Publications
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Dellon ES, Goodwin B, Liu E, de los Santos B, Korgaonkar S, Meyers J, Schaeffer-Koziol CR, Terreri B, Shah ED. Eosinophilic esophagitis-related healthcare resource utilization and associated costs for payers and patients: a retrospective cohort study of US health insurance claims data. Poster presented at the ISPOR 2025; May 14, 2025. Montréal, Canada.
Dellon ES, Goodwin B, Liu Y, de los Santos B, Korgaonkar S, Meyers J, Schaeffer-Koziol CR, Terreri B, Shah ED. Healthcare resource utilization and associated costs in patients with eosinophilic esophagitis: a retrospective cohort study of US health insurance claims data. Poster presented at the Digestive Disease Week (DDW) 2025; May 3, 2025. San Diego, CA.
Abbatemarco J, Yu J, Kleman M, Myers K, Poulos C, Conway D. Patient preferences for treatment features in Neuromyelitis Optica Spectrum Disorder (NMOSD): results from a discrete choice experiment (DCE). Poster presented at the American Academy of Neurology 2025 Annual Meeting; April 5, 2025. San Diego, CA. [abstract] Neurology. 2025 Apr 8; 104(7_Suppl 1):7-8.013. doi: 10.1212/WNL.0000000000208724
Craig T, Baptist AP, Anderson J, Zaragoza-Urdaz RH, Burnette AF, Kelbel TE, Riedl MA, Vanegas A, Boyle K, Bartsch JL, Darden C, Brown TM, Schultz BG, Blair C, Sing K, Fox D, Juethner S. Hereditary angioedema: patient health care experiences within underrepresented racial and ethnic groups in the United States. Ann Allergy Asthma Immunol. 2025 Apr;134(4):465-473.e. doi: 10.1016/j.anai.2025.01.007
Silver SM, Houghton K, Hitchens A, Ansquer VD, Ciepielewska M. Real-world assessment of the patient profile, clinical characteristics, treatment patterns, and outcomes associated with erythropoietic and X-linked protoporphyria. J Dermatol. 2025 Mar;52(3):416-22. doi: 10.1111/1346-8138.17607
Percy AK, Ryther R, Marsh ED, Neul JL, Benke TA, Berry-Kravis EM, Feyma T, Lieberman DN, Ananth AL, Fu C, Buhrfiend C, Barrett A, Doshi D, Darwish M, An D, Bishop KM, Youakim JM. Results from the phase 2/3 DAFFODIL study of trofinetide in girls aged 2-4 years with Rett syndrome. Med. 2025 Feb 28. doi: 10.1016/j.medj.2025.100608
Greenhawt M, Bansal P, Goodwin B, Korgaonkar S, Liu Y, Meyers J, Shah E, Terreri B, Dellon E, Schaeffer-Koziol C. High incidence and prevalence of eosinophilic esophagitis in the USA identified: an analysis of US health insurance claims data. Poster presented at the 2025 American Academy of Allergy Asthma & Immunology (AAAAI)/WAO Joint Congress; February 28, 2025. San Diego, CA.
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
Yee K, Poulos C, Bussberg C, Myers K. Patient characteristics associated with treatment preference for generalized Myasthenia Gravis (gMG): a multivariate analysis. Poster to be given at the ISPOR Europe 2024; November 17, 2025. Barcelona, Spain. [abstract] Value Health. 2024 Dec; 27(12 Suppl):S524-5.
Johannesen K, Herring WL, Chopra R, Elpers B, Dong O. Using patient-level simulation to estimate long-term survival in idiopathic pulmonary fibrosis. Poster presented at the ISPOR Europe 2024; November 17, 2024. Barcelona, Spain. [abstract] Value Health. 2024 Dec; 27(12 Supplement):S459-60. doi: 10.1016/j.jval.2024.10.2345
Buck P, Sweeney C, Georgieva M, Kunzweiler C, Tossonian H, Boyle K, Chirila C, Crawford R, Wolowacz S, Pesch M. Family spillover impact of congenital CMV in the United States and Canada: study methodology. Poster presented at the European Congenital Cytomegalovirus Initiative (ECCI) 2024; October 9, 2024. Leiden, the Netherlands.
Perreault S, Hukin J, Erker C, Silayuv EB, Hamilton MA, Kim Y, Jimenez MI, Candrilli SD. Treatment patterns and healthcare resource utilization of pediatric patients with neurofibromatosis type 1 and unresectable plexiform neurofibroma in Canada. Poster presented at the 2024 Global NF Conference; June 20, 2024. Brussels, Belgium.
