Sickle Cell Awareness Day

It’s World Sickle Cell Awareness Day, so today we bring back the story of Maryam Idan, a young Iraqi girl and sickle cell disease (SCD) survivor. We say “survivor” because last year Maryam had a stem cell transplant—the only cure currently available for SCD, and a complicated and risky one at that. Read on to learn more about Maryam’s story and life.

Maryam and her parents

Born in Baghdad in 2002 without access to advanced medical care, Maryam Idan stood little chance of receiving anything more than the most basic treatment for her sickle cell disease (SCD). By the time Maryam was a baby, her parents, Basim and Janin Al-Zoubaidi, had already lost three of their children to the blood disorder, so they knew the condition’s telltale signs. “When she started showing the same symptoms as her siblings, we immediately thought the worst,” says her father. “We knew there was treatment in America, but we had no idea if we would have the means to get there.”

An inherited disease, SCD causes a child’s red blood cells to malform and become stiff, taking on the shape of a sickle, like the letter C. These abnormally shaped blood cells can stick together and interrupt healthy blood flow throughout the body. This can cause a host of problems that can affect nearly every major organ, but it affects every patient differently. “Some kids don’t have pain or complications until they’re teens, while others suffer strokes and extreme pain and need blood transfusions when they are very young,” says Leslie Lehmann MD, director of the Stem Cell Transplant Program at Dana-Farber/Children’s Hospital Cancer Center (DFCHCC).

Leslie Lehmann, MD
Leslie Lehmann, MD

Like Maryam, most kids with SCD experience breathlessness, delayed growth, extreme fatigue and pain in their bones from the time they’re young. Kids with SCD also have weaker immune systems, making them susceptible to infections, and they’re at a greater risk for strokes, blindness and fatal iron-overload—another dangerous blood complication. In developed nations, where symptoms can be treated with medication, patients often still only survive until their late 40s. For patients like Maryam in developing countries, even the most basic treatment for these symptoms isn’t usually available, and death at a young age is very common.

The only cure for SCD is a stem cell transplant when the patient is young, similar to a treatment that someone with cancer would undergo. It involves the complete destruction of the immune system (as well as the sickle cells) through chemotherapy, then rebuilding the person’s immune system using healthy stem cells from a matched donor.

A test in perseverance

As a toddler, Maryam struggled with bone pain and fatigue, making it nearly impossible for her to live a carefree and energetic childhood. “There were times when she would play and feel good, but they were always followed by her being incredibly tired or struggling with pain,” says Basim. When she was 3, the war in Iraq had worsened, making quality medical care nearly impossible to find. Her parents knew that in order to keep her alive, they needed to find help immediately.

“We knew there was treatment in America, but we had no idea if we would have the means to get there.”

So Basim, Janin and Maryam, along with Maryam’s older brother, Abdullah, moved to Damascus, Syria, leaving their three oldest, and newly married, daughters behind. In Damascus, medical care was more accessible, but it still wasn’t enough: Maryam’s doctors knew that they couldn’t cure her and that she needed a stem cell transplant in the United States. “A transplant is a complicated and risky cure, and it’s only helpful in a fraction of patients,” says Venee Tubman, MD, clinical fellow in Hematology and Oncology at DFCHCC. “But right now, it’s the only cure that exists.”

Unfortunately, the likelihood of a child being a candidate for a stem cell transplant is slim because it relies on having a sibling who has identical red blood cells that can easily transfer to the sick child’s body. (In very rare circumstances, a non-sibling can donate stem cells, but these transplants are usually less successful.) The chance of a sibling match is just one in four, but because SCD is inherited, siblings are at risk of having the disease too, stretching the likelihood to one in six. But if a brother or sister is a match, there’s a good chance that his or her healthy red blood cells can replace the sick child’s sickle cells. Hoping for a match, Syrian doctors tested Maryam’s 12-year-old brother, Abdullah, but they said he was not a match. Her older sisters were tested back home too, and while one was a candidate, she couldn’t leave to come to America.

Despite these setbacks, the family continued to search for medical help. They applied for refugee status in the United States in the hope that American doctors might know a way to help Maryam, despite her lack of a donor. They boarded a plane and took off for their new home in Manchester, New Hampshire, where they planned to live.

The long trip proved extremely difficult for Maryam, who had a painfully enlarged spleen and needed extra oxygen during the 16-hour journey. Within their first few days in America, Maryam’s health quickly deteriorated, her spleen grew even larger and her pain worsened. Her parents took her to a nearby hospital, where doctors realized that blood was being absorbed into her spleen, but not being filtered out, thereby depriving her body of crucial blood supply. They quickly referred her to Boston Children’s Hospital to have her spleen removed. “All we felt was despair,” says Basim. “We had no hope, since the doctors told us that a stem cell transplant was the only way she would survive.”

