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

References

• Neurofibromatosis. Conference statement. National Institutes of health consensus development conference. Arch Neurol. 1988;45(5):575–578.3128965
   PubMedGoogle Scholar
• Korf BR. Plexiform neurofibromas. Am J Med Genet. 1999;89(1):31–37. doi:10.1002/(SICI)1096-8628(19990326)89:1<31::AID-AJMG7>3.0.CO;2-W10469434
   PubMed Web of Science ®Google Scholar
• Evans DG, O’Hara C, Wilding A, et al. Mortality in neurofibromatosis 1: in North West England: an assessment of actuarial survival in a region of the UK since 1989. Eur J Hum Genet. 2011;19(11):1187–1191. doi:10.1038/ejhg.2011.11321694737
   PubMed Web of Science ®Google Scholar
• Masocco M, Kodra Y, Vichi M, et al. Mortality associated with neurofibromatosis type 1: a study based on Italian death certificates (1995–2006). Orphanet J Rare Dis. 2011;6(1):11. doi:10.1186/1750-1172-6-1121439034
   PubMed Web of Science ®Google Scholar
• Rasmussen SA, Yang Q, Friedman JM. Mortality in neurofibromatosis 1: an analysis using U.S. death certificates. Am J Hum Genet. 2001;68(5):1110–1118. doi:10.1086/32012111283797
   PubMed Web of Science ®Google Scholar
• Gutmann DH, Ferner RE, Listernick RH, Korf BR, Wolters PL, Johnson KJ. Neurofibromatosis type 1. Nat Rev Dis Primers. 2017;3(1):17004. doi:10.1038/nrdp.2017.428230061
   PubMed Web of Science ®Google Scholar
• Uusitalo E, Leppavirta J, Koffert A, et al. Incidence and mortality of neurofibromatosis: a total population study in Finland. J Invest Dermatol. 2015;135(3):904–906. doi:10.1038/jid.2014.46525354145
   PubMed Web of Science ®Google Scholar
• Ferner RE, Huson SM, Thomas N, et al. Guidelines for the diagnosis and management of individuals with neurofibromatosis 1. J Med Genet. 2007;44(2):81–88. doi:10.1136/jmg.2006.04590617105749
   PubMed Web of Science ®Google Scholar
• DeBella K, Szudek J, Friedman JM. Use of the national institutes of health criteria for diagnosis of neurofibromatosis 1 in children. Pediatrics. 2000;105(3 Pt 1):608–614. doi:10.1542/peds.105.3.60810699117
   PubMed Web of Science ®Google Scholar
• Mautner VF, Asuagbor FA, Dombi E, et al. Assessment of benign tumor burden by whole-body MRI in patients with neurofibromatosis 1. Neuro Oncol. 2008;10(4):593–598. doi:10.1215/15228517-2008-01118559970
   PubMed Web of Science ®Google Scholar
• Karaconji T, Whist E, Jamieson RV, Flaherty MP, Grigg JRB. Neurofibromatosis type 1: review and update on emerging therapies. Asia Pac J Ophthalmol (Phila). 2019;8(1):62–72. doi:10.22608/APO.201818230387339
   PubMed Web of Science ®Google Scholar
• Nguyen R, Kluwe L, Fuensterer C, Kentsch M, Friedrich RE, Mautner VF. Plexiform neurofibromas in children with neurofibromatosis type 1: frequency and associated clinical deficits. J Pediatr. 2011;159(4):652–655 e652. doi:10.1016/j.jpeds.2011.04.00821621223
   PubMed Web of Science ®Google Scholar
• Lai JS, Jensen SE, Charrow J, Listernick R. Patient reported outcomes measurement information system and quality of life in neurological disorders measurement system to evaluate quality of life for children and adolescents with neurofibromatosis type 1 associated plexiform neurofibroma. J Pediatr. 2019;206:190–196. doi:10.1016/j.jpeds.2018.10.01930413310
   PubMed Web of Science ®Google Scholar
• Korf BR. Malignancy in neurofibromatosis type 1. Oncologist. 2000;5(6):477–485. doi:10.1634/theoncologist.5-6-47711110599
   PubMedGoogle Scholar
• Evans DG, Baser ME, McGaughran J, Sharif S, Howard E, Moran A. Malignant peripheral nerve sheath tumours in neurofibromatosis 1. J Med Genet. 2002;39(5):311–314. doi:10.1136/jmg.39.5.31112011145
   PubMed Web of Science ®Google Scholar
• Ducatman BS, Scheithauer BW, Piepgras DG, Reiman HM, Ilstrup DM. Malignant peripheral nerve sheath tumors. A clinicopathologic study of 120 cases. Cancer. 1986;57(10):2006–2021. doi:10.1002/1097-0142(19860515)57:10<2006::AID-CNCR2820571022>3.0.CO;2-63082508
   PubMed Web of Science ®Google Scholar
• Karajannis MA, Ferner RE. Neurofibromatosis-related tumors: emerging biology and therapies. Curr Opin Pediatr. 2015;27(1):26–33. doi:10.1097/MOP.000000000000016925490687
   PubMed Web of Science ®Google Scholar
• Gutmann DH, Blakeley JO, Korf BR, Packer RJ. Optimizing biologically targeted clinical trials for neurofibromatosis. Expert Opin Investig Drugs. 2013;22(4):443–462. doi:10.1517/13543784.2013.772979
   PubMed Web of Science ®Google Scholar
• U.S. Food and Drug Administration. FDA Approves First Therapy for Children with Debilitating and Disfiguring Rare Disease; 2020.
