Resumen
Rationale & Objective: Focal segmental glomerulosclerosis (FSGS) is a heterogeneous disorder with a high risk of progression to kidney failure. There are no approved therapies for FSGS, and futility of treatment is poorly defined. The Cure Glomerulonephropathy (CureGN) study offers the opportunity to describe the characteristics of participants who started immunosuppressive therapy (IST), never received IST, or in whom this treatment was discontinued. Study Design: An observational cohort. Settings & Participants: Participants enrolled in CureGN with FSGS and surveyed nephrologists. Interventions: The clinical and laboratory data from participants with FSGS who were enrolled in the CureGN observational cohort were reviewed to define features associated with withholding initial IST or terminating ongoing IST. Nephrologists were surveyed about what factors would influence their decision to prescribe or withdraw IST in patients with FSGS. Outcomes: (1) Identify factors associated with IST initiation and discontinuation in individuals with FSGS; and (2) Identify clinical and laboratory features nephrologists consider when they recommend against the use of IST at diagnosis (initiation of care) and during the course of disease. Results: Based on quantitative findings from the CureGN cohort and survey responses from practicing nephrologists, a low estimated glomerular filtration rate at presentation, significant glomerulosclerosis, and interstitial fibrosis and tubular atrophy on kidney biopsy make initiation of IST less likely. Limitations: Heterogeneous nature of the cohort and an inability to divide the patients into KDIGO subgroups of FSGS. Rationale for decision to stop or defer treatment was not available. More surveys were completed by pediatric providers, and the majority were completed by academic practitioners. Conclusions: The factors that impact decisions about IST initiation and discontinuation were consistent among pediatric and internal medicine nephrologists, namely advanced scarring and lower estimated glomerular filtration rate. We suggest that this information should be incorporated into patient management guidelines and clinical trial design. Plain Language Summary: Patients with focal segmental glomerulosclerosis (FSGS) are at high risk for progression to kidney failure and there are no approved therapies. This study from the CureGN consortium described clinical situations in which immunosuppressive therapy (IST) was started, not started, or discontinued in participants with FSGS. Furthermore, we surveyed nephrologists to better understand factors that influence the management of patients with FSGS. Participants in the CureGN cohort with a lower estimated glomerular filtration rate and advanced scarring on kidney biopsy were less likely to be started on IST and have this treatment discontinued, which was similar to what surveyed providers did in practice. Understanding the characteristics of these patients may help further develop management guidelines and assist in clinical trial design.
| Idioma original | English |
|---|---|
| Número de artículo | 100975 |
| Publicación | Kidney Medicine |
| Volumen | 7 |
| N.º | 4 |
| DOI | |
| Estado | Published - abr 2025 |
Nota bibliográfica
Publisher Copyright:© 2025 The Authors
Financiación
Support: Funding for the CureGN consortium is provided by U24DK100845 (formerly UM1DK100845), U01DK100846 (formerly UM1DK100846), U01DK100876 (formerly UM1DK100876), U01DK100866 (formerly UM1DK100866), and U01DK100867 (formerly UM1DK100867) from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). Patient recruitment is supported by NephCure Kidney International. Dates of funding for the first phase of CureGN was 9/16/2013-5/31/2019. Dates of funding for the second phase of CureGN was 6/1/2019 - 5/31/2024. Brooke Blazius, MD, Jonathan P. Troost, PhD, Jeffrey B. Kopp, MD, Rulan S. Parekh, MD, Brenda Gillespie, PhD, Isabelle Ayoub, MD, Mahmoud Kallash, MD, Rasheed Gbadegesin, MD, Pietro A. Canetta, MD, Tarak Srivastava, MD, Tracy E. Hunley, MD, Katherine E. Twombley, MD, Yonatan A. Peleg, MD, Larry A. Greenbaum, MD, Aftab S. Chishti, MD, Carla M. Nester, MD, Amy K. Mottl, MD, Susan L. Hogan, PhD, Virginie Royal, MD, Vivette D. D'Agati, MD, Kimberly J. Reidy, MD, Krzysztof Mucha, MD, PhD, Julia Steinke, MD, Simone Sanna-Cherchi, MD, David T. Selewski, MD, Dhruti P. Chen, MD, C. John Sperati, MD, Ronald J. Falk, MD, Howard Trachtman, MD, and Jason M. Kidd, MD, on behalf of Cure GlomeruloNephropathy (CureGN) Study Consortium, Research and study design: BB, JPT, HT, and JMK; data acquisition: BB, JPT, VR, VDA, HT, and JK; data analysis and interpretation: BB, JBK, RSP, BG, IA, MK, RG, PAC, TS, TEH, KET, PY, LAG, ASC, CMN, AKM, SLH, VR, VDA, KJR, KM, JS, SSC, DTS, DPC, CJS, RJF, HT, and JMK; supervision or mentorship: JPT, HT, and JMK. Each author contributed important intellectual content during article drafting or revision and accepts accountability for the overall work by ensuring that questions pertaining to the accuracy or integrity of any portion of the work are appropriately investigated and resolved. Funding for the CureGN consortium is provided by U24DK100845 (formerly UM1DK100845), U01DK100846 (formerly UM1DK100846), U01DK100876 (formerly UM1DK100876), U01DK100866 (formerly UM1DK100866), and U01DK100867 (formerly UM1DK100867) from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). Patient recruitment is supported by NephCure Kidney International. Dates of funding for the first phase of CureGN was September 16, 2013, to May 31, 2019. Dates of funding for the second phase of CureGN was from June 1, 2019, to May 31, 2024. The authors declare that they have no relevant financial interests. The authors acknowledge Dr Debbie Gipson for her assistance with the development of this study and article. We would also thank the participants of the CureGN study. Received August 5, 2024. Evaluated by 2 external peer reviewers, with direct editorial input from an Associate Editor and the Editor-in-Chief. Accepted in revised form November 4, 2024.
| Financiadores | Número del financiador |
|---|---|
| National Institute of Diabetes and Digestive and Kidney Diseases | |
| Nephcure | |
| CureGN | UM1DK100846, UM1DK100867, UM1DK100845, U01DK100876, UM1DK100866, U01DK100867, U01DK100866, UM1DK100876, U01DK100846, U24DK100845 |
ASJC Scopus subject areas
- Internal Medicine
- Nephrology
Huella
Profundice en los temas de investigación de 'Clinical Decision-Making About Immunosuppressive Treatment in Focal Segmental Glomerulosclerosis'. En conjunto forman una huella única.Citar esto
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