Dong OM, Herring W, Chopra P, Elpers B, Johannesen K. A targeted literature review of lung function decline in idiopathic pulmonary fibrosis to improve survival predictions in cost-effectiveness analyses. Poster presented at the ISPOR 2024; May 8, 2024. Atlanta, GA. [abstract] Value Health. 2024 Jun; 27(6 Supplement):S148. doi: 10.1016/j.jval.2024.03.2259
Margulis AV, Riera-Guardia N, Calingaert B, Armstrong R, Fletcher M, Patki K, Weiner E, Rivero-Ferrer E. Fetal and neonatal alloimmune thrombocytopenia: a systematic literature review and meta-analysis of adverse pregnancy-related outcomes to support the development of a novel prophylactic therapeutic. Poster presented at the AMCP Annual Meeting 2024; April 15, 2024. New Orleans, LA. [abstract] J Manag Care Spec Pharm. 2024 Apr; 30(4-a):S96-7. doi: 10.18553/jmcp.2024.30.4-a.s1
Yee KS, Lewis S, Evans E, Romano (DeMuro) C, Alexanderian D. Caregiver experiences and observations of intrathecal idursulfase-IT treatment in a phase 2/3 trial in pediatric patients with neuronopathic mucopolysaccharidosis II. Orphanet J Rare Dis. 2024 Mar 10;19(1):100. doi: 10.1186/s13023-024-03034-y
Ayodele O, Parikh RC, Esterberg E, Ajmera M, Goodwin B, Williams J, Desai NK, Katzka DA. Treatment patterns and persistent disease activity in patients with eosinophilic esophagitis: a retrospective cohort study. Gastro Hep Adv. 2024 Mar 2;3(5):659-70. doi: 10.1016/j.gastha.2024.02.007
Brown TM, Vera-Llonch M, Kanu C, Sikora Kessler A, Yarlas A, Fehnel SE. Cross-sectional quantitative evaluation of a novel patient-reported outcome measure in familial chylomicronemia syndrome. Patient Relat Outcome Meas. 2024 Feb 15;15:45-59. doi: 10.2147/PROM.S441583
Gold BD, Goodwin B, Davis K, Sweeney C, Reynolds M, Jiang J, Fan T, Boules M, Chen S, Katzka DA. Health-related quality of life and perceived stigma in eosinophilic esophagitis: a real-world, US, web-based survey. Gastro Hep Adv. 2024;3(8):1087-97. doi: 10.1016/j.gastha.2024.07.015
Davis S, Edwards T, Norcross L, Fehnel S, Beaudet A, Eckart M, Fastenau J. Use of the National Cancer Institute Patient-Reported Outcomes version of the common terminology criteria for adverse events to assess treatment tolerability in pulmonary arterial hypertension: qualitative patient research findings in current and former users of oral selexipag. J Patient Rep Outcomes. 2023 Dec 18;7(1):134. doi: 10.1186/s41687-023-00673-w.
