Clinical Burdens of β-Thalassemia Major in Affected Children

J Pediatr Hematol Oncol. 2018 Apr;40(3):182-187. doi: 10.1097/MPH.0000000000001104.

Abstract

Background: β-thalassemia major (BTM) is an inherited blood disorder leading to severe anemia. A better understanding of BTM complications can be considered an important factor in developing effective health care provision.

Method: A descriptive exploratory design was used to identify the clinical burden of BTM from affected children's perspective. A convenience sample of 45 patients with BTM, accompanied by a family member, was recruited from a governmental hospital during April-May 2015.

Results: The most reported clinical burden was facial deformity 86.9%, followed by systematic infection (48.8%), growth delay (44.4%), and liver problems (39.9%). Patient age was significantly associated with clinical burdens such as bone pain and facial deformity. The number of blood transfusions received was associated with growth delay and bone pain.

Conclusion: This study highlights the clinical burdens of thalassemia on affected children, in terms of physical appearance, growth delay and other burdens.

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Cost of Illness
  • Female
  • Humans
  • Infant
  • Male
  • beta-Thalassemia / complications*