Long-Term Study Shows Hydroxyurea Works Wonders for Children

In a groundbreaking study published in *Blood Advances*, researchers have confirmed that hydroxyurea remains remarkably effective over the long term in reducing emergency room visits and hospitalizations for children grappling with sickle cell disease (SCD).

Dr. Paul George, a pediatric hematology/oncology fellow at Emory University, stated, "This is one of the first large-scale, real-world studies examining hydroxyurea's efficacy beyond a controlled environment, reaffirming its vital benefits for pediatric patients."

The Impact of Sickle Cell Disease

Sickle cell disease is the most widespread inherited blood disorder in the U.S., affecting around 100,000 individuals. Alarmingly, it affects approximately 1 in every 365 Black or African American births and 1 in 16,300 Hispanic American births, according to the CDC.

Characterized by misshapen blood cells that obstruct blood flow, SCD can lead to debilitating pain, organ damage, and severe infections.

Hydroxyurea: A Lifesaver for SCD Patients

Hydroxyurea, a daily oral medication typically used for a lifetime, plays a crucial role in mitigating the frequency and severity of pain crises, reducing blood transfusion needs, alleviating anemia, and lowering the risk of acute chest syndrome.

The National Heart, Lung, and Blood Institute now recommends that hydroxyurea be administered to every patient with the severe HbSS/HbS variant of SCD starting as young as nine to twelve months.

Safety Concerns Addressed

Though hydroxyurea has been a cornerstone in SCD treatment for years, initial concerns about its safety and effectiveness—particularly for children—due to its background as chemotherapy, have lingered. Dr. Wilbur Lam, also part of the research team, assures that this study provides important reassurance that this therapy, one of the most accessible for SCD, is indeed safe and beneficial.

Key Findings from the Research

The study analyzed a cohort of 2,147 children under 18, all diagnosed with the HbSS/HbS variant of SCD, who had multiple clinical visits from 2010 to 2021 and hadn't undergone extensive treatments beyond hydroxyurea.

Out of these participants, 1,240 (58%) were treated with hydroxyurea, averaging 5.1 years on the medication. Remarkably, it was found that these children had fewer emergency department visits (0.36 less per patient-year) and spent fewer days in the hospital (0.84 less per patient-year) compared to those not on hydroxyurea.

Vital Importance of Medication Adherence

Even when accounting for disease severity and ensuring that data indicated regular medication intake, these effects persisted. However, Dr. George pointed out that improvements in hemoglobin levels and anemia reduction were observed only in children who adhered strictly to their medication schedules.

Conclusion: A Call to Action for Patients

The study underscores the importance of consistent hydroxyurea intake, urging healthcare providers to emphasize daily adherence to maximize treatment benefits. Despite some limitations in data due to its real-world nature, this study highlights hydroxyurea's potential as a safe and effective treatment for managing sickle cell disease in children.