Special Warning : אזהרת שימוש

4.4   Special warnings and precautions for use

Proper aseptic techniques should always be used for the preparation and infusion of Upstaza.

Monitoring
Upstaza-SPC-0424-V1
Patients undergoing gene therapy should be closely monitored for procedure-related complications, complications related to their underlying disease, and risks associated with general anaesthesia during the peri-operative period. Patients may experience exacerbations of symptoms of their underlying AADC deficiency as a result of surgery and anaesthesia (see section 4.8).

Autonomic and serotonergic symptoms of AADC may persist after treatment with eladocagene exuparvovec.

Traceability

In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.

Immunogenicity

Experience with eladocagene exuparvovec in patients with anti-AAV2 antibody levels > 1:20 prior to treatment is not available.

Cerebrospinal fluid leaks

Cerebrospinal fluid (CSF) leaks occur when there is a tear or hole in the meninges surrounding the brain or spinal cord, allowing the CSF to escape. Upstaza is administered by bilateral intraputaminal infusion using burr holes, therefore, CSF leak may occur postoperatively. Patients undergoing eladocagene exuparvovec treatment should be carefully monitored after administration for CSF leaks, particularly in relation to the risk of meningitis and encephalitis.

Dyskinesia

AADC-deficient patients may have increased sensitivity to dopamine due to their chronic dopamine deficiency. Dyskinesia has been reported in 26/30 patients after treatment with eladocagene exuparvovec (see section 4.8). The occurrence of dyskinesia is due to dopamine sensitivity and generally starts 1 month after the administration of gene therapy and gradually decreases over several months. Events of dyskinesia were managed with routine medical care, such as antidopaminergic treatment (eg, risperidone) (see section 5.1).

Risk of viral shedding

The risk of shedding is considered to be low due to very limited systemic distribution of eladocagene exuparvovec (see section 5.2). As a precautionary measure, patients/caregivers should be advised to handle waste material generated from dressings and/or any secretions (tears, blood, nasal secretions, and CSF) appropriately, which may include storage of waste material in sealed bags prior to disposal and patients/caregivers wearing gloves for dressing changes and waste disposal. These handling precautions should be followed for 14 days after administration of eladocagene exuparvovec. It is recommended that patients/caregivers wear gloves for dressing changes and waste disposal, especially in case of pregnancy, breast-feeding, or immunodeficiency of caregivers.

Blood, organ, tissue, and cell donation

Patients treated with Upstaza must not donate blood, organs, tissues, and cells for transplantation.


Upstaza-SPC-0424-V1
Sodium and potassium content

This medicinal product contains less than 1 mmol sodium (23 mg) per dose, that is to say essentially ‘sodium-free’.
This medicinal product contains less than 1 mmol potassium (39 mg) per dose, that is to say essentially ‘potassium-free’.

Effects on Driving

4.7    Effects on ability to drive and use machines

Not relevant.