Health‐related quality of life in children with sickle cell anemia: Impact of blood transfusion therapy

Abstract

The completion of the Multicenter Silent Infarct Transfusion Trial demonstrated that children with pre-existing silent cerebral infarct and sickle cell anemia (SCA) who received regular blood transfusion therapy had a 58% relative risk reduction of infarct recurrence when compared to observation. However, the total benefit of blood transfusion therapy, as assessed by the parents, was not measured against the burden of monthly blood transfusion therapy. In this planned ancillary study, we tested the hypothesis that a patient centered outcome, health-related quality of life (HRQL), would be greater in participants randomly assigned to the blood transfusion therapy group than the observation group. A total of 89% (175 of 196) of the randomly allocated participants had evaluable entry and exit HRQL evaluations. The increase in Change in Health, measured as the child’s health being better, was significantly greater for the transfusion group than the observation group (Difference Estimate = −0.54, p ≤ 0.001). This study provides the first evidence that children with SCA who received regular blood transfusion therapy felt better and had better overall HRQL than those who did not receive transfusion therapy.

Central nervous system complications and management in sickle cell disease

“…7 When compared with observation, the benefit of blood transfusion therapy included an improvement of overall quality of life, 75 as well as a statistically significant decrease in the incidence of priapism, new-onset symptomatic avascular necrosis of the hip, severe vaso-occlusive pain events that resulted in hospitalization and acute chest syndrome. 7 The number of children with SCA and SCI who needed to be transfused to prevent 1 recurrent infarct was 13.…”

Red blood cell transfusion therapy for sickle cell patients with frequent painful events

“…This finding may be relevant for patients with frequent pain who continued to request opioid prescriptions at each clinic visit for transfusion for management of their pain at home. Finally, while we did not capture school attendance and quality of life, based on prior literature we could hypothesize that both of these important patient‐centered measures would improve during transfusion and are part of the joint therapeutic decision‐making …”

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

“…The impact of a change by .1 point is considered significant on a 5-category scale. 33,34 HRQL data were analyzed for changes in mean HRQL score from pretransplant measurement and performed as an exploratory analysis, given the small sample size. A paired Student t test was used to assess changes from baseline to each posttransplant time point (day 100, 6 months, and 1 year).…”