Amyloidosis treatment

Amyloidosis has no known treatment. Treatment, on the other hand, can help control symptoms while also limiting amyloid protein synthesis. If another illness, such as rheumatoid arthritis or TB, has prompted the amyloidosis, treating the underlying ailment may be beneficial.

Overview of the treatment

Different types of doctors frequently collaborate to develop a patient’s overall treatment plan, which may include a variety of therapies. Because amyloidosis affects so many organs, this is extremely essential. A multidisciplinary team is what this is referred to as.

  • These professionals are frequently found on the treatment team:
  • Hematologist. A blood problem specialist is a physician who specializes in the treatment of blood diseases.
  • Cardiologist. A cardiologist is a doctor who specialises in the diagnosis and treatment of heart disorders.
  • Gastroenterologist. A gastroenterologist is a doctor who specializes in gastrointestinal problems.
  • Pulmonologist. A pulmonologist is a doctor who specializes in lung problems.
  • Nephrologist. A nephrologist is a physician who specializes in the treatment of kidney
  • Neurologist. A doctor who specializes in diseases of the brain and nerve system.

The hematologist is frequently in charge of organizing this interdisciplinary team. Other health care professionals, such as physician assistants, nurses, social workers, pharmacists, counselors, nutritionists, and others, may be included in health care teams.

The following are descriptions of the most prevalent amyloidosis treatment options. The kind of amyloidosis, the type of underlying illness, probable side effects, and the patient’s preferences and general health all influence treatment options and recommendations. Symptoms and side effects will be treated as part of your treatment strategy. Symptoms and side effects will be treated as part of your treatment strategy. Take the time to learn about all of your treatment choices, and don’t be afraid to ask questions if you have any concerns. Also, discuss the aims of each therapy with your doctor and what you may expect during treatment. Find out more about choosing treatment choices.

Aminoglycosides are a class of drugs that are used to treat amyloidosis.

Controlling the symptoms of amyloidosis with medication is possible. Steroids have been proved to be effective and are frequently used in conjunction with other medications.


In AL amyloidosis, many of the same drugs that are used to treat cancer are used to halt the development of aberrant cells that generate the protein that leads to the production of amyloid. Chemotherapy is the use of medicines to kill cancerous cells by preventing them from growing and multiplying. The most prevalent application is for cancer treatment. Chemotherapy, on the other hand, can help with a variety of illnesses, including amyloidosis. A hematologist or a medical oncologist, a specialist who specializes in cancer treatment, can administer chemotherapy.

Chemotherapy is used to eliminate aberrant blood cells during amyloidosis treatment. Chemotherapy is commonly administered by an intravenous (IV) tube inserted into a vein with a needle, an injection under the skin, or a tablet or capsule ingested (orally).

Medications for the heart. If your heart is damaged, your doctor may prescribe blood thinners to prevent clots and medicines to regulate your heart rhythm. You may also need to limit your salt consumption and take medications that promote urine, which can help your heart and kidneys work more efficiently.

A chemotherapy regimen (schedule) generally consists of a specified number of cycles administered during a certain time period. A patient may be given a single medication or a mixture of drugs at the same time.

Cyclophosphamide (Cytoxan, Neosar) and melphalan (Alkeran), in combination with dexamethasone (various brand names) and prednisone, are two popular forms of chemotherapy used to treat amyloidosis (multiple brand names).

Chemotherapy treatment

Side-effects of chemotherapy:

Chemotherapy can cause nausea, vomiting, and tiredness, depending on the person and the amount administered. When they do occur, they are usually tolerable and disappear after therapy is completed. Long-term adverse effects of several kinds of chemotherapy might include harm to the bone marrow and other organs.

Anemia is frequent in patients with amyloidosis, particularly those who are taking melphalan-based chemotherapy. Anemia is a condition in which the body’s red blood cell count is abnormally low (RBCs).  RBCs carry hemoglobin, an iron-containing protein that transports oxygen throughout the body. When RBC levels are too low, portions of the body do not receive enough oxygen and are unable to function correctly. Anemia causes most individuals to feel weary or weak.

Therapy that is specific to the patient. 

Targeted therapy is a type of treatment that focuses on specific genes, proteins, or the tissue environment that contribute to the development of amyloidosis. This form of therapy stops aberrant cells from growing and spreading while reducing harm to good cells.

According to recent research, not all anomalies have the same objectives. Your doctor may perform tests to discover genes, proteins, and other variables in order to determine the best effective medication. When feasible, this allows doctors to better match each patient with the most appropriate treatment. Furthermore, several research projects are now underway to learn more about specific molecular targets and potential therapies aimed at them.

Anti-angiogenesis treatment, monoclonal antibodies, and proteasome inhibitors are some of the targeted therapeutics for amyloidosis. Immunosuppressive medicines. Lenalidomide (Revlimid), pomalidomide (Pomalyst), and thalidomide are all drugs in this family (Synovir, Thalomid).

