Evidence map›Paper›PMID 39159950›Full record

ArticleBritish journal of haematology2026

Long-term outcome after allogeneic stem cell transplantation for GATA2 deficiency: An analysis of 67 adults and children from France and Belgium.

Flore Sicre de Fontbrune, Florian Chevillon, Mony Fahd, Kristell Desseaux, Xavier Poiré, Edouard Forcade, Arthur Sterin, Bénédicte Neven, Virginie Gandemer, Sylvain Thepot and 11 more

Abstract readMulticenter Study
In one paragraph

Article in British journal of haematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing 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

6 citing papers in PubMed.

  1. Article
  2. Fungal Infections in Disorders of Inborn Errors of Immunity.Clinical reviews in allergy & immunology · 2026
    Review
  3. Article
  4. Article
  5. Insights into PediatricBiomedicines · 2025
    Review
  6. 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

21 authors.

Flore Sicre de FontbruneHématologie Greffe, Hôpital Saint-Louis, APHP, Paris, France.ORCID https://orcid.org/0000-0003-2000-1556
Florian ChevillonAJA, Hôpital Saint Louis, APHP, Paris, France.
Mony FahdService d'Hématologie Pédiatrique, Hôpital Robert Debré, APHP, Paris, France.
Kristell DesseauxDBIM, Hôpital Saint Louis, APHP, Paris, France.
Xavier PoiréAdult Hematology, Clinique Universitaire Saint Luc, Bruxelles, Belgium.
Edouard ForcadeHématologie Adulte, Centre Hospitalier Universitaire de Bordeaux, Hôpital Haut-Lévêque, Pessac, France.ORCID https://orcid.org/0000-0002-8873-2868
Arthur SterinService d'Hématologie Pédiatrique, Hôpital de la Timone, Marseille, France.
Bénédicte NevenService d'Immuno-Hématologie et Rhumatologie Pédiatrique, Hôpital Necker-Enfants Malades, Laboratoire INSERM U768, Institut Imagine, Paris, France.
Virginie GandemerService d'Hematologie Oncologie Pediatrique, Hopital Sud, CHU de Rennes, Rennes, France.
Sylvain ThepotService d'Hématologie Adulte, CHU d'Angers, Angers, France.
Alice GarnierService d'Hématologie, CHU de Nantes, Nantes, France.
Bruno LioureService d'Hématologie, Hôpital Hautepierre, Hôpitaux Universitaire de Strasbourg, Strasbourg, France.
Ambroise MarcaisService d'Hématologie Adulte, Hôpital Necker Enfants Malades, Paris, France.ORCID https://orcid.org/0000-0002-6844-7920
Stephanie Nguyen-QuocService d'Hématologie, Hôpital de la Pitié Salpetrière, APHP, Paris, France.
Suzanne TavitianService d'Hématologie Adulte, CHU Purpan, Toulouse, France.
Laure VincentDepartement of Hematology, CHU de Montpellier, Montpellier, France.
Jean DonadieuService d'Hématologie et Oncologie pédiatrique, APHP Trousseau, Paris, France.
Matthieu Resche RiggonDBIM, Hôpital Saint Louis, APHP, Paris, France.
Sylvie ChevretDBIM, Hôpital Saint Louis, APHP, Paris, France.
Marlene PasquetService d'Hématologie Immunologie et Oncologie Pédiatrique, CHU Purpan, Toulouse, France.
Regis Peffault de LatourHématologie Greffe, Hôpital Saint-Louis, APHP, Paris, France.ORCID https://orcid.org/0000-0001-6222-4753

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Modalities and timing of haematopoietic stem cell transplant (HSCT) in patients with GATA2 deficiency are still subject to debate. On June 2022, 67 patients (median age 20.6 years) underwent a first allogeneic HSCT among 21 centres. Indications for HSCT were myelodysplastic syndrome (MDS) ≤5% blasts ± immunodeficiency (66%), MDS >5% blasts (15%), acute myeloid leukaemia (19%). Conditioning regimen was myeloablative in 85% and anti-thymocyte globulins were used in 67%. The cumulative incidence (CInc) of acute graft versus host disease (GvHD) grade II-IV and III-IV at day 100 were 42% and 13%, and CInc of chronic and extensive chronic GvHD at 2 years were 42% and 23%. CInc of relapses was 3% and 11% at 1 and 5 years. Overall survival (OS) at 1 and 5 years was 83% and 72% (median follow-up 5.6 years). The factors associated with worse OS in multivariable analysis were the year of HSCT, a history of excess blasts before transplant and peripheral blood stem cell (PBSC) grafts. Age at HSCT, non-myeloablative conditioning and PBSC grafts were associated with increased non-relapse mortality. In conclusion, bone marrow monitoring to identify clonal evolution and perform HSCT before the appearance of excess blast is mandatory.

Indexed as

GATA2 DeficiencyHematopoietic Stem Cell TransplantationAdolescentAdultAllograftsBelgiumChildChild, PreschoolFemaleFranceGraft vs Host DiseaseHumansInfantMaleMiddle AgedRetrospective Studiesallogeneicexcess blastsGATA2myeloid malignanciesrelapse

Identifiers

PMID39159950
PMCPMC12819089

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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 Socratic graph.