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Evaluation of The Use of Hydroxyurea in Treating Children With Sickle Cell Anemia in Central Africa's Rural Area
Gloire Mbayabo1,2,3, Paul Lumbala Kabuyi1,2,3, Mamy Ngole2,3,4
1Department of Pediatrics University of Kinshasa Kinshasa Democratic Republic of the Congo.
Insights
Hydroxyurea (HU) effectively treats sickle cell anemia (SCA) in Congolese children, improving HbF levels and clinical response. Adherence remains a challenge in rural settings, requiring targeted strategies for sustained treatment benefits.
Area of Science:
- Pediatrics
- Hematology
- Global Health
Background:
- Sickle cell anemia (SCA) is a prevalent and severe childhood disease in Central Africa.
- Hydroxyurea (HU) is the most effective treatment for SCA, but its use in rural African settings requires evaluation.
- Assessing HU's efficacy in pediatric SCA patients in the Democratic Republic of Congo (DRC) is crucial.
Purpose of the Study:
- To evaluate the clinical and biological effectiveness of hydroxyurea (HU) in children with sickle cell anemia (SCA) in rural Central Africa.
- To compare clinical and biological data before and after HU treatment to assess efficacy.
- To identify challenges and potential strategies for improving HU treatment adherence in resource-limited settings.
Main Methods:
- A clinical trial was conducted in the DR Congo involving pediatric SCA patients (6 months to 18 years) with moderate to severe disease.
- Patients received an initial HU dose of 15 mg/kg/day, with gradual escalation up to 30 mg/kg/day over 24 months.
- Clinical and biological data were collected at baseline, 12 months, and 24 months to determine treatment response.
Main Results:
- Sixty-nine patients were enrolled, with 39 completing the trial.
- Hemoglobin F (HbF) levels significantly increased by 3-fold at 12 months and 3.3-fold at 24 months compared to baseline.
- A good clinical response was observed in 80.4% of patients, while 19.6% did not respond adequately.
Conclusions:
- Hydroxyurea (HU) treatment demonstrated significant clinical and biological effectiveness in young Congolese SCA patients.
- Low patient adherence presents a major challenge to sustained HU treatment in rural areas.
- Implementing targeted strategies is essential to enhance adherence and optimize HU therapy outcomes in resource-limited settings.
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.
Abstract:
Trial Registration: ClinicalTrials.gov identifier: NCT05681598.
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