Research Centre on Public Health (CESP), University of Milano-Bicocca, Monza, Italy.
IRCCS Multimedica, Sesto San Giovanni, Italy.
Haemophilia. 2022 Mar;28(2):254-263. doi: 10.1111/hae.14504. Epub 2022 Feb 8.
Haemophilia management and patients' quality of life significantly improved. However, data on current patients', caregivers' and clinicians' satisfaction and limitations of treatments and haemophilia management are limited.
Assessing the management satisfaction and unmet needs from the perspective of Italian patients with haemophilia (PWH) without inhibitors (or caregivers if children) and of specialist physicians.
Surveys (for patients≥18 years, caregivers of children and haemophilia specialists) were developed by a multidisciplinary working group and conducted from November 2019 to June 2020.
Among 275 participants, 120 (43.6%) were PWH without inhibitors, 79 (28.7%) caregivers and 37 (13.4%) clinicians. Patients and caregivers perceived a higher control of the disease compared to clinicians. However, more than 40% of patients and caregivers reported to feel significantly conditioned by the risk of bleeding during their daily life. PWH reported a 6-month mean/median (range) of bleeds 2.3/.0 (0-24) and caregivers 1.3/.0 (0-16) in children. The treatment burden (frequency of administration) was not satisfactory for more than half adults and caregivers of children treated with prophylaxis. A good access to treatment, haemophilia centres and medical service was reported, with issues associated to the multidisciplinary approach and treatment at emergency department.
This large national study provides an updated overview of haemophilia care in Italy from different points of views, highlighting positive aspects and unmet needs. This information can guide future interventions to improve haemophilia management and the assessment of impact of new treatment options.
血友病的管理和患者的生活质量显著改善。然而，目前关于患者、照顾者和临床医生对治疗和血友病管理的满意度以及治疗的局限性的数据有限。
从无抑制剂的意大利血友病患者（PWH）（或儿童的照顾者）和血友病专家的角度评估管理满意度和未满足的需求。
由一个多学科工作组制定调查问卷，并于 2019 年 11 月至 2020 年 6 月进行调查。
在 275 名参与者中，120 名（43.6%）为无抑制剂的 PWH，79 名（28.7%）为照顾者，37 名（13.4%）为临床医生。患者和照顾者认为对疾病的控制程度高于临床医生。然而，超过 40%的患者和照顾者报告说，他们在日常生活中因出血风险而感到受到严重限制。PWH 报告在 6 个月内平均/中位数（范围）出血 2.3/0（0-24），儿童照顾者为 1.3/0（0-16）。超过一半的接受预防治疗的成年患者和儿童照顾者对治疗负担（给药频率）不满意。报告称，有良好的治疗途径、血友病中心和医疗服务，但也存在与多学科方法和急诊治疗相关的问题。
这项大型国家研究从不同角度提供了意大利血友病护理的最新概述，强调了积极方面和未满足的需求。这些信息可以指导未来的干预措施，以改善血友病管理和评估新治疗方案的影响。
