Zolgensma: Caregiver Counseling

As Zolgensma was recently approved by the FDA – in May 2019 – there has been little caregiver education on the therapy and how best to monitor SMA patients undergoing the treatment. Specifically, caregivers should be provided information on how best to mitigate risks associated with Zolgensma. There are four main areas in which caregivers should be counseled: 

Potential Liver Damage

The biggest risk associated with the use of Zolgensma is damage to the liver. Caregivers should be informed that the patient will need to take an oral corticosteroid before Zolgensma administration. They should also be warned that the patient will need to undergo regular blood tests to evaluate liver function so that any problematic changes can be rapidly addressed. 

Caregivers should be advised that medical attention is required immediately in the case that:

  • The patient’s skin or the whites of the patient’s eyes develop a yellowish hue
  • The patient misses a corticosteroid dose
  • The patient vomits up a corticosteroid dose

Potential Thrombocytopenia

As Zolgensma may also lead to thrombocytopenia, caregivers should be made aware that the therapy can reduce platelet count and increase the likelihood of bruising and bleeding. They should be told to contact their healthcare provider if the patient develops bruising or bleeding that cannot be easily explained by an injury.

Vector Shedding

It should be made clear to caregivers that the virus used in this gene therapy will be shed largely through waste, which poses risks to those who come into contact with that waste. Caregivers should be educated on proper waste handling, including the sealing of diapers before they are discarded and proper hand hygiene following contact with human waste. These caregivers should also be informed that these precautionary measures should be undertaken for a full month following Zolgensma treatment.   

Vaccination Adjustments

It is important to ensure that caregivers understand that the corticosteroids that are required for the use of Zolgensma will suppress the patient’s immune system and that because certain vaccines can be harmful when the immune system is suppressed, their vaccination schedule may need to be changed. To prevent complications that can arise from viral respiratory infection, caregivers should also be informed that infections such as cold, flu, and bronchiolitis can lead to serious complications in those undergoing Zolgensma therapy and that they should seek medical attention if the patient shows signs of infection, including fever, coughing, runny nose, sneezing, or wheezing.  

Because Zolgensma is currently approved only for use in patients who are less than 2 years of age, it is essential that caregivers understand the potential impacts of this gene therapy and what to watch for in patients undergoing the treatment. 


1. Highlights of prescribing information. fda.gov. https://www.fda.gov/media/126109/download. Published 2019. Accessed September 5, 2019.