Browsing by Author "Trachtman, Howard"

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    Association of Histologic Variants in FSGS Clinical Trial with Presenting Features and Outcomes
    (Clinical Journal of the American Society of Nephrology, 2013-03-07) D’Agati, Vivette D; Alster, Joan M; Jennette, J Charles; Thomas, David B; Pullman, James; Savino, Daniel A; Cohen, Arthur H; Gipson, Debbie S; Gassman, Jennifer J; Radeva, Milena K; Moxey-Mims, Marva M; Friedman, Aaron L; Kaskel, Frederick J; Trachtman, Howard; Alpers, Charles E; Fogo, Agnes B; Greene, Tom H; Nast, Cynthia C
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    FSGS Recurrence Collaboration: Report of a Symposium.
    (Glomerular diseases, 2024-01) Gipson, Debbie S; Wang, Chia-Shi; Salmon, Eloise; Gbadegesin, Rasheed; Naik, Abhijit; Sanna-Cherchi, Simone; Fornoni, Alessia; Kretzler, Matthias; Merscher, Sandra; Hoover, Paul; Kidwell, Kelley; Saleem, Moin; Riella, Leonardo; Holzman, Lawrence; Jackson, Annette; Olabisi, Opeyemi; Cravedi, Paolo; Freedman, Benjamin Solomon; Himmelfarb, Jonathan; Vivarelli, Marina; Harder, Jennifer; Klein, Jon; Burke, George; Rheault, Michelle; Spino, Cathie; Desmond, Hailey E; Trachtman, Howard
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    Rationale and design of the Nephrotic Syndrome Study Network (NEPTUNE) Match in glomerular diseases: designing the right trial for the right patient, today.
    (Kidney international, 2024-02) Trachtman, Howard; Desmond, Hailey; Williams, Amanda L; Mariani, Laura H; Eddy, Sean; Ju, Wenjun; Barisoni, Laura; Ascani, Heather K; Uhlmann, Wendy R; Spino, Cathie; Holzman, Lawrence B; Sedor, John R; Gadegbeku, Crystal; Subramanian, Lalita; Lienczewski, Chrysta C; Manieri, Tina; Roberts, Scott J; Gipson, Debbie S; Kretzler, Matthias; NEPTUNE investigators
    Glomerular diseases are classified using a descriptive taxonomy that is not reflective of the heterogeneous underlying molecular drivers. This limits not only diagnostic and therapeutic patient management, but also impacts clinical trials evaluating targeted interventions. The Nephrotic Syndrome Study Network (NEPTUNE) is poised to address these challenges. The study has enrolled >850 pediatric and adult patients with proteinuric glomerular diseases who have contributed to deep clinical, histologic, genetic, and molecular profiles linked to long-term outcomes. The NEPTUNE Knowledge Network, comprising combined, multiscalar data sets, captures each participant's molecular disease processes at the time of kidney biopsy. In this editorial, we describe the design and implementation of NEPTUNE Match, which bridges a basic science discovery pipeline with targeted clinical trials. Noninvasive biomarkers have been developed for real-time pathway analyses. A Molecular Nephrology Board reviews the pathway maps together with clinical, laboratory, and histopathologic data assembled for each patient to compile a Match report that estimates the fit between the specific molecular disease pathway(s) identified in an individual patient and proposed clinical trials. The NEPTUNE Match report is communicated using established protocols to the patient and the attending nephrologist for use in their selection of available clinical trials. NEPTUNE Match represents the first application of precision medicine in nephrology with the aim of developing targeted therapies and providing the right medication for each patient with primary glomerular disease.
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    Responsiveness of the PROMIS® measures to changes in disease status among pediatric nephrotic syndrome patients: a Midwest pediatric nephrology consortium study.
    (Health Qual Life Outcomes, 2017-08-23) Selewski, David T; Troost, Jonathan P; Cummings, Danyelle; Massengill, Susan F; Gbadegesin, Rasheed A; Greenbaum, Larry A; Shatat, Ibrahim F; Cai, Yi; Kapur, Gaurav; Hebert, Diane; Somers, Michael J; Trachtman, Howard; Pais, Priya; Seifert, Michael E; Goebel, Jens; Sethna, Christine B; Mahan, John D; Gross, Heather E; Herreshoff, Emily; Liu, Yang; Carlozzi, Noelle E; Reeve, Bryce B; DeWalt, Darren A; Gipson, Debbie S
    BACKGROUND: Nephrotic syndrome represents a condition in pediatric nephrology typified by a relapsing and remitting course, proteinuria and the presence of edema. The PROMIS measures have previously been studied and validated in cross-sectional studies of children with nephrotic syndrome. This study was designed to longitudinally validate the PROMIS measures in pediatric nephrotic syndrome. METHODS: One hundred twenty seven children with nephrotic syndrome between the ages of 8 and 17 years participated in this prospective cohort study. Patients completed a baseline assessment while their nephrotic syndrome was active, a follow-up assessment at the time of their first complete proteinuria remission or study month 3 if no remission occurred, and a final assessment at study month 12. Participants completed six PROMIS measures (Mobility, Fatigue, Pain Interference, Depressive Symptoms, Anxiety, and Peer Relationships), the PedsQL version 4.0, and two global assessment of change items. RESULTS: Disease status was classified at each assessment: nephrotic syndrome active in 100% at baseline, 33% at month 3, and 46% at month 12. The PROMIS domains of Mobility, Fatigue, Pain Interference, Depressive Symptoms, and Anxiety each showed a significant overall improvement over time (p < 0.001). When the PROMIS measures were compared to the patients' global assessment of change, the domains of Mobility, Fatigue, Pain Interference, and Anxiety consistently changed in an expected fashion. With the exception of Pain Interference, change in PROMIS domain scores did not correlate with changes in disease activity. PROMIS domain scores were moderately correlated with analogous PedsQL domain scores. CONCLUSION: This study demonstrates that the PROMIS Mobility, Fatigue, Pain Interference, and Anxiety domains are sensitive to self-reported changes in disease and overall health status over time in children with nephrotic syndrome. The lack of significant anchoring to clinically defined nephrotic syndrome disease active and remission status may highlight an opportunity to improve the measurement of HRQOL in children with nephrotic syndrome through the development of a nephrotic syndrome disease-specific HRQOL measure.