Insights into immunogenicity and therapeutic strategies to mitigate the immune response in infantile-onset Pompe disease: a comprehensive systematic literature review.

What was studied

This review looked at infantile-onset Pompe disease. The usual care is enzyme replacement. The body may make antibodies against it. The review asked how doctors test CRIM status. It also asked how they use it to plan drugs that calm the immune response. It used papers from 2003 to 2022.

What they found

Papers gave data for each question: 54, 51 and 69 papers. Tests have moved toward predicting CRIM status from gene variants. Drugs that calm the immune response were mostly given to CRIM-negative people. A common plan was a short course of rituximab and methotrexate. With enzyme care plus this plan, CRIM-negative people did better than without it.

What it means for patients

The authors say CRIM status matters for how doctors plan care. They say calming the immune system was mainly reported in CRIM-negative people. An early start of enzyme care and doses above the label were other key factors. For CRIM-positive people, choices vary by doctor, family history or gene variants.

Why it is not certain yet

This review gathers reports of how doctors work. The abstract gives no numbers on how people fared. It does not say how good the papers were. Practice for CRIM-positive people varies. A review can only be as good as the studies in it.

Source

Insights into immunogenicity and therapeutic strategies to mitigate the immune response in infantile-onset Pompe disease: a comprehensive systematic literature review.

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