Rare Diseases
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
BACKGROUND AND AIMS: Limited real-world nontertiary care evidence on the patient therapeutic journey and disease burden of eosinophilic esophagitis (EoE) exists. The aim was to collect real-world data on the EoE patient journey across different age groups.
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
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
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
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
OBJECTIVES: The impact of eosinophilic esophagitis (EoE) on the daily life and ability to function in adolescents and adults with EoE in the United States of America was examined.
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
GOALS: We assessed satisfaction with and adherence to off-label corticosteroids in patients with eosinophilic esophagitis (EoE) in the United States.
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
OBJECTIVES: To identify evidence gaps in the literature on the burden of illness and treatment of bullous pemphigoid (BP) to support the launch of efgartigimod to treat this rare disease.