Research ExplorerPublicationsOxalobacter formigenes treatment combined with intensive dia...

Oxalobacter formigenes treatment combined with intensive dialysis lowers plasma oxalate and halts disease progression in a patient with severe infantile oxalosis

Standard

Oxalobacter formigenes treatment combined with intensive dialysis lowers plasma oxalate and halts disease progression in a patient with severe infantile oxalosis. / Pape, Lars; Ahlenstiel-Grunow, Thurid; Birtel, Johannes; Krohne, Tim U; Hoppe, Bernd.

In: PEDIATR NEPHROL, Vol. 35, No. 6, 06.2020, p. 1121-1124.

Research output: SCORING: Contribution to journal › Short publication › Research › peer-review

Harvard

Pape, L, Ahlenstiel-Grunow, T, Birtel, J, Krohne, TU & Hoppe, B 2020, 'Oxalobacter formigenes treatment combined with intensive dialysis lowers plasma oxalate and halts disease progression in a patient with severe infantile oxalosis', PEDIATR NEPHROL, vol. 35, no. 6, pp. 1121-1124. https://doi.org/10.1007/s00467-019-04463-9

APA

Pape, L., Ahlenstiel-Grunow, T., Birtel, J., Krohne, T. U., & Hoppe, B. (2020). Oxalobacter formigenes treatment combined with intensive dialysis lowers plasma oxalate and halts disease progression in a patient with severe infantile oxalosis. PEDIATR NEPHROL, 35(6), 1121-1124. https://doi.org/10.1007/s00467-019-04463-9

Vancouver

Pape L, Ahlenstiel-Grunow T, Birtel J, Krohne TU, Hoppe B. Oxalobacter formigenes treatment combined with intensive dialysis lowers plasma oxalate and halts disease progression in a patient with severe infantile oxalosis. PEDIATR NEPHROL. 2020 Jun;35(6):1121-1124. https://doi.org/10.1007/s00467-019-04463-9

Bibtex

@article{7b7dfec759e641fdb360873e710993c0,
title = "Oxalobacter formigenes treatment combined with intensive dialysis lowers plasma oxalate and halts disease progression in a patient with severe infantile oxalosis",
abstract = "BACKGROUND: Infantile oxalosis, the most devastating form of primary hyperoxaluria type 1 (PH1), often leads to end-stage renal disease (ESRD) during the first weeks to months of life.CASE-DIAGNOSIS: Here, we report the outcome of the therapeutic use of Oxalobacter formigenes (Oxabact OC5; OxThera AB, Stockholm, Sweden) in a female infant with PH1 who exhibited severely elevated plasma oxalate (Pox) levels, pronounced nephrocalcinosis, anuretic end-stage renal disease, and retinal oxalate deposits. Following the diagnosis of PH1 at an age of 8 weeks, a combined regimen of daily peritoneal dialysis, daily pyridoxine treatment and hemodialysis (3 times a week) was unable to reduce the pronounced hyperoxalemia. After the addition of Oxalobacter formigenes therapy to the otherwise unchanged treatment regimen, Pox levels first stabilized and subsequently declined from 130 μmol/L to around 80 μmol/L. Nephrocalcinosis and retinal deposits stabilized. Oxalobacter formigenes treatment was well-tolerated and no related adverse events were observed. The patient showed nearly age-appropriate growth and development and received successful combined liver-kidney transplantation at the age of two years.CONCLUSIONS: Treatment with O. formigenes combined with intensive dialysis led to reduction of Pox, stabilization of systemic oxalosis, and improvement in the clinical disease course. O. formigenes treatment may be an option for reduction of oxalosis in infantile patients with insufficient response to conservative treatments until combined liver-kidney transplantation can be performed.",
keywords = "Calcium Oxalate/blood, Disease Progression, Female, Humans, Hyperoxaluria/etiology, Infant, Kidney Transplantation, Liver Transplantation, Oxalobacter formigenes/metabolism, Renal Dialysis/methods, Renal Insufficiency, Chronic/complications",
author = "Lars Pape and Thurid Ahlenstiel-Grunow and Johannes Birtel and Krohne, {Tim U} and Bernd Hoppe",
year = "2020",
month = jun,
doi = "10.1007/s00467-019-04463-9",
language = "English",
volume = "35",
pages = "1121--1124",
journal = "PEDIATR NEPHROL",
issn = "0931-041X",
publisher = "Springer",
number = "6",

