Light Chain Amyloidosis: Patient Experience Survey from the Amyloidosis Research Consortium
Light Chain Amyloidosis: Patient Experience Survey from the Amyloidosis Research Consortium
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DOI：
10.1007/s12325-015-0250-0
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发表时间：
2015-10-01
影响因子：
3.8
通讯作者：
Merlini, Giampaolo
中科院分区：
文献类型：
--
作者：
Lousada, Isabelle;Comenzo, Raymond L.;Merlini, Giampaolo
引言：缺乏轻链（AL）淀粉样变性患者经验的详细信息。本研究的主要目的是收集患者的经验，了解诊断的挑战，并深入了解障碍，以获得适当的care.Methods：淀粉样变性患者，家庭成员和照顾者被邀请参加一个在线的16个问题的调查（可从2015年1月29日至2月5日）。AL淀粉样变性的参与者被发送了一个八个问题的后续调查。结果：初始调查完成了533名参与者（后续调查完成了201名参与者）。AL淀粉样变性是最常见的诊断。对于37.1%的受访者，淀粉样变性的诊断直到初始症状发作后>= 1年才确定。分别有7.6%、23.5%、20.3%、16.8%和31.8%的受访者在就诊1、2、3、4或>= 5名医生后得到诊断。血液科/肿瘤科医生最常作出正确诊断（34.1%）。治疗包括化疗（63.1%）和干细胞移植（38.9%），54.1%的受访者难以耐受。相当多的受访者对临床试验感到不知情。然而，大约一半（46.1%）的人认为，参加试验将提高他们的care.Conclusions：建立淀粉样变性的诊断是困难的。目前的治疗难以耐受，并且不能显著改善大多数患者的生活质量。迫切需要具有明确治疗获益的耐受性良好的治疗方法。可以提高患者对临床试验的认识，特别是考虑到受访者表示参与意愿很高。
Introduction: Information detailing the experience of patients with light chain (AL) amyloidosis is lacking. The primary aim of this study was to gather data on the patient experience to understand the challenges in diagnosis and to gain insight into barriers to accessing appropriate care.Methods: Patients with amyloidosis, family members, and caregivers were invited to participate in an online 16-question survey (available from January 29 to February 5, 2015). Participants with AL amyloidosis were sent an eight-question follow-up survey.Results: The initial survey was completed by 533 participants (follow-up survey completed by 201 participants). AL amyloidosis was the most common diagnosis. For 37.1% of respondents, the diagnosis of amyloidosis was not established until >= 1 year after the onset of initial symptoms. Diagnosis was received after visits to 1, 2, 3, 4, or >= 5 physicians by 7.6%, 23.5%, 20.3%, 16.8%, and 31.8% of respondents, respectively. Correct diagnosis was most often made by hematologists/ oncologists (34.1%). Treatments included chemotherapy (63.1%) and stem cell transplantation (38.9%) and were difficult to tolerate for 54.1% of respondents. A significant number of respondents felt uninformed about clinical trials. Nevertheless, approximately half (46.1%) believed that enrolling in a trial would enhance their care.Conclusions: Establishing a diagnosis of amyloidosis is difficult. Current treatments are difficult to tolerate and do not substantially improve quality of life for most patients. There is an urgent need for well-tolerated therapies with clear treatment benefit. Patient awareness of clinical trials can be improved, especially given that respondents indicated high willingness to participate.