Evidence map›Paper›PMID 27105596›Full record

ReviewBiomedical journal2016

Current and future alternative therapies for beta-thalassemia major.

Edouard de Dreuzy, Kanit Bhukhai, Philippe Leboulch, Emmanuel Payen

Open access · goldAbstract readReview
In one paragraph

Review in Biomedical journal, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.

0numbers the graph read from it
0cells of the map it votes in
35citing papers in PubMed
4.4field-weighted citation impact, top 5% 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

35 citing papers in PubMed, 93 citations in OpenAlex.

  1. Prediction ofFrontiers in medicine · 2026
    Article
  2. Metal Ion Signaling in Biomedicine.Chemical reviews · 2025
    Review
  3. Article
  4. Article
  5. Article
  6. Review
  7. Approaches to Address the Anemia Challenge.The Journal of nutrition · 2023
    Article
  8. Review
  9. Article
  10. Review
  11. Erythrocyte Indices and Hemoglobin Analysis for α-Thalassemia Screening in an Area with High Carrying Rate.Indian journal of hematology & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion · 2022
    Article
  12. Article
  13. Association of Beta-Thalassaemia and Hypogonadotropic Hypogonadism.Case reports in obstetrics and gynecology · 2022
    Article
  14. Frontiers in pharmacology · 2022
    Article
  15. Article
  16. Iron metabolism: pathophysiology and pharmacology.Trends in pharmacological sciences · 2021
    Review
  17. Review
  18. Article
  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

4 authors at 3 institutions in 3 countries.

Edouard de DreuzyCEA, Institute of Emerging Diseases and Innovative Therapies, Fontenay aux Roses, France; University of Paris 11, CEA-iMETI, 92260 Fontenay aux Roses, France.
Kanit BhukhaiCEA, Institute of Emerging Diseases and Innovative Therapies, Fontenay aux Roses, France; University of Paris 11, CEA-iMETI, 92260 Fontenay aux Roses, France.
Philippe LeboulchCEA, Institute of Emerging Diseases and Innovative Therapies, Fontenay aux Roses, France; University of Paris 11, CEA-iMETI, 92260 Fontenay aux Roses, France; Department of Medicine, Harvard Medical School and Genetics Division, Brigham and Women's Hospital, Boston MA, USA; Mahidol University and Ramathibodi Hospital, Bangkok, Thailand.
Emmanuel PayenCEA, Institute of Emerging Diseases and Innovative Therapies, Fontenay aux Roses, France; University of Paris 11, CEA-iMETI, 92260 Fontenay aux Roses, France; INSERM, Paris, France. Electronic address: emmanuel.payen@cea.fr.
Université Paris-Saclay · FRCommissariat à l'Énergie Atomique et aux Énergies Alternatives · FRUniversité Paris Cité · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Beta-thalassemia is a group of frequent genetic disorders resulting in the synthesis of little or no β-globin chains. Novel approaches are being developed to correct the resulting α/β-globin chain imbalance, in an effort to move beyond the palliative management of this disease and the complications of its treatment (e.g. life-long red blood cell transfusion, iron chelation, splenectomy), which impose high costs on healthcare systems. Three approaches are envisaged: fetal globin gene reactivation by pharmacological compounds injected into patients throughout their lives, allogeneic hematopoietic stem cell transplantation (HSCT), and gene therapy. HSCT is currently the only treatment shown to provide an effective, definitive cure for β-thalassemia. However, this procedure remains risky and histocompatible donors are identified for only a small fraction of patients. New pharmacological compounds are being tested, but none has yet made it into common clinical practice for the treatment of beta-thalassemia major. Gene therapy is in the experimental phase. It is emerging as a powerful approach without the immunological complications of HSCT, but with other possible drawbacks. Rapid progress is being made in this field, and long-term efficacy and safety studies are underway.

Indexed as

Complementary TherapiesHematopoietic Stem Cell TransplantationTimeAnimalsbeta-ThalassemiaHumansTissue DonorsTreatment OutcomeAllogeneic transplantationBeta-globinBeta-thalassemiaGamma-globin inducersGene therapy

Identifiers

PMID27105596
PMCPMC6138429
OpenAlexW2332195908

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.