Does the valproic acid level assay (CPT 80164) have cross reactivity with the valproate metabolites/conjugates? Do these metabolites contribute to valproic acid toxicity?

Comment by InpharmD Researcher

Evidence assessing cross-reactivity of CPT 80164 valproic acid immunoassays with valproate metabolites is limited. Comparative studies show enzyme-multiplied immunoassay technique (EMT) assays systematically overestimate valproic acid versus chromatographic methods, likely due to antibody cross-reactivity with conjugated metabolites such as valproate glucuronide, although this interference is generally described as minor. Manufacturer cross-reactivity testing (Table 1) demonstrates minimal interference for most metabolites but measurable cross-reactivity with select unsaturated metabolites under testing conditions. Separately, the toxicology literature consistently describes valproic acid-associated toxicity as being mediated in part by specific metabolites, including unsaturated and hydroxylated metabolites implicated in hepatotoxicity, hyperammonemia, mitochondrial dysfunction, and neurologic adverse effects. However, formal validation across all clinically relevant valproate metabolites is lacking.

valproic acid metabolites toxicity; valproic acid level assay metabolites; valproate conjugates assay toxicity

Background

A 2021 study evaluated the agreement between an enzyme-multiplied immunoassay technique (EMIT) and Liquid Chromatography-Electrospray Ionization-Tandem Mass Spectrometry (LC-ESI-MS/MS) for routine therapeutic drug monitoring of valproic acid (VPA) in pediatric patients with epilepsy. The study included 774 plasma samples from 711 children, with outcomes assessing assay correlation, absolute and relative bias, and diagnostic concordance across therapeutic VPA ranges. Although a strong correlation was observed between methods (r2 = 0.9281), EMIT systematically overestimated VPA concentrations compared with LC-ESI-MS/MS, with a mean absolute bias of 14.5 mcg/mL and a relative overestimation of 27.8%, resulting in diagnostic mismatch in 32.9% of samples. The authors noted that this difference may be related to antibody cross-reactivity with valproate conjugated metabolites, including valproate glucuronide (VPAG), which were not evaluated during immunoassay validation. The study did not assess individual metabolite concentrations or clinical toxicity outcomes. [1]

The Handbook of Drug Monitoring Methods describes anticonvulsants as drugs for which therapeutic drug monitoring is appropriate, as blood concentrations correlate with pharmacodynamic effects, while dose–concentration relationships are variable. The text notes that multiple assay methodologies are used, including chromatographic methods (GC, HPLC, mass spectrometry) and immunoassays, with different sources of analytic interference depending on method. For immunoassays, cross-reactivity with structurally related drugs or metabolites is a recognized issue and is well documented for phenytoin and carbamazepine, where metabolites and prodrugs can significantly interfere with measured concentrations. In contrast, the handbook reports no documented clinically significant interferences with immunoassays for measurement of valproic acid, noting only minor cross-reactivity. [2]

A 2020 study evaluated the performance of an enzyme-multiplied immunoassay technique (EMIT) for routine therapeutic drug monitoring of valproic acid using the Emit 2000 Valproic Acid Assay. The study assessed assay calibration, quality control performance, and clinical applicability in both therapeutic monitoring and acute overdose settings, demonstrating that EMIT can reliably quantify serum valproic acid within its calibrated range. The assay uses monoclonal antibodies reactive to valproic acid and is intended to measure the parent drug; however, the authors did not perform or report formal analytical cross-reactivity testing with valproate metabolites or glucuronide conjugates, so definitive conclusions regarding metabolite interference cannot be drawn from this study. The article describes that valproic acid undergoes extensive hepatic metabolism, producing unsaturated metabolites such as 2-en-VPA and 3-keto-VPA, which have been implicated in toxicity mechanisms including hyperammonemia, hepatotoxicity, and pancreatitis. Based on these findings, the study supports that valproate metabolites contribute to clinical toxicity, but it does not establish that these metabolites are directly measured or meaningfully contribute to serum valproic acid concentrations reported by CPT 80164 immunoassays. [3]

A 1980 study evaluated an enzyme-multiplied immunoassay technique (EMIT) for the measurement of serum valproic acid by comparison with gas-liquid chromatography. Using 80 patient samples over a wide range of concentrations, the authors reported a significant correlation coefficient (r= 0.979) and state that the correlation between the two methods examined is satisfactory for routine measurement of valproic acid. The EMIT assay demonstrated high analytical specificity, with no antibody cross-reactivity detected when tested against other commonly prescribed antiepileptic drugs, including phenobarbitone, primidone, phenytoin, carbamazepine, and ethosuximide. The study did not evaluate cross-reactivity with valproic acid metabolites or conjugates and did not assess the contribution of metabolites to valproic acid toxicity. [4]

