Quality of Life Assessment in Sarcoidosis

The impact of common measures to assess sarcoidosis have not been compared longitudinally to outcomes that are meaningful to patients. We prospectively examined the relationship of baseline measurements of sarcoidosis status to outcomes of interest to patients longitudinally over 6 months.

Sarcoidosis patients cared for at 6 US medical centers were “phenotyped” at baseline with measurements of pulmonary function, organ involvement, health related quality of life (HRQoL) instruments, and their anti-sarcoidosis treatment history. These patients were followed for 6 months by monitoring outcomes of interest to patients (OIPs) including steps walked, calories expended, sleep, HRQoL measures, workdays missed and health care utilization. For each baseline phenotypic measurement, patients were dichotomized into two groups above and below a specified cutoff value. The area under the OIP versus time curve was compared between these two groups.

The cutoff values for many baseline phenotypic measures distinguished the patients into groups with significantly different 6-month OIPs. The chosen cutoff for the patient global estimate of health status distinguished the most OIPs (13/15). The 6-min walk distance cutoff was associated with more OIPs than spirometric measures. All of the HRQOL measure cutoffs were associated with many OIPs, although most of them were other HRQOL measures.

Cutoffs for most of the phenotypic measures used to assess sarcoidosis distinguished groups of sarcoidosis patients with differing OIPs over the subsequent 6 months. The patients’ global assessment of their disease was the most accurate of these measures.

NCT04342403.

Although it is the general consensus that sarcoidosis patients who present with sarcoidosis-related symptoms have a worse outcome than patients whose disease is detected incidentally without symptoms, this premise has not been rigorously examined.

Consecutive patients followed longitudinally at one US university sarcoidosis clinic were questioned concerning the onset and description of sarcoidosis-related symptoms at disease presentation. The patients were classified into those with no sarcoidosis-related symptoms at presentation (NSP group) and those with symptoms at presentation (SP group). The following outcomes were examined in the NSP and SP groups: most recent spirometry, organ involvement, need for sarcoidosis therapy, most recent health related quality of life (HRQOL) as measured by the Sarcoidosis Assessment Tool (SAT), most recent chest imaging Scadding stage results.

660 sarcoidosis patients were analyzed, with 175 in the NSP group and 485 in the SP group. Compared to the NSP group, the SP group had a more frequent requirement for any sarcoidosis treatment, corticosteroid treatment, and non-corticosteroid treatment at some time and within the most recent year of follow up (at least 50% more than the NP group with strong statistical differences with p values all 0.01 or less). In addition, the SP group had significantly more organ involvement (p < 0.001) and several worse SAT domains (p < 0.022) than the NP group. There were no differences between the groups in terms of final spirometry or development of Scadding stage 4 chest radiographs. These findings held even after adjusting for age, sex, race, and time between presentation and the most recent follow-up visit using a multivariable logistic regression framework.

In our sarcoidosis cohort, compared to the absence of symptoms at presentation, the presence of symptoms was associated with a greater need for treatment, more organ involvement, and worse HRQOL.

The Sarcoidosis Health Questionnaire (SHQ) is the first sarcoidosis-specific health status questionnaire ever developed. Worse health status, as evaluated by the SHQ, may indicate higher risk for deterioration in the following 5 years.

To evaluate the association between SHQ scores and deterioration defined clinically at 5-year follow-up.

122 patients with biopsy-supported sarcoidosis completed the SHQ and underwent evaluation with respect to organ involvement, chest radiograph, electrocardiogram, serum biomarker measurements, pulmonary function tests, and echocardiogram. Of these 122, 88 (72.1%) were available for pulmonary, cardiac, and non-pulmonary, non-cardiac deterioration assessment during the following 5 years.

Five-year deterioration was observed in 20 patients (23%). The SHQ total score was significantly associated with 5-year deterioration, after adjusting for cardiac involvement at baseline, with adjusted odds ratio (OR) of 0.54 (95% confidence interval [95% CI], 0.29–0.99). The association of the total SHQ with 5-year outcome was not significant when adjusted for left ventricular ejection fraction (LVEF) at baseline (adjusted OR, 0.61 [0.32–1.16]), whereas LVEF was significantly associated with 5-year outcome (adjusted OR, 0.92 [0.86–0.99]). The association between total SHQ score and 5-year deterioration was marginal when adjusted for baseline usage of systemic corticosteroid (CS)/immunosuppressive (IS) agents (adjusted OR, 0.58 [0.31–1.10]), whereas systemic CS/IS usage significantly predicted 5-year deterioration (adjusted odds ratio [OR], 3.46 [1.12–10.7]). There was a marginal correlation between the total SHQ and LVEF (rho = 0.19, p = 0.07) and a weak association between the total SHQ and systemic CS/IS usage (rho = −0.23, p = 0.03). The Physical Functioning domain scores of the SHQ were significantly associated with 5-year deterioration (adjusted OR, 0.45–0.51).

Worse health status, as assessed by the SHQ score, can be a risk factor for 5-year deterioration of sarcoidosis, although usage of the CS/IS at baseline and lower LVEF at baseline are more predictive of 5-year deterioration.

Many patients with pulmonary sarcoidosis experience reduced quality of life. Although oral corticosteroids are the most common agents used in sarcoidosis, very little is known on the effects on quality of life.

In this double-blind, placebo-controlled trial, newly diagnosed patients without an indication for high dose immunosuppressive therapy were randomised to once-daily dexamethasone 1 mg (6.5 mg prednisone equivalent) or placebo for 6 months. The primary study parameter was the subscale physical functioning of the 36-item Short Form health survey (SF-36). Secondary parameters included five other patient reported outcome measures, disease activity markers and plasma cytokine profiles.

A total of 16 patients was randomised to dexamethasone (n = 7) and placebo (n = 9). During follow-up no significant difference for physical functioning was measured (p = 0.18). Dexamethasone treated patients showed a decrease in fatigue score (Checklist Individual Strength) from 106 (baseline) to 88 (3 months; p = 0.03); 86 (6 months; p = 0.05); 79 (9 months; p = 0.04); 90 (12 months; p = 0.03). Placebo treated patients showed no change: 96 (baseline) to 105 (3 months; p = 0.16); 91 (6 months; p = 0.48); 92 (9 months; p = 0.61); 95 (12 months; p = 0.90). During treatment with dexamethasone significant improvements in the SF-36 subscales vitality and pain, and a significant reduction in serum angiotensin-converting enzyme, soluble interleukin 2 receptor levels and serum cytokines and chemokines were measured.

Low-dose dexamethasone results in a reduction of the inflammatory profile and has the potential to improve quality of life parameters and fatigue.