Evidence map›Paper›PMID 38786070›Full record

ReviewCells2024

Current and Future Therapeutics for Treating Patients with Sickle Cell Disease.

Mariam Barak, Christopher Hu, Alicia Matthews, Yolanda M Fortenberry

Registry-linked trialAbstract readReview
In one paragraph

Review in Cells, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06989567 (A Phase III, Multi-Center, Double-Blind, Placebo-Controlled, Randomized Study of Intravenous L-Citrulline in Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis in Children, Adolescents and Young Adults), which is not on this map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
–field-weighted citation impact
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.

NCT06989567 phase3not yet recruitingnot on this mapstarted 2025, after this paper: background citation

A Phase III, Multi-Center, Double-Blind, Placebo-Controlled, Randomized Study of Intravenous L-Citrulline in Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis in Children, Adolescents and Young Adults (6 to 21 Years)

TypeinterventionalSponsorAsklepion Pharmaceuticals, LLCRan2025 to 2029Enrolled120ConditionsSickle Cell DiseaseArmsL-citrulline, Placebo
3 · Its place in the literature

Who cites it

12 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Review
  5. Updated Review of Current Therapeutic Approaches for the Management of Sickle Cell Disease.Cardiovascular & hematological disorders drug targets · 2026
    Review
  6. Article
  7. Article
  8. Advancing Precision Medicine: Recent Innovations in Gene Editing Technologies.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Review
  9. Article
  10. Gene Therapy Techniques and Delivery Methods (Review).Sovremennye tekhnologii v meditsine · 2025
    Review
  11. Sickle cell disease in India: the journey and hope for the future.Hematology. American Society of Hematology. Education Program · 2024
    Review
  12. 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.

Mariam BarakBiology Department, Case Western Reserve University, Cleveland, OH 44106, USA.
Christopher HuBiology Department, Case Western Reserve University, Cleveland, OH 44106, USA.
Alicia MatthewsBiology Department, Case Western Reserve University, Cleveland, OH 44106, USA.
Yolanda M FortenberryBiology Department, Case Western Reserve University, Cleveland, OH 44106, USA.

Funding

The Hartwell Foundation XXXXXXXX
6 · The paper itself

Abstract

Sickle cell disease (SCD) is the most common genetic blood disorder in the United States, with over 100,000 people suffering from this debilitating disease. SCD is caused by abnormal hemoglobin (Hb) variants that interfere with normal red blood cell (RBC) function. Research on SCD has led to the development and approval of several new SCD therapies in recent years. The recent FDA-approved novel gene therapies are potentially curative, giving patients an additional option besides a hematopoietic bone marrow transplant. Despite the promise of existing therapies, questions remain regarding their long-term pharmacological effects on adults and children. These questions, along with the exorbitant cost of the new gene therapies, justify additional research into more effective therapeutic options. Continual research in this field focuses on not only developing cheaper, more effective cures/treatments but also investigating the physiological effects of the current therapies on SCD patients, particularly on the brain and kidneys. In this article, we undertake a comprehensive review of ongoing clinical trials with completion dates in 2024 or later. Our exploration provides insights into the landscape of current therapeutics and emerging novel therapies designed to combat and potentially eradicate SCD, including the latest FDA-approved gene therapies.

Indexed as

Anemia, Sickle CellGenetic TherapyClinical Trials as TopicHumansclinical trialsgene therapyhydroxyureasickle cell diseasetherapeuticsvaso-occlusionvoxelotor

Identifiers

PMID38786070
PMCPMC11120250

What OpenQuestion holds

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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.