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
Neofytos D, Pagliuca A, Houghton K, Broughton E, de Figueiredo Valente MLN, Jiang L, Enoch DA, Gruener B, Herbrecht R, Lahmer T, Lortholary O, Melenotte C, De Rosa FG, Garcia-Vidal C, Jimenez M, Fernandez M, Cornely O. Effectiveness, safety, and patterns of real-world isavuconazole use in Europe (2015-2019). Infect Dis Ther. 2024 Dec;13(12):2527-43. doi: 10.1007/s40121-024-01064-4
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
Grivas P, Barata P, Moon H, Gupta S, Hutson T, Sternberg C, Brown J, Dave V, Downey C, Shillington A, Katzenstein H, Kirker M, Hanson S, Liu F, Morris V, Bhanegaonkar A, Sonpavde G. Avelumab first-line maintenance (1LM) therapy for locally advanced/metastatic urothelial carcinoma (la/mUC): results from the realworld US PATRIOT-II study. Poster presented at the Academy of Managed Care Pharmacy (AMCP) 2024; April 15, 2024. New Orleans, LA. [abstract] J Manag Care Spec Pharm. 2024 Apr; 30(4-a):S123.
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
Zografos LJ, Andrews E, Wolin DL, Calingaert B, Davenport EK, Michel A, Latocha M, Schmidt-Ott UM, Lovic N, Brunck LR, Johnson KT, Suzart-Woischnik K. Evaluation of physician knowledge of safety and safe use information for intravitreal aflibercept injection in Europe: a second survey of physicians following dissemination of updated risk-minimization materials. Pharmaceut Med. 2024 Jan;38(1):63-73. doi: 10.1007/s40290-023-00506-7
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.
Williams V, Romano (DeMuro) C, Clark M, Korver D, Williams N, Goss D, Naujoks C, Marvel J. Psychometric evaluation of an electronic Asthma Symptom Diary for young children. J Patient Rep Outcomes. 2023 Oct 30;7(1):105. doi: 10.1186/s41687-023-00647-y
Sinclair G, Sension M, Dretler A, Schneider S, Schubert C, Merrill D, Richardson D, Sherif B, Zografos L, Garris C. Clinical outcomes at month 6 after initiation of cabotegravir and rilpivirine long-acting (CAB+RPV LA) in an observational real-world study (BEYOND). Poster presented at the IDWeek 2023; October 13, 2023. Boston, MA.
Dandachi D, Cunningham D, Valenti W, Phoenix J, Nguyen K, Teichner P, Jean-Louis A, Reynolds M, Richardson D, Garris C. Perspectives of people with HIV (PWH) 6 months following a switch to cabotegravir and rilpivirine long-acting (CAB+RPV LA) in an observational real-world US study (BEYOND). Poster presented at the IDWeek 2023; October 13, 2023. Boston, MA.
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
Sinclair G, Cunningham D, Gupta SK, Richardson D, Reynolds M, Nelson K, Puga A, Garris C. US Healthcare Provider Perspectives on the initiation of cabotegravir and rilpivirine long-acting (CAB+RPV LA) in an observational real-world study (BEYOND). Poster presented at the 17th Annual ACTHIV 2023 Conference; May 4, 2023. Phoenix, AZ.
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
Sung A, Houghton K, Cornely OA, Neofytos D, Pagliuca A, Capparella MR, Aguado J, Jimenez M, Gheorghe M. Effectiveness, safety, and patterns of real-world isavuconazole use—a retrospective medical record review study of patients with invasive aspergillosis. Poster presented at the 33rd European Congress of Clinical Microbiology & Infectious Diseases (ECCMID) 2023; April 15, 2023. Copenhagen, Denmark.
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
DeMuro C, Finelli L, Lewis S, Williams V, Martin E, Phillips M, Saretsky T, Osborne R, Norquist J. Assessing the patient experience of respiratory syncytial virus infection: development of a patient-reported outcome measure. Health Qual Life Outcomes. 2023 Feb 28;21(1):20. doi: 10.1186/s12955-022-02066-x
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
Williams V, Romano (DeMuro) C, Clark M, Goss D, Naujoks C, Marvel J. Psychometric evaluation of a new electronic pediatric asthma symptom diary. Poster presented at the ISPOR Europe 2022; November 8, 2022. Vienna, Austria. [abstract] Value Health. 2022 Dec 1; 25(12):S428. doi: 10.1016/j.jval.2022.09.2130
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
Whalley D, Morrison R, Balp MM, Joubert Y, McKenna SJ, Christen L, Naujoks C, Gardner DD, Dery E, Lacombe A. Adaptation of the urticaria patient daily diary for caregiver completion in pediatric chronic spontaneous urticaria. 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.2022.09.2077
Paller AS, Yosipovitch G, Weidinger S, DiBenedetti D, Whalley D, Gadkari A, Guillemin I, Zhang H, Eckert L, Chao J, Bansal A, Chuang CC, Delevry D. Development, psychometric validation and responder definition of worst itch scale in children with severe atopic dermatitis. Dermatol Ther. 2022 Dec;12(12):2839-50. doi: 10.1007/s13555-022-00804-z
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
Hsu RK, Phoenix J, Sinclair GI, Gupta SK, Puga A, Nguyen K, Schubert CK, Merrill D, Richardson D, Nelson K, Reynolds M, Zografos L, Jean-Louis A, Garris C. US Healthcare Provider Perspectives on the initiation of cabotegravir and rilpivirine long-acting (CAB+RPV LA) in an observational real-world study (BEYOND). Poster presented at the IDWeek 2022 Conference; October 21, 2022. Washington, DC.