Gildea L, Copley-Merriman C, Arvin-Berod C, Vande Walle K, Verheesen P, Stoykov I. Evidence gap analysis of the burden of illness and treatment of bullous pemphigoid. Poster presented at the ISPOR Europe 2023; November 13, 2023. Copenhagen, Denmark. [abstract] Value Health. 2023 Dec; 26(12 supplement):S27. doi: 10.1016/j.jval.2023.09.141
Fishman J, Anderson S, Talbird SE, Dingli D. Analysis of costs per responder in US adults with a paroxysmal nocturnal hemoglobinuria with suboptimal response to prior eculizumab treatment. Hematol Rep. 2023 Oct 13;15(4):578-91. doi: 10.3390/hematolrep15040060
Joly F, DasMahapatra P, DiBenedetti DB, Kosa K, Hill QA. Development of the cold agglutinin disease symptoms and impact questionnaire (CAD-SIQ). Eur J Haematol. 2023 Aug;111(2):211-9. doi: 10.1111/ejh.13984
Martin S, Harris N, Romanus D. Evaluating meaningful changes in physical functioning and cognitive declines in metachromatic leukodystrophy: a caregiver interview study. J Patient Rep Outcomes. 2023 Jul 17;7(1):70. doi: 10.1186/s41687-023-00595-7
Houghton K, Hitchens A, Ciepielewska M. Healthcare resource use and costs among patients with erythropoietic protoporphyria and x-linked protoporphyria in the United States. Poster presented at the ISPOR 2023 Conference; May 7, 2023. Boston, MA. [abstract] Value Health. 2023 Jun; 26(6 supplement):S155. doi: 10.1016/j.jval.2023.03.2501
Cotter SP, Schwartz L, Strong TV, Bender RH, Fehnel S. The Prader-Willi syndrome anxiousness and distress behaviors questionnaire: development and psychometric validation. Value Health. 2023 Feb 1;26(2):243-50. doi: 10.1016/j.jval.2022.08.004
Paret K, Ronquest N, Droege M. Economic modeling considerations for rare neurodegenerative diseases of infancy and early childhood. Poster presented at the ISPOR Europe 2022; November 9, 2022. Vienna, Austria. [abstract] Value Health. 2022 Dec 1; 25(12):S171. doi: 10.1016/j.jval.2022.09.830
Petrillo J, Paret K, Sawant R, Koenig A, Wolowacz S, Ronquest N, Rickards H. Estimation of health state utilities in Huntington's disease: a targeted review. Poster presented at the HSG 2022 Annual Meeting; November 2022. Tampa, FL. [abstract] J Huntingtons Dis. 2022 Oct 31; 11(s1):S31. Previously presented at the European Huntington's Disease Network (EHDN) 2022 Plenary Meeting. doi: 10.3233/JHD-229005
Joly F, DasMahapatra P, Su J, DiBenedetti D, Kosa K, Hill QA. Creation of a cold agglutinin disease (CAD) specific patient-reported outcome (PRO) symptom measure. Poster presented at the 84th Annual Meeting of the Japanese Society of Hematology; October 14, 2022. Fukuoka, Japan.
Petrillo J, Paret K, Sawant R, Koenig A, Wolowacz S, Ronquest N, Rickards H. Estimation of health state utilities in Huntington's disease: a targeted review. Poster presented at the European Huntington's Disease Network (EHDN) 2022 Plenary Meeting; September 18, 2022. Bologna, Italy. [abstract] J Neurol Neurosurg Psychiatry. 2022 Sep 12; 93(suppl 1):A45-6. doi: 10.1136/jnnp-2022-ehdn.118
Ronquest NA, Paret K, Lucas A, Ciepielewska M, Hagan M. Quantifying the value of introducing an oral drug delivery option for edaravone: a review of analyses evaluating the economic impact of oral versus intravenous formulations. Clinicoecon Outcomes Res. 2022 Jul 27;14:499-511. doi: 10.2147/CEOR.S359025
Whalley D, Belongie K, Frangiosa T, Krasa H, Mladsi DM, Twiss J, Wolowacz S. Understanding the patient experience of erythropoietic protoporphyria and x-linked protoporphyria: a qualitative study. Poster presented at the ISPOR 2022 Conference; May 15, 2022. Washington, DC. [abstract] Value Health. 2022 Jun; 25(6 S1).
Ciepielewska M, Hagan M, Houghton K, Hitchens A, Ansquer V, Silver SM. Real-world treatment patterns and healthcare resource utilization among patients diagnosed with erythropoietic protoporphyria and x-linked protoporphyria: results from a US electronic medical records study. Poster presented at the ISPOR 2022 Conference; May 17, 2022. Washington, DC. [abstract] Value Health. 2022 Jun; 25(6 S1).
Anderson-Smits C, Mitchell S, Hawe E, Hartley L. Use of intravenous immunoglobulin in patients with chronic inflammatory demyelinating polyneuropathy: a systematic literature review. Poster presented at the 2022 Peripheral Nerve Society (PNS) Annual Meeting; May 14, 2022. Miami, FL.
Anderson-Smits C, Park M, Bell J, Mitchell S, Hartley L, Hawe E. Subcutaneous immunoglobulin use in immunoglobulin-naïve patients with primary immunodeficiency: a systematic review. Immunotherapy. 2022 Apr;14(5):373-87. doi: 10.2217/imt-2021-0265
Yee KS, Alexanderian D, Hamm L, Martin S, Olayinka-Amao B, Whiteman DAH. Clinical investigator perspectives on the effects of intrathecal idursulfase-IT treatment in children with neuronopathic mucopolysaccharidosis type II. Poster presented at the 2022 18th Annual World Symposium; February 7, 2022. San Diego, CA.
