General information about amyloidosis

Reading this section first will help you understand the basics of amyloidosis, before you move on to learning about the specific types.

General information about amyloidosis

Reading this section first will help you understand the basics of amyloidosis, before you move on to learning about the specific types.

What is amyloidosis?

Amyloidosis is a syndrome caused when abnormal proteins accumulate as ‘amyloid deposits’ in body tissues and organs. Amyloid can be described as a sticky substance which is formed when proteins in the body are incorrectly formed, or ‘misfold’, and then clump together in various tissues and organs. As these amyloid deposits build up, they start to cause organ damage, this then begins to negatively impact a persons everyday activities and quality of life. Amyloidosis is still considered a rare disease, although some types are more rare than others.

Understanding Amyloidosis - 3D Animation & Overview

What is amyloidosis?

Amyloidosis is a syndrome caused when abnormal proteins accumulate as ‘amyloid deposits’ in body tissues and organs. Amyloid can be described as a sticky substance which is formed when proteins in the body are incorrectly formed, or ‘misfold’, and then clump together in various tissues and organs. As these amyloid deposits build up, they start to cause organ damage, this then begins to negatively impact a persons everyday activities and quality of life. Amyloidosis is still considered a rare disease, although some types are more rare than others.

Understanding Amyloidosis - 3D Animation & Overview

Symptoms of amyloidosis

Symptoms can vary depending on the type of the amyloidosis and which organs are affected. However, there are many common features among the different types of amyloidosis, which is why you would usually have to have many tests before an accurate diagnosis can be made. Also, some of the most common early manifestations are vague (e.g. fatigue) and/or common in other conditions (e.g. ankle swelling), and this can make getting an accurate diagnosis a lengthy and challenging process. The good news is that over the past decade knowledge and awareness of amyloidosis has grown thanks to research and innovation in diagnostic techniques and treatment. We’ll go into more detail on symptoms in later sections.

Figure 1: Some common non-specific early symptoms of amyloidosis

Symptoms of amyloidosis

Symptoms can vary depending on the type of the amyloidosis and which organs are affected. However, there are many common features among the different types of amyloidosis, which is why you would usually have to have many tests before an accurate diagnosis can be made. Also, some of the most common early manifestations are vague (e.g. fatigue) and/or common in other conditions (e.g. ankle swelling), and this can make getting an accurate diagnosis a lengthy and challenging process. The good news is that over the past decade knowledge and awareness of amyloidosis has grown thanks to research and innovation in diagnostic techniques and treatment. We’ll go into more detail on symptoms in later sections.

Figure 1: Some common non-specific early symptoms of amyloidosis

Diagnosing amyloidosis

Because amyloidosis is often hard to diagnose, a person would usually have to have a number of different tests and investigations to ensure an accurate diagnosis. Some of those are:

  • Blood and urine tests to look at how well your organs (e.g., heart, kidneys, liver) are working
  • Scans to check organ structure and function (e.g. MRI, SAP and DPD scans, echocardiogram)
  • Biopsy, where a small piece of tissue is removed and tested in the lab for amyloid deposits
  • Genetic tests on saliva or blood samples
  • Questions about your current and past medical problems and your parent’s and siblings’ medical history
  • Physical examination, which entails listening to your heart and lungs with a stethoscope, checking for visible swelling, bruising or other abnormalities, and feeling around your tummy (abdomen).

The combined results of these and other tests will show what type of amyloidosis you have and how it’s affecting you.

It’s important for patients and healthcare professionals (HCPs) alike to remember that although there are many manifestations that people with amyloidosis might have in common, EVERY person with amyloidosis is different. Even if you have the same type of amyloidosis as someone else, the way it affects you and your body will be different. Even within families it can affect people differently. For this reason, each person with amyloidosis should have a full and thorough examination to determine the effects of the condition are on them and their lives. Patients and their families play an important role in this process by making sure your doctors and nurses know about all your health issues, past and present.

Diagnosing amyloidosis

Because amyloidosis is often hard to diagnose, a person would usually have to have a number of different tests and investigations to ensure an accurate diagnosis. Some of those are:

  • Blood and urine tests to look at how well your organs (e.g., heart, kidneys, liver) are working
  • Scans to check organ structure and function (e.g. MRI, SAP and DPD scans, echocardiogram)
  • Biopsy, where a small piece of tissue is removed and tested in the lab for amyloid deposits
  • Genetic tests on saliva or blood samples
  • Questions about your current and past medical problems and your parent’s and siblings’ medical history
  • Physical examination, which entails listening to your heart and lungs with a stethoscope, checking for visible swelling, bruising or other abnormalities, and feeling around your tummy (abdomen).

