Gimenez Emmanuel, Besses Carles, Boque Concepcion, Velez Patricia, Kerguelen Ana, Cervantes Francisco, Ferrer-Marin Francisca, Perez-Encinas Manuel, Rodriguez Mercedes, Gonzalez Juan Diego, Calzada Reyes, Hernandez-Boluda Juan Carlos
Antares-Consulting , Barcelona , Spain.
J Med Econ. 2014 Jun;17(6):435-41. doi: 10.3111/13696998.2014.903257. Epub 2014 Apr 9.
Myelofibrosis is a non-frequent chronic myeloproliferative Philadelphia-negative chromosome neoplasm. It is a heavy incapacitating orphan disease and associated with high morbidity and mortality. In this context, indirect and non-medical costs are expected to be high. The main objective of this project is to estimate the economic burden of this disease in Spain.
Thirty-three patients with a diagnosis of myelofibrosis for at least 1 year participated in a questionnaire in three Spanish centers. The study consisted of analyzing in various aspects the cost and impact of the disease; indeed, daily life time limitations with a need of informal care, symtomatology. Additionally, information concerning the clinical management of the disease was collected through a focus group of eight experts.
The mean age was 65 years. 15 of 33 patients were at their productive stage. Six had difficulties at work and eight have received informal care. Bone and muscular pain were the main symptoms of patients (72%). The estimated global indirect and non-medical costs of the disease were 86,315€ per patient (20% working and 80% informal care), which reached 104,153€ at productive stage patients (45%) and 168,459€ for more symptomatic patients.
The economic burden of indirect and non-medical costs of myelofibrosis are important (15,142€/annual) as a result, and should be considered in economic evaluation, as well as in preventive plans for patients and caregivers, despite the fact that studies with larger numbers of patients should be done.
骨髓纤维化是一种不常见的慢性骨髓增殖性费城染色体阴性肿瘤。它是一种严重致残的罕见疾病，发病率和死亡率都很高。在这种情况下，间接和非医疗成本预计也会很高。本项目的主要目标是估计西班牙这种疾病的经济负担。
33名诊断为骨髓纤维化至少1年的患者参与了西班牙三个中心的问卷调查。该研究包括从各个方面分析疾病的成本和影响；实际上，包括日常生活时间限制、对非正式护理的需求、症状学。此外，通过一个由八名专家组成的焦点小组收集了有关该疾病临床管理的信息。
平均年龄为65岁。33名患者中有15名处于生产阶段。6人在工作上有困难，8人接受了非正式护理。骨骼和肌肉疼痛是患者的主要症状（72%）。该疾病估计的全球间接和非医疗成本为每位患者86,315欧元（20%为工作成本，80%为非正式护理成本），在处于生产阶段的患者中达到104,153欧元（45%），症状更严重的患者为168,459欧元。
因此，骨髓纤维化间接和非医疗成本的经济负担很重（每年15,142欧元），在经济评估以及针对患者和护理人员的预防计划中都应予以考虑，尽管应该开展更多患者数量的研究。
