Publications
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Perrone RD, Garbinsky D, Nunna S, Gandhi HK, Fernandes AW, Burgos G, Olopoenia A, DeCongelio M, Maculaitis MC, Zhou X. Effectiveness of tolvaptan for autosomal dominant polycystic kidney disease in US clinical practice with comparison to historical control data. Kidney Med. 2025 Feb 27;7(5):100988. doi: 10.1016/j.xkme.2025.100988
Chebib FT, Dahl N, Zhou X, Garbinsky D, Wang J, Nunna S, Oberdhan D, Fernandes AW. Tolvaptan and autosomal dominant polycystic kidney disease progression in individuals aged 18–35 years: a pooled database analysis. Kidney Med. 2025 Jan;7(1):100935. doi: 10.1016/j.xkme.2024.100935
Gitomer BY, Chonchol M, Zhou X, Garbinsky D, Wang J, Nunna S, Fernandes AW, Oberdhan D. Kidney stone disease and progression risk in autosomal dominant polycystic kidney disease: a post hoc analysis of OVERTURE. Kidney360. 2024 Sep;5(9):1364-6. doi: 10.34067/KID.0000000000000524
Slota C, Norcross L, Comerford E, Sasane M, Zheng Y, Gnanasakthy A. Critical comments by Food and Drug Administration Reviewers on patient-reported outcomes in Food and Drug Administration Regulatory Submissions (2018-2021) [Editor's Choice]. Value Health. 2024 Jun;27(6):755-66. doi: 10.1016/j.jval.2024.02.011
Comerford E, Norcross L, Sasane M, Zheng Y, Gnanasakthy A. Patient-reported outcome (PRO) data in oncology NDAs approved by the FDA (2018–2021): a review of criticism and concerns in comments from regulators. Poster presented at the National Comprehensive Cancer Network (NCCN) 2024 Annual Conference; April 5, 2024. Orlando, FL. [abstract] J Natl Compr Canc Netw. 2024 Apr 5; 22(2.5). doi: 10.6004/jnccn.2023.7146
Maeda-Chubachi T, McLeod L, Enloe C, Cartwright M, Siegfried E, Hebert AA, Silverberg N. Defining clinically meaningful improvement in molluscum contagiosum. J Am Acad Dermatol. 2024 Feb;90(2):443-5. doi: 10.1016/j.jaad.2023.10.033
Sherif B, Burke M, Besse B, Solomon B, Bazhenova L, Kim DW, Lin JJ, Wolf J, Popat S, Goto K, de Langen AJ, Springfeld C, Reynolds M, Odom D, Yuan Y, Lee A, Blum SI, Thamake S, Ades F, Drilon A. Health-related quality-of-life of patients treated with repotrectinib for neurotrophic tyrosine receptor kinase (NTRK)-positive advanced solid tumors: results From TRIDENT-1. Poster presented at the ISPOR Europe 2023; November 14, 2023. Copenhagen, Denmark. [abstract] Value Health. 2023 Dec; 26(12 Supplement):S484. doi: 10.1016/j.jval.2023.09.2619
Yee KS, Chirila C, Davenport E, Mladsi D, Barnett C, Kronenberger WG. A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT. Orphanet J Rare Dis. 2023 Nov 2;18(1):343. doi: 10.1186/s13023-023-02957-2
Perrone RD, Nunna S, Gandhi HK, Fernandes AW, Garbinsky D, Zhou X. Kidney function decline in autosomal dominant polycystic kidney disease (ADPKD) patients: assessment of real world effectiveness of tolvaptan. Poster presented at the Kidney Week 2023; November 2, 2023. Philadelphia, PA.
Whalley D, Balp M, Yarr S, Porter M, Barve A. Patient-reported symptom burden in chronic inducible urticaria: post hoc analysis of baseline data from a phase 3 clinical trial. Poster presented at the 2023 EADV Congress; October 11, 2023. Berlin, Germany.