Antibodies that are generated in a single cell are known as monoclonal antibodies. A form of targeted treatment is a monoclonal antibody. It identifies and binds to a particular protein in aberrant cells, but it has no effect on cells lacking that protein. Daratumumab (Darzalex) and elotuzumab are two drugs in this family (Empliciti). A different type of monoclonal antibody attacks the amyloid directly. Several of these antibodies are now being tested in clinical studies.

Inhibitors of the proteasome. Targeted treatment, such as proteasome inhibition, is a form of targeted therapy. Proteasomes are enzymes that break down proteins in cells, and the medicines in this class target them. Bortezomib (Velcade), Xiaomi (Ninlaro), and carfilzomib are all members of this class (Kyprolis).

Researchers are still researching these medicines to see how effectively they cure amyloidosis. The accumulation of amyloid proteins in tissues and organs is not reversed by these medicines. However, studies have indicated that a combination of bortezomib, dexamethasone, and melphalan may improve the function of amyloidosis-affected organs.

Targeted therapy’s side effects

Side effects from targeted treatment may differ from those associated with chemotherapy. Patients who get targeted therapy may experience issues with their skin, hair, nails, and/or eyes.


Organ transplantation may be utilized in the treatment of amyloidosis. Liver transplantation has been used to treat some types of hereditary amyloidosis. Kidney and heart transplants may also be successful. Kidney and heart transplants may also be successful. Organ transplantation, on the other hand, may not be acceptable for many patients, particularly those with high quantities of amyloid proteins accumulated in their organs. Discuss your choices with your doctor or a clinic that specializes in organ transplantation.

Bone marrow/stem cell transplant:

A bone marrow/stem cell transplant is a medical treatment in which the amyloid-producing plasma cells in the bone marrow are first killed by strong doses of chemotherapy and then replaced by highly specialized cells known as hematopoietic stem cells, which grow into the healthy bone marrow. Blood-forming cells present in the circulation and bone marrow are known as hematopoietic stem cells. Because the stem cells are taken from the circulation, rather than the actual bone marrow tissue, are generally implanted. This procedure is currently known as a peripheral blood stem cell transplant, as opposed to a bone marrow transplant.

Peripheral blood stem cell transplantation may be beneficial for certain patients with amyloidosis who have some organ function. The patient is given large doses of chemotherapy to kill blood cells that produce amyloid proteins during this treatment. After that, the patient is given healthy peripheral blood stem cells, which allow the body to begin producing healthy blood cells once more. High-dose chemotherapy and peripheral blood stem cell transplantation can considerably increase survival. Many patients, however, are unable to undergo this therapy because amyloid protein accumulation has interfered with the operation of other organs.

Before suggesting stem cell transplantation, doctors will discuss the risks with the patient and take into account a number of other variables, including the kind of disease, the outcomes of any prior treatments, and the patient’s age and general health.


 If amyloidosis has damaged your kidneys, you may need to start dialysis. A machine filters wastes, salts, and liquids from your blood on a regular basis in this process.

An organ transplant is a procedure in which a person receives If amyloid deposits have badly harmed your heart or kidneys, your doctor may recommend surgery to replace those organs. Because some forms of amyloid are generated in the liver, a liver transplant may be able to stop this process.

Receiving medical attention for symptoms and negative effects

Amyloidosis and its treatment frequently result in negative side effects. It is critical to alleviating a person’s symptoms and adverse effects as part of their treatment. Palliative care, also known as supportive care, involves assisting the patient with his or her physical, emotional, and social needs.

Palliative care refers to any therapy that aims to alleviate symptoms, improve quality of life, and provide support to patients and their families. Palliative care is available to anybody, regardless of age or illness. Palliative care works best when it is begun as soon as possible.

People frequently receive treatment for both amyloidosis and adverse effects at the same time.

Medication, nutritional therapy (see above), relaxation methods, emotional support, and other therapies are common palliative treatments. You may also get palliative therapies like chemotherapy, which is comparable to those used to cure the disease. 

Before beginning therapy, discuss the potential side effects of your individual treatment plan as well as palliative care alternatives with your health care team. If you have a problem during or after treatment, notify your doctor or another member of your health care team so it may be treated as soon as possible.

What if the treatment fails:

It is not always feasible to recover from amyloidosis. Advanced or terminal disease refers to a condition that cannot be treated or managed. This is a distressing diagnosis, and many individuals find it difficult to talk about the advanced disease. It is, nevertheless, critical to have open and honest discussions with your doctor and healthcare team about your thoughts, choices, and concerns. The healthcare team is there to assist patients and their families, and many team members have specialized skills, expertise, and knowledge to assist them. It is critical to ensure that a person is physically comfortable and pain-free.

Patients with the severe disease with a life expectancy of fewer than six months may benefit from hospice care, a kind of palliative care. Hospice care is meant to give individuals who are nearing the end of their lives the best quality of life possible. You and your family should consider where you would feel most at ease: at home, in the hospital, or in a hospice setting. For many families, living at home with nursing care and specific equipment is a viable option.


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