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
Brockbank J, Hancock H, Khare A, Joshi P, Risson V. Cost-effectiveness of [177Lu]Lu-PSMA-617 in progressive PSMA-positive metastatic castration-resistant prostate cancer. Poster presented at the ISPOR Europe 2024; November 17, 2024. Barcelona, Spain. [abstract] Value Health. 2024 Dec; 27(12 Supplement):S121. doi: 10.1016/j.jval.2024.10.619
Adelakun A, Dolph M, Matthews E, Oglebsy A, Campbell K, Davis A, Xu J, Hardy I, Danchenko N, Anderson SJ. Cost-effectiveness and public-health impact of cabotegravir long-acting injectable for HIV pre-exposure prophylaxis in Canada. Poster presented at the HIV Drug Therapy Glasgow 2024; November 10, 2024. Glasgow, Scotland. [abstract] J Int AIDS Soc. 2024 Nov; 27:234-5.
Yousif Z, Nangia A, Abeysinghe S, Jiang J, Sanchirico M, Fan T. Comparative efficacy of biologics for the induction of clinical remission and response in biologic-naïve patients with moderate-to-severe crohn's disease: outcomes from frequentist and bayesian network meta-analyses. Poster presented at the American College of Gastroenterology (ACG) Annual Scientific Meeting 2024; October 29, 2024. Philadelphia, PA.
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
O'Brien P, Campbell K, Anderson SJ, Cornic L, Davis A, Schroeder M. Cost-effectiveness of every two month Cabotegravir Long-Acting (CAB-LA) for Pre-exposure Prophylaxis (PrEP) compared with daily oral Tenofovir Disoproxil Fumarate (TDF)/ emtricitabine (FTC) as PrEP to prevent HIV-1 UK. Poster presented at the ISPOR 2024; May 7, 2024. Atlanta, GA. [abstract] Value Health. 2024 Jun; 27(6 Supplement):S109. doi: 10.1016/j.jval.2024.03.580
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
Niyazov A, Simou E, Kirker M, Houghton K, Le Moine JG, Chang J, Yarr S, Barnett C, Mladsi D. Pre-progression health state utility values from the TALAPRO-2 clinical trial in patients with metastatic castration-resistant prostate cancer unselected for DDR mutations. Poster presented at the ISPOR Europe 2023; November 14, 2023. Copenhagen, Denmark. [abstract] Value Health. 2023 Dec; 26(12 Supplement):S149. doi: 10.1016/j.jval.2023.09.772
Niyazov A, Simou E, Kirker M, Houghton K, Le Moine JG, Chang J, Yarr S, Barnett C, Mladsi D. Pre-progression health state utility values from the TALAPRO-2 clinical trial in patients with metastatic castration-resistant prostate cancer with HRR mutations. Poster presented at the ISPOR Europe 2023; November 14, 2023. Copenhagen, Denmark. [abstract] Value Health. 2023 Dec; 26(12 Supplement):S119. doi: 10.1016/j.jval.2023.09.772
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
Morris MJ, Armstrong AJ, Chi K, de Bono J, Herrmann K, Krause B, Rahbar K, Sartor O, Tagawa ST, Nagarajah J, Wei XX, Nordquist LT, Koshkin VS, Saad F, Vickers A, Ghouse R, Wu J, Mirante O, Fizazi K. Association of health-related quality of life with efficacy outcomes in the VISION study of patients with metastatic castration-resistant prostate cancer. Poster presented at the ESMO Congress 2023; October 22, 2023. Madrid, Spain.
Njue A, Nuabor W, Lyall M, Margulis A, Mauskopf J, Curcio D, Kurosky S, Gessner BD, Begier E. Systematic literature review of risk factors for poor outcomes among adults with respiratory syncytial virus infection in high-income countries. Open Forum Infect Dis. 2023 Oct 20;10(11):ofad513. doi: 10.1093/ofid/ofad513
Fan Q, Hawe E, Yang J, Burgess B, Bullano M, Mokdad A. Cost outcomes of noninhibitor patients with hemophilia A switching from prophylaxis with factor VIII to emicizumab: a meta-analysis of real-world evidence studies in the United States. Poster presented at the AMCP Nexus 2023; October 16, 2023. Orlando, FL. [abstract] J Manag Care Pharm. 2023 Oct; 29(10-a Suppl):S35.
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.
Nangia A, Simou E, Abeysinghe S, Colosia A, Bektas M, Workman J, Gao S, Bello N. Incidence rate of infections and association between lupus nephritis and serious infections in patients with systemic lupus erythematosus: systematic literature review and meta-analyses. Poster presented at the ISPOR 2023 Conference; May 7, 2023. Boston, MA. [abstract] Value Health. 2023 Jun; 26(6 supplement):S180. doi: 10.1016/j.jval.2023.03.972
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
Yousif Z, Schultz BG, Candela N, Harold S, Bell J, Turpin R, Hartley L. Systematic literature review to evaluate randomized clinical trials of advanced therapies for moderate to severe crohn's disease. Poster presented at the ISPOR 2023 Conference; May 6, 2023. Boston, MA. [abstract] Value Health. 2023 Jun; 26(6 supplement):S15. doi: 10.1016/j.jval.2023.03.085
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
Bello N, Meyers KJ, Workman J, Hartley L, McMahon M. Cardiovascular events and risk in patients with systemic lupus erythematosus: systematic literature review and meta-analysis. Lupus. 2023 Mar;32(3):325-41. doi: 10.1177/09612033221147471
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
Bello N, Meyers KJ, Workman J, Marcano Belisario J, Cervera R. Systematic literature review and meta-analysis of venous thromboembolism events in systemic lupus erythematosus. Rheum Ther. 2023 Feb 1;10(1):7-34. doi: 10.1007/s40744-022-00513-1
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
Ainsworth C, Higuchi K, Simou E, Guyot P, Araujo L, Greene N. Relative differences in brain volume loss with teriflunomide vs other disease-modifying therapies in relapsing multiple sclerosis: a systematic review and network meta-analysis. Poster presented at the AMCP Nexus 2022; October 11, 2022. National Harbor, MD. [abstract] J Manag Care Spec Pharm. 2022 Oct; 28(10-a):S73-4. Previously presented at the ACTRIMS Forum 2022.
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, 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
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
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
Hawe E, Castro CV, Johannesen J, Belisario JM, Mordin M, Sawant R, Petrillo Billet J. Driving outcomes in mild cognitive impairment/Alzheimer's disease - a review of association between driving simulator measures and clinical outcomes assessments. Poster presented at the ISPOR 2022 Conference; May 15, 2022. Washington, DC. [abstract] Value Health. 2022 Jun; 25(6 S1).
Hawe E, Castro CV, Johannesen J, Belisario JM, Mordin M, Sawant R, Billet JP. Impact of Huntington's disease on driving: a review of driving simulator measures and association with clinical outcomes assessments. Poster presented at the ISPOR 2022 Conference; May 15, 2022. Washington, DC. [abstract] Value Health. 2022 Jun; 25(6 S1).
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
Ainsworth C, Higuchi K, Simou E, Guyot P, Greene N. Relative differences in brain volume loss with teriflunomide versus other disease-modifying therapies in relapsing multiple sclerosis: a systematic review and network meta-analysis. Poster presented at the ACTRIMS Forum 2022; February 24, 2022. West Palm Beach, FL.
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