Hearing and Facial Function Outcomes for Neurofibromatosis 2 Clinical Trials

Overview

Journal Neurology

Specialty Neurology

Date 2013 Nov 20

PMID 24249803

Citations 17

Authors

Scott R Plotkin

Simone L Ardern-Holmes

Fred G Barker 2nd

Jaishri O Blakeley

D Gareth Evans

Rosalie E Ferner

Tessa A Hadlock

Chris Halpin

Affiliations

Soon will be listed here.

Abstract

Objectives: Vestibular schwannomas are the hallmark of neurofibromatosis 2 (NF2), occurring in >95% of patients. These tumors develop on the vestibulocochlear nerve and are associated with significant morbidity due to hearing loss, tinnitus, imbalance, facial weakness, and risk of early mortality from brainstem compression. Although hearing loss and facial weakness have been identified as important functional outcomes for patients with NF2, there is a lack of consensus regarding appropriate endpoints in clinical trials.

Methods: The functional outcomes group reviewed existing endpoints for hearing and facial function and developed consensus recommendations for response evaluation in NF2 clinical trials.

Results: For hearing endpoints, the functional group endorsed the use of maximum word recognition score as a primary endpoint, with the 95% critical difference as primary hearing outcomes. The group recommended use of the scaled measurement of improvement in lip excursion (SMILE) system for studies of facial function.

Conclusions: These recommendations are intended to provide researchers with a common set of endpoints for use in clinical trials of patients with NF2. The use of common endpoints should improve the quality of clinical trials and foster comparison among studies for hearing loss and facial weakness.

Citing Articles

Matrix metalloproteinase 9: An emerging biomarker for classification of adherent vestibular schwannoma.

Nguyen H, Duhon B, Kuo H, Fisher M, Brickey O, Zhang L Neurooncol Adv. 2024; 6(1):vdae058.

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Multicenter, prospective, phase II study of maintenance bevacizumab for children and adults with NF2-related schwannomatosis and progressive vestibular schwannoma.

Plotkin S, Allen J, Dhall G, Campian J, Clapp D, Fisher M Neuro Oncol. 2023; 25(8):1498-1506.

PMID: 37010875 PMC: 10398799. DOI: 10.1093/neuonc/noad066.

Early prediction of functional prognosis in neurofibromatosis type 2 patients based on genotype-phenotype correlation with targeted deep sequencing.

Teranishi Y, Miyawaki S, Nakatomi H, Ohara K, Hongo H, Dofuku S Sci Rep. 2022; 12(1):9543.

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Neurofibromatosis: Molecular Pathogenesis and Natural Compounds as Potential Treatments.

Amaravathi A, Oblinger J, Welling D, Kinghorn A, Chang L Front Oncol. 2021; 11:698192.

PMID: 34604034 PMC: 8485038. DOI: 10.3389/fonc.2021.698192.

Patient Report of Hearing in Neurofibromatosis Type 2: Recommendations for Clinical Trials.

Thompson H, Blanton A, Franklin B, Merker V, Franck K, Welling D Neurology. 2021; 97(7 Suppl 1):S64-S72.

PMID: 34230203 PMC: 8594003. DOI: 10.1212/WNL.0000000000012424.

References

Caye-Thomasen P, Dethloff T, Hansen S, Stangerup S, Thomsen J . Hearing in patients with intracanalicular vestibular schwannomas. Audiol Neurootol. 2006; 12(1):1-12. DOI: 10.1159/000096152. View

Plotkin S, Merker V, Halpin C, Jennings D, McKenna M, Harris G . Bevacizumab for progressive vestibular schwannoma in neurofibromatosis type 2: a retrospective review of 31 patients. Otol Neurotol. 2012; 33(6):1046-52. DOI: 10.1097/MAO.0b013e31825e73f5. View

Berg T, Jonsson L, Engstrom M . Agreement between the Sunnybrook, House-Brackmann, and Yanagihara facial nerve grading systems in Bell's palsy. Otol Neurotol. 2004; 25(6):1020-6. DOI: 10.1097/00129492-200411000-00027. View

House J, Brackmann D . Facial nerve grading system. Otolaryngol Head Neck Surg. 1985; 93(2):146-7. DOI: 10.1177/019459988509300202. View

