The availability of a commercial formulation of hydroxyurea will provide uninterrupted access to many pediatric patients with sickle cell disease (SCD) and Medicaid insurance, but it has created an “overwhelming financial burden” on those with private insurance, according to recent research.
The National Heart, Lung, and Blood Institute (NHLBI) and the American Society of Hematology (ASH) both strongly recommend the early and universal use of hydroxyurea in pediatric patients with SCD. The most common formulation in this population is a 100 mg/mL liquid suspension, compounded with hydroxyurea powder or opened capsules. This approach does not require refrigeration and can be bottled in various quantities.
“The cost of hydroxyurea liquid or powder is negligible, and this medication has been easily available with no associated financial burden for decades,” wrote the authors, led by Lydia Musula of Brown University Health Comprehensive Sickle Cell Center in Providence, Rhode Island.
This year saw the release of a commercial formulation of hydroxyurea called Xromi (Nova Laboratories Ltd., Leister, United Kingdom, and Rare Disease Therapeutics, Inc., Franklin, Tennessee). The formulation, which requires refrigeration, is prepackaged in 148 mL bottles that cost about $900 each ($6 per mL). This development now prevents pharmacies from compounding hydroxyurea, the authors explained. Options are now limited to capsules that must be swallowed or prior authorization for an alternative.
The authors wanted to explore and describe the challenges and barriers of transitioning their institution’s pediatric SCD patients. They identified 50 patients in their SCD center who had been prescribed liquid hydroxyurea at the time Xromi was released. The sample’s mean age was 7.2 years, and the majority (72%) had Medicaid insurance.
Prior to Xromi’s release, the outpatient pharmacy provided hydroxyurea in a 60-day supply in whatever quantity was needed for dose. The mean daily dose was 5.6 ± 3.7 mL, for a mean dispense volume of 176 ± 15 mL. Most patients had no copay, and the maximum monthly copay was $30 for one patient.
After Xromi’s release, all but one patient required prior authorization, which must be renewed every three months. Private insurance covered at least part of the therapy for about 28% of patients, and those families now have a copay of $20 to $723 per month. The transition required adjustments to dosing and timing of medication pick-up. In addition, two patients transitioned to capsules and four patients transitioned to Siklos. The team reported that they do not currently have a viable solution for about 10% of patients.
“This current situation creates real and significant risk of interruption or discontinuation of hydroxyurea for the vulnerable pediatric SCD population,” the authors concluded. “We must act together as a hematology and SCD community to advocate for our patients to prevent disruption of hydroxyurea, which would undoubtedly increase unnecessary morbidity and mortality for children with SCD.”
Reference
Musula L, Hitte R, Graves M, et al. Financial and clinical implications of Xromi as a new commercial liquid formulation of hydroxyurea. Abstract abs25-2319. Presented at: the 67th American Society of Hematology Annual Meeting and Exposition, Dec. 6-9, 2025, Orlando, FL.
