Giving Sickle Cell Patients Hope for a Cure

April 27

As director of the robust Pediatric Stem Cell Transplantation Program at Columbia University Medical Center, Monica Bhatia, MD, specializes in caring for the very sickest children with blood disorders – those who cannot be cured without a stem cell transplant.

"My passion is treating patients with sickle cell disease,” said Dr. Bhatia.

People forget how debilitating this illness can be. These children often miss school, are plagued with chronic pain and unable to achieve their goals. It’s quite moving to hear patients articulate how this disease affects them.

Dr. Bhatia and her dedicated team have seen remarkable results among children who receive a transplant – the only known cure for sickle cell disease – and go on to lead healthy, productive lives.

“Afterwards, they soar,” explained Dr. Bhatia. “These patients embrace the opportunity to have a second chance at life. “It is so nice to see them years later, watching them grow as people, go to school, get married. After what they endure, I feel as if they are destined for great things.”

While nationwide cure rates for stem cell transplantation using matched sibling donors approach 90-95%, the biggest challenge is for children with sickle cell disease who do not have a sibling donor.

“Because only 15 percent of patients with the disease have a matched sibling donor, I am also concentrating my efforts on unrelated donor transplants,” she said.

African Americans and Hispanics are most affected by this disease and unfortunately, are not well represented on the registry. It is important to educate people about what it means to be a donor, get more people registered and grow the donor pool.

Currently, the doctor and her colleagues are working to reduce the toxicity and complications of transplantation without compromising its effectiveness, and to make it available to more patients.

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Quick facts: Sickle cell disease