Vaccination proposal for patients on onasemnogene abeparvovec therapy

Area of Science:

• Neurology
• Infectious Diseases
• Immunology

Background:

• Disease-modifying therapies have transformed spinal muscular atrophy (SMA) care, improving patient outcomes.
• Newer treatments, such as onasemnogene aboparvovec, introduce novel challenges, including potential immunosuppression.
• Immunosuppression can affect vaccine efficacy and increase susceptibility to infections.

Purpose of the Study:

• To propose pretherapy screening recommendations for onasemnogene aboparvovec-treated SMA patients from an infectious disease perspective.
• To provide guidelines for vaccination strategies before, during, and after immune therapy in this patient population.

Main Methods:

• Review of current literature and guidelines on SMA treatments and infectious disease management.
• Development of a screening proposal tailored to presymptomatic newborns and symptomatic children.
• Summarization of vaccination protocols in the context of steroid-induced immunosuppression.

Main Results:

• Onasemnogene aboparvovec therapy necessitates careful consideration of infectious risks and immune modulation.
• Differential diagnostic challenges may arise due to side effects of the therapy.
• Specific screening and vaccination schedules are proposed to optimize patient safety.

Conclusions:

• Proactive infectological screening and strategic vaccination are crucial for patients undergoing onasemnogene aboparvovec treatment for SMA.
• Implementing these measures can help mitigate risks associated with immunosuppression and ensure treatment efficacy.
• This approach supports comprehensive care for individuals with spinal muscular atrophy.