Sickle Cell Patient Shows No Sign of Disease After Bone Marrow Transplant at Sylvester

A 31-year-old woman with sickle cell disease who had suffered with crippling pain all her life now shows no sign of the disease after being treated at Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine.

Brenda Alfaro was born in Nicaragua with this hereditary, red cell blood disorder and became a patient of hematologist Thomas Harrington, M.D., when she moved to Miami in her early 20s.

“She had very severe disease, excruciating pain and was hospitalized frequently,” said Dr. Harrington, who directs the adult sickle cell program at UM/Jackson Memorial Hospital.

These episodes are known as “crises” because of the severe, even life-threatening nature of the attacks. Pain, severe anemia and organ damage are common. Strokes, respiratory failure and other organ failure can be life-threatening complications.

Treatments, including Hydroxyurea and blood transfusions, were not enough to prevent Alfaro’s sickle cell crises. She was referred to Lazaros Lekakis, M.D., at Sylvester, the principal investigator at UM for the national STRIDE-2 Trial. The study compares outcomes of bone marrow transplantation versus supportive care for young adults with severe sickle cell disease.

Alfaro had the transplant in December, and four months later says she has never felt better in her life. She was very fortunate to have a perfect match in her one sibling, her brother William. The chance of a match is about 1 in 5, so most patients do not have this option.

“I can’t believe I have the opportunity for a different life with a cure,” said Alfaro.

She had put her studies on hold, but now will continue to pursue a bachelor’s degree in nursing.

“I think Brenda is likely cured, though she still needs close attention,” Dr. Harrington said. “My adult patients have been interested in bone marrow transplant for several years. Being able to offer them this option is very exciting for all of us.”

Next on the horizon for sickle cell patients: CRISPR.  In a clinical trial, the gene editing tool is used to introduce a corrective gene into a patient’s stem cells in the lab. The modified stem cells are then infused back into the patient.

Dr. Harrington says this may prove to be the “cure” available to all patients and hopes to get the study up and running at Sylvester soon.

Tags: bone marrow transplant, Dr. Thomas Harrington, sickle cell disease, STRIDE-2 trial, Sylverster Comprehensive Cancer Center