Eli Cured of Sickle Cell by Stem Cells from Brother

When Eli was born in 2010, his parents were delighted to finally have a child. They had been trying to conceive for years and had gone through multiple fertility treatments. But challenges soon confronted them when Eli was diagnosed with Sickle Cell Disease shortly after birth. Sickle Cell Disease causes red blood cells to be sickle-shaped and stiff, instead of round and flexible. The sickle cells do not carry oxygen well, causing the patient to feel chronic fatigue. Sometimes the sickle cells stick to each other and form clumps that block blood vessels; this is very painful and can be dangerous depending on where in the body it happens. There is a misconception that Sickle Cell Disease is only a problem in Africa, but it is also the most common inherited disease in the United States1. Sickle Cell Disease is caused by a genetic mutation; when both parents are carriers there is a 25% chance that their child will inherit two copies of the mutation and have the disease.

Currently the CDC estimates that 1 in 13 African Americans are carriers and 1 in 365 African American babies have the disease2. Sickle Cell impacts other ethnic groups as well, and altogether there are 100,000 Sickle Cell patients in the US2. Also, many people do not realize that Sickle Cell can impact the health-related quality of life for the patient’s entire family, especially the parents of a child with Sickle Cell3. It turned out that Eli had a more severe case of Sickle Cell Disease. He was frequently anemic from low red blood cell counts. He became sick easily and had pain crises when a blood vessel became blocked. Often his parents had to take him to the hospital emergency room and sometimes he was admitted to the hospital for long-term stays. His parents consulted with expert doctors at both Boston Children’s Hospital and Dana-Farber Cancer Institute to come up with a treatment plan that would give Eli a normal life. They knew that a stem cell transplant could cure Eli, and that a sibling would be the best donor. Finally, Eli’s parents welcomed his baby brother Gus and made sure to save his cord blood at birth. On top of having the cord blood, Gus matched Eli close enough genetically to be a bone marrow donor as well.

By now it was 2020, and Eli was nearly 10 years old. But due to the Coronavirus pandemic, there were multiple delays and Eli did not get his stem cell transplant until May of 2021. The family decided to take advantage of all their options and go ahead with a combined transplant of both cord blood and bone marrow from baby Gus. Within a year after the stem cell transplant, Eli had normal blood counts and there was no sign that he was ever diagnosed with Sickle Cell Disease. Eli has had zero pain crises and no hospital visits since his transplant.

“Since the procedure, he’s been flying with amazing colors. The doctors couldn’t be more happy that everything has gone so well. And we couldn’t be happier either!” – Eli’s Mom

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