Evidence map›Paper›PMID 32765834›Full record

ReviewF1000Research2020

Drug Therapies for the Management of Sickle Cell Disease.

Parul Rai, Kenneth I Ataga

Open access · goldAbstract readReview
In one paragraph

Review in F1000Research, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed, 1 pooled it
3.7field-weighted citation impact, top 6% 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

22 citing papers in PubMed, 1 synthesis or guideline pooled it, 41 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
  4. Article
  5. Article
  6. Article
  7. Article
  8. Using disease-modifying therapies in sickle cell disease.Hematology. American Society of Hematology. Education Program · 2023
    Article
  9. Review
  10. Review
  11. Article
  12. Article
  13. Treatment of sickle cell disease - options and perspective.American journal of blood research · 2023
    Review
  14. Review
  15. Article
  16. Review
  17. Review
  18. Review
  19. Article
  20. Review
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

2 authors at 2 institutions in 1 country.

Parul RaiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0002-5770-7280
Kenneth I AtagaCenter for Sickle Cell Disease, University of Tennessee Health Science Center, Memphis, TN, USA.ORCID 0000-0002-4501-9982
St. Jude Children's Research Hospital · USUniversity of Tennessee Health Science Center · US

Funding

THE ASSOCIATION OF BIOMARKERS OF ENDOTHELIAL FUNCTION WITH PROSPECTIVE CHANGES IN KIDNEY FUNCTION IN SICKLE CELL ANEMIAR01FD006030 · FDA · UNIV OF NORTH CAROLINA CHAPEL HILL · PI ATAGA, KENNETH I · 2017 to 2021
$2.0M
FDA HHS R01 FD006030
6 · The paper itself

Abstract

Sickle cell disease (SCD) afflicts millions of people worldwide but is referred to as an orphan disease in the United States. Over the past several decades, there has been an increasing understanding of the pathophysiology of SCD and its complications. While most individuals with SCD in resource-rich countries survive into adulthood, the life expectancy of patients with SCD remains substantially shorter than for the general African-American population. SCD can be cured using hematopoietic stem cell transplantation and possibly gene therapy, but these treatment approaches are not available to most patients, the majority of whom reside in low- and middle-income countries. Until relatively recently, only one drug, hydroxyurea, was approved by the US Food and Drug Administration to ameliorate disease severity. Multiple other drugs (L-glutamine, crizanlizumab, and voxelotor) have recently been approved for the treatment of SCD, with several others at various stages of clinical testing. The availability of multiple agents to treat SCD raises questions related to the choice of appropriate drug therapy, combination of multiple agents, and affordability of recently approved products. The enthusiasm for new drug development provides opportunities to involve patients in low- and middle-income nations in the testing of potentially disease-modifying therapies and has the potential to contribute to capacity building in these environments. Demonstration that these agents, alone or in combination, can prevent or decrease end-organ damage would provide additional evidence for the role of drug therapies in improving outcomes in SCD.

Indexed as

Anemia, Sickle CellGenetic TherapyHematopoietic Stem Cell TransplantationHumansHydroxyureaUnited StatesUnited States Food and Drug AdministrationHydroxyureaClinical TrialsDrug DevelopmentNovel DrugsSickle cell diseaseTreatment

Identifiers

PMID32765834
PMCPMC7388199
OpenAlexW3035330065

What OpenQuestion holds

Textmetadata
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.