Evidence map›Paper›PMID 23620576›Full record

Trial reportBlood2013

Patient-derived induced pluripotent stem cells recapitulate hematopoietic abnormalities of juvenile myelomonocytic leukemia.

Shilpa Gandre-Babbe, Prasuna Paluru, Chiaka Aribeana, Stella T Chou, Silvia Bresolin, Lin Lu, Spencer K Sullivan, Sarah K Tasian, Julie Weng, Helene Favre and 4 more

Abstract readClinical Trial
In one paragraph

Trial report in Blood, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 67 papers.

0numbers the graph read from it
0cells of the map it votes in
67citing papers in PubMed
8.4field-weighted citation impact, top 2% 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

67 citing papers in PubMed, 117 citations in OpenAlex.

  1. Trial
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  7. LNK/Haematologica · 2024
    Article
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7 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

14 authors at 6 institutions in 2 countries.

Shilpa Gandre-BabbeDivision of Hematology, Children’s Hospital of Philadelphia, Philadelphia, PA, USA.
Prasuna Paluru
Chiaka Aribeana
Stella T Chou
Silvia Bresolin
Lin Lu
Spencer K Sullivan
Sarah K Tasian
Julie Weng
Helene Favre
John K Choi
Deborah L French
Mignon L Loh
Mitchell J Weiss
Children's Hospital of Philadelphia · USUniversity of California, San Francisco · USSt. Jude Children's Research Hospital · USUCSF Benioff Children's Hospital · USUniversity of Padua · ITUniversity of Pennsylvania · US

Funding

UNIVERSITY OF PENNSYLVANIA CAN CTR SUPPORT GRANTP30CA016520 · NCI · UNIVERSITY OF PENNSYLVANIA · PI Robert H. Vonderheide · 1985 to 2026
$222.3M
Translational InformaticsP30CA082103 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Kate Taylor Shumate · 1999 to 2026
$209.7M
Embryonic Stem Cell-Derived Platelets as Cellular TherapeuticsU01HL099656 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI PONCZ, MORTIMER · 2009 to 2015
$8.6M
Research Training in Childhood CancerT32CA128583 · NCI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI Eric Alejandro Sweet-Cordero · 2007 to 2026
$6.0M
Research BaseP30DK090969 · NIDDK · CHILDREN'S HOSP OF PHILADELPHIA · PI CARROLL, MARTIN · 2010 to 2015
$5.7M
Mentored career development in clinical research in non-malignant hematologyK12HL087064 · NHLBI · UNIVERSITY OF PENNSYLVANIA · PI ABRAMS, CHARLES S. · 2006 to 2016
$3.9M
NICHD Institutional Training for PediatriciansT32HD044331 · NICHD · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI FERRIERO, DONNA M. · 2003 to 2012
$2.5M
Hematopoiesis from Normal and Patient-Derived Induced Pluripotent Stem CellsRC2HL101606 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI WEISS, MITCHELL J · 2009 to 2010
$2.0M
NCI NIH HHS L40 CA142226NCI NIH HHS P30 CA016520NCI NIH HHS P30 CA082103NCI NIH HHS T32 CA128583NHLBI NIH HHS K12 HL087064NHLBI NIH HHS RC2 HL101606NHLBI NIH HHS U01 HL099656NICHD NIH HHS T32 HD044331NIDDK NIH HHS P30 DK090969NIDDK NIH HHS P30DK090969
6 · The paper itself

Abstract

Juvenile myelomonocytic leukemia (JMML) is an aggressive myeloproliferative neoplasm of young children initiated by mutations that deregulate cytokine receptor signaling. Studies of JMML are constrained by limited access to patient tissues. We generated induced pluripotent stem cells (iPSCs) from malignant cells of two JMML patients with somatic heterozygous p.E76K missense mutations in PTPN11, which encodes SHP-2, a nonreceptor tyrosine phosphatase. In vitro differentiation of JMML iPSCs produced myeloid cells with increased proliferative capacity, constitutive activation of granulocyte macrophage colony-stimulating factor (GM-CSF), and enhanced STAT5/ERK phosphorylation, similar to primary JMML cells from patients. Pharmacological inhibition of MEK kinase in iPSC-derived JMML cells reduced their GM-CSF independence, providing rationale for a potential targeted therapy. Our studies offer renewable sources of biologically relevant human cells in which to explore the pathophysiology and treatment of JMML. More generally, we illustrate the utility of iPSCs for in vitro modeling of a human malignancy.

Indexed as

Mutation, MissenseCell DifferentiationCohort StudiesExtracellular Signal-Regulated MAP KinasesFemaleGranulocyte-Macrophage Colony-Stimulating FactorHeterozygoteHumansInduced Pluripotent Stem CellsLeukemia, Myelomonocytic, JuvenileMaleNeoplastic Stem CellsPhosphorylationProtein Tyrosine Phosphatase, Non-Receptor Type 1STAT5 Transcription FactorTumor Cells, CulturedExtracellular Signal-Regulated MAP KinasesGranulocyte-Macrophage Colony-Stimulating FactorProtein Tyrosine Phosphatase, Non-Receptor Type 1PTPN1 protein, humanSTAT5 Transcription Factor

Identifiers

PMID23620576
PMCPMC3682343
OpenAlexW2025762987

What OpenQuestion holds

Textmetadata
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.