Lillian Jean Kaplan International Prize for Advancement in the Understanding of Polycystic Kidney Disease
The Lillian Jean Kaplan International Prize for Advancement recognizes individuals whose seminal scientific work constitutes tangible achievement toward improving knowledge and treatment of PKD. More than one prize may be awarded to residents of any country.
2024 Award Winner: Michael Caplan, M.D., Ph.D.
C.N.H. Long Professor and Chair of Cellular and Molecular Physiology and Professor of Cell Biology at Yale University School of Medicine.
Tolvaptan is the only approved drug therapy for adults with ADPKD at CKD stages 2-4. New trials are underway to assess children and ARPKD.
Many trials against several different targets have failed in recent years, including lixivaptan, lanreotide, pasireotide (target AVPR2); Venglustat (target lipid); everolimus, sirolimus (target mTOR); bardoxolone (target TF, NRF-2); simvastatin, etanercept (target metabolic pathways).
PKD International participated in the Patient Consensus Meeting in Brussels, represented by President Flavia Galletti and board member Uwe Korst. The event was hosted by CompCure, a Danish organization dedicated to research and advocacy in rare kidney diseases, bringing together leading experts, patient organizations, kidney health advocates, and policymakers to define priorities for kidney health.
Distinguished speakers included Prof. Detlef Böckenhauer, representing the International Society of Nephrology (ISN), who delivered a presentation on "Kidney Health and the International and European Public Policy Agenda." The meeting included participation from EKHA, EKPF, FEDERG, and ERKNet.
With consultant assistance, PKD International conducted strategic planning work including analysis of the organization's actual situation using RACI methodology, identification of benefits of PKDI membership, and drafting of vision statement, mission statement, organizational values, and governance principles.
PKD International's mission is to improve the lives of an estimated 12.5 million people affected by PKD worldwide, including their families and caregivers. The organization accomplishes this by uniting patients, families, scientists, and healthcare professionals committed to ending PKD.
The KDIGO Clinical Practice Guideline for the Evaluation, Management, and Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) represents the first KDIGO guideline on this subject. The guideline was recently made available for public review and will be published in 2024.
The guideline aims to serve as a useful resource for clinicians and patients by providing actionable recommendations with infographics based on a rigorous, formal, systematic literature review. Another aim is to propose research recommendations for areas in which there are gaps in knowledge.
The ADPKD Guideline is co-chaired by Olivier Devuyst, MD, PhD (Switzerland), and Vicente Torres, MD, PhD (United States).
Patient representatives of PKD International/ ERKNET were involved
Chapters of the guideline are:
Chapter 1: Nomenclature, Diagnosis, Prognosis, and Prevalence
Chapter 2: Kidney Manifestations
Chapter 3: CKD Management and Progression, Kidney Failure, and Kidney Replacement Therapy
Chapter 4: Therapies to Delay the Progression of Kidney Disease
Chapter 5: Polycystic Liver Disease
Chapter 6: Intracranial Aneurysms (ICA) and Other Extrarenal Manifestations
Chapter 7: Lifestyle and Psychosocial Aspects
Chapter 8: Pregnancy and Reproductive Issues
Chapter 9: Pediatric Issues
Chapter 10: Approaches to the Management of People with ADPKD
