HSR20-111: Characteristics, Treatment Patterns, Healthcare Resource Use and Costs among Pediatric Patients Diagnosed with Neurofibromatosis Type 1 and Plexiform Neurofibromas: A Retrospective Database Analysis

Background: Neurofibromatosis type 1 (NF1) is a rare, clinically heterogenous, progressive genetic disorder with 20-50% of patients with NF1 developing plexiform neurofibroma (PN) which are benign nerve sheath tumors. NF1-related PN can cause substantial morbidity including pain, motor dysfunction and disfigurement. There are currently no drug treatments approved for the treatment of PN. Real-world data among pediatric patients with NF1 and PN was limited. The objectives of this study were to describe the characteristics, treatment patterns, healthcare resource use (HRU) and costs for these patients. ​ Methods: A retrospective study of individuals enrolled in the MarketScan ® Commercial Claims and Encounters claims database from 10/2014-03/2018 (study period) was conducted. Patients ≤18 years of age at the index date with ≥1 ICD-10-CM diagnosis code for NF1 and PN were included. The index date was defined as the date of the first diagnosis of NF1 or PN during the study period, whichever occurred later. Continuous enrollment from the baseline period (12 months before index) was required. Patients were observed up to the earliest date of end of data availability or end of continuous enrollment in a health plan. Descriptive analysis was conducted to describe patients’ baseline characteristics and follow-up treatment patterns. All-cause HRU (inpatient, outpatient [OP], emergency room, pharmacy and other) and their associated costs during the follow-up period were calculated per patient per year [PPPY] in 2018 USD. Results: Among 301 patients identified with a mean follow-up for 496 days, mean age ± standard deviation (SD) was 11.7± 4.6 years; 50.8% were female. Most diagnoses at index were done by a specialist (77.1%). The most common diagnosing specialists were neurologists (14.0%), ophthalmologists (9.6%), pediatricians (8.0%) and radiologists (8.0%). During the follow-up period, pain medications were used by 44.2% of patients; 23.9% were treated with chemotherapy, 5.0% received surgery for PN, 1.3% received radiation, and 0.7% received MEK inhibitors. Mean ± SD [median] total PPPY healthcare costs were $38,292 ± 80,556 [$16,037], with OP PPPY costs as the main drivers of total cost ($22,297 ± 34,199 [$13,650]). Conclusions: This study demonstrated that a proportion of pediatric patients with NF1 and PN were treated with supportive care only, highlighting a substantial unmet need. This study also highlighted the considerable economic burden of NF1 and PN.

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Corresponding Author: Xiaoqin Yang, PhD
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