Valproic acid (VPA) toxicity is primarily linked to its metabolites, which can cause significant harm to the human body. These toxic effects are dose-dependent and can impact systems like the central nervous system or specific organs like the liver. VPA metabolites arise as intermediates, byproducts, or end products of VPA metabolism, with hepatocytes playing a role in organizing these metabolic processes to mitigate or reduce the accumulation of toxic compounds. Specific metabolites like 3-hydroxy, 4-hydroxy, and 5-hydroxy valproic acids are known for their hepatotoxic effects, being products of P-oxidation and involving several cytochrome P450 isoenzymes. 3-Oxovalproic acid, while suspected to be toxic, is still under study for its full toxic potential. Valproyl-Coenzyme A is a significant mitochondrial metabolite of VPA that can inhibit succinate-CoA ligase, potentially leading to mitochondrial DNA depletion and subsequent liver failure in patients with mitochondrial deficiencies. 2-N-Propyl-4-oxopentanoic acid can cause teratogenic effects and microvesicular steatosis. 4-Ene-valproic acid is highly hydrophobic, contributing to hepatotoxicity and hyperammonemia and is under study for therapeutic potential in various conditions. 2-Propyl-2,4-pentadienoic acid, through glucuronidation and inhibition of β-oxidation, leads to impaired urea production and associated hyperammonemia. 2-Ene-valproic acid is neurotoxic, associated with neurological adverse drug reactions (ADRs), such as diplopia, seizures, and cognitive and behavioral disorders. Valproylcarnitine, by reducing L-carnitine concentrations through urinary excretion, can lead to lipid myopathy, hypoglycemia, fatty liver disease, and hyperammonemia. [5]

References: [1] Xia Y, Long JY, Shen MY, et al. Switching Between LC-ESI-MS/MS and EMIT Methods for Routine TDM of Valproic Acid in Pediatric Patients With Epilepsy: What Clinicians and Researchers Need to Know. Front Pharmacol. 2021;12:750744. Published 2021 Nov 23. doi:10.3389/fphar.2021.750744
[2] Clarke W. Interferences with measurement of anticonvulsants. In: Dasgupta A, ed. Handbook of Drug Monitoring Methods: Therapeutics and Drugs of Abuse. Humana Press; 2008:133-148. doi:10.1007/978-1-59745-031-7_7
[3] Tudosie MS. Study regarding the determination of valproic acid serum levels by emit. FARMACIA. 2020;68(5):898-904. doi:10.31925/farmacia.2020.5.17
[4] Elyas AA, Goldberg VD, Ratnaraj N, Lascelles PT. Valproic acid estimation by enzyme immunoassay. Ann Clin Biochem. 1980;17(6):307-310. doi:10.1177/000456328001700605
[5] Shnayder NA, Grechkina VV, Khasanova AK, et al. Therapeutic and Toxic Effects of Valproic Acid Metabolites. Metabolites. 2023;13(1):134. Published 2023 Jan 16. doi:10.3390/metabo13010134
Literature Review

A search of the published medical literature revealed 3 studies investigating the researchable question:

Does the valproic acid level assay (CPT 80164) have cross reactivity with the valproate metabolites/conjugates? Do these metabolites contribute to valproic acid toxicity?

Level of evidence

C - Multiple studies with limitations or conflicting results  Read more→



Please see Tables 1-3 for your response.


Valproic Acid Metabolite Cross-reactivity via VALP method

Substance Concentration mcg/mL (mcmol/L)

% Cross-reactivity

3-keto valproic acid 100 (633)

<5

3-hydroxy valproic acid 100 (625)

<8

4-hydroxy valproic acid 100 (625)

<8

2-propyl glutaric acid 500 (2874)

<8

5-hydroxy valproic acid 100 (625)

28

4-ene valproic acid* 100 (704)

47

*Metabolite not present in plasma

The presented structurally related compounds exhibited cross-reactivity at a valproic acid concentration of 100 µg/mL (693 µmol/L).

The VALP method is based on a particle-enhanced turbidimetric inhibition immunoassay (PETINIA) technique which measures the level of valproic acid and metabolites.

 

References:
[1] Adapted from: Siemens Healthcare Diagnostics Inc. Dimension clinical chemistry system Flex VALP reagent cartridge: Valproic Acid. Instructions for Use. April 1, 2019. Accessed January 7, 2026. https://doclib.siemens-healthineers.com/rest/v1/view?document-id=601647

 

Valproate Metabolites in Serum and Urine During Antiepileptic Therapy in Children with Infantile Spasms: Abnormal Metabolite Pattern Associated with Reversible Hepatotoxicity

Design

Prospective, observational, cohort study

N= 25

Objective

To identify abnormal metabolite patterns of valproate (VPA) as possible early indicators of VPA-induced liver toxicity

Study Groups

All patients (n= 25)

Inclusion Criteria

Children with infantile spasms treated with high doses of VPA (up to 100 mg/kg/day)

Exclusion Criteria

Not specified

Methods

This was a review of infants enrolled in a previous study who received valproic acid starting at 10-15 mg/kg body weight (up to 100 mg/kg/day) for at least 4-6 weeks. The daily dose of VPA was administered in three divided doses, with the last dose comprising half of the total dose. Serum and urine levels of VPA and metabolites were collected and stored during therapy. 

