management
Managing PH1 takes a team
Primary hyperoxaluria type 1 (PH1) requires lifelong management and monitoring. A well-coordinated healthcare professional team can help you approach the different aspects that managing PH1 can involve.
Since PH1 is a personal experience and affects everyone differently, you and your healthcare professional team will work together to create a personalised plan. The makeup of your care team may include the healthcare providers listed below.
Your care team
Some of the specialists you may meet in your journey with primary hyperoxaluria type 1 (PH1)
Paediatrician
When your child first visits the hospital, they may see a general paediatrician. Paediatricians are doctors who provide medical care and advice for babies, children and teenagers.
After carrying out the initial checks and tests, if they suspect a kidney-related condition such as PH1, they may refer your child to a nephrologist.
Paediatrician
Nephrologist/Paediatric Nephrologist
Kidney specialist
A nephrologist specialises in diseases like PH1 that affect the kidneys, and may have a special focus in either children or adults. You and your nephrologist will create a personalised management plan based on how your PH1 is progressing.
Typically, a nephrologist will take the lead role in the overall management of your disease. However, other specialists may be involved in your care as well.
Nephrologist/Paediatric Nephrologist
Urologist
A surgeon who specialises in disorders of the urinary tract. For some people with PH1, their urologist may treat only their kidney stones and refer them to a nephrologist to help manage PH1 as a whole.
Though some kidney stones may need medical attention, there is a lot more to managing PH1 beyond removing stones when you have them.
Urologist
Genetic counsellor
A specialist who can answer questions you might have about genetic testing and interpret your results. Genetic counsellors also help those who may be at risk for, or be affected by, PH1 to understand the risks and make decisions, including whether other family members should consider being tested.
Genetic counsellor
Dialysis Nurse
While not necessary for all people with PH1, some people may need dialysis. A dialysis nurse is specially trained to assist and monitor a person undergoing dialysis. Dialysis nurses work in hospitals or dialysis centres. Healthcare professionals at dialysis centres may not be familiar with PH1 since they are mostly responsible for conducting the dialysis process, not for treating diseases that create the need for dialysis. You can play an important role in educating others by memorising a 1 to 2-sentence description of PH1. Here are some examples:
- PH1 is a condition where the liver makes too much oxalate, which can damage the kidneys over time.
- PH1 mostly affects the kidneys, and the most common symptom is kidney stone formation. But PH1 actually starts in the liver, where an overproduction of oxalate occurs.
Dialysis Nurse
Transplant Team
A transplant team can include a urologist specialising in kidney transplant, another surgeon specialising in liver transplant, and additional physicians who provide medical support during follow-up.
Transplant Team
As you are working with your healthcare professional team, your friends and family will also be able to help you keep on track with your PH1 management plan and support you emotionally.
The goal of management is to slow disease progression
Current management options help decrease the amount of crystals formed due to oxalate overproduction, to slow down kidney damage and lower stone formation.
As part of your management plan, your healthcare professional may measure different things, including how much oxalate is in your urine and/or blood (oxalate levels) and how well your kidneys are able to filter waste products (kidney function). To determine your kidney function, blood tests will measure your glomerular filtration rate (GFR).
PH1 Patient Perspectives: Understanding Progression
Your healthcare professional may consider these management options for your PH1:
- Your healthcare professionals may manage your PH1 with a number of options at the same time, such as making sure you drink enough water, along with taking alkali citrate and Pyridoxine (Vitamin B6).
- Your PH1 management may change over time, depending on how your disease and symptoms progress.
- Most management options cannot keep up with the continuous oxalate overproduction. However consistent management of PH1 may help you slow progressive loss of kidney function.
Hyperhydration
(drinking lots of water):
Helps dilute oxalate in your urine
Pyridoxine
(vitamin B6):
May help the broken enzyme in your liver try to work normally.
Alkali citrate (potassium citrate or sodium citrate):
May help reduce crystal formation
Emerging therapies:
Target and prevent oxalate development in the liver.
Dialysis:
If PH1 has affected your kidneys' ability to work properly, you may need to undergo dialysis. Dialysis is a process that uses a machine to help remove waste products (such as oxalate) and excess fluid from the blood when your kidneys are no longer able to work properly.
Thinking ahead: when management is no longer enough
Oxalate overproduction can cause permanent damage. Transplant surgery may be necessary if your body reaches a certain point of advanced disease progression. Until recently, liver transplant was the only way to stop the production of oxalate. However more recent developments target and prevent oxalate development in the liver without the need for transplantation.
Where previously liver transplantation was the only way to stop oxalate overproduction, more recently developed treatments target and prevent oxalate development in the liver - providing a new way of treating PH1.
A liver transplant is a surgical operation where a donor liver is taken from a person without PH1 and is transplanted into a person with PH1.
Because PH1 primarily causes damage to the kidneys, both the liver and the kidneys may need to be replaced, either at the same time or during separate surgeries. This is called a dual liver-kidney transplant.
Transplant surgeries are major operations that require preparation, long-term follow-up, and lifelong medications. Talk to your healthcare professional team about developing a detailed plan to help you recover from your surgery.
WITH
PH1
Living with PH1
Living with PH1 is more than just a management plan. Discover ideas on how you can make your care team's management plan part of your life.
Living with PH1PH1-CEMEA-00004 March 2021