Evidence map›Paper›PMID 36287444›Full record

ReviewPharmacy (Basel, Switzerland)2022

Recent Advances in Sickle-Cell Disease Therapies: A Review of Voxelotor, Crizanlizumab, and L-glutamine.

Michael Migotsky, Molly Beestrum, Sherif M Badawy

Open access · goldAbstract readReview
In one paragraph

Review in Pharmacy (Basel, Switzerland), 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.

0numbers the graph read from it
0cells of the map it votes in
22citing papers in PubMed
5.0field-weighted citation impact, top 4% 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, 35 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Article
  5. Review
  6. Crosstalk Between Sickle Cell Disease and Ferroptosis.International journal of molecular sciences · 2025
    Review
  7. Article
  8. Review
  9. Review
  10. Development and IND-enabling studies of a novel Cas9 genome-edited autologous CD34Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Article
  11. Article
  12. Article
  13. Article
  14. Article
  15. Article
  16. Article
  17. Review
  18. Review
  19. Medicine Use in Chronic Diseases.Pharmacy (Basel, Switzerland) · 2023
    Article
  20. Treatment of sickle cell disease - options and perspective.American journal of blood research · 2023
    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

3 authors at 1 institution in 1 country.

Michael MigotskyDepartment of Medical Education, McGaw Medical Center, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.
Molly BeestrumGalter Health Sciences Library, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.
Sherif M BadawyDivision of Hematology, Oncology, and Stem Cell Transplant, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL 60611, USA.ORCID 0000-0002-4739-265X
Northwestern University · US

Funding

An mHealth Strategy to Improve Medication Adherence in Adolescents with Sickle Cell DiseaseK23HL150232 · NHLBI · LURIE CHILDREN'S HOSPITAL OF CHICAGO · PI BADAWY, SHERIF MOHAMED · 2020 to 2024
$842k
NHLBI NIH HHS K23 HL150232
6 · The paper itself

Abstract

Sickle-cell disease (SCD) is an inherited hemoglobinopathy, causing lifelong complications such as painful vaso-occlusive episodes, acute chest syndrome, stroke, chronic anemia, and end-organ damage, with negative effects on quality of life and life expectancy. Within the last five years, three new treatments have been approved: L-glutamine in 2017 and crizanlizumab and voxelotor in 2019. We conducted a literature search of these three medications, and of the 31 articles meeting inclusion criteria, 6 studied L-glutamine, 9 crizanlizumab, and 16 voxelotor. Treatment with L-glutamine was associated with decrease in pain crises, hospitalizations, and time to first and second crises, with a decrease in RBC transfusion rate. Barriers to filling and taking L-glutamine included insurance denial, high deductible, and intolerability, especially abdominal pain. Crizanlizumab was associated with a reduction in pain crises and time to first crisis, with reduction in need for opioid use. Adverse effects of crizanlizumab include headache, nausea, insurance difficulty, and infusion reactions. Voxelotor was associated with increased hemoglobin and decreased markers of hemolysis. Barriers for voxelotor use included insurance denial and side effects such as headache, rash, and diarrhea. These three medications represent exciting new therapies and are generally well-tolerated though price and insurance approval remain potential barriers to access. Other studies are ongoing, particularly in the pediatric population, and more real-world studies are needed. The objective of this article is to evaluate post-approval studies of crizanlizumab, voxelotor, and L-glutamine in SCD, with a focus on real-world efficacy, side effects, and prescribing data.

Indexed as

adolescentsadultschildrencrizanlizumabdisease modifying therapyhemoglobinopathyl-glutaminepediatricsickle cellvoxelotor

Identifiers

PMID36287444
PMCPMC9610018
OpenAlexW4297545591

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.