What we need to know about scleroderma… A rare autoimmune disease, how it is treated, what the state does… Boryana Boteva, who is the chairperson of the Association of Patients with Rheumatological Diseases and the chairperson of the Association for the Development of Bulgarian Health Care, spoke to FAKTI.
- Mrs. Boteva, what is the disease scleroderma?
- Systemic sclerosis (scleroderma) is a chronic progressive connective tissue disease. Most often, the disease occurs in the age group of 30-50 years. Women are affected 7 times more often than men. The cause of the disease is unknown. Some external factors are supposed to be important – chemical agents such as silicon, vinyl chloride, medications, and bacterial and viral infections. The disease is treated with immunosuppressive drugs, corticosteroids, and some new targeting drugs that target specific organs, such as the lung, for example.
- How many people in Bulgaria suffer from this disease?
- We have no data on the number of people affected by scleroderma in Bulgaria, but the disease is rare. About 200,000 people in the US are affected by scleroderma, and the systemic form – 80,000 people. For Europe, according to the various studies, the affected are from 7.2 to 33.9 per 100,000 people in different countries.
- Does the disease lead to death… What is the survival rate?
- Mortality in scleroderma is very high. Unfortunately, from 3 to 5 years after the first symptoms, there is already organ involvement.
- What is characteristic of this disease. What are the symptoms…
- Swelling, thickening of the skin, itching, severe fatigue, emotional changes, joint pain, muscle weakness, shortness of breath, cough, digestive problems, kidney problems. Pulmonary involvement in systemic sclerosis can occur as pulmonary hypertension or interstitial lung disease. Pulmonary arterial hypertension affects about 15% of patients with progressive systemic sclerosis. Interstitial lung disease affects about 80% of patients with progressive systemic sclerosis (PSS), but only about 25-30% of them develop progressive disease within 5 years of symptom onset. The disease usually manifests at the beginning through the so-called Raynaud's syndrome. Raynaud's syndrome, also called Raynaud's disease, is a condition that affects the blood flow in the fingers and toes and changes the color of the skin as a result. In some cases, it can also affect the ears, nose and lips. A very characteristic whitening of the fingers occurs at low temperatures.
- How difficult is the diagnosis?
- Diagnosing the disease can be delayed, as it affects many organs and systems in the human body and requires a multidisciplinary approach, which we unfortunately lack. It may be possible in some larger medical facilities.
- Do we have adequate access to medicines. Are they covered by NHIF?
- The National Health Insurance Fund currently covers only the specific medications aimed at organ involvement in systemic sclerosis, in this case we are talking about medications for pulmonary involvement. They are from the group of expensive drugs and patients go through specialized committees every 6 months to get a protocol. It's great that we have access to new therapies. We just wish access was much easier for patients. Standard immunosuppressive medications and corticosteroids are not paid for by the NHIF. They are often expensive and patients purchase them from Bulgaria's neighboring countries. And this is a basic treatment of systemic sclerosis, if I can describe it that way. The National Health Insurance Fund does not pay for the treatment with immunoglobulins, which is also applied in some cases. Here we are talking about an off label application, the disease does not appear in the summary of the immunoglobulins. For some patients, a course of 90 ampoules costs nearly BGN 3,000. How often immunoglobulins will need to be administered depends on the individual patient and the severity of the disease. This problem also affects patients with systemic lupus and other systemic connective tissue diseases. People either do not buy immunoglobulins, or take out loans, or rely on relatives.