Treatments of Amyloidosis

Executive Summary:

Amyloidosis is a rare disease condition, and timely diagnosis in addition to proper treatment are essential to cure this disease condition. While, several treatment methods are available for Amyloidosis Standard treatment, but medicated therapy is the most common treatment. Medicated therapies are used to eliminate abnormal cells. Medications are administered via bloodstream to reach the abnormal cells in the body. Targeted therapies and chemotherapy are other medication therapies. There are different targeted therapies used for treating Amyloidosis in addition to monoclonal antibodies, anti-angiogenesis therapy, and proteasome inhibitors. there are several treatments available for Amyloidosis. The primary surgical procedure involves organ transplantation. Stem cell transplantation and bone marrow transplantation are used in case of accumulation of high levels of amyloid protein within tissues and organs.

The treatment approach of Amyloidosis

There are many standard treatment options. Common treatments of Amyloidosis refer to the best treatment plan available at the latest ​1,2​. Patients should ask the doctors about the best treatment options available. Patients can also enquire about any ongoing clinical trials and participate in them. Clinical trials can help doctors to understand whether a particular medication or treatment strategy will work for a patient’s disease condition or not. 

Standard Treatments

Generally, Amyloidosis is treated with a combination of different treatments. While the doctor and healthcare team will develop a treatment strategy per the patient’s condition, a multidisciplinary approach is also employed to treat Amyloidosis as the disease can affect multiple body organs.

The health care team often includes the following specialists:

  • Cardiologist: A doctor who specializes in treating heart conditions.
  • Gastroenterologist: A specialist who treats diseases of the gastrointestinal tract 
  • Haematologist: A doctor who specializes in disorders related to the blood
  • Nephrologist: A health care specialist who treats kidney disorders.
  • Pulmonologist: A doctor who treats conditions of the lungs.
  • Neurologist: A specialist who focuses on the nervous system and brain issues.

Primarily, a haematologist coordinates this multidisciplinary team of specialists, including physician assistants, dietitians, nurses, nurse practitioners, social workers, counsellors, pharmacists, and others.

The treatment option for Amyloidosis

  • The types of amyloidosis condition
  • Organs affected
  • Patient’s preferences
  • Age and overall health condition
  • Possible side effects of treatment.

Patients should know that the treatment plan will include dealing with the symptoms and side effects of the disease and treatment, moreover, patients are encouraged to ask questions regarding any treatment procedure and make informed choices. They are free to ask their doctor or health care team any treatment-related queries.

Ask your doctor about what you should expect from this particular treatment. In shared decision making, the patient and doctor design a treatment plan suitable to treat a disease condition. Therefore, shared decision making is vital in choosing treatment for Amyloidosis.

The following are the treatments of Amyloidosis available to treat amyloidosis disease condition.

Medicated Therapy

Medicated therapies are used to eliminate abnormal cells, and so they are usually administered via the bloodstream to reach the abnormal cells situated throughout the body. Another way is to assist the medication locally, i.e., directly applying the medication to the abnormal areas or the affected region of the body.

Medicated therapies are generally prescribed by a medical oncologist (a doctor specializing in treating a tumour or abnormal growth with medication) or a haematologist.

The medication is injected into an intravenous tube placed in the vein, moreover, the drug is also provided in the form of a pill that can swallow orally. If you are given medicines or any oral medications, ask your health care team about how to handle and safely store them.

Medicated therapies used to treat Amyloidosis ​3,4​include:

  • Targeted therapy 
  • Chemotherapy

A person may receive only one type of treatment at a time or a combination of therapy at the same time. Procedures include medicines too. For example, bone marrow transplantation or surgery.

Patients are also required to openly talk to their doctor about any other medications or supplements they are using. They can probably react with cancer medications and can cause harmful side effects or sometimes reduce the effectiveness of the treatment.

Targeted Therapies for Amyloidosis

Targeted therapy is a treatment procedure that targets specific proteins, genes, or tissues, which influences the development of Amyloidosis additionally, this type of treatment prevents abnormal cells’ growth and spread, thereby avoiding the damage caused to healthy cells.

There are different types of targeted therapies. It depends on the disease condition or abnormalities. The doctor will carry out a few tests or scans to identify proteins, genes, or other essential factors to decide the effective targeted treatment, moreover analyzing these helps doctors to arrive at the correct procedure suiting a person’s amyloidosis condition.

Types of targeted therapies for Amyloidosis

The different targeted therapies used for treating Amyloidosis include monoclonal antibodies, anti-angiogenesis therapy, and proteasome inhibitors.

