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01.07.2023 | RESEARCH

The real world experience of pediatric primary hyperoxaluria patients in the PEDSnet clinical research network

verfasst von: Christina B. Ching, Kimberley Dickinson, John Karafilidis, Nicole Marchesani, Lisa Mucha, Nuno Antunes, Hanieh Razzaghi, Levon Utidjian, Karyn Yonekawa, Douglas E. Coplen, Samina Muneeruddin, William DeFoor, Kyle O. Rove, Christopher B. Forrest, Gregory E. Tasian

Erschienen in: European Journal of Pediatrics | Ausgabe 9/2023

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Abstract

The rarity of primary hyperoxaluria (PH) challenges our understanding of the disease. The purpose of our study was to describe the course of clinical care in a United States cohort of PH pediatric patients, highlighting health service utilization. We performed a retrospective cohort study of PH patients < 18 years old in the PEDSnet clinical research network from 2009 to 2021. Outcomes queried included diagnostic imaging and testing related to known organ involvement of PH, surgical and medical interventions specific to PH-related renal disease, and select PH-related hospital service utilization. Outcomes were evaluated relative to cohort entrance date (CED), defined as date of first PH-related diagnostic code. Thirty-three patients were identified: 23 with PH type 1; 4 with PH type 2; 6 with PH type 3. Median age at CED was 5.0 years (IQR 1.4, 9.3 years) with the majority being non-Hispanic white (73%) males (70%). Median follow-up between CED and most recent encounter was 5.1 years (IQR 1.2, 6.8). Nephrology and Urology were the most common specialties involved in care, with low utilization of other sub-specialties (12%-36%). Most patients (82%) had diagnostic imaging used to evaluate kidney stones; 11 (33%) had studies of extra-renal involvement. Stone surgery was performed in 15 (46%) patients. Four patients (12%) required dialysis, begun in all prior to CED; four patients required renal or renal/liver transplant.

Conclusion: In this large cohort of U.S. PH children, patients required heavy health care utilization with room for improvement in involving multi-disciplinary specialists.

What is Known:

• Primary hyperoxaluria (PH) is rare with significant implications on patient health. Typical involvement includes the kidneys; however, extra-renal manifestations occur.

• Most large population studies describe clinical manifestations and involve registries.

What is New:

• We report the clinical journey, particularly related to diagnostic studies, interventions, multispecialty involvement, and hospital utilization, of a large cohort of PH pediatric patients in the PEDSnet clinical research network.

• There are missed opportunities, particularly in that of specialty care, that could help in the diagnosis, treatment, and even prevention of known clinical manifestations.

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Titel: The real world experience of pediatric primary hyperoxaluria patients in the PEDSnet clinical research network
verfasst von: Christina B. Ching
Kimberley Dickinson
John Karafilidis
Nicole Marchesani
Lisa Mucha
Nuno Antunes
Hanieh Razzaghi
Levon Utidjian
Karyn Yonekawa
Douglas E. Coplen
Samina Muneeruddin
William DeFoor
Kyle O. Rove
Christopher B. Forrest
Gregory E. Tasian
Publikationsdatum: 01.07.2023
Verlag: Springer Berlin Heidelberg
Erschienen in: European Journal of Pediatrics / Ausgabe 9/2023
Print ISSN: 0340-6199
Elektronische ISSN: 1432-1076
DOI: https://doi.org/10.1007/s00431-023-05077-y

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The real world experience of pediatric primary hyperoxaluria patients in the PEDSnet clinical research network

Abstract

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Live-Webinar "Urologie und Sexualmedizin in der Praxis"

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Abstract

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