Tysome J, Macfarlane R, Durie-Gair J, Donnelly N, Mannion R, Knight R . Surgical management of vestibular schwannomas and hearing rehabilitation in neurofibromatosis type 2. Otol Neurotol. 2012; 33(3):466-72. DOI: 10.1097/MAO.0b013e318248eaaa. View

Hornigold R, Golding J, Leschziner G, Obholzer R, Gleeson M, Thomas N . The NFTI-QOL: A Disease-Specific Quality of Life Questionnaire for Neurofibromatosis 2. J Neurol Surg B Skull Base. 2013; 73(2):104-11. PMC: 3424623. DOI: 10.1055/s-0032-1301396. View

Vrabec J, Backous D, Djalilian H, Gidley P, Leonetti J, Marzo S . Facial Nerve Grading System 2.0. Otolaryngol Head Neck Surg. 2009; 140(4):445-50. DOI: 10.1016/j.otohns.2008.12.031. View

Gardner G, Robertson J . Hearing preservation in unilateral acoustic neuroma surgery. Ann Otol Rhinol Laryngol. 1988; 97(1):55-66. DOI: 10.1177/000348948809700110. View

Neary W, Hillier V, Flute T, Stephens D, Ramsden R, Evans D . Use of a closed set questionnaire to measure primary and secondary effects of neurofibromatosis type 2. J Laryngol Otol. 2010; 124(7):720-8. DOI: 10.1017/S0022215110000460. View

10.

Bray D, Henstrom D, Cheney M, Hadlock T . Assessing outcomes in facial reanimation: evaluation and validation of the SMILE system for measuring lip excursion during smiling. Arch Facial Plast Surg. 2010; 12(5):352-4. DOI: 10.1001/archfacial.2010.69. View

11.

Karajannis M, Legault G, Hagiwara M, Ballas M, Brown K, Nusbaum A . Phase II trial of lapatinib in adult and pediatric patients with neurofibromatosis type 2 and progressive vestibular schwannomas. Neuro Oncol. 2012; 14(9):1163-70. PMC: 3424212. DOI: 10.1093/neuonc/nos146. View

12.

Lee L, Susarla S, Hohman M, Henstrom D, Cheney M, Hadlock T . A comparison of facial nerve grading systems. Ann Plast Surg. 2012; 70(3):313-6. DOI: 10.1097/SAP.0b013e31826acb2c. View

13.

Brackmann D, Owens R, Friedman R, Hitselberger W, De La Cruz A, House J . Prognostic factors for hearing preservation in vestibular schwannoma surgery. Am J Otol. 2000; 21(3):417-24. DOI: 10.1016/s0196-0709(00)80054-x. View

14.

Friedman R, Goddard J, Wilkinson E, Schwartz M, Slattery 3rd W, Fayad J . Hearing preservation with the middle cranial fossa approach for neurofibromatosis type 2. Otol Neurotol. 2011; 32(9):1530-7. DOI: 10.1097/MAO.0b013e3182355855. View

15.

Vakharia K, Henstrom D, Plotkin S, Cheney M, Hadlock T . Facial reanimation of patients with neurofibromatosis type 2. Neurosurgery. 2011; 70(2 Suppl Operative):237-43. DOI: 10.1227/NEU.0b013e31823a819f. View

16.

Plotkin S, Merker V, Muzikansky A, Barker 2nd F, Slattery 3rd W . Natural history of vestibular schwannoma growth and hearing decline in newly diagnosed neurofibromatosis type 2 patients. Otol Neurotol. 2013; 35(1):e50-6. DOI: 10.1097/MAO.0000000000000239. View

17.

Kang T, Vrabec J, Giddings N, Terris D . Facial nerve grading systems (1985-2002): beyond the House-Brackmann scale. Otol Neurotol. 2002; 23(5):767-71. DOI: 10.1097/00129492-200209000-00026. View

18.

Hadlock T, Urban L . Toward a universal, automated facial measurement tool in facial reanimation. Arch Facial Plast Surg. 2012; 14(4):277-82. DOI: 10.1001/archfacial.2012.111. View

19.

Masuda A, Fisher L, Oppenheimer M, Iqbal Z, Slattery W . Hearing changes after diagnosis in neurofibromatosis type 2. Otol Neurotol. 2004; 25(2):150-4. DOI: 10.1097/00129492-200403000-00012. View

20.

Thornton A, Raffin M . Speech-discrimination scores modeled as a binomial variable. J Speech Hear Res. 1978; 21(3):507-18. DOI: 10.1044/jshr.2103.507. View