Population pharmacokinetics and exposure–response of selumetinib and its N‐desmethyl metabolite in pediatric patients with neurofibromatosis type 1 and inoperable plexiform neurofibromas

2021 ◽  
Author(s):  
Stein Schalkwijk ◽  
Li Zhou ◽  
Sarit Cohen‐Rabbie ◽  
Lokesh Jain ◽  
Tomoko Freshwater ◽  
...  
Keyword(s):  
Population Pharmacokinetics ◽  
Neurofibromatosis Type 1 ◽  
Pediatric Patients ◽  
Neurofibromatosis Type ◽  
Plexiform Neurofibromas ◽  
Exposure Response

scholarly journals Treatment, Resource Use and Costs Among Pediatric Patients with Neurofibromatosis Type 1 and Plexiform Neurofibromas

2020 ◽  
Vol Volume 11 ◽  
pp. 421-428
Author(s):  
Xiaoqin Yang ◽  
Kaushal Desai ◽  
Neha Agrawal ◽  
Kirti Mirchandani ◽  
Sagnik Chatterjee ◽  
...  
Keyword(s):  
Resource Use ◽  
Neurofibromatosis Type 1 ◽  
Pediatric Patients ◽  
Neurofibromatosis Type ◽  
Plexiform Neurofibromas

scholarly journals Management of Head and Neck Plexiform Neurofibromas in Pediatric Patients With Neurofibromatosis Type 1

2005 ◽  
Vol 131 (8) ◽  
pp. 712 ◽  
Author(s):  
Jeffrey B. Wise ◽  
Jonathan E. Cryer ◽  
Jean B. Belasco ◽  
Ian Jacobs ◽  
Lisa Elden
Keyword(s):  
Head And Neck ◽  
Neurofibromatosis Type 1 ◽  
Pediatric Patients ◽  
Neurofibromatosis Type ◽  
Plexiform Neurofibromas

scholarly journals Population Pharmacokinetics of Sirolimus in Pediatric Patients With Neurofibromatosis Type 1

2013 ◽  
Vol 35 (3) ◽  
pp. 332-337 ◽  
Author(s):  
Jeffrey R. Scott ◽  
Joshua D. Courter ◽  
Shannon N. Saldaña ◽  
Brigitte C. Widemann ◽  
Michael Fisher ◽  
...  
Keyword(s):  
Population Pharmacokinetics ◽  
Neurofibromatosis Type 1 ◽  
Pediatric Patients ◽  
Neurofibromatosis Type

scholarly journals Activity of Selumetinib in Neurofibromatosis Type 1–Related Plexiform Neurofibromas

2016 ◽  
Vol 375 (26) ◽  
pp. 2550-2560 ◽  
Author(s):  
Eva Dombi ◽  
Andrea Baldwin ◽  
Leigh J. Marcus ◽  
Michael J. Fisher ◽  
Brian Weiss ◽  
...  
Keyword(s):  
Neurofibromatosis Type 1 ◽  
Neurofibromatosis Type ◽  
Plexiform Neurofibromas

scholarly journals Neurofibromatosis Type 1-related Multiple Plexiform Neurofibromas: A Case Report

2020 ◽  
Vol 26 (1) ◽  
pp. 42-46
Author(s):  
Tamer Bayram ◽  
Derya Bayram ◽  
Hülya Tireli
Keyword(s):  
Case Report ◽  
Neurofibromatosis Type 1 ◽  
Neurofibromatosis Type ◽  
Plexiform Neurofibromas

scholarly journals Characteristics, Treatment Patterns, Healthcare Resource Use, and Costs among Pediatric Patients Diagnosed with Neurofibromatosis Type 1 and Plexiform Neurofibromas: A Retrospective Database Analysis of a Medicaid Population

2020 ◽  
Author(s):  
Xiaoqin Yang ◽  
Kaushal Desai ◽  
Neha Agrawal ◽  
Kirti Mirchandani ◽  
Sagnik Chatterjee ◽  
...  
Keyword(s):  
Resource Use ◽  
Neurofibromatosis Type 1 ◽  
Pediatric Patients ◽  
Healthcare Resource ◽  
Neurofibromatosis Type ◽  
Treatment Patterns ◽  
Healthcare Resource Use ◽  
The Mean

Abstract Background: Neurofibromatosis type 1 (NF1)-related plexiform neurofibromas (PN) can cause substantial morbidity by disfigurement and compression of vital structures. Real-world data on the burden and cost of disease among pediatric patients with NF1 and PN is limited. The objectives of this study were to describe the characteristics, treatment patterns, healthcare resource use (HCRU), and costs of these patients.Results: A total of 383 patients were included in the retrospective analysis of patients aged ≤18 with at least 1 ICD-10-CM diagnosis code for both NF1 and PN enrolled in the MarketScan® Multistate Medicaid database from October 1, 2014 to December 31, 2017. The mean follow-up was 448 days. The mean age was 11.4 years and 52.0% of patients were male. 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 per patient per year inpatient, outpatient, emergency room, pharmacy, and other visits were 1.4, 17.3, 1.6, 13.6, and 25.8, respectively. Mean ±SD (median) total per patient per year healthcare costs (2018 USD) were $17,275 ±$61,903 ($2,889), with total medical costs of $14,628 ±$56,203 ($2,334) and pharmacy costs of $2,646 ±$13,303 ($26). Inpatient costs were the largest drivers of medical cost, with a mean per patient per year cost of $6,739.Conclusions: This study showed that many pediatric patients diagnosed with NF1 and PN were 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.

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