Lisa R. Jewett

The Scleroderma Patient-centered Intervention Network (SPIN) Cohort: protocol for a cohort multiple randomised controlled trial (cmRCT) design to support trials of psychosocial and rehabilitation interventions in a rare disease context

Introduction Psychosocial and rehabilitation interventions are increasingly used to attenuate disability and improve health-related quality of life (HRQL) in chronic diseases, but are typically not available for patients with rare diseases. Conducting rigorous, adequately powered trials of these interventions for patients with rare diseases is difficult. The Scleroderma Patient-centered Intervention Network (SPIN) is an international collaboration of patient organisations, clinicians and researchers. The aim of SPIN is to develop a research infrastructure to test accessible, low-cost self-guided online interventions to reduce disability and improve HRQL for people living with the rare disease systemic sclerosis (SSc or scleroderma). Once tested, effective interventions will be made accessible through patient organisations partnering with SPIN. Methods and analysis SPIN will employ the cohort multiple randomised controlled trial (cmRCT) design, in which patients consent to participate in a cohort for ongoing data collection. The aim is to recruit 1500–2000 patients from centres across the world within a period of 5 years (2013–2018). Eligible participants are persons ≥18 years of age with a diagnosis of SSc. In addition to baseline medical data, participants will complete patient-reported outcome measures every 3 months. Upon enrolment in the cohort, patients will consent to be contacted in the future to participate in intervention research and to allow their data to be used for comparison purposes for interventions tested with other cohort participants. Once interventions are developed, patients from the cohort will be randomly selected and offered interventions as part of pragmatic RCTs. Outcomes from patients offered interventions will be compared with outcomes from trial-eligible patients who are not offered the interventions. Ethics and dissemination The use of the cmRCT design, the development of self-guided online interventions and partnerships with patient organisations will allow SPIN to develop, rigourously test and effectively disseminate psychosocial and rehabilitation interventions for people with SSc.

Psychological health and well-being in systemic sclerosis: State of the science and consensus research agenda.

Introduction Systemic sclerosis (SSc; scleroderma) is a multisystem disorder characterized by disturbance in fibroblast function, microvascular disease, and immune system activation, culminating in fibrosis of the skin and internal organs (1,2). SSc is associated with extensive morbidity, including disfiguring skin thickening, finger ulcers, joint contractures, pulmonary hypertension, interstitial lung disease, chronic diarrhea, and renal failure (1,2). The rate of disease onset is highest between 30 and 50 years of age, with the risk for women being 4 to 5 times higher than for men (3,4). Median survival time from diagnosis is 11 years, and patients are 3.7 times more likely to die within 10 years of diagnosis (44.9% mortality) than age-, sex-, and race-matched individuals without SSc (12.0% mortality) (3).

Is longer-term psychodynamic psychotherapy more effective than shorter-term therapies? Review and critique of the evidence.

Background: In 2008, Leichsenring and Rabung performed a meta-analysis of 8 studies of longer-term psychodynamic psychotherapy (LTPP). The work was published in the Journal of the American Medical Association (vol. 300, pp 1551–1565), and they concluded that LTPP was more effective than shorter-term therapies. Method: Given that such claims have the potential to influence treatment decisions and policies, we re-examined the meta-analysis and the 8 studies. Results: We found a miscalculation of the effect sizes used to make key comparisons. Claims for the effectiveness of LTPP depended on a set of small, underpowered studies that were highly heterogeneous in terms of patients treated, interventions, comparison-control groups, and outcomes. LTPP was compared to 12 types of comparison-controls, including control groups that did not involve any psychotherapy, short-term psychodynamic psychotherapy, and unvalidated treatments. Additionally, the studies failed to protect against threats to bias, and had poor internal validity. Conclusion: Overall, we found no evidence to support claims of superiority of LTPP over shorter-term methods of psychotherapy.

