Quality of life of children and adolescents with hemophilia receiving low-dose prophylactic treatment.

OBJECTIVE

Compared to episodic treatment, low-dose prophylaxis (LDP) leads to better quality of life (QOL) by decreasing spontaneous bleeding in severe hemophilia A and B. LDP has been used to treat children under 16 years of age in Iran since 2017. The present study assesses the QOL of these patients.

METHODS

Patients aged 8-16 years with severe hemophilia A and B receiving escalating LDP were included. Patients and parents completed the Hemophilia Quality of Life Questionnaire for Children (Haemo-QOL).

RESULTS

Twenty-five children and 15 adolescents were included. The total score for children and parents was 39.7 ± 14.5 and 39.1 ± 15, respectively (p = 0.88). The total score for adolescents and parents was 36.5 ± 15 and 41 ± 15.3, respectively (p = 0.42). The domains of friends and treatment in children and domains of friends and family in parents of children had the highest and lowest scores, respectively. The domains of sport/school and coping in adolescents and the domains of physical health and coping in parents of adolescents had the highest and lowest scores, respectively. The overall QOL of adolescents was better than that of children.

CONCLUSION

LDP resulted in a marked improvement in most domains of QOL. LDP can be appropriate for societies with limited access to factor concentrate.