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78/100

✓ Peer-reviewed alzhub usefulness score 78 out of 100. Treatments

Topic: Amyloid, tau and blood-test diagnosis

Elevated brain iron observed in AD has been associated with accelerated cognitive decline and may be a tractable drug target. Results Of 167 patients screened for eligibility, 81 were included, with 53 randomly assigned to the deferiprone group (mean [SD] age, 73.0 [8.0] years; 29 male [54.7%]) and 28 to the placebo group (mean [SD] age, 71.6 [7.2] years; 17 male [60.7%]); 54 participants completed the study (7 [25.0%] withdrew from the placebo group and 20 [37.7%] from the deferiprone group). In an intention-to-treat analysis, participants in the deferiprone group showed accelerated cognitive decline on the NTB primary outcome (β for interaction = -0.50; 95% CI, -0.80 to -0.20) compared with placebo (change in NTB composite z score for deferiprone, -0.80 [95% CI, -0.98 to -0.62]; for placebo, -0.30 [95% CI, -0.54 to -0.06]). Secondary analysis revealed that this result was driven by worsening performance on executive function tests. The QSM confirmed that deferiprone decreased iron in the hippocampus compared with placebo (change in hippocampal QSM for deferiprone, -0.36 ppb [95% CI, -0.76 to 0.04 ppb]; for placebo, 0.32 ppb [95% CI, -0.12 to 0.75 ppb]; β for interaction = -0.68 [95% CI, -1.27 to -0.09]). Longitudinal hippocampal volume loss was not affected by deferiprone, but exploratory analysis of other brain regions revealed increased volume loss with deferiprone in frontal areas. Conclusions These trial findings show that deferiprone 15 mg/kg twice a day decreased hippocampal QSM and accelerated cognitive decline in patients with amyloid-confirmed early AD, suggesting that lowering iron with deferiprone is detrimental to patients with AD.

Cite this study APA, Vancouver, BibTeX · download .bib or .ris

APA 7th

Ayton, S., Barton, D., Brew, B., Brodtmann, A., Clarnette, R., Desmond, P., et al. (2025). Deferiprone in Alzheimer Disease: A Randomized Clinical Trial. JAMA neurology. https://doi.org/10.1001/jamaneurol.2024.3733

Vancouver

Ayton S, Barton D, Brew B, Brodtmann A, Clarnette R, Desmond P, et al. Deferiprone in Alzheimer Disease: A Randomized Clinical Trial. JAMA neurology. 2025. doi:10.1001/jamaneurol.2024.3733. PMID: 39495531.

BibTeX

@article{ayton2025-39495531,
  author  = {Ayton, S. and Barton, D. and Brew, B. and Brodtmann, A. and Clarnette, R. and Desmond, P. and Devos, D. and Ellis, K. A. and others},
  title   = {{Deferiprone in Alzheimer Disease: A Randomized Clinical Trial}},
  journal = {JAMA neurology},
  year    = {2025},
  doi     = {10.1001/jamaneurol.2024.3733},
  pmid    = {39495531},
  url     = {https://doi.org/10.1001/jamaneurol.2024.3733}
}

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Key findings

Elevated brain iron observed in AD has been associated with accelerated cognitive decline and may be a tractable drug target.
Results Of 167 patients screened for eligibility, 81 were included, with 53 randomly assigned to the deferiprone group (mean [SD] age, 73.0 [8.0] years; 29 male [54.7%]) and 28 to the placebo group (mean [SD] age, 71.6 [7.2] years; 17 male [60.7%]); 54 participants completed the study (7 [25.0%] withdrew from the placebo group and 20 [37.7%] from the deferiprone group).
In an intention-to-treat analysis, participants in the deferiprone group showed accelerated cognitive decline on the NTB primary outcome (β for interaction = -0.50; 95% CI, -0.80 to -0.20) compared with placebo (change in NTB composite z score for deferiprone, -0.80 [95% CI, -0.98 to -0.62]; for placebo, -0.30 [95% CI, -0.54 to -0.06]).
Secondary analysis revealed that this result was driven by worsening performance on executive function tests.
The QSM confirmed that deferiprone decreased iron in the hippocampus compared with placebo (change in hippocampal QSM for deferiprone, -0.36 ppb [95% CI, -0.76 to 0.04 ppb]; for placebo, 0.32 ppb [95% CI, -0.12 to 0.75 ppb]; β for interaction = -0.68 [95% CI, -1.27 to -0.09]).

Why it matters

In this trial of 81 participants with Alzheimer disease, deferiprone accelerated cognitive decline on the primary outcome compared with placebo, driven by worsening performance on executive function tests, even though it decreased iron in the hippocampus. Hippocampal volume loss was not affected, and exploratory analysis found increased volume loss in some other brain regions with deferiprone.

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Study details

Study designlongitudinal study
Participants167
Effect size54.7%; 60.7%; 25.0%
Evidence strengthHigh

How this study scored 78/100

Published in a leading specialty journal, and it is a randomised controlled trial.

Component	Points	Why
Journal tier	9 / 10	Tier 2 — leading specialty journal (JAMA neurology)
Journal's citation record	2 / 4	JAMA Neurology: journal h-index 235 (OpenAlex, 2026-10-29)
Study design	11 / 12	A randomised controlled trial (from PubMed type “Randomized Controlled Trial”)
Strength of evidence	5 / 5	Rated “high” in our summary
Peer-review validation	10 / 10	Peer-reviewed. Published as an article in a named peer-reviewed journal.
How recent	4 / 5	2025 — within 3 year(s) of the newest study here
Sample size	2 / 4	167 participants
Effect reported	2 / 3	Effect reported as a number, without an interval or test
Studied in people	3 / 3	Studied in people (participant count stated)
Randomised / blinded	1 / 2	Randomised (as stated in the record)
Times cited	4 / 7	Cited 92 times (OpenAlex, 2026-10-10)
Citations per year	4 / 5	30.7 citations a year over 3 year(s) since 2025
Impact for its field and year	5 / 6	Cited 22.0× the average for papers of its field and year (OpenAlex FWCI)
Cited by studies here	2 / 6	Cited by 5 other studies in this library
Still being cited	3 / 4	Cited 92 times in 2024–2026 (2026 still in progress; OpenAlex)
Senior author's track record	4 / 4	Last-listed author Ashley Ian Bush: h-index 140 (OpenAlex, whole career)
Institution's track record	2 / 3	University College London: institution h-index 2,029 (OpenAlex)
Multi-centre collaboration	3 / 3	OpenAlex lists 23 institution(s) in 4 countries
Indexed in PubMed	2 / 2	Indexed in PubMed (PMID 39495531)
Free to read	0 / 2	Behind a paywall — scores zero
Total	78 / 100	—

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