Complete results of the first randomized, placebo-controlled study of bosentan, a dual endothelin receptor antagonist, in pulmonary arterial hypertension

Background: Pulmonary arterial hypertension (PAH) is a deadly disease with limited treatment options.Objectives: The objectives of this study were to assess the effects of bosentan, an orally active dual endothelin receptor antagonist, on exercise capacity and cardiopulmonary hemodynamics, as well as to assess its safety and tolerability in patients with severe PAH.Methods: In this double-blind, placebo-controlled, multicenter study, 32 patients with PAH (primary or associated with scleroderma) were randomized to receive bosentan for a minimum of 12 weeks (62.5 mg BID for 4 weeks, then 125 mg BID) or placebo. The primary end point was the change in exercise capacity. Secondary end points included changes in cardiopulmonary hemodynamics, Borg dyspnea index, and modified New York Heart Association (NYHA) functional class, as well as withdrawal due to clinical worsening.Results: Patients treated with bosentan improved their 6-minute walking distance. Fifteen bosentan-treated patients (71%) improved their walking distance from baseline to week 12 by >30 m, which is considered a clinically relevant improvement. The difference between treatment groups in the mean (±SEM) change at week 12 was 76 ± 31 m in favor of bosentan (95% CI, 12 to 139; P = 0.021). The improvement was maintained for at least 20 weeks. For cardiac index, the difference was 1.0 ± 0.2 L/min/m² in favor of bosentan (95% CI, 0.6 to 1.4; P < 0.001). Bosentan significantly decreased pulmonary vascular resistance, whereas it was increased with placebo (P ≤ 0.001). Bosentan improved both the Borg dyspnea index and the NYHA functional class. All 3 withdrawals due to clinical worsening were in the placebo group (P = 0.033). The number and nature of adverse events were similar in the 2 groups.Conclusions: In these patients with PAH, bosentan increased exercise capacity and improved hemodynamics. These results, which have been summarized previously in a brief report, include all data up to 28 weeks of treatment and support the potential clinical value of endothelin receptor antagonists in the treatment of patients with PAH.     

温肾健脾汤联合背俞穴针刺对脾肾阳虚型系统性硬皮病的效果

目的探讨自拟温肾健脾汤联合背俞穴针刺对脾肾阳虚型系统性硬皮病的效果分析。方法选取2018年1月—2019年1月在辽宁省大连市大连市中医医院针灸门诊的80例脾肾阳虚型系统性硬皮病患者,随机分为试验组和对照组。对2组患者采用临床常规的治疗方法,对照组40例系统性硬皮病患者采用临床常规口服D-青霉胺治疗;试验组40例系统性硬皮病患者采用自拟温肾健脾汤联合背俞穴针刺治疗方法,比较2组患者治疗后的临床有效率和2组患者血清抗核抗体谱、血清抗着丝点抗体转阴累积人数。结果试验组患者治疗后临床总有效率高于对照组患者,差异有统计学意义(P<0.05);试验组患者治疗后血清抗核抗体谱、血清抗着丝点抗体转阴人数多于对照组患者,差异有统计学意义(P<0.05)。结论对脾肾阳虚型系统性硬皮病患者采取自拟温肾健脾汤联合背俞穴针刺进行治疗的临床效果较好,可以增加系统性硬皮病患者疗效,值得推广。     

Disease staging and sub setting of interstitial lung disease associated with systemic sclerosis: impact on therapy

Introduction: Interstitial lung disease (ILD) is the most serious complication of systemic sclerosis (SSc). There is no accepted guidance as to which clinical, radiological or physiological thresholds should prompt initiation or changes in treatment. Furthermore, some patients with extensive disease remain stable without the need for intervention whilst others with limited disease at the outset, experience a precipitous decline.

Areas covered: In this article, evidence for the integration of a number of disease-specific and patient-related domains are discussed and proposed. Introduction and maintenance of therapy requires a nuanced understanding of these factors and is crucial when weighing up the risks and benefits of immunomodulation. The evidence for the existing treatment modalities is discussed and the future directions for management of patients with SSc-ILD, which may include antifibrotic or biologic therapy, are explored.

Expert commentary: In the management of SSc-ILD, a multidisciplinary team approach which integrates physiology and radiology with the patient at the centre of the process is crucial for effective management and provision of the best outcomes.     

Extensión de la indicación de terapia fotodinámica dirigida vascular con padeliporfina (WST11): resultados de un estudio multicéntrico latinoamericano del cáncer de próstata

Objectives

To explore the proportion of patients with higher risk localized prostate cancer (PCa) that would become safely biopsy negative 12 months after non-thermal focal therapy with padeliporfin vascular-targeted photodynamic therapy (VTP).

Reasons for attending support groups and organizational preferences: the European scleroderma support group members survey

Purpose: The objectives were to identify reasons why patients attend scleroderma support groups and to ascertain preferences for how meetings are best organized.

Methods: The survey included 30-items on reasons for attending and nine items on organizational preferences. Patients were recruited through European patient organizations. Exploratory factor analysis was used to group reasons for attendance thematically.

Results: About 213 scleroderma patients (192 women) completed the survey. A three-factor model best described reasons for attending [χ²(348)?=?586.1, p?<?0.001; Comparative Fit Index?=?0.98; Tucker Lewis Index?=?0.97; Root Mean Square Error of Approximation?=?0.06] with themes that included: (1) obtaining interpersonal and social support, (2) learning about treatment and symptom management strategies, and (3) discussing other aspects of scleroderma. Among organizational preferences, respondents emphasized that meetings should include educational aspects and the opportunity to share information and support.