The combined results of these and other tests will show what type of amyloidosis you have and how it’s affecting you.

It’s important for patients and healthcare professionals (HCPs) alike to remember that although there are many manifestations that people with amyloidosis might have in common, EVERY person with amyloidosis is different. Even if you have the same type of amyloidosis as someone else, the way it affects you and your body will be different. Even within families it can affect people differently. For this reason, each person with amyloidosis should have a full and thorough examination to determine the effects of the condition are on them and their lives. Patients and their families play an important role in this process by making sure your doctors and nurses know about all your health issues, past and present.

Treating amyloidosis

Treatment of amyloidosis varies according to type. There is no cure for amyloidosis at present so the goal of treating all types is to reduce the amount of the amyloid-forming protein (the ‘precursor’ protein) in the body. Treatments that do this are referred to as disease-modifying treatments (DMTs) because they have the potential to alter the course (or ‘natural history’) of the condition. In some types of amyloidosis there are medicines that can do this, but in other types the only DMT is to have an organ transplant. Whilst there are currently no medicines that can cure amyloidosis, there is a lot of ongoing research on new innovative treatments, which makes for a hopeful future for patients and their families. For example, an emerging class of DMTs are being developed to remove the amyloid deposits from the organs. Without effective DMTs all types of amyloidosis worsen over time.

Other medicines your doctor might prescribe are those that help counter the effects of amyloidosis on your organs – these are called supportive treatments. For example, if your heart and/or kidneys are affected (or ‘involved’ as you might hear HCPs describe it), you might be given diuretics (or ‘water’ tablets) that help the body get rid of excess fluid.The purpose of supportive therapies is to try to help reduce the impact of symptoms, delay organ failure and maintain a good quality of life for patients.

Beyond traditional medical care, people living with amyloidosis require a range of additional supportive health and social care services to help them maintain their independence and quality of life.

Treating amyloidosis

Treatment of amyloidosis varies according to type. There is no cure for amyloidosis at present so the goal of treating all types is to reduce the amount of the amyloid-forming protein (the ‘precursor’ protein) in the body. Treatments that do this are referred to as disease-modifying treatments (DMTs) because they have the potential to alter the course (or ‘natural history’) of the condition. In some types of amyloidosis there are medicines that can do this, but in other types the only DMT is to have an organ transplant. Whilst there are currently no medicines that can cure amyloidosis, there is a lot of ongoing research on new innovative treatments, which makes for a hopeful future for patients and their families. For example, an emerging class of DMTs are being developed to remove the amyloid deposits from the organs. Without effective DMTs all types of amyloidosis worsen over time.

Other medicines your doctor might prescribe are those that help counter the effects of amyloidosis on your organs – these are called supportive treatments. For example, if your heart and/or kidneys are affected (or ‘involved’ as you might hear HCPs describe it), you might be given diuretics (or ‘water’ tablets) that help the body get rid of excess fluid.The purpose of supportive therapies is to try to help reduce the impact of symptoms, delay organ failure and maintain a good quality of life for patients.

Beyond traditional medical care, people living with amyloidosis require a range of additional supportive health and social care services to help them maintain their independence and quality of life.

Getting to grips with your diagnosis

Being diagnosed with amyloidosis can be a confusing and upsetting time for patients and their families. Most people would never have heard of amyloidosis, and even pronouncing the word is difficult! Often the diagnosis comes after a long, stressful period of unexplained illness and many hospital visits. Sometimes the diagnostic process is fairly quick because others in the family have, or have had, amyloidosis – that can be scary too. It’s important to learn as much as possible about your condition.  Ask your doctor or nurse about sources of reliable and easy-to-read information about the disease, the best available treatments, and what other healthcare and social services are available to help. Remember, Amyloidosis UK is also here to support you and your family along this journey, so do get in touch!

Getting to grips with your diagnosis

Being diagnosed with amyloidosis can be a confusing and upsetting time for patients and their families. Most people would never have heard of amyloidosis, and even pronouncing the word is difficult! Often the diagnosis comes after a long, stressful period of unexplained illness and many hospital visits. Sometimes the diagnostic process is fairly quick because others in the family have, or have had, amyloidosis – that can be scary too. It’s important to learn as much as possible about your condition.  Ask your doctor or nurse about sources of reliable and easy-to-read information about the disease, the best available treatments, and what other healthcare and social services are available to help. Remember, Amyloidosis UK is also here to support you and your family along this journey, so do get in touch!