Publications

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Displaying 1 through 50 of 88 publications.

Johannesen K, Herring WL, Chopra R, Elpers B, Dong O. Using patient-level simulation to estimate long-term survival in idiopathic pulmonary fibrosis. Poster to be given at the ISPOR Europe 2024; November 17, 2024. Barcelona, Spain.

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

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. 2024 May 15. doi: 10.1097/MCG.0000000000002006

Mansfield C, Boeri M, Coulter J, Baranowski E, Sparks S, An Haack K, Hamed A. The value of knowing: preferences for genetic testing to diagnose rare muscle diseases. Orphanet J Rare Dis. 2024 Apr 22;19(1):173. doi: 10.1186/s13023-024-03160-7

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

Craig T, Baptist AP, Anderson J, Vanegas A, Schultz BG, Blair C, Sing K, Juethner S. Quality of life among patients with hereditary angioedema within underrepresented racial or ethnic groups. Poster presented at the Western Society of Allergy, Asthma & Immunology (WSAAI) 2024 Annual Meeting; February 4, 2024. Koloa, HI.

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

Mansfield C, Nalysnyk L, Joshi D, Coulter J, Pulikottil-Jacob R. Impact of potential symptoms and risks associated with acid sphingomyelinase deficiency on patients and caregivers: a best-worst scaling study. Patient Prefer Adherence. 2023 Mar 30;17:927-39. doi: 10.2147/PPA.S381371

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.

Copley-Merriman C, Yang X, Juniper M, Amin S, Yoo HK, Sen SS. Natural history and disease burden of neurofibromatosis type 1 with plexiform neurofibromas: a systematic review. Adolesc Health Med Ther. 2021 May 19;12:55-66. doi: 10.2147/AHMT.S303456

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.

Mansfield C, Nalysnyk L, Joshi D, Coulter J, Pulikottil-Jacob R. Patient and parent preferences for symptom control in ASMD Type B and A/B using best-worst scaling methodology. Poster presented at the Virtual ISPOR 2021 Conference; May 2021. [abstract] Value Health. 2021 May; 24(5).

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

Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S14-20. doi: 10.18553/jmcp.2020.26.12-b.s14

Purser M, Gallagher M, Mladsi D, Weber JM, Andemariam B, Kaye JA, Chawla A. Evaluation of published models in sickle cell disease against key criteria for an economic model for a potentially curative one-time treatment. Poster presented at the Virtual ISPOR Europe 2020 Conference; November 2020. [abstract] Value Health. 2020 Dec; 23(S2). doi: 10.1016/j.jval.2020.08.098

Sun SX, Olayinka-Amao O, DiBenedetti D. Assessing patient experiences with prophylactic treatments for hemophilia A: concept elicitation for gene therapy. Poster presented at the 62nd Annual Virtual Meeting of the American Society of Hematology (ASH); December 5, 2020. [abstract] Blood. 2020 Nov 5; 136(Suppl 1):33-4.

Su J, Kosa K, DiBenedetti D. Patient-reported disease burden: in-depth interviews of patients with CAD. Poster presented at the 2020 Virtual 62nd ASH Annual Meeting and Exposition; December 6, 2020. [abstract] Blood. 2020 Nov 5; 136(Suppl 1):29-30.