Upon arrival at Boston Children’s, Basim and Janin talked to Tubman through a translator, explaining their daughter’s recurring chest and bone pain, and their hopes that Boston Children’s could help her. Tubman arranged for Maryam to be admitted to the intensive care unit. “I can’t imagine how nervous she was and how much pain she was in, with all of the doctors swarming around talking in a different language—but she was very calm,” Tubman remembers.

Despite the fact that their daughter was in good hands, Maryam’s family felt utterly helpless, and the fate of their three other children who had succumbed to complications of SCD was at the forefront of their minds. But then they heard news that changed their lives: Doctors had re-tested Abdullah and found that the Syrian doctors were wrong: He was a match for his sister after all. And just like that, Maryam not only had a treatment option but the potential of a future free of the disease.

A complicated cure

She quickly began chemotherapy to rid her bones of her sickle cell marrow and make room for Abdullah’s healthy cells. Kids’ bodies can usually only tolerate the conditioning process once, so all hope was pinned on the outcome of this process. And it comes with a high price: The chemotherapy would likely rob Maryam of her fertility.

Matthew Heeney, MD

While the success rate for children undergoing a stem cell transplant for SCD is favorable—85 to 90 percent—some patients don’t survive the process or their bodies simply don’t accept the donor cells. Others may go through the procedure and still have SCD. “I always tell families that finding out you have a sibling match is just the beginning of a very long conversation,” says Matthew Heeney, MD, director of the Hematology and Sickle Cell Program at Boston Children’s. “Unfortunately, science hasn’t evolved enough for doctors to be able to predict exactly who will do well and who won’t.”

The chemotherapy was hard on Maryam, Basim remembers. “She was in a lot of pain, but was accepting because we told her it was the first step to get rid of her suffering.” Her doctors found her to be incredibly resilient. “I visited her during what we call the ‘dog days of the transplant’— the two or three tough weeks after chemo when she had no immune system and extreme pain and nausea,” says Tubman. “Every time I saw her, she told me she felt fine. She was a trooper.”

Once Maryam’s body was primed, Abdullah had healthy bone marrow removed from his hip. “It’s a serious procedure,” Lehmann explains. “We needed around three Coke bottles of marrow, so we had to go into his bone 50 to 100 times to extract enough cells.” Once Maryam’s doctors had what they needed, the transplant itself was over in a matter of hours. “People expect a flash of light or something,” says Heeney. “But it’s a pretty simple process, much like a blood transfusion.”

Maryam stayed at Children’s for a month while her family waited to see if her body would accept her brother’s cells. After the surgery, Abdullah wasn’t in much pain, but could do very little physical activity for two months while his bones regained their strength.

Maryam at play

Despite the ordeal, he felt honored to help his sister. “I was so happy to do this,” he says. “I was supposed to help Maryam.” Meanwhile, the wait was excruciating for the family. “Even though a sibling is a match and their immune systems are practically identical, there are always subtle differences,” says Heeney. “Those cells can look around and say ‘Hey, I’m not where I’m supposed to be,’ and attack various organs like the skin and liver.”

Luckily, Maryam’s new cells felt right at home.

It usually takes six months to a year for a child to recover from a stem cell transplant, as her immune system reboots. Maryam is now nearing the half-way point, and is taking precautions to keep germs at bay, like wearing her Mickey Mouse-printed paper face mask most of the time. Because her immune system needs gradual rebuilding, she’ll spend almost a year in a kind of isolation. She can play outside, but can’t go anywhere indoors except for her home and the doctor’s office. Even friends’ homes, grocery stores and classrooms are off limits.

She’s certainly lost a lot of things this year—her home, her freedom, her siblings back home and her hair. “I want to play like any girl,” she says. “And my hair used be down to here,” she says, pointing to her hip. In the next moment, she’s lit up again, pinching at the soft, half-inch strands on her head. “But, hey it’s coming back!”

For the first time, Maryam’s bone and chest pains are gone, and her body is getting stronger instead of weaker. She is lively and energetic. These days, she’s at the doctor’s for check-ups instead of emergencies. Choosing a stem cell transplant means that a year of her life is spent on the mend, but the rest of those years are hers for the taking. She is officially free of SCD.

While she anticipates the future, she spends hours on Skype each day with her sisters overseas, and they marvel at her transformation. But mostly, she is a little girl who lives for the moment. She loves to dress to the nines, and donned a flowing party dress for her 9th birthday in July. A master at her mother’s iPhone, she flips through it to show pictures of her elaborate cake and candles, pointing out new friends who came to see her while gushing about how beautiful the day was. Chatting away enthusiastically, she switches to one of her other favorite subjects: the game of tag. “I was slow at first,” she grins “but I’m getting faster.”