Introduction

Paroxysmal nocturnal haemoglobinurias (PNH) are described as a rare blood disorder. This disorder occurs when a part of the immune system attacks and damages the red blood cells and platelets. It is confirmed that around every 5 per 1 million people have PNH per year, and it typically affects both men and women between the ages of 30 to 40 years old.¹ Supportive care is essential for people with Paroxysmal nocturnal haemoglobinuria, such as iron supplementation in iron-deficient patients or transfusion support for severe anaemia.²

This article will highlight the importance of supportive therapies such as blood transfusions and iron management, as well as how these interventions help people with PNH. In addition, this article will touch on what PNH truly is and how it is presented.

Understanding PNH and Its Effects

Paroxysmal nocturnal haemoglobinuria is highlighted as a rare blood disease which causes the red blood cells to be damaged due to the immune system attacking them. What happens is that the surface of a person’s blood cells is missing a protein, which is called the complement system, which is a group of proteins that help white blood cells fight infections. Due to this being missing, it leaves the red blood cells vulnerable to the immune system.³

Various symptoms occur. This includes:

• Anaemia
• Haemoglobinuria (blood appears in urine)
• Thrombosis
• Fatigue
• Shortness of breath
• Kidney problems
• Stomach pains
• Back pains^1,4

As this condition destroys red blood cells in the body, if left untreated, a person may have anaemia as a symptom.¹ Anaemia is when a person has reduced amounts of red blood cells in the body, which means less oxygen is carried around the body. This can range from mild to severe; however, anaemia can be life-threatening.⁵ As PHN causes the breaking down of red blood cells, it is important to highlight the need for supportive therapies to combat this, specifically Iron supplementation and blood transfusions. Interventions that help manage the symptoms of Paroxysmal nocturnal hemoglobinuria.⁵

Role of Blood Transfusions in PNH

What is a blood transfusion?

A blood transfusion is described as a process where a person receives donated blood in a hospital or clinical setting, and it is used to help an individual if they are struggling with blood loss or have a condition that affects the amount of blood cells in the body.⁶ Blood transfusions not only help raise red blood cell levels but also help an individual feel healthier.

Why transfusions are used in PNH

Blood transfusions are used in PNH because it used to help manage symptoms such as severe anaemia for a period of time.⁷ It also helps to stabilise people with PNH when they are experiencing flare-ups or severe anaemia due to low blood counts.⁸

Benefits of transfusions

There are benefits to receiving a blood transfusion, such as:

• Increases blood flow to improve oxygen levels in the body
• Reduce fatigue
• Improved quality of life
• Management of severe blood loss^9,10

Limitations and considerations

There are a few considerations to take into account to blood transfusions, such as:

Allergic reactions:

Sometimes the immune system may react negatively to the donated blood given to it, which can cause an allergic reaction such as itchiness and hives. However, it is rare for someone to experience a severe allergic reaction to blood transfusions.⁶

Fever:

Some people may experience developing a fever during a blood transfusion, which is caused when the antibodies in the person’s blood react to the white blood cells in the donated blood.⁶

Temporary fix:

Blood transfusions are great for improving the blood count of an individual’s red blood cells; however, blood transfusions only temporarily manage anaemia in PHN, as they does not stop the continuous destruction of the blood cells in the body.⁷

Iron and PNH: Why management matters 

The iron connection

Iron is an essential part of making red blood cells. These cells carry oxygen around the body; without it, this can lead to iron deficiency anaemia. As PNH is caused by the loss of proteins in the blood, this causes symptoms such as haemolysis. This then causes iron loss due to elimination of haemoglobin (blood) in the urine. As a result, this makes people with PNH vulnerable to iron deficiency or makes diagnosed anaemia worse.^11,12

• Iron is a key part of red blood cells (haemoglobin)
• In PNH, constant destruction of red cells leads to iron being lost through urine
• This can cause iron deficiency, worsening anaemia

Iron deficiency management

Two ways to manage iron deficiency are by utilising iron supplementation or replacement. This is done by taking oral iron supplements, prescribed by a healthcare professional or by iron infusions, advised by a doctor.¹³

There are many good sources of iron through food, such as:

• Liver
• Red meat
• Beans
• Nuts
• Dried fruit
• Fortified breakfast cereals
• Soy bean flour¹²

It is important to note that although iron is great for the body, too much of it can cause an iron overload and toxicity.¹⁴

Iron overload in transfusion-dependent patients

Iron overload can happen when a person ingests too much iron or has repeated blood transfusions. This can lead to the body not have a natural way of getting rid of the excess iron. So, if the iron intake is unregulated, this can cause damage to key organs, such as the liver, heart, pancreas and endocrine glands.^15,16

Managing iron overload

There are various ways that iron overload is managed. This includes:

• Dietary changes: A professional or GP may request that a person may need to reduce iron intake, such as food or supplements
• Iron chelation therapy: This is a medication that is prescribed to help remove excess iron from the body
• Therapeutic phlebotomy: This is a procedure where a needle is used to remove some blood, which in result reduces iron levels¹⁷

How Blood Transfusions and Iron Management Work Together

People with Paroxysmal nocturnal haemoglobinuria need both iron supplementation/ management and blood transfusion to manage the condition. Their strategies work simultaneously to help. For example, people with PNH may have anaemia, which may require them to receive blood transfusions to increase their blood count and improve oxygen delivery in the body.^7-10 However, this tends to be temporary for people with PNH as their red blood cells are continuously being destroyed by the immune system, causing loss of iron through the urine. Although oral and intravenous iron supplements can help with this, they are sometimes not enough or can be too much, causing iron overload. This is why iron management may be introduced, such as chelation therapy to help remove extra iron.^7-17

Additionally, healthcare professionals will look at each patient’s symptoms and how many transfusions they’ve had and blood tests to ensure what needs to be put in place.^18,19

Living with PNH: Patient Perspective 

Although it is noted by patients that receiving a diagnosis has brought a sense of relief, living with PNH can have an emotional and physical impact. This includes

• Feeling distressed by delays in diagnosis
• Chronic fatigue
• Constant dependence on blood transfusions and complications due to travel, time and side effects
• Psychological stress due to symptoms and life expectancy²¹

Sometimes caregivers may experience a significant change to their life as they may need to reduce their work hours and days to tend to their family member with PNH.²¹

Although Paroxysmal nocturnal haemoglobinuria has no true cure, many people with it have nonetheless noted that the supportive therapies that they have received and the improvement over the years in introducing high-quality interventions over the past two years have helped. This includes reduced symptoms, increased energy after treatment and improved quality of life.²²

Summary 

To summarise, Paroxysmal nocturnal haemoglobinurias (PNH) are described as a rare blood disorder. This disorder occurs when a part of the immune system attacks and damages the red blood cells and platelets. Additionally, it has no cure.¹ Supportive therapies such as blood transfusions and iron management are essential interventions that are useful in managing PNH, and healthcare professionals will look at each patient’s symptoms and how many transfusions they’ve had and blood tests to ensure what needs to be put in place alongside other newer treatments.^7-19 Although some people with PNH may struggle with coping with their condition, it is possible to live a high quality of life. Especially with introducing high-quality interventions over the past two years that have helped.²² It is important that people understand supportive therapies such as blood transfusion and iron management, as knowledge and support are key in taking an active role in care.