Outpatient monthly plasmapheresis with post-PLEX evinacumab in pediatric homozygous familial hypercholesterolemia: a case report on port access and immunoglobulin preservation
Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder characterized by severely elevated low-density lipoprotein (LDL) cholesterol levels, predisposing patients to early cardiovascular disease. While LDL apheresis is the standard extracorporeal therapy, plasmapheresis (PLEX) is...
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Frontiers Media S.A.
2025-07-01
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| Series: | Frontiers in Pediatrics |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fped.2025.1559147/full |
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| author | Guido Filler Kambiz Norozi Kambiz Norozi |
| author_facet | Guido Filler Kambiz Norozi Kambiz Norozi |
| author_sort | Guido Filler |
| collection | DOAJ |
| description | Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder characterized by severely elevated low-density lipoprotein (LDL) cholesterol levels, predisposing patients to early cardiovascular disease. While LDL apheresis is the standard extracorporeal therapy, plasmapheresis (PLEX) is often used in younger children due to limitations in vascular access. Evinacumab, an ANGPTL3 inhibitor, has emerged as an effective adjunct for lowering LDL cholesterol. This case, to our knowledge, describes the first pediatric outpatient implementation of monthly plasmapheresis with post-PLEX Evinacumab infusions, enabled by dual-port access and Octaplasma support to maintain immunoglobulin levels. This report highlights procedural innovations that enabled sustained therapy and emphasizes the need for standardized approaches for combining Evinacumab with extracorporeal treatment in pediatric HoFH. |
| format | Article |
| id | doaj-art-c3f4bcaf1cfa42068b8f16e8537b7d68 |
| institution | DOAJ |
| issn | 2296-2360 |
| language | English |
| publishDate | 2025-07-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pediatrics |
| spelling | doaj-art-c3f4bcaf1cfa42068b8f16e8537b7d682025-08-20T03:12:46ZengFrontiers Media S.A.Frontiers in Pediatrics2296-23602025-07-011310.3389/fped.2025.15591471559147Outpatient monthly plasmapheresis with post-PLEX evinacumab in pediatric homozygous familial hypercholesterolemia: a case report on port access and immunoglobulin preservationGuido Filler0Kambiz Norozi1Kambiz Norozi2GF and KN are Professors of Pediatrics and Medicine, Departments of Pediatrics and Medicine, Western University, and Children’s Hospital, London Health Sciences Centre, London, ON, CanadaGF and KN are Professors of Pediatrics and Medicine, Departments of Pediatrics and Medicine, Western University, and Children’s Hospital, London Health Sciences Centre, London, ON, CanadaDepartment of Pediatric Cardiology and Intensive Care Medicine, Hannover Medical School, Hannover, GermanyHomozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder characterized by severely elevated low-density lipoprotein (LDL) cholesterol levels, predisposing patients to early cardiovascular disease. While LDL apheresis is the standard extracorporeal therapy, plasmapheresis (PLEX) is often used in younger children due to limitations in vascular access. Evinacumab, an ANGPTL3 inhibitor, has emerged as an effective adjunct for lowering LDL cholesterol. This case, to our knowledge, describes the first pediatric outpatient implementation of monthly plasmapheresis with post-PLEX Evinacumab infusions, enabled by dual-port access and Octaplasma support to maintain immunoglobulin levels. This report highlights procedural innovations that enabled sustained therapy and emphasizes the need for standardized approaches for combining Evinacumab with extracorporeal treatment in pediatric HoFH.https://www.frontiersin.org/articles/10.3389/fped.2025.1559147/fullhomozygous familial hypercholesterolemiaevinacumabplasmapheresispediatric hyperlipidemiacase report |
| spellingShingle | Guido Filler Kambiz Norozi Kambiz Norozi Outpatient monthly plasmapheresis with post-PLEX evinacumab in pediatric homozygous familial hypercholesterolemia: a case report on port access and immunoglobulin preservation Frontiers in Pediatrics homozygous familial hypercholesterolemia evinacumab plasmapheresis pediatric hyperlipidemia case report |
| title | Outpatient monthly plasmapheresis with post-PLEX evinacumab in pediatric homozygous familial hypercholesterolemia: a case report on port access and immunoglobulin preservation |
| title_full | Outpatient monthly plasmapheresis with post-PLEX evinacumab in pediatric homozygous familial hypercholesterolemia: a case report on port access and immunoglobulin preservation |
| title_fullStr | Outpatient monthly plasmapheresis with post-PLEX evinacumab in pediatric homozygous familial hypercholesterolemia: a case report on port access and immunoglobulin preservation |
| title_full_unstemmed | Outpatient monthly plasmapheresis with post-PLEX evinacumab in pediatric homozygous familial hypercholesterolemia: a case report on port access and immunoglobulin preservation |
| title_short | Outpatient monthly plasmapheresis with post-PLEX evinacumab in pediatric homozygous familial hypercholesterolemia: a case report on port access and immunoglobulin preservation |
| title_sort | outpatient monthly plasmapheresis with post plex evinacumab in pediatric homozygous familial hypercholesterolemia a case report on port access and immunoglobulin preservation |
| topic | homozygous familial hypercholesterolemia evinacumab plasmapheresis pediatric hyperlipidemia case report |
| url | https://www.frontiersin.org/articles/10.3389/fped.2025.1559147/full |
| work_keys_str_mv | AT guidofiller outpatientmonthlyplasmapheresiswithpostplexevinacumabinpediatrichomozygousfamilialhypercholesterolemiaacasereportonportaccessandimmunoglobulinpreservation AT kambiznorozi outpatientmonthlyplasmapheresiswithpostplexevinacumabinpediatrichomozygousfamilialhypercholesterolemiaacasereportonportaccessandimmunoglobulinpreservation AT kambiznorozi outpatientmonthlyplasmapheresiswithpostplexevinacumabinpediatrichomozygousfamilialhypercholesterolemiaacasereportonportaccessandimmunoglobulinpreservation |