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Characterization of a cohort of Angolan children with sickle cell anemia treated with hydroxyurea
Brígida Santos1, Catarina Ginete2, Elisângela Gonçalves3
1Centro de Investigação em Saúde de Angola (CISA), Caxito, Angola; Hospital Pediátrico David Bernardino (HPDB), Luanda, Angola; Global Health and Tropical Medicine (GHTM), Associate Laboratory in Translation and Innovation Towards Global Health (LA-REAL), Instituto de Higiene e Medicina Tropical (IHMT), Universidade NOVA de Lisboa (UNL), Lisbon, Portugal.
Insights
Hydroxyurea (HU) treatment in Angolan children with Sickle Cell Anemia (SCA) reduced clinical events and showed varied fetal hemoglobin (HbF) increases. HU treatment is associated with increased survival in children with SCA.
Area of Science:
- Hematology
- Pediatrics
- Genetics
Background:
- Sickle Cell Anemia (SCA) is a monogenic disease with highly variable clinical presentation and treatment response.
- Understanding SCA heterogeneity is crucial for effective management, especially in pediatric populations.
Purpose of the Study:
- To characterize Angolan children with SCA.
- To evaluate the efficacy and safety of hydroxyurea (HU) treatment in this cohort.
- To assess potential side effects and toxicity associated with HU therapy.
Main Methods:
- A cohort of 215 Angolan children (3-12 years) with SCA was enrolled.
- Patients received a fixed dose of hydroxyurea (HU) at 20 mg/kg/day for 12 months.
- Clinical events, fetal hemoglobin (HbF) levels, and treatment response were monitored.
Main Results:
- HU treatment reduced the frequency of transfusions (53.4%) and hospitalizations (47.1%).
- Hydroxyurea response varied, with a mean HbF increase of 11.9% (range 1.8%-31%).
- 57% were responders, 38.7% inadequate responders, and 4.2% non-adherent; no clinical side effects were reported, and hematological toxicities were reversible.
Conclusions:
- Hydroxyurea treatment is effective in reducing clinical events in Angolan children with SCA.
- The study contributes to understanding SCA profiles and supports HU's role in improving survival.
- Prophylactic measures alongside HU treatment are vital for enhancing survival rates in pediatric SCA patients.
Background:
Sickle Cell Anemia (SCA) is a monogenic disease, although its severity and response to treatment are very heterogeneous.
Objectives:
This study aims to characterize a cohort of Angolan children with SCA and evaluate their response to hydroxyurea (HU) treatment and the potential side effects and toxicity.
Methods:
The study enrolled 215 patients between 3 and 12 years old before and after the administration of HU, at a fix dose of 20 mg/kg/day for 12 months.
Results:
A total of 157 patients started HU medication and 141 of them completed the 12-month treatment. After initiating HU treatment, the frequency of clinical events decreased (transfusions 53.4 %, hospitalizations 47.1 %). The response to HU medication varied among patients, with some experiencing an increase in fetal hemoglobin (HbF) of <5 %. The mean increase in HbF was 11.9 %, ranging from 1.8 % to 31 %. Responders to HU treatment were 57 %, inadequate responders 38.7 % and non-adherent 4.2 %. No clinical side effects related to HU were reported. Hematological toxicities were transient and reversible. Children naïve to HU and with lower HbF reported higher number of hospitalizations caused by malaria infection. During HU treatment, the frequency of malaria episodes did not appear to be affected by HbF levels.
Conclusions:
the present study provided a valuable contribution to the understanding of the clinical and laboratory profiles of Angolan children with SCA. These findings support the evidence that the implementation of prophylactic measures and treatment with HU is associated with increased survival in children with SCA.
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