Lumasiran Therapy Demonstrates Effectiveness for Rare, Genetic Disease

Researchers recently published a report on the positive effects of lumasiran therapy on 3 infants with primary hyperoxaluria type 1 (PH1) who were aged less than 2 years in Pediatric Nephrology.

According to the report, the first patient was diagnosed prior to birth and thus received lumasiran beginning at day 9 with monthly treatment 3 times at 6mg/kg, then 3mg/kg with hyperhydration and potassium citrate. Doses and intervals for the treatment were administered according to each infant’s body weight.

Results for patient 1 included persistent normal kidney function, decreased plasma oxalate levels, and good tolerance, but after 2 months a kidney ultrasound found nephrocalcinosis without normalization of urinary oxalate (UOx). A normalization in UOx was induced after the dose was increased back to 6mg/kg and at month 10 nephrocalcinosis began improving.

The second patient in the report was diagnosed at 2.5 months of age with acute kidney failure and from diagnosis nephrocalcinosis was present. Monthly treatment of 6mg/kg resulted in substantial improvement in kidney function, a progressive decrease in UOx, and stable nephrocalcinosis after 9 injections.

The third patient was diagnosed at 3.5 months due to nephrocalcinosis and received treatment before the genetic diagnosis. Outcomes for patient 3 included decreased UOx, maintenance of normal kidney function, and an improvement in nephrocalcinosis after 5 injections.

“Lumasiran seems effective without side effects in infants but does not completely prevent the onset of nephrocalcinosis in the most severe forms,” concluded study authors. “Higher doses than those proposed in the product information template might be required because of hepatic immaturity.”

This report currently represents the youngest patients treated with lumasiran globally.

Reference:
Méaux MN, Sellier-Leclerc AL, Acquaviva-Bourdain, C, Harambat J, Allard L, Bacchetta J. The effect of lumasiran therapy for primary hyperoxaluria type 1 in small infants. Pediatr Nephrol. Published online January 11, 2022. doi:10.1007/s00467-021-05393-1