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Neurofibromatosis Type 1 with Inoperable, Progressive, Symptomatic Plexiform Neurofibromas

MedGen UID:
1643872
•Concept ID:
C4684949
•
Neoplastic Process
Synonym: NF Type 1 with Inoperable, Progressive, Symptomatic Plexiform Neurofibromas

Definition

Progressive, symptomatic neurofibromatosis type 1 associated with plexiform neurofibromas that cannot be removed surgically without risk of substantial morbidity. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVNeurofibromatosis Type 1 with Inoperable, Progressive, Symptomatic Plexiform Neurofibromas

Professional guidelines

PubMed

A Review of Selumetinib in the Treatment of Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
Anderson MK, Johnson M, Thornburg L, Halford Z
Ann Pharmacother 2022 Jun;56(6):716-726. Epub 2021 Sep 18 doi: 10.1177/10600280211046298. PMID: 34541874
Selumetinib in the Treatment of Symptomatic Intractable Plexiform Neurofibromas in Neurofibromatosis Type 1: A Prospective Case Series with Emphasis on Side Effects.
Baldo F, Grasso AG, Cortellazzo Wiel L, Maestro A, Trojniak MP, Murru FM, Basso L, Magnolato A, Bruno I, Barbi E
Paediatr Drugs 2020 Aug;22(4):417-423. doi: 10.1007/s40272-020-00399-y. PMID: 32533336

Recent clinical studies

Etiology

French cohort of children and adolescents with neurofibromatosis type 1 and symptomatic inoperable plexiform neurofibromas: CASSIOPEA study.
Wolkenstein P, Chaix Y, Entz Werle N, Amini-Adle M, Barbarot S, Boileau C, Miled A, Rashid T, Aerts I
Eur J Med Genet 2023 May;66(5):104734. Epub 2023 Mar 2 doi: 10.1016/j.ejmg.2023.104734. PMID: 36868501
Epidemiological profile and clinical characteristics of 491 Brazilian patients with neurofibromatosis type 1.
Darrigo Junior LG, Ferraz VEF, Cormedi MCV, Araujo LHH, Magalhães MPS, Carneiro RC, Sales LHN, Suchmacher M, Cunha KS, Filho AB, Azulay DR, Geller M
Brain Behav 2022 Jun;12(6):e2599. Epub 2022 May 4 doi: 10.1002/brb3.2599. PMID: 35506373Free PMC Article
Efficacy and safety profile of selumetinib in symptomatic inoperable plexiform neurofibromas.
Sharawat IK, Panda PK, Sihag RK, Panda P, Dawman L
J Neurosurg Sci 2022 Dec;66(6):501-510. Epub 2022 Mar 17 doi: 10.23736/S0390-5616.21.05528-4. PMID: 35301838
Cabozantinib for neurofibromatosis type 1-related plexiform neurofibromas: a phase 2 trial.
Fisher MJ, Shih CS, Rhodes SD, Armstrong AE, Wolters PL, Dombi E, Zhang C, Angus SP, Johnson GL, Packer RJ, Allen JC, Ullrich NJ, Goldman S, Gutmann DH, Plotkin SR, Rosser T, Robertson KA, Widemann BC, Smith AE, Bessler WK, He Y, Park SJ, Mund JA, Jiang L, Bijangi-Vishehsaraei K, Robinson CT, Cutter GR, Korf BR; Neurofibromatosis Clinical Trials Consortium, Blakeley JO, Clapp DW
Nat Med 2021 Jan;27(1):165-173. Epub 2021 Jan 13 doi: 10.1038/s41591-020-01193-6. PMID: 33442015Free PMC Article
Selumetinib in the Treatment of Symptomatic Intractable Plexiform Neurofibromas in Neurofibromatosis Type 1: A Prospective Case Series with Emphasis on Side Effects.
Baldo F, Grasso AG, Cortellazzo Wiel L, Maestro A, Trojniak MP, Murru FM, Basso L, Magnolato A, Bruno I, Barbi E
Paediatr Drugs 2020 Aug;22(4):417-423. doi: 10.1007/s40272-020-00399-y. PMID: 32533336

Diagnosis

NF1 plexiform neurofibroma growth rate by volumetric MRI: relationship to age and body weight.
Dombi E, Solomon J, Gillespie AJ, Fox E, Balis FM, Patronas N, Korf BR, Babovic-Vuksanovic D, Packer RJ, Belasco J, Goldman S, Jakacki R, Kieran M, Steinberg SM, Widemann BC
Neurology 2007 Feb 27;68(9):643-7. Epub 2007 Jan 10 doi: 10.1212/01.wnl.0000250332.89420.e6. PMID: 17215493