  • Monoclonal antibodies: It is a type of targeted therapy, and additionally, it works by recognizing and attaching to a specific protein in the abnormal cells. It doesn’t cause harm to the normal cells that don’t have the particular protein and simultaneously attacks the cell that contain the protein. Drugs in this class include elotuzumab (Empliciti) and daratumumab (Darzalex). The second type of monoclonal antibody directly targets the amyloid build-up. Subsequently, such antibodies are being tried and tested in many recent clinical trials.
  • Immunomodulatory drugs: medications/medicines in this class include pomalidomide (Pomalyst), lenalidomide (Revlimid), and thalidomide (Synovir, Thalomid).
  • Proteasome inhibitors: a type of targeted therapy that uses drugs that target specific enzymes called proteasomes that digest cell proteins. The drugs include carfilzomib (Kyprolis), bortezomib (Velcade), and ixazomib (Ninlaro).

Studies are still analyzing the impact of each of these drugs in treating Amyloidosis. While all studies claim that none of them can reverse amyloid build-up in tissues or organs, researches show that a combination of dexamethasone, bortezomib, and melphalan can help improve the working of body parts affected by Amyloidosis.

A recent study shows that a four-drug combination is effective. Hence, the treatment is extremely safe and standard in treating Amyloidosis, and additionally the effects and symptoms that the disease causes.

Many clinical trials are studying and testing other treatments of Amyloidosis and medications, subsequently, many types of research focus on devising treatment strategies that can improve the functioning of those organs affected by amyloid deposits.

Side effects of targeted therapy

Targeted therapy causes various side effects. Most of them are linked with the chemo procedure. Additionally, patients who receive targeted therapy can develop eye, skin, hair, or nail problems. While these side effects are scary, communicating with your doctor or healthcare team about it can help managing and relieving any probable side effects.

Chemotherapy for Amyloidosis

Chemotherapy or chemo procedure uses drugs or medications to eliminate abnormal cells. It prevents abnormal, unhealthy cells from growing, dividing and spreading. The process is usually and widely used to treat different forms of cancer. It is also effective in treating non-cancerous conditions like Amyloidosis.

In Treatments of Amyloidosis, a chemo procedure destroys the abnormal cells present in the blood. Chemo drugs are given intravenously, injecting the medication using a needle through an IV tube inserted into the vein, while sometimes, the remedy is provided as a shot under the skin or given as a capsule or a capsule pill be swallowed orally.

A chemo schedule or regimen usually consists of a specific set of cycles carried out over a set period, therefore the patient may receive one drug or medication at a time or a combination of different drugs simultaneously. The common types of chemotherapy procedures and medications used to treat Amyloidosis are melphalan (Alkeran) and cyclophosphamide (Cytoxan, Neosar), with steroids like prednisone (multiple brand names) and dexamethasone (numerous brand names) .

Side effects of chemotherapy

Chemotherapy can cause many side effects. The side effects depend on the type of medications used, their dosage, and the patient’s overall health condition. The common side effects include extreme fatigue, vomiting, nausea, hair loss, risk of infections, diarrhoea, appetite loss etc. Most of these side effects usually go away once the treatment ends.

People who receive chemo treatment (involving melphalan) for Amyloidosis are anemic. Anemia occurs as a result of low RBC levels in the blood. Hemoglobin, the iron-containing protein in the red blood cells, carries oxygen across different body parts. Deficient RBC levels in the blood interrupt the oxygen supply to other body parts, causing problems. Anemic people constantly feel exhausted and weak.

Patients should talk with their health care team about the probable side effects of chemo treatments and ways to manage and relieve them.   

Surgery for Amyloidosis

The primary surgical procedure used to treat Amyloidosis is organ transplantation. Liver transplantation is effective for treating certain types of hereditary Amyloidosis. Heart and Kidney transplantations also work. But organ transplantation may not be possible in the case of all patients, especially those who have a severe amyloidosis case (ones with a large amount of amyloid build-up in the organs). Talk with your doctor or healthcare team regarding the effectiveness of organ transplantation and related treatments options for Amyloidosis. 

Stem cell transplantation or Bone marrow transplantation

High doses of medicated therapies destroy plasma cells inside the bone marrow. Highly specialized cells replace abnormal cells. These cells are hematopoietic stem cells, and they further develop into the healthy bone marrow. These Hematopoietic stem cells are blood-forming cells found in the bone marrow and the bloodstream. This procedure is peripheral blood stem cell transplant. For instance, stem cells collected from the bloodstream and not the actual bone marrow tissue transplanted.

