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

IF 1.7 4区医学 Q3 HEMATOLOGY

International Journal of Hematology Pub Date : 2025-04-03 DOI:10.1007/s12185-025-03969-3

Zahra Badiei, Atefeh Mokhtari, Hamid Farhangi, Maryam Heidarian, Elham Bakhtiari

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引用次数: 0

Abstract

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.

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目的：与发作性治疗相比，低剂量预防性治疗（LDP）可减少严重A型和B型血友病患者的自发性出血，从而提高生活质量（QOL）。本研究对这些患者的 QOL 进行了评估：研究纳入了接受LDP升级治疗的8-16岁重症血友病A型和B型患者。患者和家长填写了儿童血友病生活质量问卷（Haemo-QOL）：结果：共纳入 25 名儿童和 15 名青少年。儿童和家长的总分分别为（39.7 ± 14.5）分和（39.1 ± 15）分（p = 0.88）。青少年和家长的总分分别为（36.5 ± 15）和（41 ± 15.3）（P = 0.42）。儿童的朋友和治疗领域以及儿童家长的朋友和家庭领域的得分分别最高和最低。青少年的运动/学校和应对领域以及青少年父母的身体健康和应对领域的得分分别最高和最低。青少年的总体 QOL 优于儿童：结论：LDP 能明显改善大多数人的 QOL。LDP 适用于因数集中机会有限的社会。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

International Journal of Hematology 医学-血液学

CiteScore

3.90

自引率

4.80%

发文量

223

审稿时长

6 months

期刊介绍： The International Journal of Hematology, the official journal of the Japanese Society of Hematology, has a long history of publishing leading research in hematology. The journal comprises articles that contribute to progress in research not only in basic hematology but also in clinical hematology, aiming to cover all aspects of this field, namely, erythrocytes, leukocytes and hematopoiesis, hemostasis, thrombosis and vascular biology, hematological malignancies, transplantation, and cell therapy. The expanded [Progress in Hematology] section integrates such relevant fields as the cell biology of stem cells and cancer cells, and clinical research in inflammation, cancer, and thrombosis. Reports on results of clinical trials are also included, thus contributing to the aim of fostering communication among researchers in the growing field of modern hematology. The journal provides the best of up-to-date information on modern hematology, presenting readers with high-impact, original work focusing on pivotal issues.