Building comprehensive care for patients with sickle cell disease

“Most of my fellowship experience was with these patients in the inpatient setting, where they are suffering,” she said. “It’s just a very difficult situation with what they are going through. This is a disease that really impacts a patient’s whole life, and not just from an aspect of physical health. It impacts their social life, emotional health, financial concerns, friendships and other relationships.”

Now, as a full-time faculty member at UW Madison and hematologist at UW Health, Nazeef specializes in treating sickle cell patients in the clinic. For her though, providing care means much more than just doling out medicines.

Sickle cell disease or SCD is a debilitating, inherited blood disorder that causes the red blood cells to acquire an abnormal shape and structure. Whereas normal blood cells are round, the blood cells of a patient with SCD are shaped like crescent moons or “sickles.” Because of their shape, they are unable to flow normally in blood vessels, often leading to clumping or blockages which cut off oxygen from reaching the tissues that need it.

In the short-term, this can cause immense bouts of pain, known as “crises.” But over the long-term, the lack of blood flow to the body’s critical organs can lead to permanent damage.

“Kidneys, lungs, heart, brain, bones, eyes, liver, you name it,” Nazeef said. Every organ is affected.”

Diagnosed at birth, SCD is relatively rare, affecting about 100,000 Americans, but who it affects is another story. According to the National Institutes of Health, the majority of individuals in the United States with SCD are of African ancestry or identify themselves as Black. Many others come from Hispanic, southern European, Middle Eastern, or Asian Indian backgrounds.

While SCD is a non-cancerous blood disorder, care for it is often administered by doctors who treat cancer patients, and there is some similarity at play. “Sickle cell disease affects patients in a very physical, emotional, psychological and social way, very closely to what you would see a cancer patient go through,” Nazeef said. “In an ideal setting, these patients need the same model of comprehensive care that cancer patients need.”

That said, patients haven’t always received what they’ve needed. Nazeef says historically, care for SCD has been uneven and many patients report negative clinical experiences. Because it’s a rarer – and often stigmatized – disease, patients may feel misunderstood, or even mistreated by their doctors, resulting in a lack of trust and even situations where patients forego care altogether.

To that end, Nazeef are colleagues are teaming up to build complete sickle cell care at UW Health and the UW Carbone Cancer Center, from diagnosis to end-of-life, care that really focuses on all of a patient’s needs.

From the moment a patient steps through the clinic doors, Nazeef wants them to know: there is a home for you here and we understand what you’re going through. “Our patients deserve higher quality, compassionate, comprehensive care,” she said. “We want to build a platform for them, a community really, where they feel comfortable seeking care.”

That’s important, because care for SCD is often a lifelong need. As it stands, the only curative therapy for the disease is a bone marrow or stem cell transplant at a young age. It requires a matched donor, and even then, the procedure can be incredibly risky. For many patients, it’s not a viable option.

As a result, SCD care primarily involves managing the complications of the disease. This means finding ways to manage pain and reducing hospitalizations for crises. It also means working to prevent and slow down any organ damage to prolong both the length and quality of a patient’s life.

Until a few years ago, there was only one FDA-approved medication to treat SCD symptoms, leaving doctors with few options to help their patients. “This disease has been around for a long time but maybe hasn’t received its due, in terms of awareness, attention and research,” Nazeef said. “But that’s thankfully improving now and changing.”

More recently, the FDA has approved three new drugs that can help manage various aspects and complications of SCD, and can be used in conjunction with one another.

There’s also hope on the horizon for a more permanent treatment, and it comes in the form of gene therapy. In preliminary studies, researchers collected stem cells from SCD patients and attempted to repair or modify the faulty gene, in an attempt to produce normal, functioning hemoglobin. Early data from these studies is promising, and Nazeef says she’ll be watching with great interest as future research is conducted.

In the meantime, she’ll keep helping her patients to the best of her ability, but says it must be a team effort. Comprehensive SCD care, she says, must include input from sickle cell RN case managers, social workers, health psychologists, pain treatment specialists, and other organ-specific specialists. The goal at UW is to make all of this the rule and not the exception in sickle cell care.

She also hopes to build more outreach and educational efforts, reaching patients where they are and reducing the stigma of the disease.

It all boils down to building trust – one experience at a time – and then building upon that foundation.

“I think that the one consistent message from our patients is really the fact that they want to be heard,” Nazeef said. “At the end of the day, even just having one understanding ear in the entire system, it makes such a big difference. Imagine what we can do if we all worked towards that goal.”