Abstract
Purpose
To test the feasibility, acceptability, and preliminary efficacy of a mind–body program for patients with neurofibromatosis 2 (NF2) who are deaf or have significant hearing loss (d3RP-NF2) against an attention placebo control (dHEP-NF2) in a single-blind randomized control trial. Both were delivered using Communication Access Real-Time Translation and live group videoconferencing.
Methods
Forty-five adults with NF2 were randomized. Co-primary outcomes were physical quality of life (QoL) and psychological QoL and secondary outcomes were social QoL and environmental QoL, all measured with the World Health Organization Quality of Life Abbreviated Instrument (WHOQOL-BREF). Assessments were conducted at baseline, post-treatment, and six-month follow-up.
Results
Forty-one participants (91%) completed the intervention, and 29 (64%) completed the six-month follow up. Participants in the d3RP-NF2 showed significantly greater improvements from baseline to post-treatment on physical QoL (14.79, 95% CI 5.41–24.18; p ≤ 0.001), psychological QoL (18.77, 95% CI 7.09–30.44, p ≤ 0.001), and environmental QoL (13.25, 95% CI 1.10–25.39, p = 0.03) compared to the dHEP-NF2. Social QoL also significantly increased in the d3RP-NF2 (16.32, 95% CI 6.66–25.97, p = 0.001), but improvement was not beyond the dHEP-NF2. Gains in QoL were clinically meaningful and maintained at the 6-month follow-up for d3RP-NF2 participants across all QoL domains. There were more treatment responders in the d3RP-NF2 compared to the dHEP-NF2.
Conclusions
The d3RP-NF2 was well accepted, highly feasible, and resulted in sustained improvements in QoL in patients with NF2 who are deaf or have significant hearing loss.
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References
Merker VL, Bergner AL, Vranceanu A-M et al (2016) Health-related quality of life of individuals with neurofibromatosis type 2: results from the NF2 natural history study. Otol Neurotol 37:574–579
Evans DG (1993) Neurofibromatosis 2. In: Adam MP, Ardinger HH, Pagon RA et al (eds) GeneReviews®. University of Washington, Seattle, Seattle
Page PZ, Page GP, Ecosse E et al (2006) Impact of neurofibromatosis 1 on Quality of Life: a cross-sectional study of 176 American cases. Am J Med Genet A 140:1893–1898. https://doi.org/10.1002/ajmg.a.31422
Granström S, Langenbruch A, Augustin M, Mautner V-F (2012) Psychological burden in adult neurofibromatosis type 1 patients: impact of disease visibility on body image. Dermatol Basel Switz 224:160–167. https://doi.org/10.1159/000337548
Wolkenstein P, Zeller J, Revuz J et al (2001) Quality-of-life impairment in neurofibromatosis type 1: a cross-sectional study of 128 cases. Arch Dermatol 137:1421–1425
Neary WJ, Hillier VF, Flute T et al (2010) The relationship between patients’ perception of the effects of neurofibromatosis type 2 and the domains of the Short Form-36. Clin Otolaryngol 35:291–299. https://doi.org/10.1111/j.1749-4486.2010.02176.x
Vranceanu A-M, Riklin E, Merker VL et al (2016) Mind-body therapy via videoconferencing in patients with neurofibromatosis: an RCT. Neurology 87:806–814
Skevington SM, Lotfy M, O’Connell KA, WHOQOL Group (2004) The World Health Organization’s WHOQOL-BREF quality of life assessment: psychometric properties and results of the international field trial. Qual Life Res 13:299–310
Kasesalu P, Tallinn J (2003) Skype
World Health Organization (1995) Field trial whoqol-100 february 1995: the 100 questions with response scales, 2012 revision
Attkisson C (1996) The client satisfaction questionnaire (csq) scales. In: Outcome assessment in clinical practice. Williams and Wilkins, Baltimore
Harris PA, Taylor R, Thielke R et al (2009) Research electronic data capture (REDCap)–a metadata-driven methodology and workflow process for providing translational research informatics support. J Biomed Inform 42:377–381
Mahaffey B, Macklin D, Vranceanu A-M et al (2018) The stony brook health enhancement program: the development of an active control condition for mind–body interventions. J Health Psychol. https://doi.org/10.1177/1359105318787024
Browne RH (1995) On the use of a pilot sample for sample size determination. Stat Med 14:1933–1940
Lancaster GA, Dodd S, Williamson PR (2004) Design and analysis of pilot studies: Recommendations for good practice. J Eval Clin Pract 10:307–312
Shih WJ, Ohman-Strickland PA, Lin Y (2004) Analysis of pilot and early phase studies with small sample sizes. Stat Med 23:1827–1842
Whitehead AL, Julious SA, Cooper CL, Campbell MJ (2016) Estimating the sample size for a pilot randomised trial to minimise the overall trial sample size for the external pilot and main trial for a continuous outcome variable. Stat Methods Med Res 25:1057–1073
Rounsaville B, Carrol K, Onken L (2001) A stage model of behavioral therapies research: Getting started and moving on from stage I. Clin Psychol Sci Pract 8:133–142
Acknowledgements
The authors would like to acknowledge Matt Hay and Michie O’ Day for their suggestions regarding adaptions to the mind–body program to better meet the needs of patients with NF2 who are deaf.
Funding
This study was funded by the Children’s Tumor Foundation (CTF) through a clinical research grant awarded to Ana-Maria Vranceanu.
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Funes, C.J., Mace, R.A., Macklin, E.A. et al. First report of quality of life in adults with neurofibromatosis 2 who are deafened or have significant hearing loss: results of a live-video randomized control trial. J Neurooncol 143, 505–513 (2019). https://doi.org/10.1007/s11060-019-03182-3
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DOI: https://doi.org/10.1007/s11060-019-03182-3