Question 1

When was PNH first reported?

Accepted Answer

PNH is generally believed to be first described in 1911 and was formally recognized as a disease in 1928. Before this, there have been observations similar to PNH that have been described by physicians in the 19th century.

Question 2

What does PNH stand for?

Accepted Answer

PNH stands for Paroxysmal Nocturnal Hemoglobinuria. Paroxysmal means sudden and irregular, Nocturnal means at night, and Hemoglobinuria means hemoglobin in urine making it appear dark. However, our understanding of PNH has advanced and we now know PNH does not only occur at night.

Question 3

Does PNH only occur at night?

Accepted Answer

No. Early observations showed people passed dark urine at night or early morning, but we now know this is no longer accurate. People can be impacted at different times of the day and not everyone will experience dark urine. Dark urine occurs when the immune system breaks down red blood cells through hemolysis.

Question 4

How do people get PNH?

Accepted Answer

PNH is a rare blood disease that is acquired, not inherited. This means PNH may appear over the course of a person's lifetime and is not passed down through genes.

Question 5

How is PNH diagnosed?

Accepted Answer

To diagnose PNH, your doctor may use a range of tests, including blood tests, X-rays, CT scans, ultrasounds and urine tests. You may be referred to a hematologist or PNH specialist.

Question 6

Who treats PNH?

Accepted Answer

Your family doctor may refer you to a hematologist who has experience in managing PNH. These doctors are also known as PNH specialists.

Question 7

Can PNH be cured?

Accepted Answer

PNH is considered a chronic disease. The only potential cure is a bone marrow transplant, but well-matched donors are difficult to find and the procedure carries many risks. Other strategies may be available to ease symptoms and prevent problems.

Question 8

What are my treatment options if I'm having a sub-optimal treatment response?

Accepted Answer

If you're having a sub-optimal response, your doctor may alter your treatment plan. They may change your current dose or consider other prescription medications including switching to another complement inhibitor or providing add-on therapy.

Question 9

What is a sub-optimal treatment response?

Accepted Answer

A sub-optimal treatment response is when your PNH is not being controlled adequately by your current treatment. This can lead to ongoing hemolysis or a lack of hemolytic control, which can lead to an array of symptoms.

Question 10

How do I know if I am having a sub-optimal treatment response?

Accepted Answer

When you're having a sub-optimal treatment response, you may experience symptoms that either reappear or happen persistently during treatment. The main tell-tale signs are fatigue, anemia or the need for blood transfusions.

Question 11

How do I talk about my suspected sub-optimal treatment response with my doctor?

Accepted Answer

It is important to have an open conversation with your doctor about your disease. Talk to your doctor about how you really feel, what your treatment goals are and why your treatment should be re-evaluated.

Frequently asked questions about PNH

Frequently asked questions about PNH

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About PNH

Diagnosis and treatment

Recognizing a sub-optimal treatment response