Treatment is ‘promising option,’ more studies needed, researchers say by Margarida Maia, PhD | May 14, 2024 Local injection with botulinum toxin type A (BTX-A), such as Botox, may ease the severity of pain associated with Raynaud’s phenomenon in people with scleroderma, also known as systemic sclerosis, a meta-analysis study found.
The study, “Efficacy and safety of botulinum toxin in treating scleroderma-associated raynaud’s phenomenon: a systematic review and meta-analysis,” was published as a research letter in the Archives of Dermatological Research. Scleroderma affects the connective tissue that holds tissues and organs together. While it mainly causes the skin to harden, scleroderma also can damage internal organs and blood vessels. It often starts with Raynaud’s phenomenon, which may appear long before other symptoms. Raynaud’s phenomenon limits blood flow to fingers and toes due to cold or stress. As a result, fingers and toes can change color and become numb or prickly. This is often accompanied by pain and sometimes digital ulcers or sores. Bacteria-derived BTX-A, often injected locally to smooth wrinkles on the face, among other uses, is being tested as a treatment for Raynaud’s phenomenon secondary to scleroderma. It’s thought that it may work in part by widening the blood vessels and reducing pain. Formula could ID patients in need of 'enhanced managing strategies,' study says by Lindsey Shapiro, PhD | April 30, 2024 Researchers have developed a model for predicting which systemic sclerosis (SSc) patients with interstitial lung disease (ILD) — together known as SSc-ILD — will experience progressive lung function declines that are known to be associated with a poorer prognosis.
The prediction algorithm accounts for nine clinical factors, including age, certain SSc features and symptoms, therapy use, and disease-associated antibodies. All were found to be linked to the development of this aggressive form of ILD — called progressive fibrosing interstitial lung disease or PF-ILD — in a group of about 300 patients. “This study developed the first prediction model for PF-ILD in patients with SSc-ILD,” the researchers wrote, noting that “the established formula … could help in identifying patients with the highest risk, who should receive enhanced managing strategies during follow-up.” Titled “Prediction of progressive fibrosing interstitial lung disease in patients with systemic sclerosis: insight from the CRDC cohort study,” the work was published in RMD Open. Half of patients in study found to have aggressive disease type PF-ILD In SSc, also called systemic scleroderma, an overactive immune system drives inflammation and scar tissue buildup — known as fibrosis — in the skin and internal organs. When the lungs are affected, patients may experience ILD, then named SSc-ILD, where the lung tissue is scarred and damaged, and breathing becomes more difficult. The severity and progression of interstitial lung disease can vary. PF-ILD, a progressive form, is marked by “rapidly aggravating [shortness of breath], progressive deteriorating lung function, declining physical functional capacity, worsening health-related quality of life, adverse therapeutic response and often early mortality,” the researchers wrote. An earlier identification of which patients are likely to experience this type of progression may enable a sooner start to interventions that could slow it down or stop it, preventing irreversible lung damage, according to the team. While some studies have reported a number of potential predictive factors of PF-ILD, an accurate model for identifying SSc patients who might be at a high risk of the aggressive lung disease has yet to be developed. Now, a team of scientists in China sought to develop one such model by looking at clinical information from 304 SSc-ILD patients who were registered in a Chinese database. Meta-analysis has implications for prognosis and prevention, researchers sayby Andrea Lobo, PhD | April 23, 2024 Having scleroderma increases the risk of developing diseases affecting blood vessels of the brain or heart, known as cerebrovascular and cardiovascular diseases, a review study found.
Relative to controls, the risk of stroke — caused by poor blood flow to the brain — in scleroderma patients was 64% higher, whereas that of cardiovascular disease was 112% higher. “Our findings support increased clinical surveillance and consideration of preventative strategies in this high-risk population,” researchers in Taiwan wrote. Their study, “Association between systemic sclerosis and risk of cerebrovascular and cardiovascular disease: a meta-analysis,” was published in Scientific Reports. Scleroderma, also known as systemic sclerosis (SSc), is characterized by the excessive production of collagen that leads to the accumulation of scar tissue, affecting the skin and potentially internal organs, such as the digestive tract and lungs. The disease may also damage blood vessels, both small and large, leading to complications such as pulmonary arterial hypertension and scleroderma renal crisis. Evidence has suggested that people with scleroderma may have an increased risk of stroke, heart attack, and peripheral vascular disease, which is reduced circulation in blood vessels outside the brain and heart. However, “the risk of cerebrovascular and cardiovascular complications attributed to SSc remains debated,” the researchers wrote. Calcitonin and SOST levels may point to PAH and ILD, small study find by Marisa Wexler, MS | April 9, 2024 Levels of two blood proteins, calcitonin and SOST, may be markers of lung disease in people with scleroderma, a new study shows.
