Background/Objectives: The aim of this study was to assess the unmet needs of myelodysplastic neoplasm (MDS) patients and their caregivers, focusing on how these needs impact quality of life (QoL) and daily functioning. MDS predominantly affects older adults. It is often complicated by severe red blood cell transfusion-dependent anemia and may require frequent hospital visits, conferring a substantial burden on patients and caregivers. Methods: A national survey was conducted between June 2022 and May 2023 in 46 hematology centers across Italy, involving 259 patients and 105 caregivers. The survey included validated QoL tools (QOL-E and HM-PRO) to measure the impact of disease and treatments on health-related QoL and symptoms. Results: Of the 259 patients surveyed, 42% were transfusion-dependent, with 45% reporting distress related to hospital travel, which was significantly associated with lower QoL scores (QOL-E physical score 50.0 vs. 62.5,p< 0.001). Transfusion dependency led to worse outcomes across physical, emotional, and social domains (HM-PRO Part A score 59.8 vs. 23.7,p< 0.001). Anxiety affected 66% of patients, while 56% reported feeling emotionally distressed. Forty-eight percent of patients required a caregiver, and among caregivers, 29% reported significant disruption to their work, including changing their job or reduced hours. Patients requiring frequent hospital visits showed notably worse QoL scores (HM-PRO emotional score 56.8 vs. 31.8,p< 0.001). Conclusions: This study identified substantial unmet needs for MDS patients, particularly in addressing the heavy burden of transfusions and hospital visits. Both patients and caregivers experienced significant impact on daily life and on QoL, highlighting the urgent need for treatments that reduce hospital dependency, improve patient outcomes, and alleviate the caregiver burden.
背景/目的：本研究旨在评估骨髓增生异常肿瘤（MDS）患者及其照护者未被满足的需求，重点关注这些需求如何影响生活质量及日常功能。MDS主要影响老年人群，常因严重的红细胞输注依赖性贫血而复杂化，且可能需要频繁就医，给患者和照护者带来沉重负担。方法：2022年6月至2023年5月期间，在意大利46个血液中心开展全国性调查，共纳入259名患者和105名照护者。调查采用经过验证的生活质量评估工具（QOL-E和HM-PRO），以评估疾病及治疗对健康相关生活质量和症状的影响。结果：在259名受访患者中，42%存在输血依赖，其中45%报告因就医交通问题产生困扰，这与较低的生活质量评分显著相关（QOL-E生理评分50.0 vs. 62.5，p<0.001）。输血依赖导致患者在生理、情感及社会功能领域的结果均更差（HM-PRO A部分评分59.8 vs. 23.7，p<0.001）。66%的患者存在焦虑情绪，56%报告情感困扰。48%的患者需要照护者支持，而照护者中有29%表示工作受到严重影响，包括更换工作或减少工作时间。需要频繁就医的患者生活质量评分显著更低（HM-PRO情感评分56.8 vs. 31.8，p<0.001）。结论：本研究揭示了MDS患者存在大量未被满足的需求，尤其在应对输血和就医带来的沉重负担方面。患者和照护者的日常生活及生活质量均受到显著影响，这凸显了亟需开发能减少医院依赖、改善患者预后并减轻照护者负担的治疗方案。
肿瘤(癌症)患者之家