Leukaemia Care successfully campaigns for equal access in Scotland to a new stem cell transplant conditioning treatment for leukaemia patients

Last updated: 12th June 2023

Today, Leukaemia Care welcomes the decision by the SMC to approve treosulfan in combination with fludarabine for use on the NHS in Scotland. Treosulfan will be used as a conditioning treatment before an allogeneic haematopoietic stem cell transplant (alloHSCT). This treatment is for leukaemia patients, as well as patients with other malignant diseases, who would benefit from a reduced-intensity regimen.

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 treosulfan with fludarabine could be beneficial to patients and have a positive impact on their quality of life when compared to other reduced-intensity conditioning treatments (RICs), such as the commonly used busulfan with fludarabine.

It was important to emphasise that there was an unmet need in Scotland for conditioning treatments that are effective while also having a lower level of toxicity and more manageable side effects. People who are unable to have traditional conditioning treatment need something that could offer improved quality of life and improved survival.

What is alloHSCT? And what is conditioning treatment for?

Allogeneic haematopoietic stem cell transplantation (alloHSCT) is used to treat many haematological disorders, such as leukaemias, lymphomas and myeloma.  However, alloHSCT has many risks for the patient, regardless of their condition, and is only considered as a treatment option for patients who are fit enough to withstand the side effects and whose disease is unlikely to be cured with chemotherapy alone.

Before a patient undergoes a stem cell transplant, they will go through conditioning treatment. This is where their immune system is treated and prepared to make way for the new cells from the stem cell transplant. Conditioning treatment is typically given during the week before a patient receives a transplant.

Why is the approval of this treatment important for patients in Scotland?

Existing conditioning treatments in Scotland are known to cause significant side effects, which can be extremely distressing for patients and their families. They can unfortunately lead to what is known as “treatment-related mortality” which is when patients do not survive the effects of the treatment.

Treosulfan with fludarabine would provide an alternative conditioning treatment and the evidence to date indicates that it could offer a better side-effect profile than the current RIC treatments available in Scotland. Patients treated with treosulfan would be expected to have less treatment-related toxicity, experience fewer long-term side effects, and have improved survival after alloHSCT compared to those treated with a busulfan-based regimen.

We are very pleased that patients in Scotland now have equal access to treosulfan with fludarabine as those in England, Wales, and Northern Ireland. 

How will this new treatment be given to patients?

Treosulfan is given as a drip into a vein, lasting 2 hours. Patients will receive treosulfan once a day for 3 days before their alloHSCT. Fludarabine is also given as a drip once a day for 5 days before alloHSCTs. The dose of treosulfan depends on the condition for which the alloHSCT is needed. Other factors such as the patient’s weight and height are also considered.