“This study indicates that serum calcitonin and SOST levels are promising biomarkers for [scleroderma]-related PAH [pulmonary arterial hypertension] and ILD [interstitial lung disease], respectively,” the researchers wrote, though they noted that “further research is needed to verify these results and understand the underlying mechanisms.” The study, “Protein profiling in systemic sclerosis patients with different pulmonary complications using proteomic antibody microarray,” was published in Arthritis Research & Therapy. Scleroderma, also called systemic sclerosis or SSc, is marked by abnormal scarring that usually affects skin and can also affect various other organs throughout the body. When the disorder involves the lungs, SSc can lead to complications like interstitial lung disease (ILD, marked by lung scarring) and pulmonary arterial hypertension — PAH, defined by high pressure in the blood vessels that carry blood to and through the lungs. These lung issues are leading causes of mortality in SSc, and early detection is key to facilitate optimal treatment. Scleroderma patients who feel socially isolated report more life dissatisfaction by Andrea Lobo, PhD | April 2, 2024 People with scleroderma who feel more socially isolated are more likely to report being dissatisfied with life, but this association is partially attenuated in those who show more resilience, according to a recent study.
Resilience refers to the ability to adapt to and recover from challenging life experiences, such as living with a chronic disease. These findings suggest that more adaptive coping may help protect against the negative effects of perceived social isolation, and “support the promotion of social connection and resilience to enhance life satisfaction in people with [scleroderma],” researchers wrote. The study, “Resilience partially mediates the association between perceived social isolation and life satisfaction in people with systemic sclerosis,” was published in the Journal of Scleroderma and Related Disorders. Scleroderma, or systemic sclerosis (SSc), is an autoimmune disease characterized by the accumulation of scar tissue in the skin and potentially several internal organs, including the heart, kidney, lungs, and gastrointestinal tract. Scleroderma-related changes may lead to mental health issues SSc-related physical changes and limitations, and the uncertainties about disease progression may lead to mental health issues, such as anxiety and depression, and feelings of social isolation. Therefore, assessing a patient’s well-being is an important part of disease management. In studies of other chronic disorders, resilience was found to help patients address daily life stressors and functional limitations, and improve their social functioning. However, “examination of life satisfaction in the SSc population remains limited … [and] no known study has investigated the mediating role of resilience in the relationship between perceived social isolation and life satisfaction in people with SSc,” the researchers wrote. To know more, researchers in the U.S. analyzed baseline (at the beginning of a study) data from the RENEW clinical trial (NCT04908943), in which patients were randomly assigned to a 12-week online intervention to help them manage energy and symptoms, or to no intervention. RENEW stands for Resilience-building Energy management to Enhance Well-being. The study included 163 participants, who were mainly women (93.9%), white (85.3%), married (66.7%), and with a college or higher degree (59%). Nearly half of the patients had diffuse cutaneous SSc, and 57% were within five years of their SSc diagnosis. CAR T-cell therapy for SSc designed to eliminate hyperactive B-cells by Steve Bryson, PhD | March 26, 2024 The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Cabaletta Bio’s cell therapy CABA-201 as a treatment for adults with hard-to-treat systemic sclerosis (SSc), or scleroderma.
