The SKYSONA™ Treatment Journey

ACTOR PORTRAYAL

SKYSONA treatment is a complex process, but we can help you understand what lies ahead as your child moves through 3 main phases of the treatment journey: pre-treatment, treatment, and post-treatment.

ACTOR PORTRAYAL

SKYSONA can only be administered at Qualified Treatment Centers (QTCs). These are highly specialized hospitals with care teams that have:

transplant experience
experience treating children with cerebral adrenoleukodystrophy
special training in administering SKYSONA

It’s at these hospitals your child will receive their gene therapy and recover from their treatment.

As each treatment is made specially for each child, lengths of time shown for each part of the treatment journey are approximations only.

Before being treated with SKYSONA, you should have a detailed discussion with your child’s doctor about the risks and benefits of SKYSONA and alternative treatment options. Alternative treatment may include an allogeneic hematopoietic stem cell transplant—discuss with your child’s physician the possibility of a stem cell transplant using cells from a suitable and willing matched sibling donor, if available.

BEFORE TREATMENT

Your healthcare providers will give your child other medicines, including chemotherapy medicine, as part of their treatment before your child is given SKYSONA. It’s important that you talk to your child’s healthcare providers about the risks and benefits of all medicines involved in their treatment.

Before your child is treated with SKYSONA, you should have a detailed discussion with their doctor about the risks and benefits of SKYSONA and alternative treatment options.
Because of the risk of cancer caused by SKYSONA, your child’s doctor may recommend that they’re evaluated by a hematologist to determine if they have underlying risk factors that could further increase their risk for cancer and change whether SKYSONA is appropriate for your child.

Pre-Treatment

CELL COLLECTION

(APPROXIMATELY 1 WEEK)

Mobilization/apheresis is the procedure where blood is collected from the body. Blood stem cells are then collected to be used for SKYSONA creation
Your child is brought into a QTC, where your child receives medication that moves their blood stem cells from the bone marrow into their bloodstream (clinically this is referred to as mobilization)
Once the blood stem cells are collected, a machine (known as an apheresis machine) separates the blood components to help isolate the specific stem cells needed. More than one day of apheresis and more than one cycle of mobilization may be necessary to collect enough cells to manufacture SKYSONA
Additional “back-up” stem cells are also collected and stored at the hospital. This is a precaution in case there is a problem during the treatment process. If this happens, back-up stem cells, sometimes called “rescue cells,” will be given back to your child. If your child must receive “rescue cells” they will have no benefit from SKYSONA

SKYSONA MANUFACTURING

(51-65 DAYS)

These collected stem cells are then transported to a manufacturing center, where they are modified to carry the functional ABCD1 gene needed to produce ALD protein to break down very long-chain fatty acids and to slow the progression of damage to the brain and slow the decline in neurologic function
It takes approximately 51–65 days from the time your child’s cells are collected to make and test SKYSONA before it is shipped to their healthcare providers, but the time may vary
Once the stem cells have been modified, your child’s personalized treatment of SKYSONA is frozen before being shipped back to the QTC

DISCUSSING BLOOD STEM CELL COLLECTION WITH YOUR CHILD'S DOCTOR

If you have questions about blood stem cell collection for your child, contact your doctor at the QTC.

Here are some questions you may ask:

How many days do you expect my child's blood stem cell collection to take?
Do we need to stay close to the treatment center?
What side effects can we expect?
How will the side effects be managed?
Should I change my child's diet before or after collection?
Who will be there to support us at that time?
When would my child need an infusion of rescue cells?
Is there anything else I should be aware of?
Would it be possible to tour or see the area where my child would receive treatment?

During Treatment
(PERFORMED AT A QTC)

CONDITIONING

(6-8 Days)*

*CONDITIONING DAYS WERE BASED OFF SKYSONA CLINICAL TRIALS.

This is the process by which your child’s body is prepared to receive gene therapy. This involves your child receiving chemotherapy to remove stem cells in their bone marrow to make way for the new cells that will be added
Before receiving SKYSONA, your child will be admitted to the hospital and will need to undergo conditioning
Conditioning involves 4-6 days of chemotherapy to make room in the bone marrow for SKYSONA
After conditioning, your child will need at least 2 days of rest before receiving SKYSONA
After conditioning, your child will remain in the hospital while they receive SKYSONA
After receiving the chemotherapy it may not be possible for your child to father a child. Consider discussing options for fertility preservation with your child’s doctor before treatment

SKYSONA INFUSION

(2 HOURS OR LESS)

SKYSONA will be infused into your child’s bloodstream. Your child may receive 1 or 2 bags. Each bag takes less than 1 hour

RECOVERY

(APPROXIMATELY 2 MONTHS)

Once infused, the SKYSONA blood stem cells engraft into your child’s bone marrow and begin producing ALD protein
Your child will remain in the hospital for roughly an additional 2 months, so that the healthcare provider can monitor their recovery and watch for any side effects that occur

DISCUSSING TREATMENT WITH YOUR CHILD’S DOCTOR

Here are some questions you could ask:

Where will my child’s treatment take place?
Where will my child be monitored following treatment?
How will I know that my child’s SKYSONA has been manufactured and is ready for infusion?
How long should we plan to stay at the QTC?
What is your recommendation for travel? Should we come a few days in advance? How long should we expect to stay?
What should my child expect during chemotherapy?
What should we expect during SKYSONA infusion and recovery?
What will happen if my child has side effects at any point during the treatment process?
What are we allowed to bring with us? What do you recommend we bring to the QTC?
Is there anything else I should know?

Post-Treatment

FOLLOW-UP CARE

(AT LEAST 15 YEARS)

Once SKYSONA treatment has been administered, your child can focus on their recovery from treatment. It’s important to keep in mind that each child recovers at their own rate as they work to regain their strength
Your child will also have lifelong monitoring for blood cancer and will continue to be monitored for cerebral adrenoleukodystrophy. Work with your child’s doctor to establish regular follow-up care
As a caregiver, you can talk with your child’s doctor about how your child is recovering, focusing on both their physical and mental recovery
Because of the risk of cancer, your child will need lifelong monitoring. Monitoring will include blood tests and additional testing may be recommended. Additional testing might include more frequent blood tests and a bone marrow evaluation, which can tell your child’s doctor more than blood tests about the health of their bone marrow and if there is cancer forming.

Seek help for any signs or symptoms of cancer, such as fatigue, easy bleeding, or bruising. If your child is diagnosed with a cancer, have your child’s treating physician contact bluebird bio at 1-833-999-NEST (6378) or medinfo@bluebirdbio.com.

REGISTRY (15 YEARS)

We encourage that anyone treated with SKYSONA be enrolled in the 15-year registry study that collects information on the long-term effects of SKYSONA
During participation in the registry your child will be monitored, and you are encouraged to continue lifelong follow-up monitoring after the registry ends

DISCUSSING POST-TREATMENT WITH YOUR CHILD’S DOCTOR