}

RIS

TY - JOUR

T1 - Oxalobacter formigenes treatment combined with intensive dialysis lowers plasma oxalate and halts disease progression in a patient with severe infantile oxalosis

AU - Pape, Lars

AU - Ahlenstiel-Grunow, Thurid

AU - Birtel, Johannes

AU - Krohne, Tim U

AU - Hoppe, Bernd

PY - 2020/6

Y1 - 2020/6

N2 - BACKGROUND: Infantile oxalosis, the most devastating form of primary hyperoxaluria type 1 (PH1), often leads to end-stage renal disease (ESRD) during the first weeks to months of life.CASE-DIAGNOSIS: Here, we report the outcome of the therapeutic use of Oxalobacter formigenes (Oxabact OC5; OxThera AB, Stockholm, Sweden) in a female infant with PH1 who exhibited severely elevated plasma oxalate (Pox) levels, pronounced nephrocalcinosis, anuretic end-stage renal disease, and retinal oxalate deposits. Following the diagnosis of PH1 at an age of 8 weeks, a combined regimen of daily peritoneal dialysis, daily pyridoxine treatment and hemodialysis (3 times a week) was unable to reduce the pronounced hyperoxalemia. After the addition of Oxalobacter formigenes therapy to the otherwise unchanged treatment regimen, Pox levels first stabilized and subsequently declined from 130 μmol/L to around 80 μmol/L. Nephrocalcinosis and retinal deposits stabilized. Oxalobacter formigenes treatment was well-tolerated and no related adverse events were observed. The patient showed nearly age-appropriate growth and development and received successful combined liver-kidney transplantation at the age of two years.CONCLUSIONS: Treatment with O. formigenes combined with intensive dialysis led to reduction of Pox, stabilization of systemic oxalosis, and improvement in the clinical disease course. O. formigenes treatment may be an option for reduction of oxalosis in infantile patients with insufficient response to conservative treatments until combined liver-kidney transplantation can be performed.

AB - BACKGROUND: Infantile oxalosis, the most devastating form of primary hyperoxaluria type 1 (PH1), often leads to end-stage renal disease (ESRD) during the first weeks to months of life.CASE-DIAGNOSIS: Here, we report the outcome of the therapeutic use of Oxalobacter formigenes (Oxabact OC5; OxThera AB, Stockholm, Sweden) in a female infant with PH1 who exhibited severely elevated plasma oxalate (Pox) levels, pronounced nephrocalcinosis, anuretic end-stage renal disease, and retinal oxalate deposits. Following the diagnosis of PH1 at an age of 8 weeks, a combined regimen of daily peritoneal dialysis, daily pyridoxine treatment and hemodialysis (3 times a week) was unable to reduce the pronounced hyperoxalemia. After the addition of Oxalobacter formigenes therapy to the otherwise unchanged treatment regimen, Pox levels first stabilized and subsequently declined from 130 μmol/L to around 80 μmol/L. Nephrocalcinosis and retinal deposits stabilized. Oxalobacter formigenes treatment was well-tolerated and no related adverse events were observed. The patient showed nearly age-appropriate growth and development and received successful combined liver-kidney transplantation at the age of two years.CONCLUSIONS: Treatment with O. formigenes combined with intensive dialysis led to reduction of Pox, stabilization of systemic oxalosis, and improvement in the clinical disease course. O. formigenes treatment may be an option for reduction of oxalosis in infantile patients with insufficient response to conservative treatments until combined liver-kidney transplantation can be performed.

KW - Calcium Oxalate/blood

KW - Disease Progression

KW - Female

KW - Humans

KW - Hyperoxaluria/etiology

KW - Infant

KW - Kidney Transplantation

KW - Liver Transplantation

KW - Oxalobacter formigenes/metabolism

KW - Renal Dialysis/methods

KW - Renal Insufficiency, Chronic/complications

U2 - 10.1007/s00467-019-04463-9

DO - 10.1007/s00467-019-04463-9

M3 - Short publication

C2 - 32107618

VL - 35

SP - 1121

EP - 1124

JO - PEDIATR NEPHROL

JF - PEDIATR NEPHROL

SN - 0931-041X

IS - 6

ER -