by Danielle Bradshaw from In The Cloud Copy
A year-long study has shown that Tracleer greatly reduces the skin-related symptoms of scleroderma. The results also showed that although it helped patients with more severe scleroderma symptoms, the effectiveness was lessened.
What Is Scleroderma?
Scleroderma is a disease in which a person’s immune system attacks healthy connective tissues, causing them to create an overabundance of collagen. This can result in the formation of excessive scar tissue on both the skin and organs, as well as around blood vessels, which makes them thicken and stiffen.
The signs and symptoms can differ from person to person but a large number of people who suffer from it – 40 percent – tend to have digital ulcers (DUs). Digital ulcers are small sores that form on the patient’s toes and fingers due to restricted blood flow. This also makes them very hard to heal and may necessitate surgical removal.
Raynaud’s disease is another sign of scleroderma. This occurs when blood vessels in the fingers and toes contract due to cold or emotional distress. This results in fingers and toes turning blue, with numbness, tingling, or pain. People with scleroderma also develop many types of lung diseases, two of the more common types being pulmonary arterial hypertension (PAH) and interstitial lung disease (ILD).
What Does Tracleer Treat Exactly, and How?
What Tracleer is used to treat depends on what part of the world scleroderma patients live in. While it has shown effectiveness in treating both DUs and PAH, it is used for the former in Europe, while in the United States, it has been approved by the Food and Drug Administration (FDA) to treat the latter.
The medication works by blocking the signaling molecule, endothelin. Endothelin is a naturally created substance in the human body that makes blood vessels narrow. Tracleer is able to stop endothelin from tightening blood vessels and allows improved blood flow, despite the vessels being surrounded by scar tissue.
How the Study Was Conducted
In 2016 to 2017, a team of researchers in Romania did a first-year study of the benefits of Tracleer with a group of 41 people who had either:
- Diffuse systemic scleroderma, which categorized by increased likeliness of skin fibrosis on the torso, arms, legs, and a greater chance of organ damage. 28 of the participants had diffuse.
- Limited systemic scleroderma, a more common form of scleroderma which is slower to develop and has much fewer cases in which patients develop organ damage. 13 of the patients suffered from limited.
Two women developed higher-than-normal liver enzyme levels and left the study (the issue was resolved by stopping treatment), but the 39 that remained continued taking Tracleer.
They began with 62.5mg twice a day for the first four weeks of the trial and were then put on an increased dosage of 125mg; patients were monitored for increases in their liver enzyme levels and for other potential side effects of the treatment.
All patients had their opinions on the effectiveness of the treatments regularly evaluated by three separate polls or visual analog scales (VAS):
- A 0mm to 100mm scale – 0 for no symptoms and 100 for the worst symptoms.
- Severity of their Raynaud’s disease (referred to as VAS-R)
- Severity of their digital ulcers (VAS-DU)
These measurements were then gauged alongside the Health Assessment Questionnaire Disability Index (HAQ-DI) which was used to verify how difficult it was for the patients to perform daily tasks such as bathing and eating.
The Results of The Study
At the end of the year, there was a large improvement in patients since the test first began:
- VAS-R had an increase of 38.71 points
- VAS-DU had an increase of 51.41 points
- HAQ-DI increased 0.72 points
The type of scleroderma didn’t seem to affect the results, nor did gender or any additional treatments being administered to the participants. There was, however, a predicted decrease in effectiveness around 12 months – especially in VAS-DU and VAS-R – in patients with higher disability scores.
Approximately 50% of the participants reported shortness of breath (which was found to be the biggest impairment throughout measuring the HAQ-DI) and there was a marked decrease in effectiveness in patients over 60.
Overall, while the research team acknowledged that the small group size and the absence of a control group were somewhat constraining, the fact of the matter is that the fewer symptoms a person had at the start of the test meant more improvement with treatment.