Abstract：
The objectives of this study were to retrospectively investigate the patient characteristics, treatment patterns, healthcare resource utilization (HCRU), and healthcare costs related to management of neurofibromatosis type 1 (NF1) in Japan.
Cohorts of NF1 patients with or without plexiform neurofibromas (PN) were identified from the Medical Data Vision database in 2008-2019. Baseline characteristics, NF1 medications, HCRU, and associated costs were assessed using descriptive statistics. All-cause HCRU and costs following the first confirmed NF1 diagnosis date were analyzed per patient per year (PPPY) in Japanese Yen (JPY) and United States Dollar (USD).
A total of 4394 NF1 patients without PN and 370 NF1 patients with PN were identified. The mean age was 35.0 and 36.9 years, respectively. The proportion of patients with PN treated with medications was higher than that in patients without PN (except for antirheumatic/immunologic agents). Analgesics/non-steroidal anti-inflammatory drugs were the most frequently prescribed NF1 medications (44.3% and 56.0% in patients without and with PN, respectively), followed by inpatient prescriptions of opioids/opioid-like agents (17.8% and 27.6%, respectively). Inpatient admissions accounted for the highest costs in both cohorts with the average cost PPPY being JPY 2,133,277 (USD 19,861) for patients without PN and JPY 1,052,868 (USD 9802) for patients with PN.
NF1 is treated primarily with supportive care with analgesics/non-steroidal anti-inflammatory drugs being the most frequently prescribed NF1 medications in Japan. Findings underscored the unmet need and substantial economic burden among patients with NF1 and highlighted the need for new treatment options for patients with this disease.
Cohorts of NF1 patients with or without plexiform neurofibromas (PN) were identified from the Medical Data Vision database in 2008-2019. Baseline characteristics, NF1 medications, HCRU, and associated costs were assessed using descriptive statistics. All-cause HCRU and costs following the first confirmed NF1 diagnosis date were analyzed per patient per year (PPPY) in Japanese Yen (JPY) and United States Dollar (USD).
A total of 4394 NF1 patients without PN and 370 NF1 patients with PN were identified. The mean age was 35.0 and 36.9 years, respectively. The proportion of patients with PN treated with medications was higher than that in patients without PN (except for antirheumatic/immunologic agents). Analgesics/non-steroidal anti-inflammatory drugs were the most frequently prescribed NF1 medications (44.3% and 56.0% in patients without and with PN, respectively), followed by inpatient prescriptions of opioids/opioid-like agents (17.8% and 27.6%, respectively). Inpatient admissions accounted for the highest costs in both cohorts with the average cost PPPY being JPY 2,133,277 (USD 19,861) for patients without PN and JPY 1,052,868 (USD 9802) for patients with PN.
NF1 is treated primarily with supportive care with analgesics/non-steroidal anti-inflammatory drugs being the most frequently prescribed NF1 medications in Japan. Findings underscored the unmet need and substantial economic burden among patients with NF1 and highlighted the need for new treatment options for patients with this disease.
摘要：
■这项研究的目的是回顾性调查患者的特征，治疗模式,医疗保健资源利用(HCCU)，以及与日本1型神经纤维瘤病（NF1）管理相关的医疗费用。
■在2008-2019年从医学数据视觉数据库中确定了有或没有丛状神经纤维瘤（PN）的NF1患者队列。基线特征，NF1药物，HCCU,和相关成本使用描述性统计数据进行评估。对每位患者每年（PPPY）的日元（JPY）和美元（USD）分析了首次确认NF1诊断日期后的全因HCRU和费用。
■总共有4394例无PN的NF1患者和370例有PN的NF1患者。平均年龄为35.0岁和36.9岁，分别。接受药物治疗的PN患者比例高于无PN患者（抗风湿/免疫药物除外）。止痛药/非甾体类抗炎药是最常用的NF1药物（无PN和有PN的患者分别为44.3%和56.0%，分别)，其次是阿片类药物/类阿片类药物的住院处方(17.8%和27.6%，分别)。在这两个队列中，住院费用最高，无PN患者的平均费用PPPY为2,133,277日元（19,861美元），PN患者的平均费用为1,052,868日元（9802美元）。
■NF1主要采用镇痛药/非甾体抗炎药的支持性治疗，这是日本最常用的NF1处方药。研究结果强调了NF1患者未满足的需求和巨大的经济负担，并强调了对该疾病患者的新治疗选择的需求。
■在2008-2019年从医学数据视觉数据库中确定了有或没有丛状神经纤维瘤（PN）的NF1患者队列。基线特征，NF1药物，HCCU,和相关成本使用描述性统计数据进行评估。对每位患者每年（PPPY）的日元（JPY）和美元（USD）分析了首次确认NF1诊断日期后的全因HCRU和费用。
■总共有4394例无PN的NF1患者和370例有PN的NF1患者。平均年龄为35.0岁和36.9岁，分别。接受药物治疗的PN患者比例高于无PN患者（抗风湿/免疫药物除外）。止痛药/非甾体类抗炎药是最常用的NF1药物（无PN和有PN的患者分别为44.3%和56.0%，分别)，其次是阿片类药物/类阿片类药物的住院处方(17.8%和27.6%，分别)。在这两个队列中，住院费用最高，无PN患者的平均费用PPPY为2,133,277日元（19,861美元），PN患者的平均费用为1,052,868日元（9802美元）。
■NF1主要采用镇痛药/非甾体抗炎药的支持性治疗，这是日本最常用的NF1处方药。研究结果强调了NF1患者未满足的需求和巨大的经济负担，并强调了对该疾病患者的新治疗选择的需求。