(Not) talking about sex: a systematic comparison of sexual impairment in women with systemic sclerosis and other chronic disease samples

OBJECTIVE Sexual impairment in women with SSc has received little attention. The objective of this study was to compare levels of sexual impairment in women with SSc with samples of women with medical illnesses for which sexual impairment has been researched more extensively. METHODS SSc patients completed the Sexual Relationships subscale of the Psychosocial Adjustment to Illness Scale-Self-Report (PAIS-SR). A systematic review was conducted to select comparison samples. Sexual Relationships subscale scores from SSc patients were compared with scores from comparison samples (breast or gynaecological cancer and HIV) using t-tests and Hedgess g to calculate effect sizes. RESULTS Samples from 138 female SSc patients were analysed (28.3% diffuse; mean age 52.1 +/- 12.3 years; mean time since diagnosis 9.0 +/- 8.3 years). Women with dcSSc (6.1 +/- 4.2) reported significantly greater sexual impairment (P < 0.05) than those with lcSSc (4.4 +/- 4.2), three breast cancer samples (1.8 +/- 0.1, 3.4 +/- 3.9, 1.6 +/- 0.6) and two samples of HIV-positive female patients (4.4 +/- 3.8, 4.5 +/- 3.9). Scores in dcSSc were similar to one sample of HIV-positive women (5.8 +/- 4.1) and gynaecological cancer patients (7.3 +/- 4.3). Scores in lcSSc were significantly higher than two breast cancer samples, similar to one breast cancer sample and two HIV-positive samples, and significantly lower (P < 0.05) than in one HIV sample and gynaecological cancer. CONCLUSION Women with SSc, particularly those with dcSSc, have high levels of sexual impairment compared with women with other chronic diseases, where sexual function has received greater attention. Further research is needed on sexual function among women with SSc.

Development and validation of the brief-satisfaction with appearance scale for systemic sclerosis

Body image concerns are understudied in systemic sclerosis (SSc; scleroderma). The objective was to develop and cross‐validate a brief version of the Satisfaction with Appearance Scale (SWAP) in order to reduce item redundancy, increase SSc relevancy, and improve the feasibility of body image assessment in SSc.

Sociodemographic and Disease Correlates of Body Image Distress among Patients with Systemic Sclerosis

Background Body image concerns are infrequently studied in systemic sclerosis (SSc), even though significant visible disfigurement is common. The objective of this study was to identify sociodemographic and disease-related correlates of dissatisfaction with appearance and social discomfort among people with SSc. Methods SSc patients came from the 15-center Canadian Scleroderma Research Group Registry. Sociodemographic information was based on patient self-report. Disease characteristics were obtained via physician examinations. The Brief-SWAP was used to assess dissatisfaction with appearance and social discomfort. Structural equation models were conducted with MPlus to determine the relationship of dissatisfaction with appearance and social discomfort with age, sex, education, marital status, race/ethnicity, disease duration, skin involvement, telangiectasias, skin pigmentation changes, and hand contractures. Results A total of 489 SSc patients (432 female, 57 male) were included. Extent of skin involvement was significantly associated with both dissatisfaction with appearance and social discomfort (standardized regression coefficients = 0.02, p = 0.001; 0.02, p = 0.020, respectively), as was skin involvement in the face (0.18, p = 0.016; 0.23, p = 0.006, respectively). Greater social discomfort was robustly associated with younger age (−0.017, p<0.001) and upper-body telangiectasias (0.32, p = 0.021). Dissatisfaction with appearance was associated with hand contractures (0.07, p = 0.036). Conclusion This study found that dissatisfaction with appearance and social discomfort were associated with numerous disfiguring characteristics of SSc, in addition to age. These results underline that there are multiple factors contributing to body image distress in SSc, as well as the need to attend to both disease and social contexts in understanding the impact of disfigurement among patients.

Psychosocial Aspects of Scleroderma.

Patients with systemic sclerosis (SSc; also called scleroderma) have to cope with not only the physical impacts of the disease but also the emotional and social consequences of living with the condition. Because there is no cure for SSc, improving quality of life is a primary focus of treatment and an important clinical challenge. This article summarizes significant problems faced by patients with SSc, including depression, anxiety, fatigue, sleep disruption, pain, pruritus, body image dissatisfaction, and sexual dysfunction, and describes options to help patients cope with the consequences of the disease.

