4.6 Review

Treatment of primary hyperoxaluria type 1

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Letter Urology & Nephrology

Is stiripentol truly effective for treating primary hyperoxaluria?

Cristina Martin-Higueras et al.

CLINICAL KIDNEY JOURNAL (2021)

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Effects of Oxalobacter formigenes in subjects with primary hyperoxaluria Type 1 and end-stage renal disease: a Phase II study

Bernd Hoppe et al.

Summary: In PH1 ESRD patients, long-term administration of OC5 was found to significantly reduce Pox concentrations, improve or stabilize cardiac function and clinical status, without increasing dialysis frequency. OC5 was safe and well-tolerated, showing promising results for potential clinical impact.

NEPHROLOGY DIALYSIS TRANSPLANTATION (2021)

Article Medicine, General & Internal

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

Sander F. Garrelfs et al.

Summary: Primary hyperoxaluria type 1 is a rare genetic disease caused by hepatic overproduction of oxalate, leading to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. This trial tested the efficacy of lumasiran, an RNAi therapeutic agent, in reducing hepatic oxalate production. The results showed that lumasiran significantly reduced urinary oxalate excretion, alleviating the cause of kidney failure in PH1 patients.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

Review Pediatrics

Transplantation outcomes in patients with primary hyperoxaluria: a systematic review

Elisabeth L. Metry et al.

Summary: The study suggests that combined liver-kidney transplantation leads to superior kidney graft survival compared to kidney transplantation alone. However, evidence for the advantages of sequentially performed liver-kidney transplantation or kidney transplantation in pyridoxine-responsive patients is scarce, emphasizing the need for further studies.

PEDIATRIC NEPHROLOGY (2021)

Editorial Material Urology & Nephrology

Stiripentol for the treatment of primary hyperoxaluria and calcium oxalate nephropathy

Christina M. Wyatt et al.

KIDNEY INTERNATIONAL (2020)

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Inherited conditions resulting in nephrolithiasis

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CURRENT OPINION IN PEDIATRICS (2020)

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End Points for Clinical Trials in Primary Hyperoxaluria

Dawn S. Milliner et al.

CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY (2020)

Article Medicine, Research & Experimental

Stiripentol protects against calcium oxalate nephrolithiasis and ethylene glycol poisoning

Marine Le Dudal et al.

JOURNAL OF CLINICAL INVESTIGATION (2019)

Review Urology & Nephrology

Recent advances in the identification and management of inherited hyperoxalurias

David J. Sas et al.

UROLITHIASIS (2019)

Article Urology & Nephrology

Oxalate Quantification in Hemodialysate to Assess Dialysis Adequacy for Primary Hyperoxaluria

Xiaojing Tang et al.

AMERICAN JOURNAL OF NEPHROLOGY (2014)

Article Biochemistry & Molecular Biology

Multiple mechanisms of action of pyridoxine in primary hyperoxaluria type 1

Sonia Fargue et al.

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2013)

Review Medicine, General & Internal

Primary Hyperoxaluria

Pierre Cochat et al.

NEW ENGLAND JOURNAL OF MEDICINE (2013)

Article Urology & Nephrology

Characteristics and Outcomes of Children with Primary Oxalosis Requiring Renal Replacement Therapy

Jerome Harambat et al.

CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY (2012)

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Primary hyperoxaluria Type 1: indications for screening and guidance for diagnosis and treatment

Pierre Cochat et al.

NEPHROLOGY DIALYSIS TRANSPLANTATION (2012)

Article Transplantation

Efficacy and safety of Oxalobacter formigenes to reduce urinary oxalate in primary hyperoxaluria

Bernd Hoppe et al.

NEPHROLOGY DIALYSIS TRANSPLANTATION (2011)

Article Urology & Nephrology

Calcium oxalate saturation in dialysis patients with and without primary hyperoxaluria

Y Ogawa et al.

UROLOGICAL RESEARCH (2006)

Article Urology & Nephrology

Primary hyperoxaluria - The German experience

B Hoppe et al.

AMERICAN JOURNAL OF NEPHROLOGY (2005)

Article Urology & Nephrology

Clinical implications of mutation analysis in primary hyperoxaluria type 1

CS van Woerden et al.

KIDNEY INTERNATIONAL (2004)

Article Urology & Nephrology

Phenotypic expression of primary hyperoxaluria: Comparative features of types I and II

DS Milliner et al.

KIDNEY INTERNATIONAL (2001)