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New treatment paradigms for ADPKD: moving towards precision medicine

Matthew B Lanktree1, Arlene B Chapman2

  • 1Nephrology Division, Department of Medicine, McMaster University, St Joseph's Healthcare Hamilton, 50 Charlton Avenue E, Hamilton, Ontario L8N 4A6, Canada.

Nature Reviews. Nephrology
|October 10, 2017
PubMed

Insights

Autosomal dominant polycystic kidney disease (ADPKD) management is advancing with precision medicine. Early interventions slow cyst growth, preserve kidney function, and improve quality of life, delaying the need for renal replacement therapy.

Area of Science:

  • Nephrology
  • Genetics
  • Precision Medicine

Background:

  • Autosomal dominant polycystic kidney disease (ADPKD) exhibits variable progression, including cyst development, kidney function decline, and diverse clinical manifestations.
  • Current management strategies aim to address symptoms and slow disease progression, but personalized approaches are emerging.

Purpose of the Study:

  • To review current and emerging interventions for ADPKD management before end-stage renal disease (ESRD).
  • To discuss precision medicine's role in tailoring ADPKD therapies based on individual patient characteristics.
  • To provide recommendations for managing renal complications associated with ADPKD.

Main Methods:

  • Review of pharmacological and non-pharmacological interventions for hypertension, proteinuria, and cyst growth.
  • Discussion of advances in genetics, imaging, and biomarker development for ADPKD.
  • Analysis of strategies for managing renal complications like cyst infection and nephrolithiasis.

Main Results:

  • Precision medicine approaches are nearing clinical implementation for ADPKD.
  • Early treatment, though burdensome, can slow cyst growth and delay ESRD.
  • Effective management of hypertension, proteinuria, and renal complications is crucial.

Conclusions:

  • Personalized therapeutic strategies hold significant promise for improving ADPKD patient outcomes.
  • Early intervention and proactive management of complications can preserve kidney function and enhance quality of life.
  • Advances in understanding ADPKD pathophysiology are driving the development of targeted therapies.

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