Title of article :
Natural History and Disease Burden of Neurofibromatosis Type 1 with Plexiform Neurofibromas: A Systematic Literature Review
Author/Authors :
Copley-Merriman, Catherine RTI Health Solutions - Research Triangle Park, NC, USA , Yang, Xiaoqin Merck & Co Inc., Kenilworth, NJ, USA , Juniper, Melissa RTI Health Solutions - Research Triangle Park, NC, USA , Amin, Suvina AstraZeneca, Gaithersburg, MD, USA , Yoo, Hyun Kyoo AstraZeneca, Cambridge, UK , Sen, Shuvayu S Merck & Co Inc., Kenilworth, NJ, USA
Pages :
12
From page :
1
To page :
12
Abstract :
Neurofibromatosis type 1 (NF1) is an incurable genetic condition that frequently includes the development of plexiform neurofibromas (PNs) in patients. A systematic literature review was conducted to identify data on the natural history, disease burden, and treatment patterns among patients diagnosed with NF1 and PN, as well as to identify evidence gaps in these areas. MEDLINE and MEDLINE In-Process, Embase, and Cochrane Library Searches were searched using predefined terms. Potential references underwent two phases of screening by two independent researchers. A total of 39 references focusing on populations of patients with both NF1 and PN were included in this review. The wide range of PN-related complications creates a substantial quality-of-life (QOL) burden for patients, including pain, social functioning, physical function impact, stigma, and emotional distress. The severe burden of NF1 with PN on the QOL of patients demonstrates the high unmet need for an effective treatment option that can reduce tumor burden and improve QOL. The heterogeneity of measurement tools used to evaluate QOL and the gap in data evaluating the health economic burden of PN should be the focus of future research.
Farsi abstract :
فاقد چكيده فارسي
Keywords :
neurofibromatosis type 1 , plexiform neurofibromas , quality of life , natural history , review
Journal title :
Adolescent Health, Medicine and Therapeutics
Serial Year :
2021
Full Text URL :
Record number :
2625535
Link To Document :
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