Evidence map›Paper›PMID 35979111›Full record

ArticleWorld journal of clinical cases2022

Allogeneic stem cell transplantation-A curative treatment for paroxysmal nocturnal hemoglobinuria with PIGT mutation: A case report.

Laurence Schenone, Anne-Béatrice Notarantonio, Véronique Latger-Cannard, Veronique Fremeaux-Bacchi, Marcelo De Carvalho-Bittencourt, Marie-Thérèse Rubio, Marc Muller, Maud D'Aveni

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PubMed Full text at PMC Publisher
In one paragraph

Article in World journal of clinical cases, 2022. 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
0.3field-weighted citation impact, top 46% of its field
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, 6 citations in OpenAlex.

  1. Review
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 at 3 institutions in 1 country.

Laurence SchenoneDepartment of Hematology, CHRU de Nancy, Nancy F-54000, France.
Anne-Béatrice NotarantonioIMoPA, CNRS 7365, University of Lorraine, Nancy F-54000, France.
Véronique Latger-CannardHematology Laboratory, Cytometry Platform, CHRU de Nancy, Nancy F-54000, France.
Veronique Fremeaux-BacchiImmunology Laboratory, Hôpital Européen George Pompidou, Paris 75015, France.
Marcelo De Carvalho-BittencourtImmunology Laboratory, CHRU de Nancy, Nancy F-54000, France.
Marie-Thérèse RubioDepartment of Hematology, CHRU de Nancy, Nancy F-54000, France.
Marc MullerGenetic Laboratory, CHRU de Nancy, Nancy F-54000, France.
Maud D'AveniDepartment of Hematology, CHRU de Nancy, Nancy F-54000, France.
Centre Hospitalier Régional et Universitaire de Nancy · FRCentre National de la Recherche Scientifique · FRHôpital Européen · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPatients with paroxysmal nocturnal hemoglobinuria (PNH) have a clonal population of blood cells deficient in glycosylphosphatidylinositol-anchored (GPI-anchored) proteins, most of the time resulting from a mutation in the X-linked gene PIGA. We report a patient with PNH resulting from a rare biallelic PIGT mutation on chromosome 20. CASE SUMMARY: A 47-year-old man was referred to our hospital for febrile pancytopenia. The patient reported a history of recurrent urticaria and arthralgia and he presented during 3 mo recurrent acute dermo-hypodermitis and aseptic meningitidis. Based on clinical cases published with PIGT-PNH, with clinically typical PNH and autoinflammatory symptoms, we treated our patients with repeated infusions of eculizumab to decrease autoinflammatory symptoms and then we performed an allogeneic stem cell transplantation (allo-SCT) with a mismatched unrelated donor. Our patient experienced no acute Graft

conclusionThis case report suggests that allo-SCT should be considered to cure PIGT-PNH patients.

Indexed as

Allogeneic stem cell transplantationAutoinflammatory symptomsCase reportParoxysmal nocturnal hemoglobinuriaPIGT mutationRecurrent meningitidis

Identifiers

PMID35979111
PMCPMC9258387
OpenAlexW4281634001

What OpenQuestion holds

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LicenceCC BY-NC
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Registered trials

None linked

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.