Badiei Zahra, Mokhtari Atefeh, Farhangi Hamid, Heidarian Maryam, Bakhtiari Elham
Department of Pediatrics, School of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran.
School of Medicine, Mashhad University of Medical Sciences, Mashhad, Iran.
Int J Hematol. 2025 Aug;122(2):277-283. doi: 10.1007/s12185-025-03969-3. Epub 2025 Apr 3.
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.
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).
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.
LDP resulted in a marked improvement in most domains of QOL. LDP can be appropriate for societies with limited access to factor concentrate.
与按需治疗相比，低剂量预防治疗（LDP）通过减少重度甲型和乙型血友病患者的自发性出血，可提高生活质量（QOL）。自2017年以来，LDP已在伊朗用于治疗16岁以下儿童。本研究评估了这些患者的生活质量。
纳入年龄在8 - 16岁、接受递增LDP治疗的重度甲型和乙型血友病患者。患者及其父母完成了儿童血友病生活质量问卷（Haemo-QOL）。
纳入25名儿童和15名青少年。儿童及其父母的总分分别为39.7±14.5和39.1±15（p = 0.88）。青少年及其父母的总分分别为36.5±15和41±15.3（p = 0.42）。儿童的朋友和治疗领域得分最高，而儿童父母的朋友和家庭领域得分最低。青少年的运动/学校和应对领域得分最高，青少年父母的身体健康和应对领域得分最低。青少年的总体生活质量优于儿童。
LDP使生活质量的大多数领域有显著改善。LDP适用于凝血因子浓缩剂获取受限的社会。
