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MANAGING PH1

Current management approaches to PH1 aim at enhancing clearance of oxalate, inhibiting oxalate crystallisation, or lowering oxalate production by the liver. 1–3

MEDICAL MANAGEMENT OPTIONS

PH1 medical management is based on the provider’s assessment of each patient.

Current medical management strategies aim to decrease calcium oxalate crystal deposition and stone formation or to reduce oxalate production by the liver.1–3

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Hyperhydration2

  • Recommended daily fluid intake of ≥2–3 L/m2 of body surface area aims to reduce urinary calcium oxalate crystallisation2
Alkali Citrate Icon

Alkali citrate2,3

  • Inhibits calcium oxalate crystallisation2,3
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High-dose pyridoxine (vitamin B6) therapy1,3,4

  • Can act as an AGT co-factor, which may increase residual AGT enzyme activity and reduce oxalate production by the liver in some patients1,3
  • A small subset of patients with PH1 may respond to vitamin B61,3,4
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RNA interference (RNAi) therapeutics5,6

  • Binds to messenger RNA of enzymes involved in oxalate production, preventing their expression via the RNAi pathway5,6
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DIALYSIS STRATEGIES IN PH1 MANAGEMENT1,3,7,8

  • To reduce plasma oxalate levels, PH1 patients with compromised kidney function may require up to 6 haemodialysis sessions per week, which may need to be combined with continuous peritoneal dialysis1,3,7,8
  • Dialysis may either serve as a bridge to transplantation or as an adjunct therapy to decrease oxalate burden in the presence of delayed or poor renal function after liver/kidney transplantation1
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LIVER-KIDNEY TRANSPLANTATION

  • Liver transplant addresses the defect that causes glyoxylate — an organic compound generated by the liver enzyme GO — to convert into oxalate2,3
  • As kidney function declines, a kidney transplant may be required along with a liver transplant1,3

ISOLATED KIDNEY TRANSPLANTATION

  • The proportion of kidney transplant failures caused by a missed diagnosis of PH1 before transplantation is high9
  • ~3% of all kidney transplants will fail within the first post-transplant year, due to early loss of renal function10
  • Decreasing oxalate storage increases the probability of a positive outcome from transplantation in patients with PH111,12

MANAGING PH1 CAN BE BURDENSOME2,4

Becky describes life with PH1

This is Zoe and Nathan’s story with primary hyperoxaluria type 1 (PH1) as told by their mum, Becky.

Watch Ziggy’s moving story

A brave journey through PH1, from diagnosis to life after a liver and kidney transplant.

TAKE ME TO RESOURCES & SUPPORT

Testing patients for PH1

See how genetic testing plays an important role in a PH1 diagnosis.1,3

Testing Options

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References: 1. Milliner DS, Harris PC, Sas DJ, et al. Primary hyperoxaluria type 1. GeneReviews® [Internet]. Updated 15 August 2024. Accessed January 2026. https://www.ncbi.nlm.nih.gov/books/NBK1283/ 2. Cochat P, Rumsby G. N Engl J Med. 2013;369(7):649–658. 3. Cochat P, Hulton SA, Acquaviva C, et al. Nephrol Dial Transplant. 2012;27(5):1729–1736. 4. Hoyer-Kuhn H, Kohbrok S, Volland R, et al. Clin J Am Soc Nephrol. 2014;9(3):468–477. 5. Le Dudal M, Huguet L, Perez J, et al. J Clin Invest. 2019;129(6):2571–2577. 6. Lai C, Pursell N, Gierut J, et al. Mol Ther. 2018;26(8):1983–1995. 7. Cochat P, Fargue S, Harambat J. Curr Opin Organ Transplant. 2010;15(5):590–593. 8. Plumb TJ, Swee ML, Filaus JA. Am J Kidney Dis. 2013;62(6):1155–1159. 9. Cai Z, Ding M, Chen R, et al. BMC Nephrol. 2021;22(1):393. 10. Helanterä I, Räihä J, Finne P, Lempinen M. Transpl Int. 2018;31(8):880–886. 11. Somers MJ, Devresse A, Willey R, et al. Presented at: National Kidney Foundation (NKF) Spring Clinical Meeting; April 10–13, 2025; Boston, MA. 12. Devresse A, Cochat P, Godefroid N, et al. Kidney Int Rep. 2020;5(12):2136–2145.

PH1-INTR-00031 | January 2026

Alnylam Pharmaceuticals is responsible for the funding and content of this website. The site is intended for Healthcare Professionals in Europe, Middle East and Africa. For disease awareness purposes only.

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