Evidence map›Paper›PMID 39289521›Full record

ArticleBone marrow transplantation2024

Outcomes of patients undergoing allogeneic haematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia; a study on behalf of the PDWP of the EBMT.

Clémence Aldebert, Mony Fahd, Jacques-Emmanuel Galimard, Ibrahim A Ghemlas, Marco Zecca, Juliana Silva, Alexander Mohseny, Alphan Kupesiz, Rose-Marie Hamladji, Nuno Miranda and 20 more

Abstract read
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In one paragraph

Article in Bone marrow transplantation, 2024. 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

30 authors.

Clémence AldebertPaediatric Haematology and Immunology Department, Robert-Debré Hospital, GHU APHP Nord-Université de Paris, Paris, France. aldebert.cl@gmail.com.ORCID http://orcid.org/0009-0002-6265-5881
Mony FahdPaediatric Haematology and Immunology Department, Robert-Debré Hospital, GHU APHP Nord-Université de Paris, Paris, France.
Jacques-Emmanuel GalimardEBMT Statistical Unit, Sorbonne University, Saint-Antoine Hospital, AP-HP, INSERM UMRs 938, Paris, France.ORCID http://orcid.org/0000-0001-9102-4427
Ibrahim A GhemlasKing Faisal Specialist Hospital & Research Centre (Paeds), Riyadh, Saudi Arabia.
Marco ZeccaFondazione IRCCS Policlinico San Matteo Paediatric Haematology-Oncology, Pavia, Italy.ORCID http://orcid.org/0000-0002-8818-1744
Juliana SilvaGreat Ormond Street Hospital-Bone Marrow Transplant Department, London, UK.
Alexander MohsenyWillem-Alexander Children's Hospital, Division of Stem Cell Transplantation, Leiden University Medical Center, Leiden, The Netherlands.ORCID http://orcid.org/0000-0001-7857-4256
Alphan KupesizPaediatric Haematology, Akdeniz University Medical School, Antalya, Turkey.ORCID http://orcid.org/0000-0001-8827-5567
Rose-Marie HamladjiService Hématologie Greffe de Moëlle, Centre Pierre Et Marie Curie, Algiers, Algeria.
Nuno MirandaBMT, Instituto Portugues de Oncologia, Lisbon, Portugal.ORCID http://orcid.org/0000-0001-5175-9947
Tayfun GüngörDivision of Stem Cell Transplantation, The Hospital of the Eleonore Foundation, University Children's Hospital, Zürich, Switzerland.ORCID http://orcid.org/0000-0002-3261-1186
Robert F WynnDepartment of Paediatric Haematology, Bone Marrow Unit, Royal Manchester Children's Hospital, Manchester, UK.ORCID http://orcid.org/0000-0002-6046-8428
Pietro MerliDepartment of Paediatric Haematology and Oncology, IRRCS Ospedale Pediatrico Bambino Gesù, Rome, Italy.ORCID http://orcid.org/0000-0001-6426-4046
Mikael SundinDivision Paediatric Haematology, Immunology and HCT, Astrid Lindgren Children's Hospital, Karolinska University, Stockholm, Sweden.ORCID http://orcid.org/0000-0002-9871-0961
Maura FaraciDepartment of Haematology and Oncology, HSCT Unit, IRCCS Institute G. Gaslini, Genoa, Italy.ORCID http://orcid.org/0000-0001-5715-501X
Cristina Diaz-de-HerediaDepartment of Paediatric Haematology and Oncology, Hospital Universitari Vall d'Hebron, Barcelona, Spain.ORCID http://orcid.org/0000-0001-8086-296X
Birgit BurkhardtPaediatric Haematology Oncology and BMT, University Hospital Münster, Münster, Germany.ORCID http://orcid.org/0000-0002-1151-829X
Victoria BordonDepartment of Paediatric Haematology-Oncology and Stem Cell Transplantation, Ghent University Hospital, Ghent, Belgium.ORCID http://orcid.org/0000-0001-8446-0220
Marie AngosoDepartment of Paediatric Haematology/Oncology, CHU Bordeaux, Bordeaux, France.
Peter BaderDepartment for Children and Adolescents, Division for Stem Cell Transplantation, Immunology and Intensive Care Medicine, Goethe University, University Hospital Frankfurt, Frankfurt, Germany.
Marianne IfversenDepartment of Paediatric and Adolescent Medicine, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark.ORCID http://orcid.org/0000-0002-2558-1935
Concepcion Herrera ArroyoDepartment of Haematology, Reina Sofia University Hospital, IMIBIC, University of Cordoba, Cordoba, Spain.ORCID http://orcid.org/0000-0003-0055-2270
Natalia MaximovaBone Marrow Transplant Centre, Institute for Maternal and Child Health - IRCCS Burlo Garofolo, Trieste, Italy.ORCID http://orcid.org/0000-0003-0934-1875
Susana RiescoDepartment of Paediatrics, Hospital Universitario de Salamanca - IBSAL, Salamanca, Spain.
Jerry SteinDepartment of Haemato-Oncology, Schneider Children's Medical Center of Israel, Petach Tikva, Israel.
Arnaud DalissierEBMT Paris Study Unit, Sorbonne University, Saint-Antoine Hospital, AP-HP, INSERM UMRs 938, Paris, France.
Franco LocatelliDepartment of Paediatric Haematology and Oncology, IRRCS Ospedale Pediatrico Bambino Gesù, Rome, Catholic University of the Sacred Heart, Rome, Italy.ORCID http://orcid.org/0000-0002-7976-3654
Krzysztof KalwakDepartment of Paediatric Haematology Oncology and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland.ORCID http://orcid.org/0000-0003-1174-5799
Jean-Hugues DallePaediatric Haematology and Immunology Department, Robert-Debré Hospital, GHU APHP Nord-Université de Paris, Paris, France.
Selim CorbaciogluDepartment of Paediatric Haematology, Oncology and Stem Cell Transplantation, University of Regensburg, Regensburg, Germany.ORCID http://orcid.org/0000-0003-1070-8486

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Congenital amegakaryocytic thrombocytopenia is a rare, inherited bone marrow failure syndrome. Allogeneic haematopoietic stem cell transplantation (allo-HSCT) is currently the only curative treatment. In this retrospective study, we analysed 66 patients with allo-HSCT, reported in the European Society for Blood and Marrow Transplantation (EBMT) registry. Bone marrow (BM) was the most widely used stem cell source (n = 40; 61%) followed by peripheral blood (PB) (n = 18; 27%), and unrelated umbilical cord blood (UCB) (n = 8; 12%). Most frequently was a HLA-matched graft from related (n = 26; 39%) and unrelated (n = 15; 23%) donors after a myeloablative busulfan-based conditioning regimen. GvHD prophylaxis was mostly cyclosporine and methotrexate (53%). The 6-year cumulative incidence of graft-failure and second transplant were 25% and 17%, respectively. The 6-year disease-free survival (DFS) and overall survival (OS) were 66.9% and 85.6%, respectively. The 6-year transplant-related mortality (TRM) was 8.0%. In conclusion, most patients with CAMT benefit from allo-HSCT, but with many graft failures.

Indexed as

Congenital Bone Marrow Failure SyndromesHematopoietic Stem Cell TransplantationAdolescentAdultAllograftsChildChild, PreschoolDisease-Free SurvivalFemaleHumansInfantMaleMiddle AgedRegistriesRetrospective StudiesSurvival Rate

Identifiers

PMID39289521

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