Publications
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Sauchelli S, Levy C, Gnanasakthy A, Dave V, Doward L, Fitzgerald KA, Carson R. From clinical trials to informing clinical decision-making: a review of patient-reported outcomes in nononcology medicines approved by the European Medicines Agency (2018-2022). Front Pharmacol. 2025 Apr 11;16:1536401. doi: 10.3389/fphar.2025.1536401
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
Law EH, Hanson KA, Harries M, Korver D, Sherif B, Chirila C. Patient-reported outcome improvements following scalp hair regrowth among patients with alopecia areata: analysis of the ALLEGRO-2b/3 trial. J Dermatolog Trea. 2025 Feb 3;36(1):2460577. doi: 10.1080/09546634.2025.2460577
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
Sauchelli S, Levy C, Gnanasakthy A, Doward L, Fitzgerald K. Inclusion of patient-reported outcome endpoints in the summary of product characteristics for non-oncology drugs approved by the European Medicines Agency (2018-2022). Poster presented at the ISOQOL 31st Annual Conference; October 13, 2024. Cologne, Germany.
Shore S, Ervin C, Kosa K, Fehnel S, Salberg L, Butzner M, Heitner SB, Jacoby D, Saberi S. Qualitative interview study of patient-reported symptoms, impacts and treatment goals of patients with obstructive hypertrophic cardiomyopathy. BMJ Open. 2024 Sep 17;14(9):e081323. doi: 10.1136/bmjopen-2023-081323
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
Law E, Williams N, Korver D, Bender R, Mitra D, Schaefer G, Nelson LM. Determining meaningful thresholds for evaluating treatment efficacy in patients with alopecia areata. JEACP. 2024 Sep;3(4):1119-31. doi: 10.1002/jvc2.441
Morga A, Zimmermann L, Valluri U, Siddiqui E, McLeod L, Bender RH. Validation and application of thresholds to define meaningful change in vasomotor symptoms frequency: analysis of pooled SKYLIGHT 1 and 2 data. Adv Ther. 2024 Jul;41(7):2845-58. doi: 10.1007/s12325-024-02849-2
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
Law EH, Sherif B, Mostaghimi A, King B, Sinclair R, Mesinkovshka N, Hanson KA, Korver D, Chirila C. Patients with alopecia areata treated with ritlecitinib have improved patient-reported emotional symptoms and activity limitations due to hair loss: post hoc analyses of ALLEGRO-2b/3. Poster presented at the Maui Derm Hawaii 2024; January 22, 2024. Wailea, HI.
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
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
Kamudoni P, Amtmann D, Johns J, Cook KF, Salem R, Salek S, Raab J, Middleton R, Repovic P, Alschuler KN, von Geldern G, Wundes A, Barrett A, Olayinka-Amao B, Henke C. The validity, responsiveness, and score interpretation of the PROMISnq Physical Function - Multiple Sclerosis 15a short form in multiple sclerosis. Mult Scler Relat Disord. 2022 Jun;62:103753. doi: 10.1016/j.msard.2022.103753
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
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.
Kolotkin RL, Williams VSL, von Huth Smith L, Meincke HH, Qin S, Williams N, Fehnel SE. Confirmatory psychometric evaluations of the impact of weight on quality of life-lite clinical trials version (IWQOL-Lite-CT). Clin Obes. 2021 Oct;11(5):e12477. doi: 10.1111/cob.12477
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.
Brandt T, Frangiosa T, Biggar V, Taylor A, Valentine J, Keller B, Price M, DeMuro C, Abler V. Symptoms and treatment needs of people with dementia-related psychosis: a mixed-methods study of the patient experience. Clin Gerontol. 2021 Aug 9;45(3):681-95. doi: 10.1080/07317115.2021.1957050
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.
English M, Stoykova B, Slota C, Doward L, Siddiqui E, Crawford R, DiBenedetti D. Qualitative study: burden of menopause-associated vasomotor symptoms (VMS) and validation of PROMIS sleep disturbance and sleep-related impairment measures for assessment of VMS impact on sleep. J Patient Rep Outcomes. 2021 Apr 26;5(1):37. doi: 10.1186/s41687-021-00289-y
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
English M, Stoykova M, Slota C, Doward L, Siddiqui E, Crawford R, DiBenedetti D. Qualitative study of the burden of vasomotor symptoms associated with menopause and content validity of two PROMIS sleep measures in menopausal women. Presented at the 2021 IMS Conference (Conference cancelled); February 4, 2021. Previously presented at the NAMS 2020 Virtual Meeting.
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
English M, Stoykova B, Slota C, Doward L, Siddiqui E, Crawford R, DiBenedetti D. Qualitative study of the burden of vasomotor symptoms associated with menopause and content validity of two PROMIS sleep measures in menopausal women. Poster presented at the NAMS 2020 Virtual Meeting; September 30, 2020.
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