Chebib FT, Dahl N, Zhou X, Garbinsky D, Wang J, Nunna S, Oberdhan D, Fernandes AW. Pooled data analysis of the effect of tolvaptan in patients aged 18-35 years with autosomal dominant polycystic kidney disease. Poster presented at the PKD Connect Conference 2023; June 23, 2023. Denver, CO.
Lioudis M, Zhou X, Davenport E, Nunna S, Krasa HB, Oberdhan D, Fernandes AW. Effects of tolvaptan discontinuation in patients with autosomal dominant polycystic kidney disease: a post hoc pooled analysis. BMC Neurol. 2023 Jun 22;24:182. doi: 10.1186/s12882-023-03247-6
Davenport E, Zhou X, Wang J, Nunna S, Oberdhan D, Fernandes A, Mekahli D. Disease burden and disease progression of young adult patients with autosomal dominant polycystic kidney disease (ADPKD). Poster presented at the 51st Annual Meeting of the European Working Group on Psychosocial Aspects of Children; June 8, 2023. Leuven, Belgium. Previously presented at the National Kidney Foundation 2023 Spring Clinical Meeting.
Chebib FT, Zhou X, Garbinsky D, Davenport E, Nunna S, Oberdhan D, Fernandes A. Tolvaptan and kidney function decline in older individuals with autosomal dominant polycystic kidney disease: a pooled analysis of randomized clinical trials and observational studies. Kidney Med. 2023 Jun;5(6):100639. doi: 10.1016/j.xkme.2023.100639
Maeda-Chubachi T, McLeod L, Cartwright M. Meaningful change in children's dermatology life quality index (DLQI) and lesion counts in patients with molluscum contagiosum. Poster presented at the First International Societies for Investigative Dermatology (ISID2023) Meeting; May 10, 2023. Tokyo, Japan. [abstract] J Invest Dermatol. 2023 May; 143(5 Suppl):S105. doi: 10.1016/j.jid.2023.03.622
Davenport E, Zhou X, Wang J, Nunna S, Oberdhan D, Fernandes A, Mekahli D. Disease burden and disease progression of young adult patients with autosomal dominant polycystic kidney disease. Poster presented at the National Kidney Foundation 2023 Spring Clinical Meeting; April 11, 2023. Austin, TX. [abstract] Am J Kidney Dis. 2023 Apr 1; 81(4, Supple 1):S102. doi: 10.1053/j.ajkd.2023.01.349
Bergerot C, Young Rha S, Pal S, Koralewski P, Stroyakovskiy D, Alekseev B, Parnis F, Castellano D, Lyun Lee J, Sunela K, Ciuleanu T, Heng D, Glen H, Wang J, Bennett L, Pan J, O'Hara K, Puente J. Health-related quality of life outcomes with two different starting doses of lenvatinib in combination with everolimus for previously treated renal cell carcinoma. Oncologist. 2023 Jan 18;28(1):59-71. doi: 10.1093/oncolo/oyac142
Forsythe E, Haws RM, Argente J, Beales P, Martos-Moreno GA, Dollfus H, Chirila C, Gnanasakthy A, Buckley BC, Mallya UG, Clement K, Haqq AM. Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results. Orphanet J Rare Dis. 2023 Jan 16;18(1):12. doi: 10.1186/s13023-022-02602-4
Morrison R, Allas S, Crawford R, Tremel N, Weiss B, Robo C, Doward L. International adaptation and linguistic validation of the hypoparathyroidism daily diary of symptom experience (HPT-DD-SE) and the hypoparathyroidism life impact questionnaire (HPT-LIQ). Poster presented at the ISPOR Europe 2022; November 8, 2022. Vienna, Austria. [abstract] Value Health. 2022 Dec 1; 25(12):S418. doi: 10.1016/j.jval.2022.09.2079
