{"id":54239,"date":"2026-01-24T06:47:44","date_gmt":"2026-01-24T05:47:44","guid":{"rendered":"https:\/\/inmuno.es\/index.php\/2026\/01\/24\/a-comprehensive-study-on-the-effect-of-alglucosidase-alpha-and-immunomodulation-on-survival-motor-and-cardiac-outcome-creatine-kinase-and-antibody-titers-in-classic-infantile-pompe-disease-the-monz\/"},"modified":"2026-01-24T06:47:44","modified_gmt":"2026-01-24T05:47:44","slug":"a-comprehensive-study-on-the-effect-of-alglucosidase-alpha-and-immunomodulation-on-survival-motor-and-cardiac-outcome-creatine-kinase-and-antibody-titers-in-classic-infantile-pompe-disease-the-monz","status":"publish","type":"post","link":"https:\/\/inmuno.es\/index.php\/2026\/01\/24\/a-comprehensive-study-on-the-effect-of-alglucosidase-alpha-and-immunomodulation-on-survival-motor-and-cardiac-outcome-creatine-kinase-and-antibody-titers-in-classic-infantile-pompe-disease-the-monz\/","title":{"rendered":"A comprehensive study on the effect of alglucosidase alpha and immunomodulation on survival, motor and cardiac outcome, creatine kinase and antibody titers in classic infantile Pompe disease: the Monza experience"},"content":{"rendered":"<div>\n<p><b>Curr Opin Immunol<\/b>. 2026 Jan 22;99:102725. doi: 10.1016\/j.coi.2026.102725. Online ahead of print.<\/p>\n<p><b>ABSTRACT<\/b><\/p>\n<p>We report on 13 classic infantile Pompe patients, including four cross-reactive immunological material negative (31%), treated with alglucosidase alpha (rhGAA) at Fondazione IRCCS San Gerardo, Monza, between 2003 and 2024. Median age at rhGAA initiation was 3.3 months, with nine patients (69%) starting on doses &gt; 20 mg\/kg\/every other week. With a median follow-up of 6.9 years, the 5- and 10-year survival was 92%. Four patients died, and three became ventilator-dependent. Hypertrophic cardiomyopathy normalized in all patients, though electrocardiogram abnormalities persisted in 36%. Walking was achieved by 10 (77%). Ten patients received immune tolerance induction (77%, five primary, two secondary, three both), and seven received long-term sirolimus. Nine developed anti-rhGAA, of whom five (38%) had high-sustained antibody titers (HSAT). All had elevated creatine kinase at diagnosis; creatine phosphokinase normalized over time in four patients on 40 mg\/kg\/week since start without HSAT. This study offers real-world insight into managing classic infantile Pompe disease and compares the cohort&#8217;s outcome to international experiences.<\/p>\n<p>PMID:<a href=\"https:\/\/pubmed.ncbi.nlm.nih.gov\/41576647\/?utm_source=SimplePie&amp;utm_medium=rss&amp;utm_content=8900118&amp;ff=20260124004743&amp;v=2.18.0.post22+67771e2\">41576647<\/a> | DOI:<a href=\"https:\/\/doi.org\/10.1016\/j.coi.2026.102725\">10.1016\/j.coi.2026.102725<\/a><\/p>\n<\/div>","protected":false},"excerpt":{"rendered":"<p>Curr Opin Immunol. 2026 Jan 22;99:102725. doi: 10.1016\/j.coi.2026.102725. Online ahead of print. ABSTRACT We report on 13 classic infantile Pompe patients, including four cross-reactive immunological material negative (31%), treated with alglucosidase alpha (rhGAA) at Fondazione IRCCS San Gerardo, Monza, between 2003 and 2024. Median age at rhGAA initiation was 3.3 months, with nine patients (69%) &#8230; <a title=\"A comprehensive study on the effect of alglucosidase alpha and immunomodulation on survival, motor and cardiac outcome, creatine kinase and antibody titers in classic infantile Pompe disease: the Monza experience\" class=\"read-more\" href=\"https:\/\/inmuno.es\/index.php\/2026\/01\/24\/a-comprehensive-study-on-the-effect-of-alglucosidase-alpha-and-immunomodulation-on-survival-motor-and-cardiac-outcome-creatine-kinase-and-antibody-titers-in-classic-infantile-pompe-disease-the-monz\/\" aria-label=\"Read more about A comprehensive study on the effect of alglucosidase alpha and immunomodulation on survival, motor and cardiac outcome, creatine kinase and antibody titers in classic infantile Pompe disease: the Monza experience\">Read more<\/a><\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[59,42],"tags":[],"class_list":["post-54239","post","type-post","status-publish","format-standard","hentry","category-current-opinion-in-immunology","category-publicaciones"],"_links":{"self":[{"href":"https:\/\/inmuno.es\/index.php\/wp-json\/wp\/v2\/posts\/54239","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/inmuno.es\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/inmuno.es\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/inmuno.es\/index.php\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/inmuno.es\/index.php\/wp-json\/wp\/v2\/comments?post=54239"}],"version-history":[{"count":0,"href":"https:\/\/inmuno.es\/index.php\/wp-json\/wp\/v2\/posts\/54239\/revisions"}],"wp:attachment":[{"href":"https:\/\/inmuno.es\/index.php\/wp-json\/wp\/v2\/media?parent=54239"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/inmuno.es\/index.php\/wp-json\/wp\/v2\/categories?post=54239"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/inmuno.es\/index.php\/wp-json\/wp\/v2\/tags?post=54239"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}