Evidence map›Paper›PMID 39828781›Full record

ReviewAnnals of hematology2025

A review on disease modifying pharmacologic therapies for sickle cell disease.

Himil Mahadevia, Ben Ponvilawan, Ujjwal Madan, Parth Sharma, Hana Qasim, Anuj Shrestha

Abstract readReview
In one paragraph

Review in Annals of hematology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.

0numbers the graph read from it
0cells of the map it votes in
9citing 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.

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

9 citing papers in PubMed.

  1. Article
  2. Article
  3. Review
  4. Article
  5. Review
  6. Optimizing the "right" patient selection for treatment for sickle cell disease.Hematology. American Society of Hematology. Education Program · 2025
    Article
  7. Review
  8. Article
  9. 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

6 authors.

Himil MahadeviaDepartment of Internal Medicine, University of Missouri-Kansas City, Kansas City, MO, 64108, USA. himil7777@gmail.com.ORCID http://orcid.org/0000-0002-2083-5510
Ben PonvilawanDepartment of Internal Medicine, University of Missouri-Kansas City, Kansas City, MO, 64108, USA.
Ujjwal MadanDepartment of Internal Medicine, University of Missouri-Kansas City, Kansas City, MO, 64108, USA.
Parth SharmaDepartment of Internal Medicine, University of Missouri-Kansas City, Kansas City, MO, 64108, USA.
Hana QasimDepartment of Internal Medicine, University of Missouri-Kansas City, Kansas City, MO, 64108, USA.
Anuj ShresthaDepartment of Internal Medicine, Section of Hematology/Oncology, University of Missouri-Kansas City, Kansas City, MO, 64108, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Sickle cell disease (SCD) is an inherited hematologic disease caused by sickle hemoglobin as the predominant RBC hemoglobin or by sickle hemoglobin in combination with other abnormal β-hemoglobin variants like HbC, HbD and others. Sickling of erythrocytes under deoxygenated conditions is the basis of inflammatory and thrombotic cascades which result in multiple serious complications, leading to early morbidity and mortality. While HLA-matched allogeneic bone marrow transplantation is potentially curative, it has considerable limitations due to potential severe toxicities. Despite slow progress towards novel therapeutic strategies for SCD and hydroxyurea being the sole medication that is shown to reduce vaso-occlusive events and mortality for almost 20 years, several pharmacological agents targeting different mechanisms have been examined in clinical trials and recently US- US-FDA-approved, including L-glutamine and crizanlizumab. Voxelotor was previously US-FDA-approved but has been voluntarily withdrawn from the market as the overall benefit did not outweigh the risks. Gene therapies based on CRISPR-Cas9 and lentiviral vectors have been very recently approved. However, excessive costs are a barrier to widespread use. Nonetheless, there is still a large area of unmet needs for patients with SCD, and further research towards better care is warranted.

Indexed as

Anemia, Sickle CellAntisickling AgentsAntibodies, Monoclonal, HumanizedBenzaldehydesGenetic TherapyGlutamineHumansHydroxyureaNitrilesPyrazinesPyrazolesAntibodies, Monoclonal, HumanizedAntisickling AgentsBenzaldehydescrizanlizumabGlutamineHydroxyureaNitrilesPyrazinesPyrazolesvoxelotorCrizanlizumabHydroxyureaL-glutaminePharmacologic therapySickle cell diseaseVoxelotor

Identifiers

PMID39828781
PMCPMC11971234

What OpenQuestion holds

Textmetadata
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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.