Evaluation of Quality of Life in Children with Hemophilia

Aim: Hemophilia is an X-linked hereditary coagulation disorder characterized by recurrent bleeding episodes that significantly impair patients’ quality of life. This study aimed to evaluate the quality of life levels of pediatric patients with hemophilia and their parents according to clinical variables such as disease severity, prophylaxis status, and the presence of target joints.Materials and Methods: A total of 40 hemophilia patients aged 8-12 years and their parents were included in this cross-sectional study. Data were collected using the Pediatric Quality of Life Inventory (PedsQL™) Hematology/Oncology Module. The form included 27 questions covering domains such as pain and hurt, nausea, procedural concern, treatment concern, anxiety, cognitive problems, perceived physical appearance, and communication.Results: Of the patients included in our study, 23 (57.5%) were being followed up for severe hemophilia. While 24 (60%) patients were receiving regular prophylaxis, 3 (7.5%) were inhibitor-positive. Thirteen patients (32.5%) had target joint damage. The prophylaxis (+) group reported significantly less pain and hurt than the non-prophylaxis (-) group (p=0.038). Similarly, the group without target joints (target joint -) had significantly better scores for pain and hurt than the group with target joints (target joint +) (p=0.025). In the comparison based on disease severity, patients with severe hemophilia experienced significantly more pain than those with mild or moderate forms (p<0.001).Conclusions: Our findings indicate that the most significant problems adversely affecting the quality of life of children with hemophilia are frequent hospitalizations, recurrent treatment needs, joint damage, and consequent pain. Our results strongly support that the prevention of joint damage should be a primary goal to improve quality of life. We conclude that early diagnosis, effective and individualized treatment, and regular prophylaxis can prevent these adverse outcomes and enhance patients’ living standards.