Crizanlizumab (Adakveo, Novartis) for the Prevention of Pain Crises in Sickle Cell Disease

Introduction

The purpose of this leaflet is to provide our patients who have sickle cell disease (SCD) with a brief background of the disease and a guide to the new treatment available with the use of Crizanlizumab. In conjunction with discussions with your specialist team, this leaflet aims to give you some understanding to help you decide on the best treatments available to manage pain crisis in sickle cell disease.

What is sickle cell disease?

Inherited from both parents, sickle cell disease (SCD) is a health condition that affects the red blood cells. SCD is particularly common in people with an African or Caribbean family background but it can also affect anyone.

Healthy red blood cells are round, disc shape, and flexible that can move through small blood vessels to carry oxygen to all parts of the body. In someone who has SCD, the red blood cells become hard, rigid, sticky and look like a C-shape called a “sickle” – which is why it is called “sickle” cell disease. These affected red blood cells also do not live as long as healthy blood cells.

SCD can cause serious health issues throughout the body because it can reduce the flow of blood or block blood vessels – when this happens a patient has a sickle cell crisis or vaso-occlusive crisis (VOC). These are painful and can cause a debilitating condition that has a huge impact on patients’ long term health and quality of life. The frequency and severity of VOC is unpredictable and each episode can take days or weeks to recover from, and can sometimes require hospitalisation. They may also cause blood clotting which increases the risk of stroke (opens in a new tab) or organ damage.

Current treatments to prevent sickle cell crisis include a tablet called hydroxycarbamide/ hydroxyurea (HC/HU) and regular blood transfusions or exchange transfusions.

What is Crizanlizumab?

Crizanlizumab (Adakveo, Novartis) is claimed to be a “revolutionary” life-changing drug treatment recommended by NICE as another treatment option for preventing recurrent sickle cell crises in people aged 16 or over. Clinical evidence suggests that people treated with crizanlizumab have significantly fewer sickle cell crises in a year than those receiving other standard treatment options though at the moment there is high uncertainty about its long-term effectiveness.

Crizanlizumab works by binding to a protein in the blood called P-selectin. This protein is what causes the blood cells to stick together. Blocking the P-selectin prevents blood cell restrictions and therefore improves blood supply throughout the body. This also lowers the risk of pain crisis needing a hospital stay and other severe complications of sickle cell disease such as stroke and organ damage.

How it is given?

Crizanlizumab can be given as an add-on therapy to hydroxycarbamide/hydroxyurea or can be taken on its own if HC/HU is inappropriate or inadequate. It comes as a liquid solution mixed in compatible fluid and given as an intravenous (IV) (opens in a new tab) drip into your arm. The infusion will take about 30 minutes but you will be monitored for an hour after your dose. Your doctor will prescribe your dosage depending on several factors including your body weight and other medical conditions that you may have.

When you start treatment with crizanlizumab you will get one dose, followed by a second dose two weeks later. After that, you will get a dose once every four weeks.

Your medication will be administered in a day case infusion unit facility with staff specifically trained to give these infusions. Your appointments will be given to you by your haematology team.

What are the common side effects?

The most frequently reported adverse reactions (≥10%) in patients treated with crizanlizumab were nausea (18%), arthralgia (joint pain) (18%), back pain (15%), abdominal pain (12%), and pyrexia (fever) (11%). Most of these side effects should go away within a few days or a couple of weeks. But if they become more severe or don’t go away, talk with your doctor, nurse or pharmacist.

It is not known how many people may have experienced an allergic reaction in the clinical study (opens in a new tab) of crizanlizumab. Call your doctor right away if you have an allergic reaction as the reaction could become severe. Call 999 or your local emergency number if your symptoms feel life threatening or you think you’re having a medical emergency.

Special considerations

If you’re pregnant or planning a pregnancy, talk with your doctor before starting the treatment. If you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control (opens in a new tab) needs while you’re on this treatment.

It is not known if this treatment is safe to use during pregnancy. Because of this, you should only use crizanlizumab if the benefits are greater than the possible risk of harm to your baby. It is also important to note that, in rare cases, sickle cell disease may raise the risk of miscarriage. If you have any concerns about this, please speak to your doctor.

It’s also unknown whether the treatment is safe to use while breastfeeding.It has not yet been studied to see if it passes into breast milk. If you are breastfeeding, consider the benefit versus the risks of harm to your baby while taking the drug. Talk with your doctor about the benefits and risks of being on this treatment while breastfeeding.

There are no known interactions between crizanlizumab and alcohol. Alcohol, however, is a trigger (opens in a new tab) of sickle cell disease (opens in a new tab) and drinking alcohol may raise your risk of pain crisis related to sickle cell disease.

References

• National institute for health and care excellence (opens in a new tab)
• NHS website (opens in a new tab)
• Sickle Cell Society (opens in a new tab)

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