Sirolimus treatment for children with vascular malformations information sheet

Contact details

Oncology Treatment Centre: (02) 7825 2115  | Monday - Friday, 8am – 4pm

Oncologist on call: (02) 7825 0000 – ask switchboard to page the Oncology Fellow on call

Camperdown Ward: (02) 7825 1123 (24hr cover for general advice)

Vascular Anomalies CNC:  0450 259 124 | Monday and Tuesday, 8am – 4pm

Email: SCHN-CHW-VascularAnomalies@health.nsw.gov.au (for non-urgent vascular anomalies enquiries)

Introduction

This Information sheet explains the use of sirolimus to treat vascular anomalies at The Children’s Hospital at Westmead. It includes information on sirolimus, its possible side effects and when to contact your specialist doctor.

This information is for educational purposes only. Please consult with your doctor or other health professionals to make sure this information is right for  your child. 

About sirolimus treatment

Sirolimus (also known as Rapamune) is a medication that suppresses the immune system. It is commonly used following kidney transplant to prevent rejection, but it is also used for other medical conditions, including vascular anomalies. 

Sirolimus interferes with the pathways involved in the growth of cells and blood vessels, making it an effective treatment for overgrowth of body tissues commonly associated with vascular tumours and malformations.

Sirolimus is not a cure, but it can help reduce some of the problematic symptoms associated with vascular anomalies, such as pain, swelling, reduced mobility, and clotting abnormalities.

Before starting sirolimus

  • If your child hasn’t had any medical imaging, like Magnetic Resonance Imaging (MRI), or ultrasound, this will usually be arranged before sirolimus starts. 
  • Your child will also need to have a blood test. This will give us baseline information that will guide treatment, and later check if treatment is working. 

 

During treatment with sirolimus

During treatment, your child may be asked to take an antibiotic 3 times a week to reduce the risk of them developing an infection. This is not given to every child. If you have any questions, please speak with your specialist doctor. 

During treatment, your child will have regular clinic appointments and blood tests to check their progress and monitor the effectiveness of the medication. 

Blood tests

  • Blood tests are important to see if sirolimus is working and to adjust the dose of sirolimus as required.
  • At the start of treatment blood tests will be done every 2-4 weeks, then monthly, then 3 monthly or at an interval determined by your specialist. 
  • Blood tests need to be taken before the morning dose of sirolimus is given to your child. You can give the dose of sirolimus as normal after the blood test has been taken.
  • Results of the sirolimus blood level will be sent to your doctor within a few days. The doctor will let you know if the dose of sirolimus needs to change.  

Imaging 

  • Your specialist will request follow up MRI scans and/or ultrasounds at different times during your treatment to see if the sirolimus is working.

Duration of sirolimus treatment

The duration of sirolimus depends on many factors and will be determined by your doctor, although you can expect to take Sirolimus for at least two years.

Important things to note:

  1. If your child has ANY allergies, please tell your specialist doctor. If your child is allergic to soy products, please tell your doctor.
  2. Your child should NOT receive live vaccinations whilst taking sirolimus. Please discuss any vaccinations with your Oncologist before giving them.
  3. Sirolimus can be given with or without food but should be taken the same way every day to ensure the same amount of active ingredient is absorbed each time.
  4. DO NOT take sirolimus with Grapefruit juice as there is a chemical within grapefruit that will increase the amount of sirolimus in the bloodstream. This can make side effects more likely.

Do not give sirolimus to your child if they are allergic to sirolimus or any of the ingredients of the oral solution or tablets. See the consumer medication information summary (CMI) for more information. 

Side effects

Sirolimus is given to help treat your child’s vascular malformation, however there might be some unwanted side effects. If you have any of the side effects, please tell your specialist doctor or nurse.

Not every child will experience side effects and if your child’s side effects are serious, the specialist may decide to stop sirolimus. 

Below are some of the side effects to be aware of.

Please note: all possible side effects and precautions of this medicine are not covered in this information sheet. For a complete list of side effects, ask your healthcare professional or refer to the consumer medication information summary (CMI). 

