologist and (b) determine intra-observer variability. MATERIAL AND METHOD: Between June and August 2013, we conducted a descriptive study of Thai adult SSc patients and all rheumatologists in the Scleroderma Research Group at Srinagarind Hospital, Khon Kaen University, Northeast Thailand. Eleven rheumatologists assessed the mRSS of 22 SSc patients three times (i.e., before and after training, and eight weeks after training). The intra-class correlation coefficient (ICC) and its 95% CI were estimated at week 8 after training. RESULTS: The mean and standard deviation (SD) of mRSS for inter-observer variability analysis was slightly decreased from before training, after training (by an experienced rheumatologist), and at week 8 after training (17.3 ± 11.9, 16.5 ± 11.1, and 16.2 ± 10.3, respectively). Intra-observer variability had moderate agreement before training (ICC 0.59; 95% CI 0.38-0.78), which increased to good agreement after training and at week 8 after training (ICC 0.60; 95% CI 0.42-0.76 vs. 0.68; 95% CI 0.53-0.82, respectively). CONCLUSION: Inter-observer variability for mRSS assessment decreased after training and the reduction persisted for eight weeks after training. The ICC rose from moderate agreement at baseline to good agreement at the end of the study. The mRSS assessment by members of the Scleroderma Research Group was reliable.