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Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
Published on: March 14, 2017
Reducing transfusion utilization for children with sickle cell anemia in sub-Saharan Africa with hydroxyurea:
Alexandra Power-Hays1,2, George A Tomlinson3, Leon Tshilolo4
1Division of Hematology and Global Health Center, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Insights
Hydroxyurea treatment significantly reduces blood transfusion needs in African children with sickle cell anemia (SCA). This therapy, especially at maximum tolerated dose, could prevent a substantial percentage of pediatric transfusions in sub-Saharan Africa.
Area of Science:
- Hematology
- Pediatric Medicine
- Public Health
Background:
- Children with sickle cell anemia (SCA) in Africa often require blood transfusions due to complications.
- Limited blood supplies in the region necessitate strategies to reduce transfusion dependency.
- The effectiveness of such strategies, like hydroxyurea treatment, has not been widely evaluated in this population.
Purpose of the Study:
- To analyze transfusion utilization in children with SCA before and during hydroxyurea treatment.
- To evaluate the impact of hydroxyurea, particularly at maximum tolerated dose (MTD), on reducing transfusion needs.
- To assess the potential public health impact of widespread hydroxyurea implementation for SCA in sub-Saharan Africa.
Main Methods:
- Analysis of transfusion data from the REACH trial (NCT01966731), a longitudinal study of hydroxyurea in African children with SCA.
- Comparison of transfusion rates across different treatment periods: pre-treatment, fixed-dose, dose escalation, and MTD.
- Statistical analysis using univariate and multivariable models to identify factors associated with transfusions and calculate incidence rate ratios (IRR).
Main Results:
- Among 635 children, 40.4% received 545 transfusions over 4124 person-years.
- Transfusion rates per 100 person-years decreased significantly from 43.2 (pre-treatment) to 10.8 (MTD).
- Hydroxyurea at MTD reduced transfusion incidence by 75% compared to pre-treatment (IRR 0.25) and 50% compared to fixed-dose (IRR 0.50).
Conclusions:
- Hydroxyurea treatment at the maximum tolerated dose significantly decreases blood transfusion utilization in African children with SCA.
- Widespread implementation of hydroxyurea could potentially prevent 21% of all pediatric transfusions in sub-Saharan Africa.
- Increasing access to hydroxyurea for SCA management is crucial to alleviate the transfusion burden and improve blood supply availability.
Abstract:
Children with sickle cell anemia (SCA) in Africa frequently require transfusions for SCA complications. Despite limited blood supplies, strategies to reduce their transfusion needs have not been widely evaluated or implemented. We analyzed transfusion utilization in children with SCA before and during hydroxyurea treatment. REACH (Realizing Effectiveness Across Continents with Hydroxyurea, NCT01966731) is a longitudinal Phase I/II trial of hydroxyurea in children with SCA from Angola, Democratic Republic of Congo, Kenya, and Uganda. After enrollment, children had a two-month pre-treatment screening period followed by 6 months of fixed-dose hydroxyurea (15-20 mg/kg/day), 18 months of dose escalation, and then stable dosing at maximum tolerated dose (MTD). Characteristics associated with transfusions were analyzed with univariate and multivariable models. Transfusion incidence rate ratios (IRR) across treatment periods were calculated. Among 635 enrolled children with 4124 person-years of observation, 258 participants (40.4%) received 545 transfusions. The transfusion rate per 100 person-years was 43.2 before hydroxyurea, 21.7 on fixed-dose, 14.5 during dose escalation, and 10.8 on MTD. During MTD, transfusion incidence was reduced by 75% compared to pre-treatment (IRR 0.25, 95% confidence interval [CI] 0.18-0.35, p < .0001), and by 50% compared to fixed dose (IRR 0.50, 95% CI 0.39-0.63, p < .0001). Hydroxyurea at MTD decreases transfusion utilization in African children with SCA. If widely implemented, universal testing and hydroxyurea treatment at MTD could potentially prevent 21% of all pediatric transfusions administered in sub-Saharan Africa. Increasing hydroxyurea access for SCA should decrease the transfusion burden and increase the overall blood supply.