Li D, Darden C, Osman N, Sayeed S, Jackson L, Garbinsky D, Chauhan A. Real-world clinical and patient-reported outcomes from the longitudinal telotristat ethyl treatment registry of patients with neuroendocrine tumors. Cancer Manag Res. 2022 Oct 17;14:3009-20. doi: 10.2147/CMAR.S386419
Crawford R, Allas S, Tremel N, Weiss B, Robo C, Morrison R, Murphy D, Doward L. Development of the Hypoparathyroidism daily diary of symptom experience (HPT-DD-SE) and the Hypoparathyroidism life impact questionnaire (HPT-LIQ). Poster presented at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting 2022; September 9, 2022. Austin, TX.
Paller A, Siegried E, Marron SE, Clark M, Kosa K, Whalley D, Chao J, Bansal A, Sun Y, Chuang C, Wang Z. Development and validation of a caregiver-reported numeric rating scale for measuring sleep quality in children aged 6 months to <6 years with atopic dermatitis. Poster presented at the 2022 Society for Investigative Dermatology (SID) Annual Meeting; May 19, 2022. Portland, OR. [abstract] J Invest Dermatol. 2022 Aug; 142(8 supplement):S42. doi: 10.1016/j.jid.2022.05.256
Paller A, Siegfried E, Marron SE, Clark M, DiBenedetti D, Nelson L, Chao J, Bansal A, Sun Y, Chuang C, Wang Z. Development and validation of a caregiver-reported numeric rating scale for measuring pruritus in children aged 6 months to <6 years with atopic dermatitis. Poster presented at the 2022 Society for Investigative Dermatology (SID) Annual Meeting; May 2022. Portland, OR. [abstract] J Invest Dermatol. 2022 Aug; 142(8 supplement):S25. doi: 10.1016/j.jid.2022.05.154
Paller A, Siegfried E, Marron SE, Clark M, Harris N, Quin S, Chao J, Bansal A, Sun Y, Chuang C, Wang Z. Development and validation of a caregiver reported numeric rating scale for measuring skin pain in children aged 6 months to 5 years with atopic dermatitis. Poster presented at the 2022 Society for Investigative Dermatology (SID) Annual Meeting; May 19, 2022. Portland, OR. [abstract] J Invest Dermatol. 2022 Aug 1; 142(8 supplement):S42. doi: 10.1016/j.jid.2022.05.257
Crawford R, Allas S, Tremel N, Weiss B, Robo C, Morrison R, Murphy D, Doward L. Living with an "invisible disease”: a qualitative study with patients with hypoparathyroidism. Poster presented at the Endocrine Society Annual Meeting (ENDO) 2022; June 11, 2022. Atlanta, GA.
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
Clark M, Romano C, Olayinka-Amao O, Whalley D, Crawford R, Pathak P, Brindicci C, Garg K, Kordy K, Everhard F, Patalano F, Roesler Z, Sutton T, Goransson O, Landles R, Naujoks C, Marvel J, Keininger DL. Development and content validation of a self-completed, electronic Pediatric Asthma Symptom Diary. J Patient Rep Outcomes. 2022 Mar 20;6(1):25. doi: 10.1186/s41687-022-00432-3
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
Wyrwich KW, Winnette R, Bender R, Gandhi K, Williams N, Harris N, Nelson L. Validation of the alopecia areata patient priority outcomes (AAPPO) questionnaire in adults and adolescents with alopecia areata. Dermatol Ther. 2022 Jan;12(1):149-66. doi: 10.1007/s13555-021-00648-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.
Li D, Darden C, Sayeed S, Jackson L, Garbinsky D, Osman N. Patient-reported clinical and productivity outcomes from the longitudinal telotristat ethyl treatment registry. Poster presented at the 2021 Virtual Annual Symposium of the North American Neuroendocrine Tumor Society (NANETS); November 3, 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.