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Amyloidosis (amyloidosis) is a disease in which a biologically inactive protein (amyloid) is deposited in the cells of various organs (most often the kidneys, liver, spleen). There are three types of this disease: primary, secondary and familial. The goal of treatment is to stop the development of amyloidosis.
Amyloidosis – the definition of the disease
Amyloidosis is a condition otherwise known as amyloidosis, it is a multi-system condition where the abnormal protein amyloid is deposited in various tissues. This causes damage to the organs they use, most often the liver, kidneys and heart. Depending on what type of protein accumulates in organs and what symptoms occur in individual people, we can distinguish three types of amyloidosis: primary, secondary and family. Amyloidosis usually occurs around the age of 45 and affects women more than men.
Causes of Amyloidosis
So far, the causes of poor protein structure and its deposition in organs are not fully known. The accumulated amount of protein in given organs oppresses them, which causes their function to be disturbed, and then the organ’s parenchyma atrophy.
The indirect causes of amyloidosis include:
- chronic inflammation,
- autoimmune factors,
- genetics.
Breakdown of amyloidosis
1. Primary amyloidosis – the bone marrow produces defective antibodies that do not fulfill their function and instead of protecting against various pathogens, they accumulate in the blood, successively reaching the tissues where they destroy them under the influence of pressure.
2. Secondary amyloidosis – there are simultaneously other disorders of the nature of chronic systemic inflammations, therefore it is not known which disease causes the formation of the latter. It is important that treating the accompanying ailment reduces the risk of developing amyloidosis.
3. Familial amyloidosis – it appears due to a genetic predisposition, patients inherit the disease in an autosomal dominant manner, i.e. a sick parent has a 50% risk of transmitting the disease to their offspring.
Symptoms of amyloidosis
Depending on the organs involved, amyloidosis can cause different symptoms:
• kidneys: proteinuria, renal failure;
• heart: heart failure with prognosis, heart rhythm disturbances;
• peripheral nervous system: neuropathies and polyneuropathies (impaired function of nerve fibers);
• the brain: Alzheimer’s disease;
• language: magnification (macroglossia);
• liver: enlargement (hepatomegaly);
• spleen: enlargement (splenomegaly).
In addition, patients complain of:
- clotting problems,
- trouble feeling in the hands and feet,
- shortness of breath
- exercise intolerance,
- dizziness (when changing position),
- swelling of the legs (especially ankles)
- weight loss,
- weakness.
Circulatory symptoms of amyloidosis
Typical features that affect the circulatory system are:
- reduction of the QRS amplitude visible on the ECG,
- edema due to congestive heart failure,
- problems with proper filling of the heart cavities with blood due to its stiff walls,
- ventricular tachycardia,
- ventricular fibrillation (can lead to sudden death).
Amyloidosis and damage to the nervous system
Symptoms of the nervous system damage caused by amyloidosis mainly concern its peripheral part. There is peripheral polyneuropathy, which is characterized by the abnormality of many nerves, especially the limbs, which become weak and numb. Pathological changes, however, do not apply to spinal birth and the brain.
Peripheral polyneuropathy can be divided into:
- autonomic – responsible for disorders in the circulatory system, bladder, and rapid fatigue and trouble breathing;
- sensory – in contrast to motor polyneuropathy, it is more symmetrical, attacks both limbs and sides along the midline of the body;
- motor – occurs less frequently than sensory polyneuropathy, does not attack symmetrically.
Diagnosis of amyloidosis
To diagnose amyloidosis, it is necessary to carry out:
- genetic research;
- biochemical research;
- a biopsy of a diseased organ.
A biopsy allows you to make a clear diagnosis. The material is usually taken from the abdominal subcutaneous fat and then examined under a microscope. If amyloidosis is genetic, genetic testing should be performed to confirm the presence of the gene mutation. The biochemical test involves the examination of biochemical markers: the concentration of troponin T or I and NT-pro-BNP. Controlling these indicators allows you to monitor the advancement of the disease and the effectiveness of the implemented treatment. In addition, doctors recommend that you have an EKG or echo of the heart to be able to better see how your heart is working. It is also important to have a basic blood count and a urine test to have an overview of the patient’s general condition.
Treatment of amyloidosis
Treatment of amyloidosis is primarily to inhibit the development of the disease and support the function of the organs affected by the disease. The current treatment methods include:
- chemotherapy followed by transplantation of stem cells derived from the patient’s blood. This is called an autologous transplant;
- administration of monoclonal antibodies, immunosuppressants and cytostatics;
- administration of glucocorticosteroids (anti-inflammatory effect).
If a particular organ is involved, its function should be supported and the development of amyloidosis should be inhibited. Patients who develop renal failure are treated with dialysis or diuretics (in the case of a milder course). Cardiac arrhythmias require the use of antiarrhythmic preparations. In contrast, familial amyloidosis requires a liver transplant because damaged protein is produced in it.