Short-term safety and beneficial effects of hydroxyurea therapy in children with sickle cell disease

Introduction: Worldwide, sickle cell disease (SCD) is the most common hemoglobinopathy among which SS pattern is more common. Although hydroxyurea (HU) is approved by the Food and Drug Administration for the treatment of recurrent moderate-to-severe painful crises in pediatric sickle cell anemia, there is a fear of toxicities. Objectives: The objectives of the study were to evaluate the short-term safety and beneficial effects of low-dose HU therapy in SCD (SS pattern) children. Materials and Methods: This prospective cohort study enrolled 40 cases of severe SCD and started HU in a fixed dose of 10 mg/kg/day. During follow-up, cases were evaluated for compliance of HU, its toxic effects and adverse events from their histories, clinical examinations, and laboratory parameters. Furthermore, beneficial effects of HU therapy were evaluated by assessing blood transfusion rate, frequency of painful events, strokes, acute chest syndrome, avascular necrosis of femur, and estimation of hemoglobin F (HbF) level after 2 years of therapy. HU was discontinued temporarily if any toxicity or minor adverse drug events occurred during therapy and was restarted at the same dose after normalization of deranged laboratory parameters. Results: The clinical adverse drug events seen were nausea (8.33%), diarrhea (2.78%), and hematuria (2.78%). The most common hematological toxicity was anemia and thrombocytopenia. Renal and hepatic toxicities were transient in nature. The mean acute painful events and blood transfusion rate reduced significantly on HU therapy. It increased Hb and HbF level significantly in SCD children. Conclusion: HU is a safe drug without significant toxicity or adverse events in a dose of 10 mg/kg/day for short duration and it is beneficial in SCD (SS pattern) children in reducing acute painful events and decrease blood transfusion rate

Risk Factors for Predicting Mortality in a sick children in Paediatric Intensive Care Unit in developing country

Aim: The aim of this study is to assess risk factors contributing to increased mortality and their association with PRISM III 24 hour score in PICU. Materials and Methods: In this observational prospective study, 200 children admitted fulfilling inclusion criteria during 24 months period were enrolled in this study. PRISM III-24 score and mortality risk factor were calculated. Follow up was noted as discharge or death. Result: of 200 patients 115 male and 85 female.45 died and155 survived. PRISM III-24 score showed an increase of mortality from 4% in 0-9 score patients to 83.3% in ≥ 30 score. The presence of GCS score ≤ 8,MODS on admission ,need of inotropes and mechanical ventilation was significantly associated with poor outcome and well correlated with PRISM III score on univariate linear regression analysis. MODS on admission,mechanical ventilation and need of inotropes are independant predictors for PICU mortality on multiple logistic regression analysis. Conclusion: GCS score ≤ 8 ,MODS on admission ,need of inotropes and mechanical ventilation was significantly associated with poor outcome and well correlated with PRISM III-24 score. High PRISM III -24 score, MODS on admission ,mechanical ventilation and need of inotropes are independant predictors for PICU mortality