Background: Sickle cell anaemia (SCA) is a prevalent genetic disorder in sub-Saharan Africa, characterised by frequent vaso-occlusive crises and other complications. Hydroxycarbamide (hydroxyurea) has proven effective in managing SCA but is underutilised in low- and middle-income countries (LMICs) due to cost and monitoring challenges. This study aims to ascertain the impact of a fixed low-dose hydroxycarbamide regimen on emergency room visits and treatment adherence for paediatric SCA patients in a resource-limited setting. Methods: An observational study was conducted over two years (January 2022 to December 2023) in a tertiary hospital in South East Nigeria. One hundred children aged 1 to 18 years with confirmed SCA (HbSS genotype) were administered a fixed low-dose hydroxycarbamide regimen (20 mg/kg/day, capped at 500 mg/day). The primary outcome was the number of emergency room visits due to SCA-related crises. Secondary outcomes included treatment adherence and factors influencing adherence. Patients experiencing breakthrough crises had their doses increased to 25 mg/kg/day or from 500 mg to 750 mg. Results: Over the study period, only one patient (0.01%) on the fixed low-dose regimen presented to the clinical emergency room with a vaso-occlusive crisis, necessitating a dose increase. Treatment adherence was high, with 84.2% of patients reporting consistent adherence throughout the study period. Younger patients were more likely to adhere to treatment (mean age of compliant patients: 8.49 ± 5.28 years vs. non-compliant: 11.62 ± 4.21 years, p=0.027). Father’s age was also associated with adherence, with non-compliant patients having older fathers (median age 49.0 vs. 44.0 years, p=0.02). No significant clinical side effects were observed. Conclusion: The fixed low-dose hydroxycarbamide regimen significantly reduced emergency room visits and demonstrated high adherence rates among paediatric SCA patients in a resource-limited setting. This approach appears to be a safe, effective, and feasible treatment strategy for managing SCA in LMICs, potentially improving accessibility and sustainability of care.

Background: Adherence to follow-up visits is crucial for optimizing clinical outcomes in paediatric patients with sickle cell disease (SCD). This study aimed to evaluate compliance with follow-up visits among paediatric SCD patients over a five-year period. Methodology: A retrospective analysis was conducted using data from the sickle cell disease clinic of Nnamdi Azikiwe University Teaching Hospital, spanning from January 2015 to December 2019. A total of 271 unique paediatric patients with SCD and 1117 follow-up visits were included. Compliance with follow-up visits, defined as attendance within 100 days of the previous visit, was assessed using Kaplan-Meier curves and Cox regression analysis. Results: During the study period, a total of 1117 follow-up visits were documented among 271 children diagnosed with sickle cell anaemia. The median age of the study cohort was 8 years (IQR: 4 to 12 years). Male patients constituted 57% of the study population. Fifty percent of subjects remained compliant with follow-up visits at 14.2 months (95% CI = 8.9-24.1 months) and this compliance decreased to 40% at 24 months (95% CI = 31-49 months). Males showed a lower likelihood to be compliant to follow-up visits than females (HR: 0.83; CI: 0.56-1.23). Following adjustments, patients who were sicker and young adolescents (10 to 14 years) had an increased likelihood of complying to follow up visits (HR: 1.05; CI: 0.71-1.57 vs HR: 1.15; CI: 0.682-1.940 respectively) but this was not statistically significant. Conclusion: The overall poor compliance rate to follow up visits underscores the urgent need for interventions aimed at improving adherence to follow-up visits in this vulnerable population. Counselling interventions and further exploration of factors influencing compliance are warranted to enhance the management and outcomes of children living with sickle cell anaemia.

Background: Sickle cell disease (SCD) presents a formidable healthcare challenge, with stroke being a significant complication. While transcranial Doppler ultrasound (TCD) is the gold standard for stroke risk assessment, its availability is often limited by resource constraints. This study investigates the platelet-neutrophil ratio (PNR) as a cost-effective and accessible alternative for stroke risk assessment in SCD patients. Methods: We conducted a retrospective study of 144 SCD HbSS children at Children’s National Hospital, Washington DC, USA. TCD values were categorized as normal, conditional, or abnormal. PNR was calculated from routine blood tests. Statistical analyses were performed to evaluate the association between PNR and stroke risk. Results: PNR values varied significantly across stroke risk categories: 83.83 (58.15-127.65) for normal, 75.69 (64.89-88.6) for conditional, and 62.57 (28.12-64.47) for abnormal (p = 0.031). After adjusting for age and hydroxyurea intake, a unit increase in PNR was significantly associated with a decreased stroke risk (OR = 0.975, 95% CI: 0.953-0.998, p = 0.0345). PNR demonstrated superior discriminative performance for stroke risk compared to neutrophils and platelets, with AUC values of 0.81, 0.57, and 0.19, respectively. Multivariate analysis confirmed that higher PNR was independently associated with a reduced likelihood of abnormal TCD values (OR = 0.975, 95% CI: 0.953-0.998, p = 0.0345). Conclusion: Our study highlights the potential of the platelet-neutrophil ratio (PNR) as a promising, cost-effective, and accessible alternative for stroke risk assessment in SCD management, offering a more equitable healthcare solution.