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Deep Dives

Antifolates & the slow erosion of the tubule

Pemetrexed tubular nephrotoxicity

Pemetrexed leaves the body through the kidney, so the tubule sees the drug at every cycle — and the injury accumulates quietly, cycle by cycle, into a tubulointerstitial scar that often does not heal once the drug is finally stopped.

21%
clinically relevant renal declineShare of 359 pemetrexed-treated patients with a ≥25% fall in eGFR during treatment in a retrospective cohort
5.66×
risk with cumulative doseAdjusted odds ratio for renal decline once patients pass ≥10 cycles of pemetrexed-based therapy (95% CI 1.73–18.54) — the injury is cumulative
8.1%
stopped for nephrotoxicityPatients who discontinued pemetrexed-based treatment because of declining renal function
often irreversible
does not recoverIn a biopsy series of maintenance pemetrexed, kidney function remained impaired but stable after the drug was stopped — the tubulointerstitial injury does not reverse
12.6%
early AKI with immuno-chemoEarly AKI in front-line ICI-based therapy for metastatic NSCLC; highest (16.7%) with pemetrexed + pembrolizumab + carboplatin

Teaching case · illustrative composite, not a real patient

A 66-year-old man with metastatic non-squamous NSCLC, ten months into maintenance pemetrexed (well past his tenth cycle), is noted to have a serum creatinine that has crept from 0.9 to 1.6 mg/dL over successive cycles. He feels well; the urinalysis is bland, without significant proteinuria, hematuria, or cellular casts, and he has no eosinophilia or rash.

Because the decline is gradual and the sediment is bland, a drug-induced tubular process is suspected. If a kidney biopsy is done it shows tubulointerstitial injury — tubular simplification and loss of brush border with interstitial fibrosis — rather than a florid interstitial nephritis. Pemetrexed is held. Over the following months the creatinine stabilizes but does not return to baseline: he is left with chronic kidney disease.

Teaching point — Pemetrexed nephrotoxicity is insidious and cumulative, not a single dramatic event: watch the eGFR trend across cycles rather than any one value, since risk climbs with cumulative dose. The urine is often bland and the injury is a tubulointerstitial one that frequently does not recover after discontinuation. In combination with a checkpoint inhibitor, the harder question is which drug to blame — chemotherapy tends to cause acute tubular injury, whereas the ICI causes acute tubulointerstitial nephritis, and telling them apart decides which agent to hold.

01

How it happens

The pathophysiology as a cascade — select a step to follow the mechanism.

02

How we learned it

  1. 2006

    Vootukuru et al. report a patient who developed acute renal failure with nephrogenic diabetes insipidus and distal renal tubular acidosis after pemetrexed, postulating renal tubular toxicity as the unifying lesion.

    An early, detailed description that pemetrexed injures the tubule — producing not just azotemia but concentrating and acidification defects.

    PMID 17018900
  2. 2011

    Glezerman et al. biopsy three patients on maintenance pemetrexed and find tubulointerstitial injury with tubular simplification, brush-border loss, and atrophy; function stayed impaired but stable after stopping.

    Pathologically defined the lesion as a tubulointerstitial injury and showed it does not reverse on drug withdrawal.

    PMID 21849225
  3. 2014

    Chauvet et al. describe two patients — including one on single-agent pemetrexed — whose AKI progressed to chronic kidney disease, with biopsy showing acute tubular necrosis and interstitial fibrosis.

    Confirmed that pemetrexed alone can cause AKI that leaves permanent CKD, decoupling it from co-administered cisplatin.

    PMID 24424085
  4. 2020

    de Rouw et al. show in 359 patients that ≥10 cycles of pemetrexed-based therapy is an independent risk factor for renal decline (adjusted OR 5.66), with 21% declining and 8.1% discontinuing.

    Quantified the dose-dependence and flagged that longer survival on immuno-chemotherapy means more cycles — and more nephrotoxicity.

    PMID 32505078
  5. 2020

    Dumoulin et al. lay out how to distinguish chemotherapy-induced acute tubular injury from checkpoint-inhibitor tubulointerstitial nephritis when the two are given together.

    Made the combination-era diagnostic problem explicit: which drug injured the kidney determines which one you can safely continue.

    PMID 32360753
  6. 2025

    Li et al. find early AKI in 12.6% of patients on front-line ICI-based therapy (16.7% with pemetrexed + pembrolizumab + carboplatin), associated with worse 12-month survival (HR 1.73).

    Linked early renal injury in these regimens to survival, reinforcing close monitoring in the modern combination era.

