Evidence map›Paper›PMID 27156964›Full record

ReviewTransfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis2016

New approaches in the diagnosis, pathophysiology, and treatment of pediatric hematopoietic stem cell transplantation-associated thrombotic microangiopathy.

Sonata Jodele, Christopher E Dandoy, Kasiani C Myers, Javier El-Bietar, Adam Nelson, Gregory Wallace, Benjamin L Laskin

Registry-linked trialOpen access · greenAbstract readReview
In one paragraph

Review in Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03384693 (A Pilot Trial of Using Pre-Transplant Risk Stratification and Prophylactic Defibrotide to Prevent Serious Thrombotic Microangiopathy in High-Risk Hematopoietic Stem Cell Transplant Patients), which is not on this map. Cited by 60 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
60citing papers in PubMed, 1 pooled it
6.8field-weighted citation impact, top 3% 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.

NCT03384693 phase2completednot on this mapstarted 2018, after this paper: background citation

A Pilot Trial of Using Pre-Transplant Risk Stratification and Prophylactic Defibrotide to Prevent Serious Thrombotic Microangiopathy in High-Risk Hematopoietic Stem Cell Transplant Patients

TypeinterventionalSponsorUniversity of California, San FranciscoRan2018 to 2020Enrolled25ConditionsThrombotic MicroangiopathiesArmsDefibrotide
3 · Its place in the literature

Who cites it

60 citing papers in PubMed, 1 synthesis or guideline pooled it, 114 citations in OpenAlex.

  1. Pooled it
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  19. Diagnosis and treatment of thrombotic microangiopathy.International journal of laboratory hematology · 2022
    Review
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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

7 authors at 2 institutions in 1 country.

Sonata JodeleDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH. Electronic address: sonata.jodele@cchmc.org.
Christopher E DandoyDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
Kasiani C MyersDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
Javier El-BietarDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
Adam NelsonDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
Gregory WallaceDivision of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children's Hospital Medical Center, Cincinnati, OH.
Benjamin L LaskinDivision of Nephrology, The Children's Hospital of Philadelphia, Philadelphia, PA.
Cincinnati Children's Hospital Medical Center · USChildren's Hospital of Philadelphia · US

Funding

LCenter for Clinical and Translational Science and TrainingUL1TR001425 · NCATS · UNIVERSITY OF CINCINNATI · PI HEUBI, JAMES E., KISSELA, BRETT M · 2015 to 2024
$37.5M
Pediatric Center--Gene Expression /Developmental ScienceK12HD028827 · NICHD · CINCINNATI CHILDRENS HOSP MED CTR · PI Tina Lee Cheng · 2002 to 2026
$9.6M
NCATS NIH HHS UL1 TR001425NICHD NIH HHS K12 HD028827
6 · The paper itself

Abstract

Hematopoietic stem cell transplantation (HSCT)-associated thrombotic microangiopathy (TA-TMA) is an understudied complication of HSCT that significantly affects transplant-related morbidity and mortality. Over the past several decades, the cause of TA-TMA has remained unknown, limiting treatment options to non-specific therapies adapted from other diseases. Recent prospective studies dedicated to the study of TA-TMA have provided new insights into the pathogenesis of, and genetic susceptibility to TA-TMA, raising awareness of this important transplant complication and allowing for the identification of potentially novel therapeutic targets. Specifically, many patients with TA-TMA develop multi-organ tissue injury through endothelial damage mediated by the activation of the complement pathway, leading to rational therapeutic strategies including complement blockade. This new knowledge has the potential to favorably influence clinical practice and change the standard of care for how patients with TA-TMA are managed. In this review, we summarize novel approaches to the recognition and management of TA-TMA, using case examples to illustrate key clinical points that hopefully lead to improved short and long-term outcomes for these complex HSCT patients, who remain at significant risk for treatment-related morbidity and mortality.

Indexed as

Thrombotic MicroangiopathiesAdolescentAllograftsChildChild, PreschoolFemaleHematopoietic Stem Cell TransplantationHumansInfantMaleComplementEculizumabHematopoietic stem cell transplantTA-TMAThrombotic microangiopathy

Identifiers

PMID27156964
PMCPMC5710737
OpenAlexW2343260633

What OpenQuestion holds

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LicenceTDM
Read underepoch 390

Registered trials

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.