Management of neurofibromatosis type 1-associated plexiform neurofibromas

被引：72

作者：

Fisher, Michael J. ^{[1
,2
]}

Blakeley, Jaishri O. ^{[3
]}

Weiss, Brian D. ^{[6
]}

Dombi, Eva ^{[7
]}

Ahlawat, Shivani ^{[4
]}

Akshintala, Srivandana ^{[7
]}

Belzberg, Allan J. ^{[5
]}

Bornhorst, Miriam ^{[9
]}

Bredella, Miriam A. ^{[10
,11
]}

Cai, Wenli ^{[10
,11
]}

Ferner, Rosalie E. ^{[12
]}

Gross, Andrea M. ^{[7
]}

Harris, Gordon J. ^{[10
,11
]}

Listernick, Robert ^{[13
]}

Ly, Ina ^{[14
]}

Martin, Staci ^{[7
]}

Mautner, Victor F. ^{[15
]}

Salamon, Johannes M. ^{[16
]}

Salerno, Kilian E. ^{[8
]}

Spinner, Robert J. ^{[17
]}

Staedtke, Verena ^{[3
]}

Ullrich, Nicole J. ^{[18
]}

Upadhyaya, Meena ^{[19
]}

Wolters, Pamela L. ^{[7
]}

Yohay, Kaleb ^{[20
]}

Widemann, Brigitte C. ^{[7
]}

机构：

[1] Childrens Hosp Philadelphia, Div Oncol, 3615 Civ Ctr Blvd, Philadelphia, PA 19103 USA

[2] Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA

[3] Johns Hopkins Univ, Sch Med, Dept Neurol, Div Neurooncol, Baltimore, MD 21205 USA

[4] Johns Hopkins Univ, Russell H Morgan Dept Radiol & Radiol Sci, Sch Med, Baltimore, MD USA

[5] Johns Hopkins Univ, Sch Med, Dept Neurosurg, Baltimore, MD 21205 USA

[6] Univ Cincinnati, Cincinnati Childrens Hosp Med Ctr, Div Oncol, Coll Med, Cincinnati, OH USA

[7] NCI, Pediat Oncol Branch, Bethesda, MD 20892 USA

[8] NCI, Radiat Oncol Branch, Bethesda, MD 20892 USA

[9] Childrens Natl Hosp, Ctr Neurosci & Behav Med, Family Neurofibromatosis Inst, Washington, DC USA

[10] Massachusetts Gen Hosp, Dept Radiol, Boston, MA USA

[11] Harvard Med Sch, Boston, MA 02115 USA

[12] Guys & St Thomas NHS Fdn Trust, Dept Neurol, Neurofibromatosis Serv, Guys Hosp, London, England

[13] Northwestern Univ, Ann & Robert H Lurie Childrens Hosp Chicago, Feinberg Sch Med, Dept Pediat, Chicago, IL USA

[14] Massachusetts Gen Hosp, Stephen E & Catherine Pappas Ctr NeuroOncol, Boston, MA USA

[15] Univ Med Ctr Hamburg Eppendorf, Dept Neurol, Hamburg, Germany

[16] Univ Med Ctr Hamburg Eppendorf, Dept Diagnost & Intervent Radiol & Nucl Med, Hamburg, Germany

[17] Mayo Clin, Dept Neurol Surg, Rochester, MN USA

[18] Boston Childrens Hosp, Dept Neurol, Boston, MA USA

[19] Cardiff Univ, Div Canc & Genet, Cardiff, Wales

[20] Grossman Sch Med, Dept Neurol, New York, NY USA

来源：

NEURO-ONCOLOGY | 2022年 / 24卷 / 11期

关键词：

MEK inhibitor; neurofibroma; neurofibromatosis; 1; plexiform; review; PHASE-I TRIAL; FARNESYLTRANSFERASE INHIBITOR TIPIFARNIB; NERVE SHEATH TUMORS; WHOLE-BODY MRI; CLINICAL-TRIALS; CHRONIC PAIN; NF1; GENE; SURGICAL-MANAGEMENT; CORD COMPRESSION; VOLUMETRIC MRI;

D O I：

10.1093/neuonc/noac146

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Plexiform Neurofibromas (PN) are a common manifestation of the genetic disorder neurofibromatosis type 1 (NF1). These benign nerve sheath tumors often cause significant morbidity, with treatment options limited historically to surgery. There have been tremendous advances over the past two decades in our understanding of PN, and the recent regulatory approvals of the MEK inhibitor selumetinib are reshaping the landscape for PN management. At present, there is no agreed upon PN definition, diagnostic evaluation, surveillance strategy, or clear indications for when to initiate treatment and selection of treatment modality. In this review, we address these questions via consensus recommendations from a panel of multidisciplinary NF1 experts.

引用

页码：1827 / 1844

页数：18

共 117 条

[21] Cognitive-Behavioral Therapy for Individuals With Chronic Pain Efficacy, Innovations, and Directions for Research [J].

