She’s usually pretty shy, but when someone mentions dance videos, 12-year-old Anaiya Buchanan lights up. She adores busting a move to music and has posted over 340 videos on TikTok, the social media platform that’s all the rage with tweens.
Anaiya’s feisty side has helped her withstand years of painful and life-threatening complications from severe sickle cell disease.
The diagnosis came when she was just 1 week old. Monthly blood transfusions over the years lessened her symptoms and reduced her risk of stroke but led to a buildup of iron in her organs, putting her at risk for liver and heart damage.
Anaiya is one of the first patients to take part in the Sickle Cell Disease Hematopoietic Cell Transplantation (SCDHCT) study at St. Jude Children’s Research Hospital. This clinical trial aims to cure children with severe sickle cell disease. Researchers hope to achieve high success rates with a transplantation approach that uses a gentler chemotherapy and radiation conditioning regimen than standard transplants. The goal is to maximize the odds for a cure while reducing side effects and subsequent health issues.
Gotta dance: How does an aspiring video star generate social media posts while undergoing a bone marrow transplant? By enlisting the help of her care team, of course. Joining Anaiya in the fun are (from left) Rebecca Elrod, RN; Katelyn Park, RN; Christin Mays, RN; and Jackie Hopson, PCA.
Sickle cell disease is the most common inherited blood disorder in the U.S., affecting an estimated 100,000 people. About 1 in 365 Black babies in the U.S. is born with the condition. Decades ago, half of all children with the disease did not live past age 20, and most did not survive to 50. Today, with early diagnosis and innovative treatments, most live to 50 or beyond.
Sickle cell disease causes normally round or donut-shaped red blood cells to take on a crescent or sickle shape. This hampers their ability to move through the body and deliver oxygen to tissues. When sickled cells block blood flow to joints, organs or bones, the result is a pain episode or crisis. If blood supply to the brain is compromised, it could result in a stroke. People with sickle cell disease are also at a higher risk of developing infections because many have their spleen removed or damaged early in life.
From her earliest days, Anaiya often experienced pain crises and pneumonia. She had a life-threatening concentration of sickle cell blood in her spleen and required hospitalization when she was 9 months old. By the time she was 2, St. Jude doctors discovered the velocity of blood flow to her brain was elevated, meaning she was at high risk of having a stroke. Anaiya visited St. Jude every six weeks for blood transfusions to replace her sickled blood with normal blood from donors to reduce her pain episodes and the risk of stroke.
Stem cell transplantation, also known as bone marrow transplantation, is the only known cure for sickle cell disease, with about 95% of children treated achieving long-term cure. However, transplantation requires a suitably matched donor and carries risks of long-term complications. Until recently, most transplants used a high dose of chemotherapy drugs, also called a conditioning regimen, to prepare patients to accept donor stem cells and prevent rejection.
“Typical pre-transplant conditioning can cause a lot of organ injury and longer-term problems, even for patients who tolerate the transplant procedure well,” says Akshay Sharma, MBBS, who leads the study.
Legacy of firsts
St. Jude has been researching sickle cell disease since before the hospital opened in 1962. One of the institution’s earliest clinicians, Lemuel Diggs, MD, received the institution’s first grant, which was earmarked to study the disorder. A St. Jude patient was also the first in the world to be cured of sickle cell disease with a bone marrow transplant. When the 8-year-old girl underwent a transplant for acute myeloid leukemia in 1983, she was also cured of her sickle cell disease.
Since then, most clinicians have used high-intensity chemotherapy before sickle cell disease transplants, just like transplants done to treat patients with leukemia. By the early 2000s, however, investigators started exploring less-intense regimens, testing chemotherapy doses that were 70 to 90% of standard doses.
About six years ago, sickle cell disease researchers at the National Institutes of Health (NIH) discovered that no up-front chemotherapy-based conditioning was required to cure some individuals with sickle cell disease who received fully matched transplants from their siblings.
