Every August, National Immunization Awareness Month (NIAM) puts a spotlight on one of the most effective tools we have to protect our health — vaccines. For the Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart. community, that message carries extra weight. Respiratory muscle weaknessA loss of strength that can affect movement, posture, and sometimes breathing. and impaired cough are common in both infantile-onset (IOPDA severe form of Pompe disease that begins in infancy and often affects the heart and muscles.) and late-onset (LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly.) Pompe disease, raising the risk of severe illness from infections like flu, RSV, COVID-19, and pneumonia. Staying current on recommended vaccines is one of the most effective ways patients, caregivers, and families can reduce that risk.

The guidance below has been reviewed by Pompe disease specialists at Duke and reflects the current recommendations established by the Centers for Disease Control (CDC) and the Advisory Committee on Immunization Practices (ACIP).

This is general information, not personalized medical advice. Vaccine decisions should always be made with your treating physician or geneticist, since individual immune status, enzymeA protein that helps the body carry out chemical reactions. replacement therapy(ERT)/immune tolerance induction (ITI) schedules, and medications vary from patient to patient.

The recommendation is straightforward: stay current on all age-appropriate vaccines. For most patients with Pompe disease, that means following the standard CDC-recommended schedule, including

•     Annual influenza vaccine: recommended for everyone six months and older; especially important given the aspiration and cough-impairment risk in neuromuscular disease.
•     Pneumococcal vaccine (PCV20, PCV21, or PCV15+PPSV23 depending on age and history): pneumococcal vaccination is recommended for people with chronic neuromuscular conditions.
•     COVID-19 vaccine: additional/updated doses are recommended under current ACIP guidance for people who are moderately to severely immunocompromised.
•     RSV vaccine: recommended for eligible older adults and during pregnancy (to protect newborns).

•      Routine childhood/adult schedule: Tdap, MMR, varicella, shingles, and others as appropriate for age.

One important detail for families of infants with IOPD: Ask your care team about palivizumab (Synagis)A monoclonal antibody given by monthly injection during RSV season to help prevent severe respiratory illness in infants at high risk, including those with infantile-onset Pompe disease. pronunciation: pal-ih-VIZ-yoo-mab during RSV season. Synagis is a monoclonal antibody given by monthly injection. It is a preventive treatment, not a vaccine, and is distinct from the RSV vaccines (Arexvy, Abrysvo) or nirsevimab given to the general infant population. The American Academy of Pediatrics (AAP) guidance supports palivizumab specifically for infants under 12 months with neuromuscular disease that impairs the ability to clear airway secretions — a category IOPD falls squarely into. RSV season typically runs fall through spring.

Some infants who are CRIM-negativeA classification used in Pompe disease that describes whether a person's body produces any form of the GAA enzyme. People who are CRIM-positive produce some version of the enzyme, while those who are CRIM-negative do not. CRIM status is important because it can affect how the immune system responds to enzyme replacement therapy (ERT). undergo ITI (a short course of rituximab, methotrexate, and/or IVIG) before or alongside ERT, to prevent the immune system from attacking the replacement enzyme.

For patients undergoing ITI, live vaccines should be avoided both during treatment and during the recovery period afterward while the immune system is still suppressed or reconstituting. This includes MMR, varicella, and live nasal-spray flu vaccine. Standard (non-live) vaccines like flu shots, pneumococcal, and COVID vaccines are still recommended in this population; it’s specifically the live-attenuated ones to hold off on, and for longer than just the treatment window. Work closely with your immunology/genetics team to time these correctly.

For patients on long-term ERT without ongoing immunosuppression, there’s no broad contraindicationA medical reason to avoid a specific treatment or medication because it could cause harm, given a person's condition or other treatments. to standard vaccines, though antibody response can be blunted in some cases, so it’s worth a quick conversation with your care team about timing relative to infusionsA method of delivering medication through an IV..

Making sure caregivers and close contacts are vaccinated (flu, COVID, Tdap) adds an extra layer of protection for patients with reduced respiratory reserve, a strategy often called “cocooningA protective strategy where caregivers and close contacts get vaccinated to reduce the risk of exposing a patient with reduced respiratory reserve to preventable infections..”

•     Vaccines for Children (VFC): federally funded program providing no-cost vaccines to children who are uninsured, underinsured, on Medicaid, or American Indian/Alaska Native, through age 18. Ask your pediatrician if they’re a VFC provider. (CDC VFC program)
•     Vaccines for Adults (VFA): several states (California, New York, Minnesota, and others) run parallel no-cost vaccine programs for uninsured/underinsured adults 19+, funded through CDC Section 317 grants. Availability and covered vaccines vary by state health department.
•     Affordable Care Act (ACA) no-cost preventive vaccines: under the ACA, most private insurance and Medicare Part D/B plans must cover ACIP-recommended vaccines with no out-of-pocket cost when given in-network.
•     Local health department and Section 317-funded clinics: offer low- or no-cost vaccines regardless of ability to pay.
•     Vaccines.gov: CDC’s vaccine finder tool to locate nearby pharmacies and clinics offering flu, COVID, RSV, and other vaccines. (vaccines.gov)

•      Manufacturer and pharmacy assistance programs: many pharmacy chains and vaccine manufacturers, including the maker of Synagis, offer patient assistance for uninsured or underinsured families; ask your pharmacist or care coordinator directly

•  CDC National Immunization Awareness Month: resources for professionals
•  CDC: Immunizations for Respiratory Virus Prevention
•  AAP: Palivizumab Prophylaxis in Infants and Young Children at Increased Risk
•  NFID: National Immunization Awareness Month Resource Round-Up

As always, talk with your Pompe care team before making vaccine decisions; timing and eligibility depend on your specific treatment plan.

This document was created by Samuela Fernandes, M.D., Duke University School of Medicine.