Evidence map›Paper›PMID 29883237›Full record

ReviewExpert review of hematology2018

Stem cell transplantation in sickle cell disease: therapeutic potential and challenges faced.

Alexis Leonard, John F Tisdale

Open access · greenAbstract readReview
In one paragraph

Review in Expert review of hematology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
29citing papers in PubMed, 1 pooled it
3.3field-weighted citation impact, top 7% 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

29 citing papers in PubMed, 1 synthesis or guideline pooled it, 56 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
  4. Article
  5. Review
  6. Review
  7. Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  8. Article
  9. Article
  10. Article
  11. Review
  12. Autologous gene therapy for hemoglobinopathies: From bench to patient's bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Review
  13. Review
  14. Article
  15. Article
  16. CRISPR/Cas systems: Delivery and application in gene therapy.Frontiers in bioengineering and biotechnology · 2022
    Review
  17. Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing.International journal of molecular sciences · 2021
    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.

Alexis Leonarda Molecular and Clinical Hematology Branch, National Heart, Lung, and Blood Institute , National Institutes of Health , Bethesda , MD , USA.
John F Tisdalea Molecular and Clinical Hematology Branch, National Heart, Lung, and Blood Institute , National Institutes of Health , Bethesda , MD , USA.
Children's National · USNational Heart Lung and Blood Institute · US

Funding

Nonmyeloablative allogeneic PBSC in globin disordersZIAHL006007 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI TISDALE, JOHN · 2009 to 2025
$18.4M
Isolation, characterization, and transplantation of candidate stem cellsZIAHL006009 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI TISDALE, JOHN · 2009 to 2025
$13.5M
Intramural NIH HHS Z99 HL999999
6 · The paper itself

Abstract

introductionSickle cell disease (SCD) is the most common inherited hemoglobinopathy worldwide, and is a life-limiting disease with limited therapeutic options to reduce disease severity. Despite being a monogenic disorder, the clinical phenotypes of SCD are variable, with few reliable predictors of disease severity easily identifying patients where the benefits of curative therapy outweigh the risks. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative option, though significant advances in gene therapy raise the promise for additional curative methods. Areas covered: Allogeneic transplantation in SCD has evolved and improved over the last two decades, now offering a standard of care curative option using a human leukocyte antigen (HLA)-matched sibling donor. Many of the seminal transplantation studies are reviewed here, demonstrating how initial failures and successes have influenced and led to current HSCT strategies. Such strategies aim to overcome setbacks and limitations, and focus on conditioning regimens, immune suppression methods, the use alternative donor sources, and gene therapy approaches. Expert commentary: SCD is a curable disease. Each dedicated effort to refine transplantation methods, expand the donor pool, and bring gene therapy models to fruition will make enormous impacts reducing disease burden and improving outcomes and quality of life for patients with SCD.

Indexed as

Hematopoietic Stem Cell TransplantationAllograftsAnemia, Sickle CellGenetic TherapyHistocompatibility TestingHumansSiblingsAllogeneic transplantationautologous transplantationgene therapyhematopoietic stem cell transplantationsickle cell disease

Identifiers

PMID29883237
PMCPMC8459571
OpenAlexW2805791633

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.