Motzer R, Porta C, Alekseev B, Rha SY, Choueiri TK, Mendez-Vidal MJ, Hong SH, Kapoor A, Goh JC, Eto M, Bennett L, Wang J, Pan JJ, Saretsky TL, Perini RF, He CS, Mody K, Cella D. Health-related quality-of-life outcomes in patients with advanced renal cell carcinoma treated with lenvatinib plus pembrolizumab or everolimus versus sunitinib (CLEAR): a randomised, phase 3 study. Lancet Oncol. 2022 Jun;23(6):768-80. doi: 10.1016/S1470-2045(22)00212-1
Zhou X, Davenport E, Ouyang J, Hoke ME, Garbinsky D, Agarwal I, Krasa HB, Oberdhan D. Pooled data analysis of the long-term treatment effects of tolvaptan in ADPKD. Kidney Int Rep. 2022 Feb 19;7(5):1037-48. doi: 10.1016/j.ekir.2022.02.009
Kuhnen P, Wabitsch M, von Schnurbein J, Chirila C, Mallya UG, Callahan P, Gnanasakthy A, Poitou C, Krabusch PM, Stewart M, Clement K. Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency. Orphanet J Rare Dis. 2022 Feb 5;17(1):38. doi: 10.1186/s13023-022-02186-z
Sarda SP, Heyes A, Bektas M, Thankur T, Chao W, Intorcia M, Wronski S, Jones DL. Humanistic and economic burden of geographic atrophy: a systematic literature review. Clin Opththa. 2021 Dec 8;2021(15):4629-44.
Kummar S, Berlin J, Mascarenhas L, van Tilburg CM, Geoerger B, Lassen UN, Schilder RJ, Turpin B, Nanda S, Keating K, Childs BH, Chirila C, Laetsch TW, Hyman DM, Drilon A, Hong DS. Quality of life in adult and pediatric patients with tropomyosin receptor kinase fusion cancer receiving larotrectinib. Curr Probl Cancer. 2021 Dec;45(6):100734. doi: 10.1016/j.currproblcancer.2021.100734
Forsythe E, Haws RM, Argente J, Beales P, Chirila C, Clement K, Dollfus H, Martos-Moreno GA, Gnanasakthy A, Buckley BC, Mallya U, Haqq AM. Quality of life in patients with bardet-biedl syndrome in a setmelanotide phase 3 trial. Poster presented at the 2021 Obesity Week Virtual Conference; November 1, 2021.
Kuhnen P, Wabitsch M, von Schnurbein J, Chirila C, Mallya UG, Callahan P, Gnanasakthy A, Poitou C, Krabusch PM, Stewart M, Clement K. Quality of life in POMC or LEPR deficiency: setmelanotide phase 3 trials. Poster presented at the 2021 Virtual Overcoming Obesity Conference; October 14, 2021.
Xiao H, Blum S, Davenport E, Wang J, Hunter S, Li M, Kondo K. Health care resource utilization of patients with advanced gastric cancer/gastroesophageal junction cancer or esophageal adenocarcinoma: results of nivolumab plus chemotherapy versus chemotherapy from CheckMate 649. Poster presented at the AMCP Nexus 2021; October 18, 2021. [abstract] J Manag Care Pharm. 2021 Oct; 27(10-a):S19.
Kupperman BD, Keyloun KR, Campbell JH, Zhou X, Davenport E, Bakri SJ. Decreased vision improvement in DME eyes with longer duration or larger amounts of excess edema: a post-hoc analysis of protocol T. Presented at the 2021 54th Retina Society Annual Scientific Meeting; September 29, 2021. Chicago, IL.
Elimova E, Wyrwicz L, Blum SI, Xiao H, Li M, Kondo K, Davenport E, Wang J, Hunter S, Moehler MH. Health-related quality of life (HRQOL) in patients (pts) with advanced gastric cancer/gastroesophageal junction cancer (GC/GEJC) or esophageal adenocarcinoma (EAC): results of nivolumab plus chemotherapy (NIVO+chemo) versus chemo from CheckMate 649. Poster presented at the 2021 ASCO Quality Care Symposium; September 24, 2021.
Jones DL, Bektas M, Heyes A, Wronski S, Sarda SP. Clinical & humanistic burden of geographic atrophy (GA). Poster presented at the 2021 EURETINA Virtual Congress; September 9, 2021.