For people with Amyloidosis having some degree of normal organ functions, peripheral blood stem cell transplantation may be effective. High doses of chemotherapy eliminate abnormal blood cells. After this procedure, the patient receives healthy peripheral blood stem cells, allowing the body to begin making healthy blood cells. With high doses of chemotherapy and peripheral blood stem cell transplantations, survival rates of Amyloidosis cases are significantly improved. But most patients cannot receive the treatment due to high levels of amyloid protein build up in their tissues and organs, which has dramatically affected their organ functioning. 

The doctors will (and should) talk about every aspect related to stem cell transplantation before recommending it to a patient. The doctor will explain the efficiency of the treatment, its risk factors, and factors that decide whether or not to recommend the treatment like the type of disease condition, patient’s overall health condition, past treatment history etc.

Physical, emotional, and social effects of Amyloidosis

A disease condition like Amyloidosis can cause a range of physical, emotional and social side effects. Managing and relieving any associated side effects is crucial for treatment and care; this part is supportive or palliative care. It starts alongside active treatment.

Palliative care primarily focuses on what and how you feel during treatments of Amyloidosis and provides aid in managing any symptoms or side effects. Palliative care supports the patients and the people associated with them, assisting them in non-medical needs. Any person independent of age, type of disorder, gender will receive this type of care. Palliative care usually starts right after diagnosis. Patients who receive proper care and active treatment often have less severe symptoms or side effects. It plays a vital role in improving the comfort and quality of the patient’s life.

Palliative care will include medications, diet changes, mental spiritual support, relaxation methods and other soothing therapies. 

Patients should talk about the effectiveness of each procedure ​5​ , and also enquire about the possible side effects, benefits of Amyloidosis treatments, and the mode of supportive care provided. Patients should openly talk with a social worker about whatever they feel and participate in different support groups. Your doctor can help you find these resources.

Also, during the treatment, the healthcare team will ask you several questions about the side effects and symptoms that you are experiencing. Be sure to tell everything openly, as only this will help the team deal with these symptoms and side effects and cure your disease condition.

What If the treatment does not work

Amyloidosis is a rare disease condition, and complete recovery from the situation may be impossible at times. It is especially true in the case of terminal or advanced Amyloidosis. The diagnosis and treatment of advanced Amyloidosis are difficult. Patients under extreme stress find it difficult to open up once they are diagnosed with an advanced stage of the disorder. 

However, open and honest conversations with your doctor and health care team is critical to express your feelings, concerns and preferences. The healthcare team will include specialists who have expertise in handling such situations and provide support and care to the patients and their families. In treatments of Amyloidosis, making the patient physically and mentally strong is exceptionally vital.

Patients who have a terminal amyloidosis condition and whose survival rate is less than six months can avail of hospice care. The primary goal of hospice care is to provide those nearing life end with the best possible care and support. Patients and their loved ones can decide where to provide this care- at home, in the hospital, or a proper hospice environment. If provided the appropriate nursing care and equipment, staying at home can be a workable, effective option for many families. Family members of a patient who is at the end of life or has passed away can also avail support and care.


  1. 1.
    Merlini G, Seldin DC, Gertz MA. Amyloidosis: Pathogenesis and New Therapeutic Options. JCO. Published online May 10, 2011:1924-1933. doi:10.1200/jco.2010.32.2271
  2. 2.
    Hirschfield GM, Hawkins PN. Amyloidosis: new strategies for treatment. The International Journal of Biochemistry & Cell Biology. Published online December 2003:1608-1613. doi:10.1016/s1357-2725(03)00169-9
  3. 3.
    Ihne S, Morbach C, Sommer C, Geier A, Knop S, Störk S. Amyloidosis—the Diagnosis and Treatment of an Underdiagnosed Disease. Deutsches Ärzteblatt international. Published online March 6, 2020. doi:10.3238/arztebl.2020.0159
  4. 4.
    Baker KR, Rice L. The Amyloidoses: Clinical Features, Diagnosis and Treatment. Methodist DeBakey Cardiovascular Journal. Published online July 1, 2012:3. doi:10.14797/mdcj-8-3-3
  5. 5.
    Tan SY, Pepys MB, Hawkins PN. Treatment of amyloidosis. American Journal of Kidney Diseases. Published online August 1995:267-285. doi:10.1016/0272-6386(95)90647-9