Evidence map›Paper›PMID 33015364›Full record

ArticleExpert review of precision medicine and drug development2019

Sickle cell disease in the era of precision medicine: looking to the future.

Martin H Steinberg, Sara Kumar, George J Murphy, Kim Vanuytsel

Abstract read
In one paragraph

Article in Expert review of precision medicine and drug development, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
5citing papers in PubMed, 1 pooled it
0.3field-weighted citation impact, top 36% 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

5 citing papers in PubMed, 1 synthesis or guideline pooled it, 10 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
  4. Microfluidic methods to advance mechanistic understanding and translational research in sickle cell disease.Translational research : the journal of laboratory and clinical medicine · 2022
    Review
  5. 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

4 authors at 1 institution in 1 country.

Martin H SteinbergDepartment of Medicine, Division of Hematology/Oncology, Center of Excellence for Sickle Cell Disease and Center for Regenerative Medicine, Boston University School of Medicine and Boston Medical Center, Boston MA.
Sara KumarDepartment of Medicine, Division of Hematology/Oncology, Center of Excellence for Sickle Cell Disease and Center for Regenerative Medicine, Boston University School of Medicine and Boston Medical Center, Boston MA.
George J MurphyDepartment of Medicine, Division of Hematology/Oncology, Center of Excellence for Sickle Cell Disease and Center for Regenerative Medicine, Boston University School of Medicine and Boston Medical Center, Boston MA.
Kim VanuytselDepartment of Medicine, Division of Hematology/Oncology, Center of Excellence for Sickle Cell Disease and Center for Regenerative Medicine, Boston University School of Medicine and Boston Medical Center, Boston MA.
Boston University · US

Funding

Globin Gene Expression in Sickle Cell Genotype-Specific iPS cellsU01HL107443 · NHLBI · BOSTON UNIVERSITY MEDICAL CAMPUS · PI CHUI, DAVID H K, MOSTOSLAVSKY, GUSTAVO · 2011 to 2015
$8.3M
Genetic Modulation of Sickle Cell DiseaseR01HL068970 · NHLBI · BOSTON UNIVERSITY MEDICAL CAMPUS · PI STEINBERG, MARTIN H. · 2001 to 2010
$7.0M
Genome-Wide Association Studies in Sickle Cell Anemia and in CentenariansR01HL087681 · NHLBI · BOSTON UNIVERSITY MEDICAL CAMPUS · PI STEINBERG, MARTIN H. · 2007 to 2010
$6.0M
Determinants of HbF Response to HydroxyureaR01HL070735 · NHLBI · BOSTON UNIVERSITY MEDICAL CAMPUS · PI STEINBERG, MARTIN H. · 2002 to 2005
$2.9M
Mechanisms of cis-acting HbF regulation in sickle cell anemiaR01HL133350 · NHLBI · BOSTON MEDICAL CENTER · PI MURPHY, GEORGE J, STEINBERG, MARTIN H. · 2017 to 2020
$1.7M
NHLBI NIH HHS R01 HL068970NHLBI NIH HHS R01 HL070735NHLBI NIH HHS R01 HL087681NHLBI NIH HHS R01 HL133350NHLBI NIH HHS U01 HL107443
6 · The paper itself

Abstract

introductionSickle cell anemia is a mendelian disease that is noted for the heterogeneity of its clinical expression. Because of this, providing an accurate prognosis has been a longtime quest. AREAS COVERED: Reviewed are the benefits and shortcomings of testing for the major modulators of the severity of disease, like fetal hemoglobin and α thalassemia, along with studies that have attempted to link genetic variation with sub-phenotypes of disease in a predictive fashion. Induced pluripotent stem cells driven to differentiate into erythroid precursor cells provide another area for potential patient-specific drug testing. EXPERT OPINION: Fetal hemoglobin is the strongest modulator of sickle cell anemia but simply measuring its blood levels is an insufficient means of forecasting an individual's prognosis. A more precise method would be to know the distribution of fetal hemoglobin levels across the population of red cells, an assay not yet available. Prognostic measures have been developed using genetic and other signatures, but their predictive value is suboptimal. Widely applicable assays must be developed to allow a tailored approach to using the several new treatments that are likely to be available in the near future.

Indexed as

Fetal hemoglobingenetic signaturesGWAShemolysisiPSCsSNPsvasoocclusion

Identifiers

PMID33015364
PMCPMC7531762
OpenAlexW2989394984

What OpenQuestion holds

Textmetadata
LicenceTDM
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.