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

References

• Gutmann DH, Ferner RE, Listernick RH, et al. Neurofibromatosis type 1. Nat Rev Dis Primers. 2017;3:17004.
   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.
   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;6:11.
   PubMed Web of Science ®Google Scholar
• Rasmussen SA, Yang Q, Friedman JM. Mortality in neurofibromatosis 1: an analysis using U.S. death certificates. American Journal of Human Genetics. 2001;68(5):1110–1118.
   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.
   PubMed Web of Science ®Google Scholar
• Orphanet [Internet]. Neurofibromatosis type 1. New York (NY): Orphanet; 2014 [cited 2020 October 8]. Available from: https://www.orpha.net/consor/cgi-bin/Disease_Search.php?lng=EN&data_id=185&Disease_Disease_Search_diseaseGroup=Neurofibromatosis-type-1&Disease_Disease_Search_diseaseType=Pat&Disease(s)/group%20of%20diseases=Neurofibromatosis-type-1&title=Neurofibromatosis%20type%201&search=Disease_Search_Simple
   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):608–614.
   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.
   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-oncology. 2008;10(4):593–598.
   PubMed Web of Science ®Google Scholar
• Korf BR. Plexiform neurofibromas. Am J Med Genet. 1999;89(1):31–37.
   PubMed Web of Science ®Google Scholar
• Nguyen R, Kluwe L, Fuensterer C, et al. Plexiform neurofibromas in children with neurofibromatosis type 1: frequency and associated clinical deficits. The Journal of Pediatrics. 2011;159(4):652–655.
   PubMed Web of Science ®Google Scholar
• Boulanger JM, Larbrisseau A. Neurofibromatosis type 1 in a pediatric population: Ste-Justine’s experience. Can J Neurol Sci. 2005;32(2):225–231.
   PubMed Web of Science ®Google Scholar
• Karaconji T, Whist E, Jamieson RV, et al. Neurofibromatosis type 1: review and update on emerging therapies. Asia-Pacific Journal of Ophthalmology. 2019;8(1):62–72.
   PubMed Web of Science ®Google Scholar
• Evans DG, Baser ME, McGaughran J, et al. Malignant peripheral nerve sheath tumours in neurofibromatosis 1. J Med Genet. 2002;39(5):311–314.
   PubMed Web of Science ®Google Scholar
• Korf BR. Malignancy in neurofibromatosis type 1. Oncologist. 2000;5(6):477–485.
   PubMedGoogle Scholar
• Ly KI, Blakeley JO. The diagnosis and management of neurofibromatosis type 1. Med Clin North Am. 2019;103(6):1035–1054.
   PubMed Web of Science ®Google Scholar
• Canavese F, Krajbich JI. Resection of plexiform neurofibromas in children with neurofibromatosis type 1. J Pediatr Orthop. 2011;31(3):303–311.
   PubMed Web of Science ®Google Scholar
• Nguyen R, Ibrahim C, Friedrich RE, et al. Growth behavior of plexiform neurofibromas after surgery. Genet Med. 2013;15(9):691–697.
   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.
   PubMed Web of Science ®Google Scholar
• Gutmann DH, Blakeley JO, Korf BR, et al. Optimizing biologically targeted clinical trials for neurofibromatosis. Expert Opin Investig Drugs. 2013;22(4):443–462.
   PubMed Web of Science ®Google Scholar
• US Food and Drug Administration [Internet]. FDA approves first therapy for children with debilitating and disfiguring rare disease. Silver Spring (MD): FDA; 2020 [cited 2021 June 30]. Available from: https://www.fda.gov/news-events/press-announcements/fda-approves-first-therapy-children-debilitating-and-disfiguring-rare-disease
   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.
   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.
   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.
   PubMed Web of Science ®Google Scholar
• Wolkenstein P, Durand-Zaleski I, Moreno JC, et al. Cost evaluation of the medical management of neurofibromatosis 1: a prospective study on 201 patients. Br J Dermatol. 2000;142(6):1166–1170.
   PubMed Web of Science ®Google Scholar
• IBM [Internet]. IBM MarketScan research databases. Armonk (NY): IBM; 2020 [cited 2020 January 29]. Available from: https://www.ibm.com/products/marketscan-research-databases
   Google Scholar
• IBM [Internet]. Data brochure: research databases for life sciences researchers. Armonk (NY): IBM; 2019 [cited 2020 May 2]. Available from: https://www.ibm.com/downloads/cas/OWZWJ0QO
   Google Scholar
• Neurofibroma Midwest [Internet]. Insurance coverage for neurofibroma removal. Saint Charles (IL): Neurofibroma Midwest; 2015. [cited 2020 September 3]. Available from: https://www.nfmidwest.org/wp-content/uploads/2016/03/Insurance-Coverage-of-Fibroma-Removal.pdf
   Google Scholar
• Rasu RS, Sohraby R, Cunningham L, et al. 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.
   PubMed Web of Science ®Google Scholar
• Cancer Research Network [Internet]. Cancer therapy look-up tables. Kaiser Permanente Division of Research. 2020 [cited 2020 January 29]. Available from: http://www.hcsrn.org/crn/en/RESEARCH/LookupTables/
   Google Scholar
• US Bureau of Labor Statistics [Internet]. Consumer price index. 2020 [cited 2020 October 3]. Available from: https://www.bls.gov/cpi/home.htm
   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.
   PubMed Web of Science ®Google Scholar
• Lai JS, Jensen SE, Charrow J, et al. 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.
   PubMed Web of Science ®Google Scholar
• Lai JS, Jensen SE, Patel ZS, et al. Using a qualitative approach to conceptualize concerns of patients with neurofibromatosis type 1 associated plexiform neurofibromas (pNF) across the lifespan. Am J Med Genet A. 2017;173(1):79–87.
   PubMed Web of Science ®Google Scholar
• Dagalakis U, Lodish M, Dombi E, et al. Puberty and plexiform neurofibroma tumor growth in patients with neurofibromatosis type I. J Pediatr. 2014;164(3):620–624.
   PubMed Web of Science ®Google Scholar
• Prada CE, Rangwala FA, Martin LJ, et al. Pediatric plexiform neurofibromas: impact on morbidity and mortality in neurofibromatosis type 1. The Journal of Pediatrics. 2012;160(3):461–467.
   PubMed Web of Science ®Google Scholar
• Yang X, Desai K, Agrawal N, et al. Treatment, resource use and costs among pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas. Pediatric Health Med Ther. 2020;11:421–428.
   PubMed Web of Science ®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.
   PubMed Web of Science ®Google Scholar