Evidence map›Paper›PMID 41823567›Full record

ArticleJournal of inherited metabolic disease2026

Acute Neurological Complications After Transplantation in Methylmalonic Acidemia: A 35-Patient French Cohort.

Adélaïde Vissac, Eugénie Sarda, Charles-Joris Roux, Manuel Schiff, Claire-Marine Bérat, Juliette Bouchereau, Jean-Baptiste Arnoux, Chloé de Puyraimond, Margaux Gaschignard, Tristan Mekdade and 16 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of inherited metabolic disease, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

26 authors.

Adélaïde VissacReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Eugénie SardaPediatric Neurology Unit, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Charles-Joris RouxDepartment of Radiology, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Manuel SchiffReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.ORCID https://orcid.org/0000-0001-8272-232X
Claire-Marine BératReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Juliette BouchereauReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Jean-Baptiste ArnouxReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Chloé de PuyraimondReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Margaux GaschignardReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Tristan MekdadeReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Ugo CucinottaPediatric Hepatology and Gastroenterology-Nutrition Units, Reference Center for Rare Pediatric Liver Diseases, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Claire MayerPediatric Hepatology and Gastroenterology-Nutrition Units, Reference Center for Rare Pediatric Liver Diseases, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Valérie BarbierReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Jean François BenoistReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Mehdi OualhaPediatric Intensive Care Unit, Necker-Enfants Malades Hospital, Paris Cité University, Paris, France.
Carmen CapitoDepartment of Pediatric Liver Surgery, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Christophe ChardotDepartment of Pediatric Liver Surgery, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.ORCID https://orcid.org/0000-0002-3248-5650
Myriam DaoUnit of Adult Nephrology, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Aude ServaisUnit of Adult Nephrology, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Claire FrancozUnit of Adult Hepatology and Transplantation, Beaujon Hospital, APHP, Paris Cité University, Paris, France.ORCID https://orcid.org/0000-0001-7391-8507
Laurène DehouxReference Center of Pediatric Nephrology, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Florence LacaillePediatric Hepatology and Gastroenterology-Nutrition Units, Reference Center for Rare Pediatric Liver Diseases, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Isabelle DesguerrePediatric Neurology Unit, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Nathalie BoddaertDepartment of Radiology, Necker-Enfants Malades Hospital, APHP, Paris Cité University, Paris, France.
Pascale de LonlayReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.
Anaïs BrassierReference Center for Inborn Error of Metabolism, Hôpital Necker-Enfants Malades, APHP and Université Paris Cité, Filière G2m, MetabERN, Paris, France.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Transplantation is an effective therapeutic option to improve quality of life in patients with severe methylmalonic acidemia (MMA). However, data regarding neurological complications following transplantation remain limited. A retrospective, single-center study was conducted at Necker Hospital (France), including MMA patients who underwent kidney (KT), liver (LT), or liver-kidney transplantation (LKT) between 2007 and 2022. Our aim was to evaluate acute neurological complications occurring after transplantation, focusing on clinical features, laboratory parameters and neuroimaging. Sensorineural complications were also assessed. Thirty-five patients were included, 21 had undergone LKT, 10 LT and 4 KT. The median age at transplantation was 10.1 years, with a median follow-up of 5 years. Tacrolimus was used in 91% of patients. Acute neurological complications likely related to MMA occurred in 17% of patients. They include Leigh syndrome with identifiable triggers, observed in 4 patients (2 early and 2 late-post-transplantation), leading to one early- and one late-onset death. Stroke-like episodes occurred in 2 patients. Non-epileptic myoclonus, likely related to calcineurin inhibitor (CNI), were reported in 31% of patients. Pre-transplant brain MRI showed nonspecific abnormalities in 31% of patients and remained stable afterwards. Post-transplant ophthalmological data available for 17 patients showed 3 optic atrophies. No acute vision or hearing loss was reported. Although transplantation improves metabolic control in MMA, acute neurological complications can still occur following a triggering event, even years after transplantation. The risk may arise from sensitivity to CNI-induced neurotoxicity. Pre- and post-transplant neuroimaging, emergency metabolic protocols, and tailored immunosuppression are essential for long-term management.

Indexed as

Amino Acid Metabolism, Inborn ErrorsKidney TransplantationLiver TransplantationNervous System DiseasesAdolescentAdultChildChild, PreschoolFemaleFranceHumansImmunosuppressive AgentsInfantMaleQuality of LifeRetrospective StudiesImmunosuppressive Agentscombined liver kidney transplantationimmunosuppressive treatmentLeigh syndromeliver transplantationmethylmalonic acidemiastroke‐like episodes

Identifiers

PMID41823567
PMCPMC12984472

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.