Orphan drug designation is intended to support the accelerated development of investigational treatments for rare diseases, defined as those affecting fewer than 200,000 people in the U.S. The designation provides Cabaletta with regulatory support, financial incentives, and seven years of market exclusivity if CABA-201 is approved. “Orphan drug designation is an important recognition for investigational therapies for rare diseases, and provides us with potentially valuable benefits as we develop CABA-201 for patients with systemic sclerosis,” David J. Chang, MD, chief medical officer of Cabaletta, said in a company press release. CABA-201 received FDA fast-track designation to reduce SSc-associated organ dysfunction in January. That status is designed to speed the development of therapies that address unmet medical needs for serious conditions. The FDA in October cleared Cabaletta’s request to launch a Phase 1/2 clinical trial, RESET-SSc (NCT06328777), to evaluate CABA-201 in 12 adults, ages 18-70. Location information on the study, expected to start in June, is not yet available. Results could help researchers develop targeted scleroderma therapies by Patricia Inácio, PhD | March 19, 2024 Lung disease severity in scleroderma (SSc) patients may be linked to levels of immune cells known as follicular T-helper cells (Tfh cells), according to a small study.
The findings “may guide the development of targeted therapies for this aspect of the disease,” the researchers wrote. Titled “The relationship between peripheral T follicular helper cells and disease severity in systemic sclerosis,” the study was published in the journal Clinical and Experimental Medicine. In scleroderma, the immune system produces self-targeting antibodies, or autoantibodies, that lead to hardening and fibrosis (thickening or scarring) of the skin. It sometimes affects internal organs as well. B-cells are responsible for producing antibodies, but evidence suggests that Tfh also may play a role in scleroderma. Tfh cells are a specialized subset of T-cells that support the antibody production of B-cells and have been implicated in autoimmune diseases such as multiple sclerosis and myasthenia gravis. However, a link between t-helper cells and disease severity in SSc has not been established. Inflammatory arthritis — a condition marked by joint inflammation that causes swelling, pain, and damage — affects about one-third of people with scleroderma, also known as systemic sclerosis (SSc), and is associated with worse health-related quality of life, or HRQoL for short, according to a new study from Australia.
“Our results reveal the significant impact that [inflammatory arthritis] has on patients, with the presence of [such inflammation] being negatively associated with employment, physical function, and HRQoL,” the researchers wrote. Given that such arthritis is treatable, “further research is needed to understand the extent of joint involvement in SSc and optimal strategies for its management,” the researchers wrote. Their study, “Inflammatory Arthritis in Systemic Sclerosis: Its Epidemiology, Associations, and Morbidity,” was published in the journal Arthritis Care & Research. Cognition is a problem for 50% of patients in study, while 29% were malnourished by Patricia Inácio, PhD | March 5, 2024 Cognitive difficulties affected half of all scleroderma patients in a recent study in Italy, while malnutrition was found in more than one quarter.
While no correlation was seen between cognitive impairment and malnutrition in the 100 patients involved, the presence of these complications associated with greater functional disability and a poorer quality of life. Identifying them early is “pivotal to better address the chronic needs of patients affected by this disease,” researchers wrote in the study, “Role of cognitive impairment and malnutrition as determinants of quality of life in patients with systemic sclerosis,” published in the Journal of Scleroderma and Related Disorders. The condition is associated with inflammation, end-organ involvement by Patricia Inácio, PhD | February 27, 2024 Muscle disease is common in people with scleroderma and is associated with inflammation and specific end-organ involvement, according to an Australian study.
The findings underscore the “clinical, functional, and prognostic importance of simple biomarkers ” for identifying SSc-related muscle disease, the study’s researchers wrote in “Proximal weakness and creatine kinase elevation in systemic sclerosis: clinical correlates, prognosis and functional implications,” which was published in Seminars in Arthritis and Rheumatism. Scleroderma, also called systemic sclerosis (SSc), is a chronic disease that features excessive scarring of the skin and other organs due to abnormal immune responses against the body’s own tissues. Muscle disease, or myopathy, can be common in SSc and develop due to the indirect effects of the disease, such as malnutrition. A previous study using MRI scans showed muscle involvement was found in up to 40% of asymptomatic SSc patients. A lack of consensus remains about what SSc-myopathy is and of biomarkers for muscle disease in SSc, leading researchers to assess whether two routine clinical biomarkers of muscle disease — elevated blood levels of creatine kinase (CK) and proximal muscle weakness (PW) — could help identify those at risk for more severe outcomes. The scientists analyzed data from patients enrolled in the Australian Scleroderma Cohort Study between 2007 and 2023 who had at least one assessment of PW and CK levels during follow-up. |
AuthorScleroderma Queensland Support Group Archives
May 2024
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