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The effect of lumasiran therapy for primary hyperoxaluria type 1 in small infants

dc.rights.licenseopenen_US
dc.contributor.authorMEAUX, Marie-Noelle
dc.contributor.authorSELLIER-LECLERC, Anne-Laure
dc.contributor.authorACQUAVIVA-BOURDAIN, Cecile
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorHARAMBAT, Jerome
IDREF: 110567358
dc.contributor.authorALLARD, Lise
dc.contributor.authorBACCHETTA, Justine
dc.date.accessioned2022-03-07T10:02:09Z
dc.date.available2022-03-07T10:02:09Z
dc.date.issued2022-01-11
dc.identifier.issn1432-198X (Electronic) 0931-041X (Linking)en_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/128840
dc.description.abstractEnBACKGROUND: Lumasiran, a sub-cutaneous RNA-interference therapy, has been recently approved for primary hyperoxaluria type 1 (PH1), with doses and intervals according to body weight. Little is known as to its use in infants; the aim of this study was to describe treatment outcome in 3 infants who received lumasiran therapy before 2 years of age. CASE-DIAGNOSIS/TREATMENT: Patient 1 was diagnosed antenatally and received lumasiran from day 9. According to the product information template (PIT), he received monthly lumasiran (3 times at 6 mg/kg, then 3 mg/kg), with hyperhydration and potassium citrate. Despite decreased plasma oxalate levels, persistent normal kidney function, and good tolerance, kidney ultrasound performed after 2 months found nephrocalcinosis, without normalization of urinary oxalate (UOx). The dose was increased back to 6 mg/kg, inducing a normalization in UOx. Nephrocalcinosis started to improve at month 10. Patient 2 was diagnosed at 2.5 months (acute kidney failure); nephrocalcinosis was present from diagnosis. She received monthly lumasiran (6 mg/kg), with progressive decrease in UOx and substantial improvement in kidney function but stable nephrocalcinosis after 9 injections. Patient 3 was diagnosed fortuitously (nephrocalcinosis) at 3.5 months and received lumasiran before genetic diagnosis, leading to decreased UOx and maintenance of normal kidney function. Nephrocalcinosis improved after 5 injections. CONCLUSIONS: This report presents the youngest children treated with lumasiran worldwide. Lumasiran seems effective without side effects in infants but does not completely prevent the onset of nephrocalcinosis in the most severe forms. Higher doses than those proposed in the PIT might be required because of hepatic immaturity.
dc.language.isoENen_US
dc.subject.enInfant
dc.subject.enNephrocalcinosis
dc.subject.enRNA-interference
dc.subject.enUrinary oxalate
dc.title.enThe effect of lumasiran therapy for primary hyperoxaluria type 1 in small infants
dc.typeArticle de revueen_US
dc.identifier.doi10.1007/s00467-021-05393-1en_US
dc.subject.halSciences du Vivant [q-bio]/Santé publique et épidémiologieen_US
dc.identifier.pubmed35015123en_US
bordeaux.journalPediatric Nephrologyen_US
bordeaux.hal.laboratoriesBordeaux Population Health Research Center (BPH) - UMR 1219en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.institutionINSERMen_US
bordeaux.teamLEHA_BPHen_US
bordeaux.peerReviewedouien_US
bordeaux.inpressnonen_US
hal.identifierhal-03599498
hal.version1
hal.date.transferred2022-03-07T10:02:11Z
hal.exporttrue
dc.rights.ccPas de Licence CCen_US
bordeaux.COinSctx_ver=Z39.88-2004&rft_val_fmt=info:ofi/fmt:kev:mtx:journal&rft.jtitle=Pediatric%20Nephrology&rft.date=2022-01-11&rft.eissn=1432-198X%20(Electronic)%200931-041X%20(Linking)&rft.issn=1432-198X%20(Electronic)%200931-041X%20(Linking)&rft.au=MEAUX,%20Marie-Noelle&SELLIER-LECLERC,%20Anne-Laure&ACQUAVIVA-BOURDAIN,%20Cecile&HARAMBAT,%20Jerome&ALLARD,%20Lise&rft.genre=article


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