Evidence map›Paper›PMID 39345003›Full record

ArticleHeadache2025

Efficacy of galcanezumab in proline-rich transmembrane protein 2 (PRRT2)-associated familial hemiplegic migraine: A case series.

Costanza Sottani, Giulia Di Lazzaro, Paolo Calabresi, Maria Grazia Pomponi, Francesco Danilo Tiziano, Anna Rita Bentivoglio, Serenella Servidei, Catello Vollono

Abstract readCase Reports
In one paragraph

Article in Headache, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed, 1 pooled it
–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

3 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. 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

8 authors.

Costanza SottaniNeurology Section, Department of Neuroscience, Università Cattolica del Sacro Cuore, Rome, Italy.
Giulia Di LazzaroNeurology Section, Department of Neuroscience, Università Cattolica del Sacro Cuore, Rome, Italy.
Paolo CalabresiNeurology Section, Department of Neuroscience, Università Cattolica del Sacro Cuore, Rome, Italy.
Maria Grazia PomponiUOC Genetica Medica, Fondazione Policlinico Universitario "A. Gemelli" IRCCS, Rome, Italy.
Francesco Danilo TizianoUOC Genetica Medica, Fondazione Policlinico Universitario "A. Gemelli" IRCCS, Rome, Italy.
Anna Rita BentivoglioNeurology Section, Department of Neuroscience, Università Cattolica del Sacro Cuore, Rome, Italy.
Serenella ServideiNeurology Section, Department of Neuroscience, Università Cattolica del Sacro Cuore, Rome, Italy.
Catello VollonoNeurology Section, Department of Neuroscience, Università Cattolica del Sacro Cuore, Rome, Italy.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundFamilial hemiplegic migraine (FHM) is a rare subtype of migraine with aura. Variants in calcium voltage-gated channel subunit alpha1 A (CACNA1A), ATPase Na+/K+ transporting subunit alpha 2 (ATP1A2), and sodium voltage-gated channel alpha subunit 1 (SCN1A) genes have a well-established association with the development of FHM. Recent studies suggest that other genes may also have a significant role in the pathogenesis of FHM, including proline-rich transmembrane protein 2 (PRRT2). To our knowledge, there are currently no documented reports of the use of monoclonal antibodies targeting calcitonin gene-related peptide in FHM caused by a specific identified genetic mutation - and in particular not in FHM associated with PRRT2 mutations. The aim of our work is to describe the efficacy of galcanezumab as a prophylaxis treatment on patients from an Italian family consisting of six patient carriers of a PRRT2 pathogenic variant.

methodsInclusion criteria for treatment eligibility consisted of a confirmed diagnosis of genetically confirmed FHM as defined by the International Classification of Headache Disorders, third edition, number of headache days/month ≥4, and at least two previously failed migraine prophylaxis treatments. We evaluated clinical data of patients treated with galcanezumab regarding number of headache days/month, frequency of aura, disability caused by HM using the Migraine Disability Assessment (MIDAS), attack severity through a numerical rating scale (NRS), acute medications intake, and response to acute medications at baseline (t0) and after 3 (t1) and 6 (t2) months of treatment.

resultsThree out of six family members met inclusion criteria for treatment with galcanezumab. The average number of headache days/month, acute medications, and MIDAS significantly decreased in all treated patients, as well as the average NRS score. Aura frequency reduced by ≥50% compared to the baseline in all three patients. No adverse events related to galcanezumab were reported.

conclusionGalcanezumab is a valid and well-tolerated treatment option in PRRT2-associated FHM.

Indexed as

Antibodies, Monoclonal, HumanizedMembrane ProteinsMigraine with AuraNerve Tissue ProteinsFemaleHumansMiddle AgedTreatment OutcomeYoung AdultAntibodies, Monoclonal, HumanizedgalcanezumabMembrane ProteinsNerve Tissue ProteinsPRRT2 protein, humancalcitonin gene‐related peptidegalcanezumabhemiplegic familial migraineproline‐rich transmembrane protein 2

Identifiers

PMID39345003
PMCPMC11794975

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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.