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It has been announced this week that deferasirox, an iron chelation treatment for iron overload due to frequent blood transfusions has been approved for routine use for low or intermediate risk Myelodysplastic Syndrome (MDS) patients in Scotland.
Deferasirox was recently appraised by the Scottish Medicines Consortium (SMC), which is the appraisal body that assess what drugs and health technologies are accessible to NHS patients in Scotland. The appraisal was an opportunity for patient organisations to submit the patient perspective of what it is like to live with MDS, as well as to highlight the need for more convenient treatment options for iron chelation therapy. Following the consideration of evidence put forward by Novartis, the manufacturer company and the collaborative submission by Leukaemia CARE and MDS UK, the oral iron chelation therapy will soon be available for Scottish MDS patients that meet the criteria of the recommendation.
Myelodysplastic syndromes (MDS) are a group of diseases where the production of blood cells by the bone marrow doesn’t work properly. The bone marrow in a patient with MDS is usually more active than normal but there are fewer blood cells in the circulation. This is because the stem cells become unable to produce a high enough quantity and/or are poor quality and so they are destroyed before they leave the bone marrow. This disruption of the cells causes a range of symptoms including feeling tired, weak and breathless, severe fatigue, night sweats, frequent infections, itchy skin, bone pain and unusual bruising and bleeding.
There is no known cure for MDS other than a stem cell transplant. Not all patients with MDS will receive active treatment immediately after diagnosis but most patients will require treatment at some point to control and improve their symptoms. The majority of patients, for example, will need to have frequent blood transfusions. Many MDS patients that receive these blood transfusions will experience iron overload because the body is unable to process all of the iron contained in each pint of blood they receive. Iron chelation therapy is the process of removing the excess iron in the body which is necessary to avoid high ferritin levels and their potentially toxic effects.
The standard treatment for iron chelation currently used is deferoxamine. It is administered by subcutaneous injection. For the patient, this involves inserting a needle (usually into the stomach) that is connected to an infusion pump, which then delivers the chelation over a period of 8–12 hours each day. Because the administration of the chelation takes such a long time, patients usually have to use the pump overnight or wear it underneath clothes for eight hours during the day. This current method is generally associated with an inferior quality of life, being painful, uncomfortable and inconvenient. Other wider issues cited by some patients included the emotional impact of having to use a pump – some patients find it necessary to receive emotional support to address this.
Deferasirox, marketed as ExJade®, is an oral iron chelation therapy – in the form of a dispersible tablet. Patients describe the oral administration for the iron chelation treatment more practical than the currently available treatment option, enabling them to travel, continue to work, be more active or carry out their daily routines as normal.
The final recommendation of deferasirox for MDS patients was made following the SMC’s Patient and Clinician Engagement (PACE) meeting, a part of the appraisal process where patient organisations and patients had the opportunity to further explain to the committee meeting what it is like to live with MDS, the patient experience of using the existing method of chelation therapy, as well as the benefits of the therapy under review.
As specified in Leukaemia CARE and MDS UK’s joint written submission to the SMC, iron chelation with deferasirox is beneficial in patients with MDS, successfully lowering ferritin levels, demonstrating improvements to overall survival and significantly improving the ease of administration and quality of life of patients, who are already having to cope with the emotional impact of living with a blood cancer.
Zack Pemberton-Whiteley, Leukaemia CARE’s Head of Campaigns and Advocacy, comments:
“The decision by the Scottish Medicine Consortium (SMC) to recommend deferasirox as an effective and more convenient iron chelation therapy is a welcome one. Low or intermediate risk MDS patients in Scotland will soon have access to this effective chelation therapy – hopefully improving their quality of life and relieving some of the burden of living with MDS.”