Department of Psychosomatic Medicine and Psychotherapy, University Medical Center Hamburg-Eppendorf, Martinistr. 52, 20251, Hamburg, Germany.
Department of Cardiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
Orphanet J Rare Dis. 2021 Jan 22;16(1):44. doi: 10.1186/s13023-020-01660-w.
In the absence of a cure for the majority of rare diseases, the disease management aims to provide optimal supportive care. The goal of this study was to assess supportive care needs in patients with chronic rare diseases.
Cross-sectional mixed-method study was conducted using validated self-report scales and open-ended questions to assess supportive care needs. Participants affected by rare diseases across Germany were contacted via patient organizations and centers for rare diseases. N = 304 participants with 81 different rare diseases completed the study, 81.6% were female, mean age was 44.2 years (SD = 12.8, range 16-74). The quantitative results regarding supportive care needs were compared to a reference population of patients affected by cancer (N = 888). Main outcomes were unmet supportive care needs of patients with rare diseases, as assessed by the Supportive Care Needs Survey (SNCS-SF34) and an open-ended question on support wishes.
Patients with rare diseases did not feel sufficiently supported with regard to psychological support, health system and information, physical and daily living, patient care and support, and sexuality needs. The unmet supportive care needs were significantly higher in the patient sample with rare diseases compared to the SCNS-SF34 reference sample of patients with cancer. 60% of patients with rare diseases did not feel sufficiently socially supported.
Patients affected by rare diseases have high unmet support needs in all areas studied. Multidisciplinary care, including psychological support and the provision of information regarding the healthcare system, treatment options, disease course and sexuality, might help address these needs.
在大多数罕见病缺乏治愈方法的情况下，疾病管理旨在提供最佳的支持性护理。本研究的目的是评估慢性罕见病患者的支持性护理需求。
采用经过验证的自我报告量表和开放式问题进行横断面混合方法研究，以评估支持性护理需求。通过患者组织和罕见病中心联系德国各地患有罕见病的参与者。共有 304 名患有 81 种不同罕见病的参与者完成了这项研究，其中 81.6%为女性，平均年龄为 44.2 岁（SD=12.8，范围 16-74）。关于支持性护理需求的定量结果与癌症患者的参考人群（N=888）进行了比较。主要结果是罕见病患者的支持性护理需求未得到满足，由支持性护理需求调查（SNCS-SF34）和关于支持愿望的开放式问题评估。
罕见病患者在心理支持、卫生系统和信息、身体和日常生活、患者护理和支持以及性需求方面感到支持不足。罕见病患者样本中的未满足支持性护理需求明显高于癌症患者的 SNCS-SF34 参考样本。60%的罕见病患者感到社会支持不足。
受罕见病影响的患者在所有研究领域都存在高度未满足的支持需求。多学科护理，包括心理支持以及提供有关卫生系统、治疗选择、疾病过程和性的信息，可能有助于满足这些需求。
