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Kidney Week

Abstract: TH-PO639

The Natural History of Focal and Segmental Glomerulosclerosis (FSGS)

Session Information

Category: Glomerular Diseases

  • 1402 Glomerular Diseases: Clinical, Outcomes, and Trials

Authors

  • Poyan-Mehr, Ali, The Permanente Medical Group Inc, San Francisco, California, United States
  • Li, Tingting, Washington University in St Louis, St Louis, Missouri, United States
  • Hsu, Raymond K., University of California San Francisco, San Francisco, California, United States
  • Singh, Tripti, University of Wisconsin-Madison, Madison, Wisconsin, United States
  • Messias, Nidia Cordeiro, Washington University in St Louis, St Louis, Missouri, United States
  • Wiegley, Nasim, University of California Davis, Davis, California, United States
  • Jen, Kuang-Yu, University of California Davis, Davis, California, United States
  • Lecker, Stewart H., Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States
  • Arora, Swati, Allegheny Health Network, Pittsburgh, Pennsylvania, United States
  • Waguespack, Dia Rose, The University of Texas Health Science Center at Houston John P and Katherine G McGovern Medical School, Houston, Texas, United States
  • Tchakarov, Amanda, The University of Texas Health Science Center at Houston John P and Katherine G McGovern Medical School, Houston, Texas, United States
  • Mendelssohn, Nancy D., The Permanente Medical Group Inc, San Francisco, California, United States
  • Seth, Divya, University of California San Francisco, San Francisco, California, United States
  • Mansour, Iyad S. M., Banner University Medical Center Tucson, Tucson, Arizona, United States
  • Legenzoff, Timothy, Allegheny Health Network, Pittsburgh, Pennsylvania, United States
  • Sandhu, Tejwinder S., The Permanente Medical Group Inc, San Francisco, California, United States
  • Drozd, Alice J., The Permanente Medical Group Inc, San Francisco, California, United States
  • Mena, Jose D., Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States
  • Sood, Ria Sonya, The Permanente Medical Group Inc, San Francisco, California, United States
  • Sanchez, Gabriela, The Permanente Medical Group Inc, San Francisco, California, United States
  • Yip, Jennifer, The Permanente Medical Group Inc, San Francisco, California, United States
  • Stillman, Isaac Ely, Mount Sinai Health System, New York, New York, United States
Background

FSGS is the tissue manifestation of various kidney diseases that ultimately lead to end-stage kidney disease in most patients. Their rare and heterogeneous presentations and unpredictable responses to therapies pose a tremendous challenge. Here we report on one of the most extensive natural history studies of patients with a biopsy diagnosis of FSGS.

Methods

Nine collaborating sites are enrolling 800 patients with primary, genetic, or FSGS of unknown cause (Fig. 1A) diagnosed between Jan. 1, 2000, and Aug. 1, 2021. The time of the first kidney biopsy is the index date, flanked by pre-biopsy baseline patient characteristics and longitudinal follow-up data of outcomes of interest (Fig. 2B & C).

Results

We have collaboratively created a decentralized registry with 366 data elements for patients with glomerular diseases, secured regulatory approval for deidentified data sharing, and enrolled 103 eligible patients to date.

Conclusion

Large retrospective studies of rare diseases face difficult-to-overcome challenges, including variability in data, uniform definitions, and a need for a regulatory framework for data sharing. Here, we have leveraged a decentralized registry to exchange deidentified patient information, creating a large multi-site retrospective study for patients with FSGS across highly diverse populations and healthcare settings.

Funding

  • Commercial Support – Travere Therapeutics