Abstract：
BACKGROUND: Tumor-induced osteomalacia (TIO) is an ultra-rare, paraneoplastic syndrome caused by tumors that secrete fibroblast growth factor 23 (FGF23). Initial signs and musculoskeletal symptoms can be non-specific and unrecognized, leading to long delays in diagnosis and treatment, which results in severe and progressive disability in patients with TIO. This review aimed to identify published evidence on healthcare resource use in TIO to better understand the burden of the disease.
METHODS: A targeted literature review was conducted to identify publications reporting on disease characteristics and healthcare resource use associated with TIO.
RESULTS: In total, 414 publications were included in the review, of which 376 were case reports. From the case reports, data on 621 patients were extracted. These patients had a mean (standard deviation) age of 46.3 (15.8) years; 57.6% were male. Mean time from first symptoms to diagnosis of TIO was 4.6 (4.7) years and, in cases where imaging tests were reported, patients underwent a mean of 4.1 (2.7) procedures. Tumor resection was attempted in 81.0% of patients and successful in 67.0%. Fracture was reported in 49.3% of patients. Results from association analyses demonstrated that longer time to diagnosis was associated with poorer tumor resection outcomes and a higher probability of tumor recurrence. Unfavorable tumor resection outcomes were associated with greater use of pharmacologic treatment and a greater likelihood of orthopedic surgery.
CONCLUSIONS: TIO is associated with a substantial healthcare resource burden. Improvements in the diagnostic process could lead to better management of TIO, thereby benefiting patients and reducing that burden.
METHODS: A targeted literature review was conducted to identify publications reporting on disease characteristics and healthcare resource use associated with TIO.
RESULTS: In total, 414 publications were included in the review, of which 376 were case reports. From the case reports, data on 621 patients were extracted. These patients had a mean (standard deviation) age of 46.3 (15.8) years; 57.6% were male. Mean time from first symptoms to diagnosis of TIO was 4.6 (4.7) years and, in cases where imaging tests were reported, patients underwent a mean of 4.1 (2.7) procedures. Tumor resection was attempted in 81.0% of patients and successful in 67.0%. Fracture was reported in 49.3% of patients. Results from association analyses demonstrated that longer time to diagnosis was associated with poorer tumor resection outcomes and a higher probability of tumor recurrence. Unfavorable tumor resection outcomes were associated with greater use of pharmacologic treatment and a greater likelihood of orthopedic surgery.
CONCLUSIONS: TIO is associated with a substantial healthcare resource burden. Improvements in the diagnostic process could lead to better management of TIO, thereby benefiting patients and reducing that burden.
摘要：
背景：肿瘤诱导的骨软化症（TIO）是一种非常罕见的，由分泌成纤维细胞生长因子23（FGF23）的肿瘤引起的副肿瘤综合征。最初的体征和肌肉骨骼症状可以是非特异性的和无法识别的，导致诊断和治疗的长期延误，导致TIO患者严重和进行性残疾。这篇综述旨在确定TIO中有关医疗保健资源使用的已发表证据，以更好地了解该疾病的负担。
方法：进行了有针对性的文献综述，以确定报告与TIO相关的疾病特征和医疗资源使用的出版物。
结果：总计，审查中包括414份出版物，其中376例为病例报告。从病例报告来看,提取了621例患者的数据.这些患者的平均（标准差）年龄为46.3（15.8）岁；57.6％为男性。从首次症状到诊断TIO的平均时间为4.6（4.7）年，在报告影像学检查的情况下，患者平均接受4.1(2.7)次手术.81.0%的患者尝试进行肿瘤切除，67.0%成功。49.3%的患者报告骨折。关联分析的结果表明，诊断时间较长与较差的肿瘤切除结果和较高的肿瘤复发概率相关。不利的肿瘤切除结果与药物治疗的更多使用和骨科手术的更大可能性相关。
结论：TIO与巨大的医疗资源负担相关。诊断过程的改进可能会导致TIO的更好管理，从而使患者受益并减轻负担。
方法：进行了有针对性的文献综述，以确定报告与TIO相关的疾病特征和医疗资源使用的出版物。
结果：总计，审查中包括414份出版物，其中376例为病例报告。从病例报告来看,提取了621例患者的数据.这些患者的平均（标准差）年龄为46.3（15.8）岁；57.6％为男性。从首次症状到诊断TIO的平均时间为4.6（4.7）年，在报告影像学检查的情况下，患者平均接受4.1(2.7)次手术.81.0%的患者尝试进行肿瘤切除，67.0%成功。49.3%的患者报告骨折。关联分析的结果表明，诊断时间较长与较差的肿瘤切除结果和较高的肿瘤复发概率相关。不利的肿瘤切除结果与药物治疗的更多使用和骨科手术的更大可能性相关。
结论：TIO与巨大的医疗资源负担相关。诊断过程的改进可能会导致TIO的更好管理，从而使患者受益并减轻负担。
