June 19 is World Sickle Cell Day.
At 18 months, I was diagnosed with the most severe type of sickle cell anemia: HbSS. It’s a mutation that occurs when a child inherits two genes of hemoglobin S from each parent. The rigid red blood cells clump together, causing blockages in blood flow that lead to chronic anemia, episodic pain crises, and widespread organ damage.
With the disease progression, my symptoms became harder to manage as I aged. For years, I arduously searched for a cure and in 2019, I participated in a clinical research trial, receiving an allogeneic stem cell transplant from a matched sibling donor at the National Institute of Health (NIH) in Bethesda, Maryland.
A Stranger in My Own Body
With sickle cell behind me, I was optimistic and hopeful but the road to recovery was challenging. I suddenly experienced an identity crisis; questioning my new normal and struggling to adapt to life after sickle cell anemia.
Physically, I had the immune system of a two-year-old but the body of a middle-aged woman because this debilitating blood disease wreaked havoc on my tissues, bones, and organs. Mentally, I was crippled by an influx of opposing emotions (i.e. anger, fear, sadness, joy) and couldn’t escape survivor’s guilt. As I tried to push past fear, my friends called me brave. How ironic.
Curative treatment therapies like hematopoietic transplantation and gene therapy eliminate symptoms and stop disease progression. However, after transplantation, the patient becomes a carrier of the sickle cell trait (AS), meaning the disease can still be passed on to children. Education around the transplant experience has inspired a niche community of advocates; patients who are turning their pain into purpose.
As a sickle cell thought-leader, I’ve used my patient experience as an added advantage by consulting for healthcare technology platforms and genomic biotech companies. For researchers, advisory panels can simplify educational materials, refine trial designs, train members of the health team, and share insights that optimize the clinical trial experience for upcoming therapies.
The Power of Media
Growing up, I was truly disheartened by the depiction of sickle cell disease (SCD) in the media. Characters in movies and TV shows were given story arcs that purported stigma and misinformation among the general public, so my advocacy goal was to take control of the narrative through proactive engagement with the media.
When we hear the phrase “representation matters,” it is not limited to race, religion, or sexual orientation; it also applies to rare diseases and the disabled community. Diverse perspectives in the media have the power to transform prejudice into empathy and make the invisible visible.
In my professional opinion, showcasing gamuts of the patient experience using perceptive awareness campaigns can encourage the healthcare industry to see patients as people first and lean toward providing compassionate and respectful care.
What is Optimal Advocacy?
In the sickle cell community, advocacy is multi-dimensional with a focus placed on protecting the patient’s rights, improving communication between patients and providers, activating a network of support, and proposing policies that deliver high-quality care.
While working on a project with my mentor, Vence Bonham (JD), acting deputy director of the National Human Genome Research Institute, I got a lesson in real advocacy. He believes that optimal advocacy should empower key stakeholders at every level including patients, caregivers, providers, researchers, healthcare organizations, pharmaceutical companies, community groups and congressional committees.
For decades, the standard of care was breached by implicit bias and racist attitudes expressed by health care workers who ignorantly dehumanized people with sickle cell disease. Patients were labeled as drug seekers and accused of faking pain, resulting in inadequate treatment and more suffering. Effective advocacy should facilitate change and address the needs of the community.
As a facilitator in Bonham’s group, I met three extraordinary women who are advocates for change on every level – family, system, community and policy. Their exceptional leadership and grassroots efforts are actively combating the stigma of sickle cell disease across the country.
Wanda Whitten-Shurney, M.D., is the medical director of the Michigan chapter of the Sickle Cell Disease Association of America (SCDAA). She is a general pediatrician who coordinates the newborn hemoglobinopathy screening program for the Michigan Department of Health and Human Services. If a child is born with sickle cell disease, the SCDAA alerts the family, gets the child into pediatric care and coordinates penicillin prophylaxis.
Inspired by the legacy of her late father, Charles Whitten, M.D., founder of the National Association for Sickle Cell Disease, advocacy is very important to Whitten-Shurney.
“Our patients are dying and a lot of these deaths could have been prevented,” she says. “I think the reason patient care is so poor is because of the constant stigmatization of patients as drug-seekers. Doctors don’t pay enough attention to sickle cell patients so my role is to teach them how to advocate for better care.”
Whitten-Shurney is a big part of fighting the stigma of sickle cell disease in Michigan by getting patients out of the emergency room and focusing on holistic wellness. In the future, she wants to create an adult multidisciplinary care center – a statewide hub and spoke model – where patients can receive primary care, specialized sickle cell care, and psychosocial support.
Kim Smith-Whitley, M.D., is the executive vice president and head of research and development for Global Blood Therapeutics (GBT). She is a world-renowned hematologist and an active sickle cell advocate with over 30 years of research experience in pulmonary complications, transfusion-related complications, and health care utilization.
In 2019, Global Blood Therapeutics announced the approval of Oxbryta, a disease-modifying prescription medication that stops hemoglobin from sickling in adults and children over the age of four. Oxbryta is an oral therapy taken once daily that directly inhibits sickle hemoglobin polymerization, which is the root cause of SCD.
“As a pediatric hematologist, I saw the social inequities and stigma that impacted the sickle cell community because it mostly consists of Black and brown people,” she says. “I think we need to find a way to increase awareness so that we can draw companies that are interested in developing and delivering therapies that truly address the underlying cause of sickle cell, thereby improving survival.” She believes that raising the voice of individuals with sickle cell will empower them to advocate for high-quality care and shared decision-making.
Teonna Woolford is the CEO of the Sickle Cell Reproductive Health Education Directive (SCRED), the first 501(c)3 non-profit that focuses on reproductive health. The organization offers fertility preservation grants to sickle cell patients undergoing curative therapies.
“Some people think that given the complications of sickle cell disease, women shouldn’t be encouraged to conceive. Sickle cell patients are whole and to honor that wholeness, our reproductive health must be prioritized,” she says.
SCRED provides education on reproductive health concerns, establishes standards for effective and high-quality reproductive health care, and advocates for policies that can improve access to comprehensive care. Woolford’s passion for advocacy has inspired discussions with world leaders like former First Lady Michelle Obama and the late congressman John Lewis.
Advocates play a critical role in convincing lawmakers and health leaders to reverse policies and allocate resources to fund treatment advancements for SCD patients globally. Whether you’re a physician, caregiver, or patient, a seat at the table is a rare opportunity to effect change and improve healthcare.