Conclusion: People with scleroderma attend support groups to give and obtain social support and for education about managing their disease and other aspects of living with scleroderma. Support groups should be structured to facilitate both educational and informational aspects and to provide opportunities for sharing and support between members.

• Implications for rehabilitation

• Local peer-led support groups are an important support and informational resource for patients living with scleroderma.

• People with scleroderma attend support groups in order to: (1) obtain interpersonal and social support, (2) learn about disease treatment and symptom management strategies, and (3) discuss other aspects of living with scleroderma outside of symptom management.

• Most support group members prefer groups with a trained facilitator, that include family members or loved ones in the groups, that include between 11and 20 members, that last between 1 and 2?h, and that meet once every 1–3 months.

• Rehabilitation professionals can support the formation and management of local support groups or can refer patients to national scleroderma patient organizations for information on support groups that they may be able to access.

     

X线立体定位活检在乳腺微小病变的临床应用

目的　探讨应用乳腺摄片结合立体定位穿刺活检对不可触及的乳腺微小病变的诊断价值。方法　回顾性总结对 3 0例乳腺摄片发现微小病变的病人进行立体定位穿刺活检。结果　 3 0例病人中发现早期乳腺癌 9例 ,其中原位癌 3例 ,临床I期 6例 ,另外 ,乳腺纤维瘤 6例 ,不典型增生 9例 ,纤维腺病 4例 ,囊肿 2例 ,而乳腺癌、纤维瘤、不典型增生及囊肿均有明确的手术指征。结论　对不可触及的乳腺微小病变应积极活检 ,X线立体定位活检术是提高早期乳腺癌诊断率首选而有效的方法。     

Multiple bowel intussusceptions from metastatic localized malignant pleural mesothelioma: A case report

Localized malignant pleural mesothelioma (LMPM) is a rare occurrence, and gastrointestinal intra-luminal metastases have not previously been reported. Herein, we report a patient with LMPM who presented with a local recurrence 10 mo after initial en bloc surgical resection. Abdominal computed tomography was performed for intractable, vague abdominal pain with episodic vomiting, which showed a "target sign" over the left lower quadrant. Laparotomy revealed several intra-luminal metastatic tumors in the small...     

Scleroderma lung disease,variation in screening,diagnosis and treatment practices between rheumatologists and respiratory physicians

Background: Interstitial lung disease (ILD) and pulmonary arterial hypertension (PAH) represent the leading causes of death in systemic sclerosis (SSc). Screening for these complications has assumed greater importance, but is not universal. The aim of this study is to determine the self‐reported screening, diagnosis and treatment practices of rheumatologists and respiratory physicians for SSc‐related lung disease. Methods: Email survey of 270 rheumatologists and 600 respiratory physicians. Results: Responses were received from 42 (16%) rheumatologists and 68 (11%) respiratory physicians. Of SSc patients seen by rheumatologists, 17% had ILD and 7.5% had a diagnosis of PAH compared with 31% and 21% for respiratory physicians. Forty per cent of all physicians screened asymptomatic SSc patients without a known diagnosis of ILD or PAH less than annually or not at all. The most commonly used screening investigations were pulmonary function tests (PFT) (95%) and transthoracic echocardiogram (TTE) (78%). In suspected ILD, both groups used high‐resolution computed tomography scans and PFT in >90% of patients. In suspected PAH, both used TTE and PFT (>90%); right heart catheterisation was used by only 50% of physicians. In treatment of ILD, rheumatologists used intravenous (IV) cyclophosphamide more often (CYC) (59% vs 28%, P= 0.003) and more respiratory physicians used oral CYC (44% vs 28%, P= 0.012). In PAH, more respiratory physicians used warfarin (68% vs 40%, P= 0.006). Only approximately 65% of physicians had used specific PAH therapy, which may reflect lack of access to a designated PAH treatment centre. Conclusion: The heterogeneity of responses revealed in this study raises the importance of screening, diagnosis and treatment algorithms in the management of this potentially life‐threatening disease.     

Factors related to self-efficacy in persons with scleroderma

Purpose: Scleroderma (SSc) is rare, and few studies have examined self‐efficacy in persons with the disease. Self‐efficacy is one precursor that has been shown to initiate changes in behaviour when managing chronic disease. The objective of this study was to explore the levels of self‐efficacy in persons with SSc, compare self‐efficacy in persons with limited or diffuse SSc and determine correlations between self‐efficacy, physical function and psychological variables. Methods: Sixty‐two participants with SSc completed measures assessing self‐efficacy, depression, fatigue, pain, hand function and activity limitations. The mean age of participants was 52.9 years. The mean educational level was 15.8 years. Sixty‐seven per cent were married and 87.1% were women. Thirty participants had diffuse SSc, 27 had limited SSc and five were unclassified. Results: The only significant differences between the two disease subtypes were in hand function and self‐efficacy function subscale scores. Total self‐efficacy scores significantly correlated with marital status, employment, self‐reported health, depression, functional ability, fatigue, pain and hand function. Similarly, self‐efficacy function scale scores correlated significantly with employment, self‐reported health, functional ability, pain and hand function. Self‐efficacy pain scale scores correlated significantly with fatigability. The self‐efficacy other scale scores correlated significantly with depression and fatigability. Participants with higher levels of pain and depression, more fatigue, more general disability and more hand disability had lower self‐efficacy. Conclusion: Self‐efficacy correlates with physical function and psychological variables, and could predict how patients manage their health. Self‐efficacy may increase through participation in educational programmes focusing on self‐management of these variables. Copyright © 2010 John Wiley & Sons, Ltd.