If the children were not seizure-free after 4-6 weeks, then dexamethasone was added. Carbamazepine was also used to treat focal point seizures during VPA therapy.

Duration

Follow-up period: 18 to 54 months

Outcome Measures

Primary: Identification of abnormal metabolite patterns

Secondary: Association of metabolite patterns with hepatotoxicity

Baseline Characteristics  

All patients (n= 25)

Sex - Female

13 (52%)

Age at initiation of VPA therapy, mean months (range)

7 (4-12)
EEG-pattern - Hypearrhythmia

25/25 (100%)

Seizure-free after 3 months of VPA monotherapy

18/25 (72%)

Results Concentration (pg/mL) after 8 weeks

Monotherapy (n= 18)

Polytherapy (n= 7)
VPA

89.33 ± 32.06

84.17 ± 6.65
2-en (E)

5.54 ± 2.63

12.74 ± 8.57
3-keto

5.73 ± 3.48

12.47 ± 4.50
2,3'-dien

5.18 ± 2.67

5.63 ± 1.46
4-en

0.24 ± 0.12

0.17 ± 0.05

Notable increases in the concentrations of unsaturated metabolites like 2-en, 2,3'-dien, and 3-en were documented, particularly in two patients. These children showed elevated liver enzyme activity alongside transient hepatomegaly yet had no changes in other liver function tests.

One child experienced a febrile adenovirus infection, and the other suffered prolonged pyelonephritis due to E. coli. The rise in unsaturated metabolite levels aligned with febrile infections and increased liver enzyme activity, normalizing after the VPA dose was reduced and fever subsided.

No adverse reactions or unusual metabolite patterns were observed during a follow-up period of up to 54 months. Patients undergoing polytherapy with dexamethasone exhibited significantly increased mono- or diunsaturated metabolites, primarily through the P-oxidation pathway. These patients had severe seizure disorders and were administered high VPA doses, reflected by their low VPA concentration/dose ratios, suggestive of high clearance rates.

Adverse Events

Two children had transiently aberrant metabolite profiles indicating altered β-oxidation, associated with hepatomegaly and increased liver enzyme activities during febrile infections and dexamethasone comedication.

Study Author Conclusions

Establishing the VPA metabolite profile may aid in evaluating patients showing signs of liver dysfunction during VPA therapy. Early detection of abnormal metabolite patterns might decrease the risk of severe hepatic injury.

Critique

The study provides valuable insights into the potential for using metabolite profiles as early indicators of hepatotoxicity (but not other toxicities). However, the small sample size and lack of a control group limit the generalizability of the findings. The study's reliance on single-point measurements may also affect the accuracy of the metabolite pattern assessments.

 

References:
[1] Fisher E, Siemes H, Pund R, Wittfoht W, Nau H. Valproate metabolites in serum and urine during antiepileptic therapy in children with infantile spasms: abnormal metabolite pattern associated with reversible hepatotoxicity. Epilepsia. 1992;33(1):165-171. doi:10.1111/j.1528-1157.1992.tb02301.x

 

L-Carnitine supplementation to reverse hyperammonemia in a patient undergoing chronic valproic acid treatment: A case report

Design

Case report

Case presentation

A 42-year-old woman with an 18-year history of epilepsy was receiving chronic valproic acid therapy (1500 mg/day). After a change in anticonvulsant therapy, she developed hyperammonemia associated with increased seizures and gastrointestinal symptoms.

Laboratory evaluation showed elevated ammonia levels, detectable concentrations of the valproic acid metabolite 4-en-valproic acid, and L-carnitine levels at the inferior limit of the population reference range with an increased acylcarnitine/L-carnitine ratio. Valproic acid and 4-en-valproic acid concentrations were measured using validated chromatographic methods.

L-carnitine supplementation (1 g/day) was initiated. After supplementation, ammonia levels decreased to within the reference range, 4-en-valproic acid concentrations decreased, L-carnitine levels increased, and seizure control improved.

Study Author Conclusions

The authors concluded that hyperammonemia during chronic valproic acid treatment may be related to carnitine depletion and increased production of the toxic metabolite 4-en-valproic acid. L-carnitine supplementation was useful in reversing hyperammonemia and reducing metabolite levels, allowing continuation of valproic acid therapy in this patient.

 

References:
[1] Maldonado C, Guevara N, Silveira A, Fagiolino P, Vázquez M. L-Carnitine supplementation to reverse hyperammonemia in a patient undergoing chronic valproic acid treatment: A case report. J Int Med Res. 2017;45(3):1268-1272. doi:10.1177/0300060517703278