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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

  • Recommend daily fluid intake of ≥2-3L/m2 of body surface area aims to reduce urinary calcium oxalate crystallisation2
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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 transplant.1,3

MANAGING PH1 CAN BE BURDENSOME2,4

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

Play Video about Zoe and Nathan’s Story With Primary Hyperoxaluria Type 1 (PH1) Video Thumbnail
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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1. Milliner DS, Harris PC, Cogal AG, Lieske JC. https://www.ncbi.nlm.nih.gov/books/NBK1283/. Updated February 10, 2022. Accessed June 21, 2023. 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;9:468–477.5. 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.

PH1-CEMEA-00129 | November 2023

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PH1-CEMEA-00124

November 2023

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