Sickle cell disease can have a devastating effect on the health of patients. The disease can cause chronic health conditions, including anemia, organ damage, and severe pain events. If left untreated, conditions are likely to worsen and lead to strokes or even death.
Too often, sickle cell disease patients are unable to access the necessary treatments. Many patients live far from the medical facilities and cannot afford to travel. Often, rare-matched blood is required for red cell exchange in patients to manage their disease, and there is not a sufficient supply of this blood or the donors who give it. Furthermore, some towns and cities in the US lack specialist treatment centers, alongside low reimbursements available.
A further key issue with sickle cell treatments is the severe lack of blood donors in the US. Despite an estimated 62% of the population being eligible to give blood, just 3% actually do.1 Therefore, there is a drive to raise awareness about the different ways that people and organizations can help.
The difference made by apheresis in sickle cell disease patients
One treatment option proven to make that makes a difference in sickle cell patients is apheresis, which is an approved and proven treatment option that can make a significant difference for sickle cell patients. This specialized blood transfusion uses a machine to selectively remove red blood cells, replacing them simultaneously with healthy donor cells. Procedure intervals vary. Some patients benefit from less frequent treatments, while others may require weekly sessions to maintain optimal health.
However, apheresis is typically only offered in extreme cases to patients who are already known by healthcare services. An apheresis procedure requires trained medical personnel to administer, meaning more resources and expenses are involved. These procedures are lengthy.
Simple blood transfusions are more common, but they’re not always effective and can come with side-effects. In these procedures, healthy red blood cells are added without removing unhealthy ones, often requiring frequent treatments that can limit patients’ lifestyles. Each session can take two to four hours in the hospital. Regular transfusions also increase the risk of iron overload, posing serious long-term health risks.
“Not all the patients coming to the facilities are local,” explains Doreen Condon, gene therapy apheresis expert from Bluebird Bio. “They’re coming in from a fair distance across state borders. So, if they have to return for another whole cycle, that’s a big deal for everybody.”
Another barrier for patients is the low availability of apheresis units at medical facilities in some states. There can also be a lack of education from medical staff not only on therapy but using peripheral vascular access and optimizing the use of blood.
“Not every hospital has an apheresis unit, and, unfortunately, not every hematologist knows about gene therapy, let alone about red cell exchange,” adds Condon. “
Just even having the knowledge in the community is going to have a huge impact on how the patient is treated and what treatments are offered. Because if the hospital doesn’t have red cell exchange, they want to send them to another hospital where they can get red cell exchange.”
The importance of patient advocacy groups in raising awareness on sickle cell disease
For someone diagnosed with sickle cell disease, it can be hugely beneficial to speak with other patients who have had similar experiences and undergone successful treatments to understand what the process involves and the possible results.
“They want to talk to a patient that’s been through it,” adds Condon. “Whenever we’re able to connect a patient to another patient that that ends up being super valuable.”
At an online event in September 2024 to coincide with Sickle Cell Awareness Month, members of patient advocacy groups shared their stories about the challenges of living with the disease and the difficulties in accessing effective blood-based treatments – with some patients even joining the event from hospitals.
Group members stated they would like to create a national framework or coalition that brings together a multidisciplinary group of stakeholders to address the multiple issues facing the sickle cell community. Such a coalition could investigate opportunities to utilize existing hemophilia treatment centers or day hospitals, potentially expanding access points for sickle cell patients.
Groups would seek to develop comprehensive educational materials and resources to inform patients, providers, and the public about treatment options such as apheresis, the benefits, and how to access them.
The future of sickle cell disease treatments
Patients said they wished to work alongside Terumo Blood and Cell Technologies (TBCT) and industry partners to leverage their influence and resources in support of the patient organizations in the sickle cell space.
Terumo BCT’s Spectra Optia® Apheresis System is used for sickle cell treatments. Uses in the UK were estimated to save the National Health Service £13m a year compared with other treatments such as red blood cell transfusions or top-ups.2
In other divisions, Terumo BCT is also involved with machines that support the development of cell and gene therapies, which could transform the future of sickle cell treatments. There has been some notable progress in recent years. In December 2023, it was announced that the US Food and Drug Administration had approved two breakthrough treatments.3
Lyfgenia and Casgevy are the first gene therapies available for treating sickle cell disease in patients aged 12 years and above. While the short-term costs would likely be higher for patients receiving treatments, there could be significant savings in the long term due to the dramatic reduction in therapy sessions required as Lyfgenia and Casgevy are both one-time therapies.
Treatment options such as these provide hope for sickle cell patients in the future, but the first step is increasing awareness of the disease.
To learn more about ways to increase access to cell and gene therapies, download the document below.