Less serious side effects

  • Mouth ulcers
  • Headaches
  • Tiredness and fatigue
  • Infections (as this medication increases the risk of getting an infection, avoid crowds and people with an infection, a cold, or the flu)
  • Nausea, stomach pain, diarrhea, constipation
  • Skin sensitivity
  • Acne
  • Slow wound healing
  • Low blood counts (e.g., haemoglobin and platelets)
  • Increase cholesterol
  • Lack of appetite
  • Weakness or muscle pain
  • Fast heart rate
  • Abnormal electrolytes and liver function test.

Serious side effects

  • Trouble breathing (this is an emergency, please call 000)
  • Severe infection
  • Blood clots
  • Signs of allergy such as rash, itching, hives, swelling face, lips, tongue or other parts of the body, shortness of breath, wheezing, and difficulty breathing 
  • Signs of excessive fluid on lungs such as coughing, wheezing, and difficulty breathing 
  • Swelling of the hands, feet, limbs, abdomen, and/or puffiness or swelling around the eyes
  • May increase risk of developing certain cancers, including skin cancer and lymphoma (limit your exposure to sunlight and UV light by wearing protective clothing and using sunscreen). 

When to contact the Hospital

If your child has difficulty breathing, this is an emergency, please call 000.

Please present to the Emergency Department If your child has:

  • A temperature of more than 38 degrees and is unwell, and/or you are worried
  • Any bleeding/bruising or small red spots on their skin
  • Mouth ulcers that affect their ability to eat and drink
  • Persistent vomiting or diarrhoea

Note: If your child has a temperature of 38 degrees or more but appears well (may only have a runny nose), you should see your GP within 24hrs so they can assess your child. 

Taking sirolimus safely

 Pregnancy and breastfeeding: women who are family planning, pregnant or breastfeeding should not touch hazardous medications or bodily fluids that might have hazardous residue in them.

Safe handling of hazardous medications at home information sheet

 How to give the tablets

  1. Wash hands with soap and water
  2. Dispense tablet from packaging
  3. Take tablet immediately with water or orange juice
  4. Do not crush, chew, or cut tablets.

Storage: store sirolimus tablets at room temperature.

 How to give the oral solution

Preparing the solution

  • It is recommended that sirolimus be diluted with approx. 60mls of water or orange juice, however, discuss the amount of fluid to use with your pharmacist.
  • When mixed with water or orange juice, sirolimus produces a white to off-white mixture. 
  • Sirolimus should not be taken with grapefruit juice as there is a chemical within grapefruit that increases the amount of Sirolimus in the bloodstream and can make side effects more likely.

Instructions

  1. Wash your hands with soap and water
  2. Put on the gloves provided by the hospital
  3. Shake bottle and remove lid
  4. Insert syringe adapter into top of bottle and leave it in place until the bottle is finished
  5. Draw up required dose of medicine in the syringe provided using a new syringe every time
  6. Pour water or orange juice into glass or cup. Do not mix with any other liquids
  7. Empty medicine from syringe into glass or cup
  8. Stir quickly
  9. Immediately give it to your child to drink
  10. Refill the glass with a small amount of water/orange juice, stir it to pick up any remaining solution, and give it to your child to drink.
  11. Replace lid on bottle and put back into the fridge
  12. Discard used syringe into the purple bag provided by the hospital
  13. Bring the sealed bag of used syringes to the hospital on your next visit so they can dispose of them correctly for you.

Storing sirolimus

  • Store Sirolimus bottle in fridge.
  • Sirolimus can develop a hazy-like appearance in the fridge. This is not harmful and returns to a consistency when at room temperature.
  • Discard after 30 days after opening.

Interactions with other medications

Some medicines can react with sirolimus altering how it works. Always check with your specialist or pharmacist before giving your child any other medicine. 

The following medicines are known to react with sirolimus:

  • Certain antibiotics or antifungal medicines
  • Medicines for high blood pressure
  • Anti-epileptic medicines
  • Medicines for stomach ulcers or reflux
  • Medicines to prevent nausea and vomiting
  • Other immunosuppressant medicines.

Missed or vomited doses:

  • If your child forgets to take their sirolimus, they should take their normal dose the following day. Do not double dose to make up for the forgotten dose.
  • If your child vomits within 20 minutes of taking sirolimus, repeat the dose. If it has been more than 20 minutes, do not repeat the dose as it is likely the medication has been absorbed.