Patient characteristics, treatment patterns, healthcare resource utilization, and costs among patients diagnosed with neurofibromatosis type 1 with and without plexiform neurofibromas in Japan

Abstract

Objectives

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.

Methods

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).

Results

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.

Conclusions

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.

Keywords:

• Neurofibromatosis 1
• neurofibroma, plexiform
• von Recklinghausen’s disease
• real-world evidence
• healthcare resource utilization

Transparency

Declaration of financial/other relationships

KT, RK, CI, and VB are full-time employees of AstraZeneca. YJK and LB are employees of Evidera, a business unit of PPD, which received consulting fees related to this work. YY received honoraria (lecture fee) from AstraZeneca. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

YY, KT, CI, and YJK contributed to the analysis of results. YJK wrote the manuscript with support and critical revisions by YY, KT and CI. RK and LB conceived the study and contributed to the analysis of the results. All authors had access to the data and gave final approval of the version to be published and agreed to be accountable for all aspects of the work.

Acknowledgements

None.

Data availability statement

The deidentified individual data that support the findings of this study are available from Medical Data Vision Co., Ltd. (MDV; Tokyo, Japan). Restrictions apply to the availability of these data, which were used under license for this study. Researchers looking to access the data should contact MDV via their website at: https://en.mdv.co.jp/.

Additional information

Funding

This study was funded by AstraZeneca as part of an alliance between AstraZeneca and Merck Sharp & Dohme Corp, a subsidiary of Merck & Co., Inc., Kenilworth, NJ, USA (MSD). AstraZeneca was also involved in study conception and protocol development.