Past that, providers reported concerns about the blood supply being insufficient or about alloimmunisation, which is a problem caused by transfusing blood with different antigens.

“People with sickle cell disease develop antibodies,” said Lanzkron. “They often need rare blood. It's expensive. So, you know, I can't remember exactly what the cost is for each antigen match, but people get antigen-matched blood. That is standard of care to match for specific antigens that we know patients make antibodies toward, and that makes the blood more expensive.”

Based on what he’s seen at Terumo, though, Haubner believes the blood access issues are surmountable.

“What we do see is that the apheresis departments that have really good communication and really good operations and are really proactive in making sure they get the blood they need for the patients, they can generally get the blood they need,” he said. “It is still a challenge. It takes a long time, takes some planning. But yeah, all of the challenges can be overcome, I believe, with the current blood supply, with the way it's being managed now.”

The top two answers were concerns about the long-term effects of the procedure and concerns about insurance coverage. And under that, a number of more logistical concerns – scheduling, transportation, time off work or school.

Lanzkron’s organisation, the National Alliance of Sickle Cell Centers (NASCC), is focused on creating sickle cell centres of excellence where patients can be advised about their treatment.

“We try to help [patients] navigate around the barriers to care, but the first barrier that has to be overcome is being seen by a sickle cell expert at a sickle cell centre,” she said. “And it’s really amazing how many patients or people living with sickle cell disease do not have or do not go to one of the centres that they really should be going to for care.”

Doctors are sometimes dismissive of sickle cell cases they perceive to be mild, she said, and many patients believe that any haematologist is a sickle cell expert, which is far from the case.

A sickle cell expert can help assuage patients’ concerns about the process and direct them towards aRBCX, but it’s still a procedure that they have to find the time and transportation to undergo on a monthly basis.

“Transportation to clinic visits is a huge problem and not just in the city of Philadelphia,” Lanzkron said. “Just being able to manage to get to that appointment when it's a mile or two miles away, and being able to offer that resource, often working with community-based organisations, is really essential to help patients get to their care. But paying for that two-hour Uber trip back and forth to get an exchange transfusion just isn't something that a lot of community organisations and clinical groups can have the resources to provide to patients. So, it's a real struggle for those who live outside of major urban centres to get that kind of care.”

The fact that the condition affects primarily Black people is something of an elephant in the room when it comes to discussing these access challenges.

Black patients are more likely to have socio-economic-related access challenges like the ones described above. But it goes deeper than that. Part of the reason that generalists, and even haematologists, don’t have strong knowledge of sickle cell is that the research itself is underfunded.

“Unfortunately, we have to rely on a lot of consensus recommendations as opposed to evidence-based recommendations or guidelines because there just isn't that much of an evidence base, which is why people need to be seen by someone who does this a lot,” Lanzkron said.

Lanzkron’s team published a paper comparing both Federal and foundation-level funding for sickle cell with another rare disease, cystic fibrosis.

“When we looked at the federal funding based on per patient, per individual living with the disease in this country, there is a huge discrepancy,” she said. “The discrepancy grows even larger when you look at foundational funding […] Draw conclusions as you will about why there's that discrepancy, but – very different looking people.”

“Next steps, we're going to continue to investigate this with partners to better understand why there's an underutilisation,” he said. “We call it an underutilisation of red blood cell therapy relative to the medical need. And then to get beyond simply defining the factors; that's going to be part of our efforts to understand what we can do to educate and inform and be involved in any way that we can.”

Lanzkron believes the next step is to get a fuller understanding of the scope of the disease and the access challenges with better surveillance. That will allow organisations like hers to set up “hub and spoke” models to make sure everyone has access to treatment.

“Sometimes the Red Cross can provide that service to certain hospitals, so we could set up that infrastructure in a way that makes sense in places where there isn't that current infrastructure. But the first step is to understand where people are living who have the disease,” she said.