   Google Scholar
• U.S. Food and Drug Administration. KOSELUGO (selumetinib) capsules [Prescribing information]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213756s000lbl.pdf. Accessed 5 4, 2020.
   Google Scholar
• Gross AM, Singh G, Akshintala S, et al. Association of plexiform neurofibroma volume changes and development of clinical morbidities in neurofibromatosis 1. Neuro Oncol. 2018;20(12):1643–1651. doi:10.1093/neuonc/noy06729718344
   PubMed Web of Science ®Google Scholar
• Wolters PL, Burns KM, Martin S, et al. Pain interference in youth with neurofibromatosis type 1 and plexiform neurofibromas and relation to disease severity, social-emotional functioning, and quality of life. Am J Med Genet A. 2015;167A(9):2103–2113. doi:10.1002/ajmg.a.3712325976979
   PubMed Web of Science ®Google Scholar
• Martin S, Wolters P, Baldwin A, et al. Social-emotional functioning of children and adolescents with neurofibromatosis type 1 and plexiform neurofibromas: relationships with cognitive, disease, and environmental variables. J Pediatr Psychol. 2012;37(7):713–724. doi:10.1093/jpepsy/jsr12422353803
   PubMed Web of Science ®Google Scholar
• IBM. Data brochure: research databases for life sciences researchers. Available from: https://www.ibm.com/downloads/cas/OWZWJ0QO. Accessed 52, 2020.
   Google Scholar
• Sundararajan V, Henderson T, Perry C, Muggivan A, Quan H, Ghali WA. New ICD-10 version of the Charlson comorbidity index predicted in-hospital mortality. J Clin Epidemiol. 2004;57(12):1288–1294. doi:10.1016/j.jclinepi.2004.03.01215617955
   PubMed Web of Science ®Google Scholar
• Deyo RA, Cherkin DC, Ciol MA. Adapting a clinical comorbidity index for use with ICD-9-CM administrative databases. J Clin Epidemiol. 1992;45(6):613–619. doi:10.1016/0895-4356(92)90133-81607900
   PubMed Web of Science ®Google Scholar
• Quan H, Sundararajan V, Halfon P, et al. Coding algorithms for defining comorbidities in ICD-9-CM and ICD-10 administrative data. Med Care. 2005;43(11):1130–1139. doi:10.1097/01.mlr.0000182534.19832.8316224307
   PubMed Web of Science ®Google Scholar
• Neurofibroma Midwest. Insurance coverage for neurofibroma removal. Available from: https://www.nfmidwest.org/wp-content/uploads/2016/03/Insurance-Coverage-of-Fibroma-Removal.pdf. Accessed 93, 2020.
   Google Scholar
• Rasu RS, Sohraby R, Cunningham L, Knell ME. Assessing chronic pain treatment practices and evaluating adherence to chronic pain clinical guidelines in outpatient practices in the United States. J Pain. 2013;14(6):568–578. doi:10.1016/j.jpain.2013.01.42523578958
   PubMed Web of Science ®Google Scholar
• Cancer Research Network. Cancer therapy look-up tables. Available from: http://www.hcsrn.org/crn/en/RESEARCH/LookupTables/. Accessed 1 29, 2020.
   Google Scholar
• US Bureau of Labor Statistics. Consumer Price Index. Available from: https://www.bls.gov/cpi/home.htm. Accessed 103, 2020.
   Google Scholar
• Kongkriangkai AM, King C, Martin LJ, et al. Substantial pain burden in frequency, intensity, interference and chronicity among children and adults with neurofibromatosis type 1. Am J Med Genet A. 2019;179(4):602–607. doi:10.1002/ajmg.a.6106930737893
   PubMed Web of Science ®Google Scholar
• Kim A, Gillespie A, Dombi E, et al. Characteristics of children enrolled in treatment trials for NF1-related plexiform neurofibromas. Neurology. 2009;73(16):1273–1279. doi:10.1212/WNL.0b013e3181bd132619841379
   PubMed Web of Science ®Google Scholar
• Dombi E, Baldwin A, Marcus LJ, et al. Activity of selumetinib in neurofibromatosis type 1-related plexiform neurofibromas. N Engl J Med. 2016;375(26):2550–2560. doi:10.1056/NEJMoa160594328029918
   PubMed Web of Science ®Google Scholar
• Robertson KA, Nalepa G, Yang FC, et al. Imatinib mesylate for plexiform neurofibromas in patients with neurofibromatosis type 1: a Phase 2 trial. Lancet Oncol. 2012;13(12):1218–1224. doi:10.1016/S1470-2045(12)70414-X23099009
   PubMed Web of Science ®Google Scholar
• Gross AM, Wolters PL, Dombi E, et al. Selumetinib in children with inoperable plexiform neurofibromas. N Engl J Med. 2020;382(15):1430–1442. doi:10.1056/NEJMoa191273532187457
   PubMed Web of Science ®Google Scholar