Ehde, Dawn M. ;

Dillworth, Tiara M. ;

Turner, Judith A. .

AMERICAN PSYCHOLOGIST, 2014, 69 (02) :153-166

[22] Synergistic Interplay between Curcumin and Polyphenol-Rich Foods in the Mediterranean Diet: Therapeutic Prospects for Neurofibromatosis 1 Patients [J].

Esposito, Teresa ;

Schettino, Carla ;

Polverino, Paola ;

Allocca, Salvatore ;

Adelfi, Laura ;

D'Amico, Alessandra ;

Capaldo, Guglielmo ;

Varriale, Bruno ;

Di Salle, Anna ;

Peluso, Gianfranco ;

Sorrentino, Giuseppe ;

Lus, Giacomo ;

Sampaolo, Simone ;

Di Iorio, Giuseppe ;

Melone, Mariarosa Anna Beatrice .

NUTRIENTS, 2017, 9 (07)

[23] Cancer and Central Nervous System Tumor Surveillance in Pediatric Neurofibromatosis 1 [J].

Evans, D. Gareth R. ;

Salvador, Hector ;

Chang, Vivian Y. ;

Erez, Ayelet ;

Voss, Stephan D. ;

Schneider, Kami Wolfe ;

Scott, Hamish S. ;

Plon, Sharon E. ;

Tabori, Uri .

CLINICAL CANCER RESEARCH, 2017, 23 (12) :E46-E53

[24] Malignant peripheral nerve sheath tumours in neurofibromatosis 1 [J].

Evans, DGR ;

Baser, ME ;

McGaughran, J ;

Sharif, S ;

Howard, E ;

Moran, A .

JOURNAL OF MEDICAL GENETICS, 2002, 39 (05) :311-314

[25] GIANT PLEXIFORM NEUROFIBROMA WITH HEMORRHAGE IN CRANIO-MAXILLOFACIAL REGION AS DEPICTED ON CT AND MRI [J].

Feng, Y. ;

Yang, Z. ;

Chen, T. ;

Wang, Q. ;

Deng, W. .

EUROPEAN JOURNAL OF MEDICAL RESEARCH, 2010, 15 (02) :84-87

[26] Evaluation of quality of life in adults with neurofibromatosis 1 (NF1) using the Impact of NF1 on Quality Of Life (INF1-QOL) questionnaire [J].

Ferner, Rosalie E. ;

Thomas, Mary ;

Mercer, Gemma ;

Williams, Victoria ;

Leschziner, Guy D. ;

Afridi, Shazia K. ;

Golding, John F. .

HEALTH AND QUALITY OF LIFE OUTCOMES, 2017, 15

[27]

Ferner RE, 2013, HAND CLINIC, V115, P939, DOI 10.1016/B978-0-444-52902-2.00053-9

[28] MR neurography and muscle MR imaging for image diagnosis of disorders affecting the peripheral nerves and musculature [J].

Filler, AG ;

Maravilla, KR ;

Tsuruda, JS .

NEUROLOGIC CLINICS, 2004, 22 (03) :643-+

[29] Cabozantinib for neurofibromatosis type 1-related plexiform neurofibromas: a phase 2 trial [J].

Fisher, Michael J. ;

Shih, Chie-Schin ;

Rhodes, Steven D. ;

Armstrong, Amy E. ;

Wolters, Pamela L. ;

Dombi, Eva ;

Zhang, Chi ;

Angus, Steven P. ;

Johnson, Gary L. ;

Packer, Roger J. ;

Allen, Jeffrey C. ;

Ullrich, Nicole J. ;

Goldman, Stewart ;

Gutmann, David H. ;

Plotkin, Scott R. ;

Rosser, Tena ;

Robertson, Kent A. ;

Widemann, Brigitte C. ;

Smith, Abbi E. ;

Bessler, Waylan K. ;

He, Yongzheng ;

Park, Su-Jung ;

Mund, Julie A. ;

Jiang, Li ;

Bijangi-Vishehsaraei, Khadijeh ;

Robinson, Coretta Thomas ;

Cutter, Gary R. ;

Korf, Bruce R. ;

Blakeley, Jaishri O. ;

Clapp, D. Wade .

NATURE MEDICINE, 2021, 27 (01) :165-+

[30] Functional outcome measures for NF1-associated optic pathway glioma clinical trials [J].

Fisher, Michael J. ;

Avery, Robert A. ;

Allen, Jeffrey C. ;

Ardern-Holmes, Simone L. ;

Bilaniuk, Larissa T. ;

Ferner, Rosalie E. ;

Gutmann, David H. ;

Listernick, Robert ;

Martin, Staci ;

Ullrich, Nicole J. ;

Liu, Grant T. .

NEUROLOGY, 2013, 81 (21) :S15-S24

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