Dataset Information

PRIMARY STROKE PREVENTION IN CHILDREN WITH SICKLE CELL ANEMIA LIVING IN AFRICA: THE FALSE CHOICE BETWEEN PATIENT-ORIENTED RESEARCH AND HUMANITARIAN SERVICE-PART II.

ABSTRACT: In the United States, primary stroke prevention in children with sickle cell anemia (SCA) is the standard of care and includes annual transcranial Doppler ultrasound to detect elevated velocities. For children with velocities ≥200 cm/sec, initial monthly blood transfusion therapy for at least a year, followed by the option of hydroxyurea therapy, results in a significant decline in the stroke rate. In Africa, no prior strategy exists for primary stroke prevention because regular blood transfusion therapy is not feasible. We completed a randomized controlled trial for primary stroke prevention using initial low- and moderate-dose hydroxyurea as an alternative to blood transfusion therapy. The trial results demonstrated equal primary stroke prevention efficacy with both doses. After the trial, we provided a state-supported stroke screening service and treatment for over 20,000 children in Kano, Nigeria. We will review the strategies implemented to conduct clinical research and provide humanitarian assistance to children with SCA in Nigeria.

SUBMITTER: Debaun MR

PROVIDER: S-EPMC9480538 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature

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Publications

PRIMARY STROKE PREVENTION IN CHILDREN WITH SICKLE CELL ANEMIA LIVING IN AFRICA: THE FALSE CHOICE BETWEEN PATIENT-ORIENTED RESEARCH AND HUMANITARIAN SERVICE-PART II.

Debaun Michael R MR

Transactions of the American Clinical and Climatological Association 20220101

In the United States, primary stroke prevention in children with sickle cell anemia (SCA) is the standard of care and includes annual transcranial Doppler ultrasound to detect elevated velocities. For children with velocities ≥200 cm/sec, initial monthly blood transfusion therapy for at least a year, followed by the option of hydroxyurea therapy, results in a significant decline in the stroke rate. In Africa, no prior strategy exists for primary stroke prevention because regular blood transfusio ...[more]

PMID: 36196180

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Project description:BackgroundHydroxyurea is an effective treatment for sickle cell anemia, but few studies have been conducted in sub-Saharan Africa, where the burden is greatest. Coexisting conditions such as malnutrition and malaria may affect the feasibility, safety, and benefits of hydroxyurea in low-resource settings.MethodsWe enrolled children 1 to 10 years of age with sickle cell anemia in four sub-Saharan countries. Children received hydroxyurea at a dose of 15 to 20 mg per kilogram of body weight per day for 6 months, followed by dose escalation. The end points assessed feasibility (enrollment, retention, and adherence), safety (dose levels, toxic effects, and malaria), and benefits (laboratory variables, sickle cell-related events, transfusions, and survival).ResultsA total of 635 children were fully enrolled; 606 children completed screening and began receiving hydroxyurea at a mean (±SD) dose of 17.5±1.8 mg per kilogram per day. The retention rate was 94.2% at 3 years of treatment. Hydroxyurea therapy led to significant increases in both the hemoglobin and fetal hemoglobin levels. Dose-limiting toxic events regarding laboratory variables occurred in 5.1% of the participants, which was below the protocol-specified threshold for safety. During the treatment phase, 20.6 dose-limiting toxic effects per 100 patient-years occurred, as compared with 20.7 events per 100 patient-years before treatment. As compared with the pretreatment period, the rates of clinical adverse events decreased with hydroxyurea use, including rates of vaso-occlusive pain (98.3 vs. 44.6 events per 100 patient-years; incidence rate ratio, 0.45; 95% confidence interval [CI], 0.37 to 0.56), nonmalaria infection (142.5 vs. 90.0 events per 100 patient-years; incidence rate ratio, 0.62; 95% CI, 0.53 to 0.72), malaria (46.9 vs. 22.9 events per 100 patient-years; incidence rate ratio, 0.49; 95% CI, 0.37 to 0.66), transfusion (43.3 vs. 14.2 events per 100 patient-years; incidence rate ratio, 0.33; 95% CI, 0.23 to 0.47), and death (3.6 vs. 1.1 deaths per 100 patient-years; incidence rate ratio, 0.30; 95% CI, 0.10 to 0.88).ConclusionsHydroxyurea treatment was feasible and safe in children with sickle cell anemia living in sub-Saharan Africa. Hydroxyurea use reduced the incidence of vaso-occlusive events, infections, malaria, transfusions, and death, which supports the need for wider access to treatment. (Funded by the National Heart, Lung, and Blood Institute and others; REACH ClinicalTrials.gov number, NCT01966731 .).

Dataset Information

PRIMARY STROKE PREVENTION IN CHILDREN WITH SICKLE CELL ANEMIA LIVING IN AFRICA: THE FALSE CHOICE BETWEEN PATIENT-ORIENTED RESEARCH AND HUMANITARIAN SERVICE-PART II.

Publications

PRIMARY STROKE PREVENTION IN CHILDREN WITH SICKLE CELL ANEMIA LIVING IN AFRICA: THE FALSE CHOICE BETWEEN PATIENT-ORIENTED RESEARCH AND HUMANITARIAN SERVICE-PART II.

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