Simpson EL, de Bruin-Weller M, Bansal A, Chen Z, Nelson L, Whalley D, Prescilla R, Guillemin I, Delevry D. Definition of clinically meaningful within-patient changes in POEM and CDLQI in children 6 to 11 years of age with severe atopic dermatitis. Dermatol Ther. 2021 Aug;11(4):1415-22. doi: 10.1007/s13555-021-00543-7
Smith MR, Shore N, Tammela TL, Ulys A, Vjaters E, Polyakov S, Jievaltas M, Luz M, Alekseev B, Kuss I, Le Berre MA, Mohamed AF, Odom D, Bartsch J, Snapir A, Sarapohja T, Fizazi K. Darolutamide and health-related quality of life in patients with non-metastatic castration-resistant prostate cancer: an analysis of the phase III ARAMIS trial. Eur J Cancer. 2021 Jul 14;154:138-46.
Hofheinz RD, Bruix J, Demetri GD, Grothey A, Marian M, Bartsch J, Odom D. Effect of regorafenib in delaying definitive deterioration in health-related quality of life in patients with advanced cancer of three different tumor types. Cancer Manag Res. 2021 Jul 12;13:5523-33. doi: 10.2147/CMAR.S305939
Ciruelos EM, Rugo HS, Mayer IA, Levy C, Forget F, Delgado Mingorance JI, Safra T, Masuda N, Park YH, Juric D, Conte P, Campone M, Loibl S, Iwata H, Zhou X, Park J, Ridolfi A, Lorenzo I, Andre F. Patient-reported outcomes in patients with PIK3CA-mutated hormone receptor-positive, human epidermal growth factor receptor 2–negative advanced breast cancer from SOLAR-1. J Clin Oncol. 2021 Jun 20;39(18):2005-15. doi: 10.1200/JCO.20.01139
Yee KS, Chirila C, Davenport E, Mladsi D, Barnett C, Kronenberger WG. Assessment of cognitive development in patients with neuronopathic Mucopolysaccharidosis II treated with intrathecal idursulfase-IT using Projected Retained Ability Score (PRAS): a post hoc analysis. Poster presented at the 2021 World Conference on Quality and Improvement; May 24, 2021.
Yee K, Chirila C, Davenport E, Mladsi D, Barnett C, Kronenberger WG. Using projected retained ability score to assess cognitive development in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT. Poster presented at the 2021 ACMG Annual Clinical Genetics Virtual Meeting; April 2021.
Samuelson TW, Singh IP, Williamson BK, Falvey H, Lee WC, Odom D, McSorley D, Katz LJ. Quality of life in primary open-angle glaucoma and cataract: an analysis of VFQ-25 and OSDI from the iStent inject® Pivotal Trial. Am J Ophthalmol. 2021 Mar 15;229:220-9. doi: 10.1016/j.ajo.2021.03.007
Baraliakos X, Van den Bosch F, Machado PM, Gensler LS, Marzo-Ortega H, Sherif B, Quebe-Fehling E, Porter B, Gaillez C, Deodhar A. Achievement of remission endpoints with secukinumab over 3 years in active ankylosing spondylitis: pooled analysis of two phase 3 studies. Rheum Ther. 2021 Mar;8(1):273-88. doi: 10.1007/s40744-020-00269-6
Wyrwicz L, Elimova E, Blum SI, Xiao H, Davenport E, Wang J, Hunter S, Li M, Kondo K, Mohler M. Health-related quality of life (HRQOL) in patients (pts) with advanced gastric cancer/gastroesophageal junction cancer (GC/GEJC) or esophageal adenocarcinoma (EAC): interim results of nivolumab plus chemotherapy (N+C) versus (C) from CheckMate 649. Poster presented at the 2021 ASCO Conference; June 4, 2021. [abstract] J Clin Oncol. 2021; 39(Suppl 15):4066. doi: 10.1200/JCO.2021.39.15_suppl.4066
Zhou X, Garbinsky D, Ouyang J, Davenport E, Agarwal I, Oberdhan D. Longitudinal data on treatment duration and compliance from autosomal dominant polycystic kidney disease clinical trials with tolvaptan. Nephrol Dial Transplant. 2020 Jun;35(Supplement_3):gfaa139.S. doi: 10.1093/ndt/gfaa139.SO093
Zhou X, Davenport E, Ouyang J, Hoke M, Garbinsky D, Agarwal I, Oberdhan D. Population characteristics across an expanded pooled database of multiple autosomal dominant polycystic kidney disease clinical studies. Nephrol Dial Transplant. 2020 Jun;35(Supplement_3):gfaa142.P. doi: 10.1093/ndt/gfaa142.P0037
Zhou X, Davenport EK, Ouyang J, Krasa H, Garbinsky D, Agarwal I, Oberdhan D. Population characteristics across a pooled database of multiple Autosomal Dominant Polycystic Kidney Disease (ADPKD) clinical studies. Poster presented at the 2020 National Kidney Foundation Spring Clinical Meeting; March 2020. New Orleans, LA. [abstract] Am J Kidney Dis. 2020 Apr; 75(4):663.