Prevalence of current, 12-month and lifetime major depressive disorder among patients with systemic sclerosis

OBJECTIVES Patients with SSc experience a range of problems affecting their quality of life, but only one small study has assessed the prevalence of major depressive disorder (MDD) in SSc. The objectives of this study were: (i) to assess the prevalence of current (30-day), 12-month and lifetime MDD in a large sample of Canadian SSc patients; and (ii) to investigate socio-demographic and disease factors associated with 12-month MDD. METHODS SSc patients were recruited from seven Canadian Scleroderma Research Group Registry sites (April 2009 to May 2012). MDD and history of a prior depression episode (major or minor) were assessed with the Composite International Diagnostic Interview. RESULTS Among 345 patients, prevalence of 30-day, 12-month and lifetime MDD was 3.8% (95% CI 2.2%, 6.3%; n = 13), 10.7% (95% CI 7.9%, 14.4%; n = 37) and 22.9% (95% CI 18.8%, 27.6%; n = 79), respectively. Patients with 12-month MDD had more severe gastrointestinal track involvement than patients without 12-month MDD, but there were no other significant differences on socio-demographic or disease variables. Among patients with 12-month MDD, 81.1% (95% CI 65.8%, 90.3%) reported a prior depression episode compared with 3.9% (95% CI 2.2%, 6.7%) among patients without 12-month MDD (P < 0.01). CONCLUSION The prevalence of 30-day, 12-month and lifetime MDD among Canadian SSc patients is approximately twice that of the Canadian general population and somewhat higher than in arthritis. SSc patients face a range of psychosocial problems and may benefit from a broad supportive care approach.

Analyzing Effectiveness of Long-term Psychodynamic Psychotherapy

To the Editor: Drs Leichsenring and Rabung reported that long-term psychodynamic psychotherapy (LTPP) is more effective than shorter forms of psychotherapy for complex mental disorders based on a between-group effect size of 1.8 from 7 comparative trials that they meta-analyzed. The authors did not indicate that they were concerned about this and other surprisingly large effect sizes they reported. Between-group effect sizes can be presented as group differences in terms of standard deviations or as point biserial correlations between group (eg, LTPP vs shorter-term therapies) and treatment effect. They are equivalent and convertible using a formula or tables. The authors, however, apparently erroneously calculated within-group pre-post effect sizes and point biserial correlations between group and within-group effect sizes, which is altogether different. It seems that they converted these correlations between group and within-group pre-post effect sizes to produce deviation-based effect sizes that do not appear reasonable. As a result, although none of the 7 studies had an overall effect size greater than 1.45, the authors reported a combined effect size of 1.8, which is statistically impossible. In a slightly larger set of 8 trials, the authors reported that LTPP had a larger overall effect than shorter-term therapies (0.96 vs 0.47) but a point biserial correlation (0.60) equivalent to a between-group effect size of 1.5. However, betweengroup effect sizes must be smaller than within-group effect sizes when both groups have positive effects. Similarly, these methods generated an implausible between-group effect size of 6.9 for personality functioning based on 3 trials, none of which reported an effect size for personality functioning larger than approximately 2. In addition, we believe that this collection of studies was not suitable for meta-analysis. Each reviewed study had 15 to 30 patients in the LTPP treatment group. It seems unlikely that investigators would attempt to publish a negative study with so few patients (or that such a study would be accepted for publication), which means that all published studies would have an effect size of at least 0.50 to 0.75, the minimum for statistical significance. This is an artificial floor that guarantees a large effect when these studies are combined.

Major Depression Diagnoses Among Patients With Systemic Sclerosis: Baseline and One‐Month Followup

Depression is common in many rheumatic diseases and is associated with poor prognosis. No studies of patients with any rheumatic diseases, however, have assessed the stability of major depressive disorder (MDD) diagnoses over time. The objective of the present study was to assess the stability of MDD diagnoses among patients with systemic sclerosis (SSc; scleroderma), a rare autoimmune rheumatic disease, across 2 assessments approximately 1 month apart.