Evaluation of The Use of Hydroxyurea in Treating Children With Sickle Cell Anemia in Central Africa's Rural Area.

BACKGROUND: Sickle cell anemia (SCA) is highly prevalent in Central Africa. This disease causes severe manifestations in children, requiring treatment. Hydroxyurea (HU) is currently the most effective treatment for SCA. We evaluated the use of HU in children living in rural Central Africa. METHODS: We conducted a clinical trial using HU in the Kisantu Saint Luc Hospital (KSLH) in the DR Congo (DRC) from November 2017 to February 2020. SCA patients aged 6 months to 18 years, with a moderate to severe form of SCA (Adegoke score), were treated with an entry HU dose of 15 mg/kg per day, followed with gradual dose escalation of 5 mg/kg per day increments every 6 months, up to a maximally tolerated dose of 30 mg/Kg/day. To determine the clinical and biological response to the treatment (efficacy of HU), we compared the clinical and biological data collected during the first and second year of treatment to the baseline values. RESULTS: Sixty-nine patients (37 boys and 32 girls; sex-ratio M/F 1.15) were eligible for the clinical trial using HU. Only 39 patients remained at the end of the clinical trial (56.5%). On average, the HbF increased to 3-fold at 12 months and 3.3-fold at 24 months, which was significantly different to the baseline. Thirty-seven (80.4%) patients presented a good clinical response, whereas 9 (19.6%) did not. CONCLUSION: This clinical demonstrated the clinical and biological effectiveness of HU treatment in a cohort of young Congolese patients. In addition to tremendous logistical challenges, low adherence is the major threat to HU treatment in a rural area. There is a need to implement appropriate strategies to increase the adherence to the HU treatment in these rural settings. UNLABELLED: Trial Registration: ClinicalTrials.gov identifier: NCT05681598.