Abstract

Objectives

The objectives of this study were to describe the characteristics and initial treatment patterns, healthcare resource use (HCRU), and costs of patients newly diagnosed with neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (PN).

Methods

This was a retrospective study of individuals enrolled in the IBM MarketScan Multi-State Medicaid database from 1 October 2014 to 31 December 2017. Patients aged ≤18 years at the index date (first diagnosis of NF1 or PN, whichever occurred later) with at least 1 ICD-10-CM diagnosis code for both NF1 and PN were included. All-cause HCRU and the associated direct costs during the follow-up period were calculated per patient per year (PPPY) in 2018 USD.

Results

A total of 383 patients were included with a mean follow-up of 448 days. Most patients were diagnosed by a specialist (63.5%). During the follow-up period, pain medications were used by 58.5% of patients, 25.1% were treated with chemotherapy, 7.1% received surgery for PN, 1.6% received MEK inhibitors, and 0.8% received radiation. Mean PPPY inpatient, outpatient, ER, pharmacy, and other visits were 1.4, 17.3, 1.6, 13.6, and 25.8, respectively. Mean ± SD (median) total PPPY healthcare costs were $17,275 ± $61,903 ($2889), with total medical costs of $14,628 ± $56,203 ($2334) and pharmacy costs of $2646 ± $13,303 ($26).

Conclusions

This study showed that many pediatric patients newly diagnosed with NF1 and PN were initially treated with supportive care only, highlighting a substantial unmet medical need. This study also highlights the considerable economic burden among patients with NF1 and PN.

中文翻译：

---

诊断为 1 型神经纤维瘤病和丛状神经纤维瘤的儿科患者的特征、治疗模式、医疗资源使用和成本：对医疗救助人群的回顾性数据库分析

摘要

目标

本研究的目的是描述新诊断为 1 型神经纤维瘤病 (NF1) 相关丛状神经纤维瘤 (PN) 的患者的特征和初始治疗模式、医疗资源使用 (HCRU) 和成本。

方法

这是一项对 2014 年 10 月 1 日至 2017 年 12 月 31 日在 IBM MarketScan Multi-State Medicaid 数据库中登记的个人的回顾性研究。包括 1 个 ICD-10-CM NF1 和 PN 诊断代码。全因 HCRU 和随访期间的相关直接成本按 2018 美元计算每名患者每年 (PPPY)。

结果

总共包括 383 名患者，平均随访时间为 448 天。大多数患者由专科医生诊断（63.5%）。在随访期间，58.5% 的患者使用止痛药，25.1% 接受化疗，7.1% 接受手术治疗 PN，1.6% 接受 MEK 抑制剂，0.8% 接受放射治疗。平均 PPPY 住院、门诊、急诊室、药房和其他就诊次数分别为 1.4、17.3、1.6、13.6 和 25.8。平均±标准差（中位数）PPPY 医疗保健总成本为 17,275 美元 ± 61,903 美元（2889 美元），总医疗成本为 14,628 美元 ± 56,203 美元（2334 美元），药房成本为 2646 美元 ± 13,303 美元（26 美元）。

结论

该研究表明，许多新诊断为 NF1 和 PN 的儿科患者最初仅接受支持性护理，突出了大量未满足的医疗需求。该研究还强调了 NF1 和 PN 患者的巨大经济负担。