Louisville father recounts 32‑day ICU stay and difficult path to gene therapy for daughter with sickle cell
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Summary
A parent speaker described his daughter’s severe sickle cell complications, a prolonged hospital stay and the multi‑step insurance and medical review required to obtain FDA‑approved gene therapy after she turned 12, and said the family has temporarily paused treatment to reassess.
Antoine Sarton, a sickle cell disease advocate from Louisville, Kentucky, described the family journey that led to approval for a recently FDA‑authorized gene therapy for his 12‑year‑old daughter.
"We was in the hospital for 32 days…We ended up in the ICU for a whole week. That was a point where I thought I was gonna lose her," Sarton said, recounting a July 4, 2024 hospitalization tied to complications that included hemolytic anemia and frequent transfusions. He said his daughter later developed avascular necrosis and had a hip replacement, which delayed a planned gene therapy.
Sarton said the family pursued care at specialty centers, including consultations at Cincinnati Children’s Hospital, and faced extensive testing and insurance review before receiving approval. He described the therapy as costly, saying insurers must evaluate a treatment that can run into the millions: "Most insurance, which we are on Medicaid, they not just gonna fork out $2,630,000 for a drug treatment," he said.
He explained the personal and practical considerations that shaped the family decision. Because the U.S. approvals for these gene therapies require patients to be at least 12 years old, the family waited until his daughter met the age criterion, completed the required evaluations and navigated Medicaid paperwork. He said clinicians asked the family to pause the gene therapy while his daughter recovered from surgery and because her blood counts had stabilized on hydroxyurea: "We just chose to reevaluate this in the next 6 months," Sarton said.
Sarton urged caregivers to educate themselves and to build relationships with treating physicians and specialty centers. "The only thing I can say is, educate yourself as much as you can. Build the relationships as much as you can with your doctors," he said.
The caregiver testimony illustrated barriers patients face in accessing high‑cost, complex therapies: medical eligibility assessments, prolonged pre‑treatment evaluations, coordination among multiple doctors, and insurer reviews. Sarton also highlighted community resources such as local sickle cell associations that helped the family navigate care.
Next steps for the family include continued specialty follow‑up and a reassessment of readiness for gene therapy after recovery from orthopedic surgery and a period of clinical stability.