Samuelson T, Singh IP, Katz LJ, Falvey H, Odom D, McSorley D. Effect of iStent inject trabecular micro-bypass on patient-reported outcomes for those with open-angle glaucoma and coexisting cataracts. Poster presented at the ISPOR 2019 European Conference; November 6, 2019. Copenhagen, Denmark. [abstract] Value Health. 2019 Dec; 22(S3).
Simpson EL, de Bruin-Weller M, Eckert L, Whalley D, Guillemin I, Reaney M, Chen Z, Nelson L, Qin S, Bansal A, Gadkari A. Responder threshold for Patient-Oriented Eczema Measure (POEM) and Children's Dermatology Life Quality Index (CDLQI) in adolescents with atopic dermatitis. Dermatol Ther. 2019 Dec;9(4):799-805. doi: 10.1007/s13555-019-00333-2
Nelson LM, DiBenedetti D, Pariser DM, Glaser DA, Hebert AA, Hofland H, Drew J, Ingolia D, Gillard KK, Fehnel S. Development and validation of the axillary sweating daily diary: a patient-reported outcome measure to assess axillary sweating severity. J Patient Rep Outcomes. 2019 Sep 5;3(1):59. doi: 10.1186/s41687-019-0148-8
Himmler S, Mueller M, Sherif B, Ostwald D. A case study applying a novel approach to estimate the social impact of a medical innovation - the use of secukinumab for psoriatic arthritis in Germany. Expert Rev Pharmacoecon Outcomes Res. 2019 Jul 29. doi: 10.1080/14737167.2019.1644169
Hofheinz R, Marian M, Bartsch J, Odom D. Effect of regorafenib in delaying definitive deterioration in health-related quality of life across three tumor types. Poster presented at the 2019 ESMO World Congress on Gastrointestinal Cancer Conference; July 2019. Barcelona, Spain.
Houghton K, Ainsworth C, Stull D, Haberland C, Filonenko A, Seitz C, Gerlinger C. Exploring variability in patient-reported pain and treatment efficacy in patients with endometriosis. Poster presented at the 2019 ISPOR 24th Annual International Meeting; May 20, 2019. New Orleans, LA.
Clark M, Berry P, Martin S, Harris N, Sprecher D, Olitsky S, Hoag JB. Nosebleeds in hereditary hemorrhagic telangiectasia: development of a patient-completed daily eDiary. Laryngoscope Investig Otolaryngol. 2018 Nov 14;3(6):439-45. doi: 10.1002/lio2.211
Coates L, Gossec L, Kirkham B, Sherif B, Gaillez C, Mpofu S, Jugl SM, Karyekar C, Gandhi KK. Minimal disease activity among active psoriatic arthritis patients treated with Secukinumab: 2-year results from a multicenter, randomized, double-blind, parallel-group, placebo-controlled phase III study. Arthritis Care Res (Hoboken). 2018 Oct;70(10):1529-35. doi: 10.1002/acr.23537