PNHSource

Palliative Therapies to Consider

Some of the palliative treatment options that are currently being used to help ease the symptoms of PNH are listed below. Each comes with its own set of pros and cons to help you and your doctor decide which options are best for you.

• Transfusions
• Steroid hormones
• Androgen hormones
• Anticoagulants
• Supplements

Transfusions

• Recommended during times of severe hemoglobin (brownish-red substance in healthy red blood cells that carries oxygen to other cells) depletion when your body cannot generate enough new cells to make up for those lost to hemolysis¹
• May be used on a periodic basis when your hemoglobin level is steadily decreasing¹

Pros: Transfusions have become routine procedures and their associated side effects may be less than those of chronic steroid use.¹

Cons: Frequent transfusions increase the risk of transfusion-related health problems.¹

Steroid hormones

• May be used to treat hemolysis (the destruction of red blood cells) in patients with PNH

Pros: Steroids may reduce the rate at which red blood cells are destroyed.¹

Cons: High doses are required to produce a benefit. Continual use of steroids has many potential side effects such as cataracts, osteoporosis, and diabetes.^2,3

Androgen hormones

• A type of steroid medication used either alone or in combination with other steroids
• Have been used successfully to treat the anemia associated with PNH, though some believe these are of little value^1,2,4

Pros: Androgens may work quickly to offset the effects of hemolysis (the destruction of red blood cells)^1,2

Cons: They can potentially affect the liver. In men, androgens may cause an enlargement of the prostate. In women, they may cause some masculine traits, such as increased body and facial hair.²

Anticoagulants (Blood thinners)

• Your doctor may want to put you on medication to either reduce the risk of getting blood clots (thromboses) or may need to dissolve a clot when it happens
• Medicines such as heparin or enoxaparin sodium injection are anticlotting medications (anticoagulants) — agents that can be used to treat blood clots (thromboses) when they occur^2,5
• Warfarin tablets are a type of anticlotting medication (anticoagulant) that your doctor may prescribe for you to take on a regular basis to prevent clots in the future^2,5

Pros: Using an anticlotting medication (anticoagulant) may help prevent blood clots (thromboses), the leading cause of life-threatening complications in people with PNH.^1,2

Cons: Warfarin may interact with some foods, alcohol, vitamins, herbal supplements and certain drugs, such as corticosteroids and androgen derivatives that are also prescribed to ease the signs and symptoms of PNH.⁵ Warfarin may also cause serious bleeding problems, and death of skin tissue. Regular blood tests to monitor your condition are needed to ensure your blood levels are in the target range.⁶

Supplements

• Folic acid and iron supplements may help bone marrow replace red blood cells destroyed by hemolysis^1,5

Pros: Supplements may help people with PNH who often lose iron when passing urine, resulting in iron deficiency.¹

Cons: Taking iron supplements could result in iron overload, a dangerous condition. Iron levels should be monitored on a continual basis.