    PMID 40719750
03

The landmark studies

Biopsy case series, 3 patients on maintenance pemetrexed

Kidney tubular toxicity of maintenance pemetrexed therapy

Glezerman IG, et al. · Am J Kidney Dis 2011 · PMID 21849225

Kidney biopsies showed tubulointerstitial injury (tubular simplification, brush-border loss, atrophy); renal function remained impaired but stable after stopping the drug.

3/3 with biopsy-proven tubulointerstitial injury; function impaired-but-stable after discontinuation

Case series with biopsy, 2 patients (one single-agent)

Pemetrexed-induced acute kidney injury leading to chronic kidney disease

Chauvet S, et al. · Clin Nephrol 2014 · PMID 24424085

AKI after pemetrexed — including as a single agent — progressed to persistent CKD; biopsy showed acute tubular necrosis and interstitial fibrosis.

2/2 with persistent CKD despite discontinuation; ATN + interstitial fibrosis on biopsy

Retrospective cohort, 359 patients, logistic regression

Cumulative pemetrexed dose increases the risk of nephrotoxicity

de Rouw N, et al. · Lung Cancer 2020 · PMID 32505078

A clinically relevant fall in eGFR occurred in 21% of patients and 8.1% discontinued for nephrotoxicity; ≥10 cycles of therapy independently raised the risk.

21% with ≥25% eGFR decline; 8.1% discontinued; ≥10 cycles adjusted OR 5.66 (1.73–18.54)

Retrospective cohort, 310 patients

Early acute kidney injury and its association with survival in metastatic NSCLC on front-line immunotherapy-based therapies

Li M, et al. · Cancer Med 2025 · PMID 40719750

Early AKI occurred in 12.6% of patients (highest with pemetrexed + pembrolizumab + carboplatin) and was associated with reduced 12-month survival.

AKI 12.6% overall, 16.7% with pem/pembro/carbo; HR for death 1.73 (1.06–2.84)

04

What the data says now

How disproportionately each agent's FAERS reports name these phenotypes vs. all other drugs (reporting odds ratio; significant signals only, 95% CI lower bound > 1; as of 2026-07-10). A reporting signal, not incidence or proven causation.

Per-agent FAERS reporting odds ratio for each injury phenotype in this syndrome.
AgentATNCINLYTE
PemetrexedPemetrexed, Acute Tubular Necrosis: ROR 10.15, 171 reportsPemetrexed, Chronic Interstitial Nephropathy: no significant signalPemetrexed, Electrolyte Disturbance: ROR 1.72, 572 reports
21%

Clinically relevant renal decline

359 pemetrexed-treated patients (≥25% eGFR fall)

PMID 32505078
8.1%

Discontinuation for nephrotoxicity

Same pemetrexed cohort

PMID 32505078
Adjusted OR 5.66

Risk with ≥10 cycles

Cumulative-dose risk analysis (95% CI 1.73–18.54)

PMID 32505078
12.6% (16.7% with pem/pembro/carbo)

Early AKI on front-line ICI-based therapy

310 metastatic NSCLC patients

PMID 40719750
Impaired but stable (often permanent)

Recovery after discontinuation

Maintenance pemetrexed biopsy series

PMID 21849225
05

How it's managed

  1. 1

    Track the eGFR trend across cycles, not a single value

    Because risk climbs with cumulative dose and longer treatment, monitor renal function every cycle and read the trajectory — a slow, stepwise creep is the characteristic signal.

    Cumulative dose (≥10 cycles) an independent risk factor for renal decline · PMID 32505078

  2. 2

    Treat a decline as potentially permanent — hold early

    Once eGFR falls, the tubulointerstitial injury frequently does not reverse; withholding or stopping pemetrexed to prevent further, permanent loss is the main lever, since there is no specific antidote.

    Function impaired-but-stable after discontinuation in biopsy series · PMID 21849225

  3. 3

    In combination with a checkpoint inhibitor, identify the culprit

    Chemotherapy tends to cause acute tubular injury and the ICI acute tubulointerstitial nephritis; use the clinical, laboratory, and (where needed) biopsy picture to decide which drug to hold — steroids help ICI nephritis but not pemetrexed tubular injury.

    Proposed diagnostic/treatment algorithm distinguishing chemo- from ICI-nephrotoxicity · PMID 32360753

  4. 4

    Respect the renal-function floor before dosing

    Pemetrexed's renal clearance means it is avoided at low creatinine clearance (per label); confirm adequate renal function before each course, and note that folate and B12 supplementation reduce systemic toxicity but do not prevent the renal injury.

    Renal elimination requires adequate function; cumulative renal risk · PMID 32505078

06

What the guidelines say

Society and consensus recommendations addressing this syndrome, each with its key recommendation quoted verbatim.

Every citation on this page is a real, PubMed-verified reference. The teaching case is an illustrative composite, not a real patient. Medical-education content — not medical advice.