Publications

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Displaying 1 through 50 of 68 publications.

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.

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.

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. 2022 Oct 4. doi: 10.1016/j.jval.2022.08.004

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

Hakimi Z, Wilson K, McAughey E, Pochopien M, Wojciechowski P, Toumi M, Knight C, Sarda SP, Patel N, Wiseman C, de Castro NP, Nazir J, Kelly RJ. The cost-effectiveness, of pegcetacoplan compared with ravulizumab for the treatment of paroxysmal nocturnal hemoglobinuria, in a UK setting. J Comp Eff Res. 2022 Sep;11(13):969-85. doi: 10.2217/cer-2022-0076

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).

Hillmen P, Jia X, Knight C, Sarda S, Mody-Patel N, Intorcia M, Dingli D. Model structure considerations for cost-effectiveness evaluation of C3 inhibitor pegcetacoplan versus C5 inhibitor in patients with paroxysmal nocturnal hemoglobinuria. Poster presented at the 2021 Virtual AMCP; April 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

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.

Ayodele O, Parikh RC, Ajmera A, Esterberg E, Desai NK, Williams J, Katzka DA. Assessing the diagnostic journey, burden of disease, and treatment patterns in patients with eosinophilic esophagitis in a real world setting. Poster presented at the ACG 2020 Virtual Annual Scientific Meeting; October 23, 2020.

Ayodele O, Parikh RC, Esterberg E, Ajmera M, Goodwin B, Desai NK, Williams J, Katzka DA. Characterizing the burden of disease in younger children, adults, and older adults with eosinophilic esophagitis in a real world setting. Poster presented at the ACG 2020 Virtual Annual Scientific Meeting; October 23, 2020.

Mader G, Purser MF, Mladsi DM, Sanon M, Oberdhan D, Watnick TJ, Seliger SL. Benefit of tolvaptan on time to ESRD for patients with rapidly progressing autosomal dominant polycystic kidney disease (ADPKD): a disease progression model. Poster presented at the 2020 Virtual American Society of Nephrology Kidney Week Conference; October 22, 2020. [abstract] J Am Soc Nephrol. 2020 Oct 22; 31:492.

Mader G, Mladsi DM, Zhou X, Sanon M, Wang J, Willey CJ, Seliger SL. Mortality risk in elderly ESRD patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD): study findings using data from the United States Renal Data System. Poster presented at the 2020 Virtual American Society of Nephrology Kidney Week Conference; October 22, 2020. [abstract] J Am Soc Nephrol. 2020 Oct; 31:503-4.

Ueda K, Takura T, Fujikoshi S, Meyers J, Nagar SP, Enomoto H. Longitudinal assessment of pain management among the employed Japanese population with knee osteoarthritis. Clin Interv Aging. 2020 Jun 26;2020(15):1003-12. doi: 10.2147/CIA.S242083

Copley-Merriman C, Yang X, Juniper M, Amin S, Yoo HK, Sen S. Impact of neurofibromatosis type 1 and plexiform neurofibromas on patient-reported health-related quality of life. Poster presented at the 2020 ISPOR Virtual Conference; May 2020. [abstract] Value Health. 2020 May 1; 23(Suppl 1):S344. doi: 10.1016/j.jval.2020.04.371

Mansfield C, Boeri M, Coulter J, Baranowski E, Sparks S, Hamed A. Preferences for genetic tests: trading off speed of diagnosis, availability of treatment, and cost. Poster presented at the 2020 ISPOR Virtual Conference; May 2020. [abstract] Value Health. 2020 May 1; 23(Suppl 1):S345. doi: 10.1016/j.jval.2020.04.094

Purser M, Gallagher M, Mladsi D, Weber JM, Andermariam B, Kaye JA, Chawla A. Development of key criteria for an economic model for a potentially curative one-time treatment for sickle cell disease and evaluation of published models with respect to these criteria. Hemasphere. 2020;p.1098-9.

Kabadi SM, Goyal RK, Nagar SP, Davis KL, Le H, Du XL, Jain P, Wang ML, Romaguera JE, Kaye JA. Overall survival, adverse events, and economic burden in Medicare-insured patients with mantle cell lymphoma receiving cancer-directed therapy. Presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition; December 7, 2019. Orlando, FL. [abstract] Blood. 2019 Nov 13; 134(Supplement_1):63. doi: 10.1182/blood-2019-122841

Goyal RK, Nagar SP, Kabadi SM, Davis KL, Le H, Kaye JA. Overall survival, adverse events, and economic burden in Medicare patients with chronic lymphocytic leukemia receiving cancer-directed therapy. Presented at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition; December 9, 2019. Orlando, FL. [abstract] Blood. 2019 Nov 13; 134(Supplement 1):796. doi: 10.1182/blood-2019-122842

Ueda K, Takura T, Fujikoshi S, Meyers J, Nagar SP, Enomoto H. A claims database longitudinal study of the economic burden among patients with knee osteoarthritis in Japan. Poster presented at the 34th Annual Research Meeting of the Japanese Orthopaedic Association; October 17, 2019. Yokohama, Japan.

Martin S, Chen K, Harris N, Vera-Llonch M, Krasner A. Development of a patient-reported outcome measure for chronic hypoparathyroidism. Adv Ther. 2019 Aug;36(8):1999-2009. doi: 10.1007/s12325-019-00999-2

Harrington M, Hareendran A, Skalicky A, Wilson H, Clark M, Mikl J. Assessing the impact on caregivers caring for patients with rare pediatric lysosomal storage diseases: development of the Caregiver Impact Questionnaire. J Patient Rep Outcomes. 2019 Jul 23;3(1):44. doi: 10.1186/s41687-019-0140-3

Zuluaga Sanchez S, Purser M, Mader G, Gould IG, Knight C, Johnson NB, Patel M, Jensen J, Odom T. Improved quality of life and life-years in patients with infantile-onset SMA following treatment with nusinersen. Poster presented at the 2019 ISPOR 24th Annual International Meeting; May 21, 2019. New Orleans, LA.

Zuluaga Sanchez S, Purser M, Mader G, Gould IG, Knight C, Patel M, Odom T, Jensen J, Johnson NB. Improved quality of life for patients and caregivers among patients with later-onset SMA following treatment with Nusinersen. Poster presented at the 2019 ISPOR 24th Annual International Meeting; May 21, 2019. New Orleans, LA.

Mader G, Purser M, Mladsi D, Sanon M, Oberdhan D, Watnick T, Seliger S. Modeling disease progression in patients at risk of rapid progression in Autosomal Dominant Polycystic Kidney Disease (ADPKD): using the mayo classification prediction equation. Poster presented at the 2019 National Kidney Foundation Spring Clinical Meeting; May 9, 2019. Boston, MA.