“That made transplant much more appealing to patients, but it had a problem: As conditioning intensity decreases, the risk of graft rejection increases,” Sharma says. “So, we increased the conditioning intensity by adding just a little bit of chemotherapy to prevent rejection.”
The SCDHCT clinical trial aims to enroll 40 patients under age 25 who have severe sickle cell disease — 20 individuals with fully matched sibling donors and 20 with half-matched, or haploidentical, donors.
Not every patient has a brother or sister who is a perfect match, so the ability to use a parent (a half-matched donor) is an exciting option. The SCDHCT clinical trial is also appealing to many patients because it offers a gentler treatment regimen than has been used in the past.
“Patients in our study receive reduced-intensity conditioning, than what is used in standard transplants,” Sharma explains. “We hope this approach will successfully cure sickle cell disease with much less short- and long-term toxicity.”
St. Jude has a long-standing commitment to improving the lives of children with sickle cell disease. The nurse-to-patient ratio of 1:3 for treatments to manage symptoms and 1:1 for patients in the ICU after transplant is unmatched by most other hospitals.
Physician Assistant Darla Pickett oversees chronic blood transfusions for St. Jude patients with sickle cell disease. She provided care to Anaiya for five years leading up to the transplant.
“Medication helped to lower Anaiya’s iron levels but then caused some kidney issues,” Pickett says. “Chronic transfusions were helpful to alleviate her symptoms, but they were never going to be a cure.”
Sonja Carruthers, Anaiya’s grandmother and guardian, heard about SCDHCT during a talk on sickle cell disease at the hospital.
“I signed her up for the study right away,” Carruthers says. “Transplant was her best chance to be cured and to leave behind transfusions and their side effects.”
Like Anaiya’s dance moves, transplantation involves carefully executed, well-timed steps that begin a couple of months in advance. First, the patient receives a two-drug preconditioning treatment by mouth to reduce the risk of rejecting the donor stem cells, known as a graft.
Next, the individual receives reduced-intensity chemotherapy and low-dose total body radiation. This prepares the patient to receive the donor’s stem cells on transplant day.
After the transplant, the patient receives medications to help prevent graft-versus-host disease and graft rejection.
Anaiya received a haploidentical transplant from her mom in August of 2020. She was the first person in the clinical trial to receive a half-matched transplant, and her treatment team says she has responded well to the treatment.
Nurse practitioner Amanda Kennedy met Anaiya a couple of months before her transplant and will continue to care for her during the next several years. The bond they have developed is strong.
“Anaiya didn’t want to talk with me at first. She’s very picky about who she lets in,” Kennedy says. “But once she does, that girl’s got attitude. She’s a little stubborn and sassy, in all the right ways.
“Anaiya’s a fighter,” Kennedy continues. “She lost her hair, had nausea, pain and other issues, but she bounced back quickly, and now she wants to give back. She spoke with another of one our young patients who was coming for transplant on this protocol. Anaiya gave her a heads up, sharing what she had been through and what to expect.”
It’s very fulfilling as a physician and researcher to see that our trial is helping kids get back to being kids again. They are my inspiration.
Dance like everyone’s watching
During her time at St. Jude, Anaiya convinced members of the nursing staff to make fun dance videos with her, and now she aspires to be a St. Jude nurse. Her more immediate wish list includes a rollerblading party, getting involved in basketball and track, and of course, shooting more videos with her phone and adding special effects.
“Since receiving her transplant, Anaiya hasn’t had any pain crises and has lots more energy,” Carruthers observes.
Sharma says he hopes to see similar positive responses from other patients in the clinical trial.
“It’s remarkable how the transplant process has completely changed Anaiya’s life,” he says, “Earlier, she needed to come to the hospital every few weeks because of pain crises or for blood transfusions; now, she is out and about — doing her thing as a preteen and enjoying life.
“It’s very fulfilling as a physician and researcher to see that our trial is helping kids get back to being kids again. They are my inspiration.”
From Promise, Summer 2021