Therapy

Efficacy and safety profile of selumetinib in symptomatic inoperable plexiform neurofibromas.
Sharawat IK, Panda PK, Sihag RK, Panda P, Dawman L
J Neurosurg Sci 2022 Dec;66(6):501-510. Epub 2022 Mar 17 doi: 10.23736/S0390-5616.21.05528-4. PMID: 35301838
A Review of Selumetinib in the Treatment of Neurofibromatosis Type 1-Related Plexiform Neurofibromas.
Anderson MK, Johnson M, Thornburg L, Halford Z
Ann Pharmacother 2022 Jun;56(6):716-726. Epub 2021 Sep 18 doi: 10.1177/10600280211046298. PMID: 34541874
Cabozantinib for neurofibromatosis type 1-related plexiform neurofibromas: a phase 2 trial.
Fisher MJ, Shih CS, Rhodes SD, Armstrong AE, Wolters PL, Dombi E, Zhang C, Angus SP, Johnson GL, Packer RJ, Allen JC, Ullrich NJ, Goldman S, Gutmann DH, Plotkin SR, Rosser T, Robertson KA, Widemann BC, Smith AE, Bessler WK, He Y, Park SJ, Mund JA, Jiang L, Bijangi-Vishehsaraei K, Robinson CT, Cutter GR, Korf BR; Neurofibromatosis Clinical Trials Consortium, Blakeley JO, Clapp DW
Nat Med 2021 Jan;27(1):165-173. Epub 2021 Jan 13 doi: 10.1038/s41591-020-01193-6. PMID: 33442015Free PMC Article
MEK inhibitors in RASopathies.
Bergqvist C, Wolkenstein P
Curr Opin Oncol 2021 Mar 1;33(2):110-119. doi: 10.1097/CCO.0000000000000711. PMID: 33395032
Selumetinib in Children with Inoperable Plexiform Neurofibromas.
Gross AM, Wolters PL, Dombi E, Baldwin A, Whitcomb P, Fisher MJ, Weiss B, Kim A, Bornhorst M, Shah AC, Martin S, Roderick MC, Pichard DC, Carbonell A, Paul SM, Therrien J, Kapustina O, Heisey K, Clapp DW, Zhang C, Peer CJ, Figg WD, Smith M, Glod J, Blakeley JO, Steinberg SM, Venzon DJ, Doyle LA, Widemann BC
N Engl J Med 2020 Apr 9;382(15):1430-1442. Epub 2020 Mar 18 doi: 10.1056/NEJMoa1912735. PMID: 32187457Free PMC Article

Clinical prediction guides

Efficacy and safety profile of selumetinib in symptomatic inoperable plexiform neurofibromas.
Sharawat IK, Panda PK, Sihag RK, Panda P, Dawman L
J Neurosurg Sci 2022 Dec;66(6):501-510. Epub 2022 Mar 17 doi: 10.23736/S0390-5616.21.05528-4. PMID: 35301838
Cabozantinib for neurofibromatosis type 1-related plexiform neurofibromas: a phase 2 trial.
Fisher MJ, Shih CS, Rhodes SD, Armstrong AE, Wolters PL, Dombi E, Zhang C, Angus SP, Johnson GL, Packer RJ, Allen JC, Ullrich NJ, Goldman S, Gutmann DH, Plotkin SR, Rosser T, Robertson KA, Widemann BC, Smith AE, Bessler WK, He Y, Park SJ, Mund JA, Jiang L, Bijangi-Vishehsaraei K, Robinson CT, Cutter GR, Korf BR; Neurofibromatosis Clinical Trials Consortium, Blakeley JO, Clapp DW
Nat Med 2021 Jan;27(1):165-173. Epub 2021 Jan 13 doi: 10.1038/s41591-020-01193-6. PMID: 33442015Free PMC Article
Selumetinib in the Treatment of Symptomatic Intractable Plexiform Neurofibromas in Neurofibromatosis Type 1: A Prospective Case Series with Emphasis on Side Effects.
Baldo F, Grasso AG, Cortellazzo Wiel L, Maestro A, Trojniak MP, Murru FM, Basso L, Magnolato A, Bruno I, Barbi E
Paediatr Drugs 2020 Aug;22(4):417-423. doi: 10.1007/s40272-020-00399-y. PMID: 32533336
Selumetinib in Children with Inoperable Plexiform Neurofibromas.
Gross AM, Wolters PL, Dombi E, Baldwin A, Whitcomb P, Fisher MJ, Weiss B, Kim A, Bornhorst M, Shah AC, Martin S, Roderick MC, Pichard DC, Carbonell A, Paul SM, Therrien J, Kapustina O, Heisey K, Clapp DW, Zhang C, Peer CJ, Figg WD, Smith M, Glod J, Blakeley JO, Steinberg SM, Venzon DJ, Doyle LA, Widemann BC
N Engl J Med 2020 Apr 9;382(15):1430-1442. Epub 2020 Mar 18 doi: 10.1056/NEJMoa1912735. PMID: 32187457Free PMC Article
NF1 plexiform neurofibroma growth rate by volumetric MRI: relationship to age and body weight.
Dombi E, Solomon J, Gillespie AJ, Fox E, Balis FM, Patronas N, Korf BR, Babovic-Vuksanovic D, Packer RJ, Belasco J, Goldman S, Jakacki R, Kieran M, Steinberg SM, Widemann BC
Neurology 2007 Feb 27;68(9):643-7. Epub 2007 Jan 10 doi: 10.1212/01.wnl.0000250332.89420.e6. PMID: 17215493

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