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Sézary syndrome (SS) is an aggressive cutaneous T-cell lymphoma with poor survival. We performed a retrospective review of SS patients at Emory University from 1990 to 2020. We collected data on race, clinical characteristics, therapy, and social determinants of health. Clinical endpoints were overall survival (OS) and time to next treatment (TTNT). Univariate association and multivariable analyses were assessed by Cox proportional hazards models. Among 62 patients, 45.2% were AA. The median OS and TTNT were 3.1 years and 6.3 months, respectively, with no difference by race. AA patients had a higher median baseline LDH (360 232, = 0.002) and a longer delay in initiation of systemic therapy compared to CC patients (3.17 2.14 months, = 0.039), but a shorter commute (<10 miles) and no difference in insurance coverage ( = 0.260). AA patients at an academic center had unique clinical features and treatment patterns, but similar survival to CC SS patients.
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http://dx.doi.org/10.1080/10428194.2022.2067999 | DOI Listing |
J Clin Invest
September 2025
Department of Clinical and Biomedical Sciences, Faculty of Health and Life Sciences, University of Exeter, Exeter, United Kingdom.
Understanding the genetic causes of diseases affecting pancreatic β cells and neurons can give insights into pathways essential for both cell types. Microcephaly, epilepsy and diabetes syndrome (MEDS) is a congenital disorder with two known aetiological genes, IER3IP1 and YIPF5. Both genes encode proteins involved in endoplasmic reticulum (ER) to Golgi trafficking.
View Article and Find Full Text PDFQual Life Res
September 2025
The Kids Research Institute Australia, The University of Western Australia, P.O. Box 855, West Perth, WA, 6872, Australia.
Purpose: CDKL5 deficiency disorder (CDD) is a rare developmental and epileptic encephalopathy. Greater understanding of the smallest meaningful improvements for individuals with CDD in clinical trials and practice is needed for a person-centred approach to treatment efficacy. This study explored how parent/caregivers of people with CDD understood meaningful improvements and described change for priority functional domains including communication, gross motor, fine motor, feeding.
View Article and Find Full Text PDFTissue Eng Regen Med
September 2025
Department of Ophthalmology and Visual Science, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, #505 BanPo-Dong, SeoCho-Gu, Seoul, 06591, Republic of Korea.
Background: Sjögren's syndrome (SS) is a chronic autoimmune disease delineated by excessive lymphocyte infiltration to the lacrimal or salivary glands, leading to dry eye and dry mouth. Exosomes secreted from mesenchymal stem cells (MSC) are known to have anti-inflammatory and tissue regeneration abilities. This study endeavored to demonstrate the effect of MSC-derived exosomes on the clinical parameter of dry eyes and associated pathology in SS mouse model.
View Article and Find Full Text PDFPharmacoeconomics
September 2025
Center for Innovation and Value Research, Alexandria, VA, USA.
Manufacturers of orphan drugs face several obstacles in meeting health technology assessment requirements because of poor availability of natural history data, small sample sizes, single-arm trials, and a paucity of established disease-specific endpoints. There is a need for specific considerations and modified approaches in health technology assessments that would account for the challenges in orphan drug development. Multistakeholder collaborations can benefit patients, their families, and the broader society and reduce the inequity faced by patients with rare diseases.
View Article and Find Full Text PDFSchmerz
September 2025
Forschungsgruppe Psychosomatische Rehabilitation, Abteilung für Psychosomatische Medizin, Zentrum für Innere Medizin und Dermatologie, Charité - Universitätsmedizin Berlin, 10098, Berlin, Deutschland.