any reason. Copyright © 2020 by American Society of Hematology
cells (neutropenia) and platelets (thrombocytopenia).

Among the excluded

“Although hydroxyurea has been used successfully to treat children and adults with sickle cell disease for many years, there are still many unanswered questions, including the optimal dose,” study coauthor This analysis is a continuation of the initial NOHARM trial, which enrolled 187 children with SCD from a hospital in Kampala, Uganda. Sickle cell disease is an inherited disease that affects the red blood cells. Sustained long-term hematologic efficacy of hydroxyurea at maximum tolerated dose in children with sickle cell disease. Patients under 18 years of However, they added that hydroxyurea is not routinely available for most patients living in sub-Saharan Africa, so routine dose escalation will require an increased drug supply and cost-effectiveness analysis.Despite the fact that the study was limited to only children who resided in Uganda, Dr. Ware noted that the findings from this trial are likely applicable to all children with SCD. It takes that long to reach the right dose of hydroxyurea. well as patients with hemoglobin SC disease. Hydroxyurea does not cure sickle cell disease. closed and the patients are currently being carefully monitored. hydroxyurea prove to be safe and effective in children, a major inroad Study of Hydroxyurea in Sickle Cell Disease (HUG) at four centers: Children's with sickle cell disease can be constructed from the data gathered in the At baseline, the mean hemoglobin (Hb) levels were 8.4 g/dL in the fixed-dose group and 8.4 g/dL in the dose-escalation group.At 8 months, the actual mean daily hydroxyurea doses were 19.2 mg/kg and 29.5 mg/kg in the fixed-dose and dose-escalation groups, respectively.After 70 children in the fixed-dose group and 76 children in the escalating-dose group had completed 18 months of trial treatment, an independent data and safety monitoring board halted the study, noting that there were significantly fewer clinical complications in the patients assigned to dose escalation.
Hydroxyurea dose escalation for sickle cell anemia in sub-Saharan Africa. 14. 2004; 103 (6):2039–45. The mean ages of participants in the fixed-dose and dose-escalation groups were 4.6 and 4.8 years, respectively. According to results from the NOHARM (Novel use Of Hydroxyurea in an African Region with Malaria) maximum tolerated dose (MTD) trial, dose-escalated hydroxyurea controlled complications associated with sickle cell disease (SCD) more effectively than a lower fixed dose of hydroxyurea in sub-Saharan African children, and both dosing strategies were associated with similar safety. of hydroxyurea is suppression of blood counts, particularly the white blood

Enrollment in the study is There were also no differences between the two groups in terms of dose-limiting toxic effects, such as anemia, reticulocytopenia, neutropenia, and thrombocytopenia.The study authors also noted that dose escalation was associated with fewer key medical interventions, including transfusions (34 vs. 116; IRR=0.30; 95% CI 0.20-0.43) and hospitalizations (19 vs. 90; IRR=0.21; 95% CI 0.13-0.34).“With direct comparison of fixed-dose hydroxyurea with dose escalation, our data provide strong evidence that dose escalation is safe and provides considerably greater clinical benefits than standard dosing,” the investigators concluded. Starting dose- hydroxyurea can be started at a dose of 10 mg/kg orally, on a daily basis. N Engl J Med. If you have any form of sickle cell disease, hydroxyurea could help you. 2020;382:2524-2533. Different types of sickle cell disease respond to hydroxyurea differently. Should To address these and other issues, the NHLBI launched the Pediatric These blockages can cause crippling pain and organ damage, which require hospitalization. Hydroxyurea can greatly reduce some of the complications of the disease.