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
Gold BD, Goodwin B, Davis K, Sweeney C, Ziemiecki R, Jiang J, Fan T, Boules M, Chen ST, Katzka DA. Satisfaction with and adherence to off-label corticosteroids in adolescents and adults with eosinophilic esophagitis: results of a web-based survey in the United States. J Clin Gastroenterol. 2025 Feb;59(2):138-46. doi: 10.1097/MCG.0000000000002006
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
Chapman KR, Guyot P, Ledanois O, Soliman M, Wang Z, Xu Y, Nangia A, Cheng W-H. Efficacy of dupilumab versus tezepelumab in asthma patients: matching-adjusted indirect comparison of long-term extension trials. Poster presented at the American College of Allergy, Asthma & Immunology (ACAAI) 2024 Annual Scientific Meeting; October 25, 2024. Boston, MA.
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.
Kohli RK, Hawe E, Harold S, Simou E, Chrysos AE, Orfanos P. Utility scores in chronic spontaneous urticaria: a systematic literature review and statistical analysis. Poster presented at the European Academy of Dermatology & Venereology (EADV) Congress 2024; September 25, 2024. Amsterdam, Netherlands.
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.
Krause T, Fleming A, Hurst M, Zema C, Costello J, Hawe E, Ling C. Prognostic factors and surrogate endpoints in obstructive hypertrophic cardiomyopathy: a systematic literature review. Poster presented at the ISPOR Europe 2023; November 14, 2023. Copenhagen, Denmark. [abstract] Value Health. 2023 Dec; 26(12 Supplement):S36. doi: 10.1016/j.jval.2023.09.189
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.
Fassett MJ, Reed SD, Rothman KJ, Pisa F, Schoendorf J, Wahdan Y, Peipert JF, Gatz J, Ritchey ME, Armstrong MA, Raine-Bennett T, Postlethwaite D, Getahun D, Shi JM, Xie F, Chiu VY, Im TM, Takhar HS, Wang J, Anthony MS. Risks of uterine perforation and expulsion associated with intrauterine devices. Obstet Gynecol. 2023 Sep 1;142(3):641-51. doi: 10.1097/AOG.0000000000005299
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
Herrera-Restrepo O, Davis K, Sweeney C, Davenport E, Ghaswalla P, Buck PO. Hepatitis A and B vaccination in adults at risk: a survey of US healthcare providers' attitudes and practices. Hum Vaccin Immunother. 2022 Nov 30;18(6):2123180. doi: 10.1080/21645515.2022.2123180
Anthony MS, Zhou X, Schoendorf J, Reed SD, Getahun D, Armstrong MA, Gatz J, Peipert JF, Raine-Bennett T, Fassett MJ, Saltus CW, Ritchey ME, Ichikawa L, Shi JM, Alabaster A, Wahdan Y, Wang J, Xie F, Merchant M, Hunter S, Chiu VY, Postlethwaite D, Rothman KJ, Im TM, Chillemi G, Takhar HS, Asiimwe A, Pisa F. Demographic, reproductive, and medical risk factors for intrauterine device explusion. Obstet Gynecol. 2022 Nov 3;140(6):1017-30. doi: 10.1097/AOG.0000000000005000
Bell T, Fernandez M, Khan S, Tumminello B, Bektas M, Heyes CE, Oton AB. Patient burden of illness associated with desmoid tumors. Poster presented at the 2022 Desmond Tumor Research Foundation (DTRF) Annual Meeting; September 24, 2022. Philadelphia, PA.
Gatz JL, Armstrong MA, Postlethwaite D, Raine-Bennett T, Chillemi G, Alabaster A, Merchant M, Reed SD, Ichikawa L, Getahun D, Fassett MJ, Shi JM, Xie F, Chiu VY, Im TM, Takhar HS, Wang J, Saltus CW, Ritchey ME, Asiimwe A, Pisa F, Schoendorf J, Wahdan Y, Zhou X, Hunter S, Anthony MS, Peipert JF. Association between intrauterine device type and risk of perforation and device expulsion: results from the APEX-IUD study. Am J Obstet Gynecol. 2022 Jul 1;227(1):57.e1-57.. doi: 10.1016/j.ajog.2022.03.062
Reed SD, Zhou X, Ichikawa L, Gatz JL, Peipert JF, Armstrong MA, Raine-Bennett T, Getahun D, Fassett MJ, Postlethwaite DA, Shi JM, Asiimwe A, Pisa F, Schoendorf J, Saltus CW, Anthony MS, APEX-IUD study team. Intrauterine device-related uterine perforation incidence and risk (APEX-IUD): a large multisite cohort study. Lancet. 2022 Jun 4;399(10341):2103-12. doi: 10.1016/S0140-6736(22)00015-0
Saidha S, Bell J, Harold SE, Marcano Belisario JS, Hawe E, Shao Q, Wyse K, Maiese EM. A systematic literature review of immunoglobulin levels among B-cell–depleting therapies and risk of infections in relapsing multiple sclerosis. Poster presented at the 2022 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting; June 1, 2022. National Harbor, MD.
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
Getahun D, Fassett MJ, Gatz J, Armstrong MA, Peipert JF, Raine-Bennett T, Reed SD, Zhou X, Schoendorf J, Postlethwaite D, Shi JM, Saltus CW, Wang J, Xie F, Chiu VY, Merchant M, Alabaster A, Ichikawa LE, Hunter S, Im TM, Takhar HS, Ritchey ME, Chillemi G, Pisa F, Asiimwe A, Anthony MS. Association between menorrhagia and risk of intrauterine device-related uterine perforation and device expulsion: results from the APEX-IUD Study. Am J Obstet Gynecol. 2022 Jun;399(10341):2103-12. doi: 10.1016/j.ajog.2022.03.025
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
Armstrong MA, Raine-Bennett T, Reed SD, Gatz J, Getahun D, Schoendorf J, Postlethwaite D, Fassett MJ, Peipert JF, Saltus CW, Merchant M, Alabaster A, Zhou X, Ichikawa L, Shi JM, Chiu VY, Xie F, Hunter S, Wang J, Ritchey ME, Chillemi G, Im TM, Takhar HS, Pisa F, Asiimwe A, Anthony MS. Association of the timing of postpartum intrauterine device insertion and breastfeeding with risks of intrauterine device expulsion. JAMA Netw Open. 2022 Feb 28;5(2):e2148474. doi: 10.1001/jamanetworkopen.2021.48474
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
Ghaswalla P, Davis K, Sweeney C, Davenport E, Trofa A, Herrera-Restrepo O, Buck PO. Health care providers' knowledge, practices, and barriers to hepatitis vaccination guidelines. J Nurse Pract. 2022 Feb 16;18:403-10.
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
Herrera-Restrepo O, Ghaswalla P, Davis K, Sweeney C, Davenport E, Andani A, Buck PO. What can Europe learn from HCP knowledge and attitudes towards hepatitis A vaccination in the US? Poster presented at the 14th European Public Health Virtual Conference 2021 (EPH); November 10, 2021.
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.
Gold BD, Goodwin B, Davis K, Sweeney C, Ziemiecki R, Jiang J, Fan T, Boules M, Desai NK, Katzka DA. Patient satisfaction and adherence to corticosteroids in adolescents and adults with eosinophilic esophagitis in a real-world setting in the US. Poster presented at the 2021 Annual Scientific Meeting & Postgraduate Course (ACG); October 24, 2021. Las Vegas, NV. [abstract] Am J Gastroenterol. 2021 Oct; 116:S177.
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