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Treatment

There are several treatment options available to people living with PNH, depending on the progress and severity of the disease. The only cure for PNH is a bone marrow transplant; other treatments are supportive only but have significant benefits including a reduction in the severity of symptoms.

Supportive

Most PNH patients receive one or several forms of the following treatment options.

Blood transfusions:

A patient may require a blood transfusion to replace blood cells lost through haemolysis. Usually only red blood cells are transfused, as the plasma and leukocytes (white blood cells) have been removed from whole blood. Some patients need occasional transfusions after an illness, injury or surgery and other patients may be transfusion-dependent, requiring regular transfusions every few months or weeks.

Prescription medication and supplements:

Eculizumab (pronounced E-coo-liz-oo-mab) is a monoclonal antibody that reduces haemolysis, considered by PNH experts to be the only treatment proven to effectively return patient life expectancy to that of a normal person. Eculizumab is administered intravenously every 14 days and although a relatively new treatment, it has been very successful for patients with moderate to severe PNH.  The treatment reduces symptoms significantly and in many cases haemoglobin levels are maintained without the need for blood transfusions.

Some patients are prescribed corticosteroids (such as prednisolone or dexamethasone) to reduce the rate of haemolysis by suppressing the body’s immune response, which in turn suppresses complement activation. Anticoagulants (for example, warfarin) may be prescribed to reduce the risk of a blood clot, known as thrombosis, particularly if a patient has already had a thrombotic event. Folic acid and iron supplements may be prescribed to assist the body’s production of blood cells to counteract the effects of haemolysis.

Natural therapies:

Many patients turn to complementary and natural therapies such as acupuncture, massage, meditation and drug-free pain management techniques to help ease the symptoms of PNH.

Natural therapies may help to reduce pain and stress. However, certain herbal medications can enhance some prescription drugs, particularly anticoagulants and others can exacerbate PNH symptoms, leading to life-threatening complications.

Since PNH is unpredictable and differs between individuals, it is important to discuss your condition in detail with your medical practitioner and treating specialist prior to taking any non-prescribed supplements.

Curative

Bone marrow / stem cell transplant:

A bone marrow transplant (BMT) is the only cure for PNH, however it involves significant risk and it is often difficult for a patient to obtain a well-matched donor. A BMT is considered a “last resort” for a PNH patient and is usually only performed where a patient has an excellent match (twins and siblings are best) or their PNH is immediately life-threatening and/or not responding to other treatments. A basic explanation of a bone marrow transplant is that a patient’s own bone marrow is replaced by a healthy matching donor’s bone marrow. The patient is usually treated with high dose chemotherapy to destroy the bone marrow tissue and often also anti-thymocyte globulin (ATG) to prevent rejection of the donor cells. Donor cells are collected directly from the bone marrow or blood stream.

There are many complications associated with bone marrow transplants, the most evident being Graft vs. Host Disease. There are many factors to take into account when considering a bone marrow transplant.

Disclaimer

PLEASE NOTE: This information is intended for general knowledge only and is not to be substituted for medical advice and its accuracy is not guaranteed. Please consult with your medical practitioner for further information regarding your personal circumstances.

This information has been prepared by PNHSAA Inc., is copyright protected and is used with their permission.