Graham CN, Watson C, Barlev A, Stevenson M, Dharnidharka VT. Mean lifetime survival estimates following solid organ transplantation in the US and UK. J Med Econ. 2022 Jan;25(1):230-7. doi: 10.1080/13696998.2022.2033050
Lunan M, Hartley L, Pearson I, Doyle S. Targeted literature review of the burden of illness in late-stage Duchenne Muscular Dystrophy (DMD). Poster presented at the Virtual ISPOR Europe 2021; November 3, 2021. [abstract] Value Health. 2021 Dec; 24(12):S2.
Anderson S, Talbird SE, Fishman J, Mody-Patel N, Sarda SP. Budget impact of pegcetacoplan, a complement c3 inhibitor, for the treatment of paroxysmal nocturnal hemoglobinuria in US adults. Poster presented at the Virtual ISPOR Europe 2021; November 30, 2021. [abstract] Value Health. 2021 Dec; 24(12):S2.
Joly F, DasMahapatra P, Su J, DiBenedetti D, Kosa K, Hill Q. Development of a cold Agglutinin disease-specific patient-reported outcome symptom measure. Poster presented at the 2021 63rd American Society of Hematology (ASH) Annual Meeting & Exposition; December 13, 2021. [abstract] Blood. 2021 Nov 23; 138(supplement 1):4057. doi: 10.1182/blood-2021-147199
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.
Foreman PK, Margulis AV, Alexander K, Shediac R, Calingaert B, Harding A, Pladevall-Vila M, Landis S. Birth prevalence of phenylalanine hydroxylase deficiency: a systematic literature review and meta-analysis. Orphanet J Rare Dis. 2021 Jun 3;16(1):253. doi: 10.1186/s13023-021-01874-6
Goyal RK, Jain P, Nagar SP, Le H, Kabadi SM, Davis K, Kaye JA, Du XL, Wang M. Real-world evidence on survival, adverse events, and health care burden in Medicare patients with mantle cell lymphoma. Leuk Lymphoma. 2021 Jun;62(6):1325-34. doi: 10.1080/10428194.2021.1919662
Davis A, Jiang J, Talbird S, Turpin RS, Mellott CE, Boules M, Wojtowicz AM, Fan T. Pharmacological costs for the treatment of eosinophilic esophagitis in the USA. Poster presented at the 2021 Virtual Digestive Disease Week (DDW) Conference; May 21, 2021. Previously presented at the 2021 AMCP Conference.
Wu B, Morland K, Classi P, Sketch MR, Nelson A, Goyal RK, Davis KL, Oudiz R. Economic burden of patients with pulmonary arterial hypertension prior to initiation of treprostinil: a retrospective analysis of US claims. Poster presented at the ATS International 2021 Conference; May 14, 2021.
DuBrock HM, Nathan SD, Reeve BB, Kolaitis NA, Mathai SC, Classi PM, Nelsen AC, Olayinka-Amao B, Norcross LN, Martin SA. Pulmonary hypertension due to interstitial lung disease or chronic obstructive pulmonary disease: a patient experience study of symptoms and their impact on quality of life. Pulm Circ. 2021 Apr 1;11(2):204589402. doi: 10.1177/20458940211005641
Goyal RK, Nagar SP, Kabadi SM, Le H, Davis KL, Kaye JA. Overall survival, adverse events, and economic burden in patients with chronic lymphocytic leukemia receiving systemic therapy: real-world evidence from the medicare population. Cancer Med. 2021 Apr;10(8):2690-702. doi: 10.1002/cam4.3855
Davis AE, Jiang J, Talbird SE, Turpin R, Mellott CE, Boules M, Wojtowicz AM, Fan T. Pharmacological costs for the treatment of eosinophilic esophagitis in the USA. Poster presented at the 2021 Virtual AMCP; April 2021.
Anderson S, Talbird S, Fishman J. Cost per responder analysis for pegcetacoplan and eculizumab in the treatment of adults with paroxysmal nocturnal hemoglobinuria. Blood. 2021;138(Suppl 1):4956. doi: 10.1182/blood-2021-153190
Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S8-S14. doi: 10.18553/jmcp.2020.26.12-b.s8
Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: role of the complement system, pathogenesis, and pathophysiology. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S3-8. doi: 10.18553/jmcp.2020.26.12-b.s3