Health-related quality of life in children with sickle cell anemia: impact of blood transfusion therapy.

Journal Article (Clinical Trial;Journal Article;Multicenter Study)

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.

Full Text

Duke Authors

Cited Authors

  • Beverung, LM; Strouse, JJ; Hulbert, ML; Neville, K; Liem, RI; Inusa, B; Fuh, B; King, A; Meier, ER; Casella, J; DeBaun, MR; Panepinto, JA; SIT trial investigators,

Published Date

  • February 2015

Published In

Volume / Issue

  • 90 / 2

Start / End Page

  • 139 - 143

PubMed ID

  • 25345798

Pubmed Central ID

  • PMC4304929

Electronic International Standard Serial Number (EISSN)

  • 1096-8652

Digital Object Identifier (DOI)

  • 10.1002/ajh.23877


  • eng

Conference Location

  • United States