Evidence map›Paper›PMID 30420857›Full record

ReviewFrontiers in immunology2018

Pre-emptive Allogeneic Hematopoietic Stem Cell Transplantation in Ataxia Telangiectasia.

Shahrzad Bakhtiar, Sandra Woelke, Sabine Huenecke, Matthias Kieslich, Alexander Malcolm Taylor, Ralf Schubert, Stefan Zielen, Peter Bader

Open access · goldAbstract readCase ReportsReview
In one paragraph

Review in Frontiers in immunology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
23citing papers in PubMed, 3 pooled it
2.0field-weighted citation impact, top 12% 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

23 citing papers in PubMed, 3 syntheses or guidelines pooled it, 37 citations in OpenAlex.

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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 2 institutions in 2 countries.

Shahrzad BakhtiarDivision for Stem Cell Transplantation and Immunology, Children's Hospital, Goethe-Universität Frankfurt am Main, Frankfurt, Germany.
Sandra WoelkeDepartment of Allergology, Pneumology and Cystic Fibrosis, Children's Hospital, Goethe-Universität Frankfurt am Main, Frankfurt, Germany.
Sabine HueneckeDivision for Stem Cell Transplantation and Immunology, Children's Hospital, Goethe-Universität Frankfurt am Main, Frankfurt, Germany.
Matthias KieslichDepartment of Neuropaediatrics, Children's Hospital, Goethe-Universität Frankfurt am Main, Frankfurt, Germany.
Alexander Malcolm TaylorInstitute of Cancer and Genomic Sciences, University of Birmingham, Birmingham, United Kingdom.
Ralf SchubertDepartment of Allergology, Pneumology and Cystic Fibrosis, Children's Hospital, Goethe-Universität Frankfurt am Main, Frankfurt, Germany.
Stefan ZielenDepartment of Allergology, Pneumology and Cystic Fibrosis, Children's Hospital, Goethe-Universität Frankfurt am Main, Frankfurt, Germany.
Peter BaderDivision for Stem Cell Transplantation and Immunology, Children's Hospital, Goethe-Universität Frankfurt am Main, Frankfurt, Germany.
Goethe University Frankfurt · DEUniversity of Birmingham · GB

Funding

Cancer Research UK 14346
6 · The paper itself

Abstract

Ataxia telangiectasia (A-T) is a primary immunodeficiency with mutations in the gene encoding the A-T mutated (ATM) protein that interacts with immune, hematopoietic, and endocrine targets resulting in broad multi-systemic clinical manifestations with a devastating outcome. Apart from a progressive neurodegenerative disorder, A-T leads to significantly increased susceptibility to malignancies. It is a matter of discussion whether pre-emptive allogeneic hematopoietic stem cell transplantation (alloHSCT) using a reduced intensity conditioning regimen would be an option to restore immune-competence and prevent malignancy, as shown in animal models, because conventional treatment protocols of malignant diseases using radio- and/or chemotherapy have a high rate of therapy-related morbidity and mortality in these patients. We present the course of the disease, including immune reconstitution and neurological outcome following pre-emptive alloHSCT in a 4-year-old boy with A-T on a 6 year follow-up. Our manuscript provides a proof-of-concept of alloHSCT as an individual pre-emptive treatment strategy from which some A-T patients might benefit.

Indexed as

Hematopoietic Stem Cell TransplantationAnimalsAtaxia TelangiectasiaAtaxia Telangiectasia Mutated ProteinsChildChild, PreschoolChimerismHematologic NeoplasmsHumansMaleNeurodegenerative DiseasesPrecision MedicineTransplantation ConditioningTransplantation, HomologousTreatment OutcomeAtaxia Telangiectasia Mutated Proteinsataxia telangiectasiacombined immunodeficiencymalignancypre-emptive allogeneic hematopoietic stem cell transplantationprimary immunodeficiency

Identifiers

PMID30420857
PMCPMC6215822
OpenAlexW2898977402

What OpenQuestion holds

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LicenceCC BY
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 OpenQuestion graph.