Leukaemia Care successfully campaigns for access to a new treatment for patients with cGvHD in Scotland

Last updated: 10th July 2023

Leukaemia Care today welcomes the news that the Scottish Medicines Consortium (SMC) has approved the use of belumosudil (Rezurock) on the NHS in Scotland. It is available for a period of 3 years initially, whilst more data on how well it works is collected. It will be used for patients aged 12 and over with chronic graft-versus-host disease (cGvHD) who have received at least two prior lines of systemic (whole body) treatment.

Once more data on the treatment has been collected, the use of belumosudil will be reviewed. We will report if access to the treatment changes in the future. 

How was Leukaemia Care involved in the decision to approve this treatment?

Leukaemia Care worked alongside the charity Anthony Nolan to gather data, patient experience and put together a written response to the SMC before making their decision. We focused on why belumosudil could be beneficial for patients when compared to the existing treatments currently available in Scotland.

We highlighted that many patients that are due to receive their third therapy for GVHD are likely to be battling multiple, significant side effects already, such as severe inflammation of their eyes and skin.

As such, we argued that a quick and effective third line therapy is needed to reduce the most significant side effects and control the impact and severity of GvHD. As some patients currently continue to be referred onwards to 4th and 5th line therapies, belumosudil holds the potential of resolving or managing a patient’s advanced chronic GvHD without further treatment interventions.

What is Graft versus Host Disease?

Graft versus Host Disease (GvHD) happens when a particular type of white blood cell, called T cells, in the donated stem cells or bone marrow begin to attack the patients’ own body cells. This happens because the donated cells (the graft) see the patients’ own body cells (the host) as foreign and start to attack them.

Chronic GvHD tends to develop more slowly than acute GVHD. Chronic GvHD symptoms can often include skin rashes, mouth sores, dry eyes, liver inflammation, the development of scar tissue in the skin and joints, and damage to the lungs.

Although many patients with chronic GvHD experience mild to moderate symptoms, a small number of patients can develop more severe symptoms. 

Why is the approval of this treatment important for patients?

Before the approval of belumosudil, the only other treatment recommended in Scotland for managing chronic GvHD for patients who have received two prior lines of therapy was Extracorporeal Photopheresis (ECP). ECP involves removing blood from the patient, treating the blood with chemicals to remove the white blood cells causing the GvHD, and then returning this blood to the patient. 

ECP can take up to 4 hours and can also require patients to attend hospital twice a week every two weeks. This process can last 6 months or longer and with only 2 health centres in Scotland that can provide this treatment, patients may have to travel long distances to receive ECP. 

Belumosudil is a new treatment that is likely to improve patients’ experience of treatment and quality of life, due to its convenience and the option to possibly take it at home.

Belumosudil comes in the form of a tablet that patients take once a day. Patients we spoke to expressed a preference for oral ‘at home’ treatments that allowed them to spend more time away from the  hospital. This treatment could be of benefit to patients’ well-being by allowing them to spend more time with family, friends, and continue a normal social life. 

This new treatment could also remove the financial burden on patients and their families, who have to take time off work to travel with the patient to hospital appointments if they are receiving ECP.

Clinical trials have suggested that belumosudil is well tolerated in comparison to existing treatments available in Scotland. Patients we spoke to highlighted the extremely unpleasant side effects of many of the currently available treatments and wanted new treatments that have less serious and unpleasant side effects.