ReviewExperimental hematology2020
Efficacy and safety of recently approved drugs for sickle cell disease: a review of clinical trials.
Review in Experimental hematology, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 33 papers, 1 of them a synthesis that pooled 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.
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.
Who cites it
33 citing papers in PubMed, 1 synthesis or guideline pooled it, 64 citations in OpenAlex.
- Impact of the SARS-CoV-2 infection in individuals with sickle cell disease: an integrative review.Frontiers in medicine · 2023Pooled it
- Molecular and therapeutic frontiers in anemia therapy.The Journal of clinical investigation · 2026Review
- Dissolved Oxygen-Dependent Magnetophoresis of Sickle Red Blood Cells and Proof-of-Concept Separation Using a Magnetic Column.ACS measurement science au · 2026Article
- Updated Review of Current Therapeutic Approaches for the Management of Sickle Cell Disease.Cardiovascular & hematological disorders drug targets · 2026Review
- In Silico Post-screening of Anti-polymerization Agents to Treat Sickle Cell Disease.bioRxiv : the preprint server for biology · 2025Article
- Role ofLife (Basel, Switzerland) · 2025Article
- Real-World Experience of L-Glutamine in Sickle Cell Disease: A Retrospective Observational Study.Pharmacy (Basel, Switzerland) · 2025Article
- Base editing HbS to HbG-Makassar improves hemoglobin function supporting its use in sickle cell disease.Nature communications · 2025Article
- Hemoglobin Variants as Targets for Stabilizing Drugs.Molecules (Basel, Switzerland) · 2025Review
- PGC-1α agonism induces fetal hemoglobin and exerts antisickling effects in sickle cell disease.Science advances · 2024Article
- Sickle cell disease: combination new therapies vs. CRISPR-Cas9 potential and challenges - review article.Annals of hematology · 2024Review
- Biophysical chemistry behind sickle cell anemia and the mechanism of voxelotor action.Scientific reports · 2024Article
- Sickle Cell Disease Update: New Treatments and Challenging Nutritional Interventions.Nutrients · 2024Review
- The Role of Non-genetic Therapies to Reduce the Incidence of Sickle Cell Crisis: A Systematic Review.Cureus · 2023Review
- Gene therapy in sickle cell disease: Attitudes and informational needs of patients and caregivers.Pediatric blood & cancer · 2023Article
- Are Genetic Modifiers the Answer to Different Responses to Hydroxyurea Treatment?-A Pharmacogenetic Study in Sickle Cell Anemia Angolan Children.International journal of molecular sciences · 2023Article
- Targeting the von Willebrand Factor-ADAMTS-13 axis in sickle cell disease.Journal of thrombosis and haemostasis : JTH · 2023Article
- Allogeneic hematopoietic stem cell transplantation to cure sickle cell disease: A review.Frontiers in medicine · 2023Review
- First Clinical Nutrition Outpatient Consultation: A Review of Basic Principles in Nutritional Care of Adults with Hematologic Disease.TheScientificWorldJournal · 2023Review
- Novel Strategies for the Treatment of COVID-19.Drugs in R&D · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sickle cell disease is prevalent in several parts of the world. Most hospitalizations of these patients are related to pain crisis episodes. Moreover, levels of hemoglobin are lower in sickle cell disease patients as compared with the general population. Complications related to sickle cell disease are managed with blood transfusions, hydroxyurea, and opioids. Despite these therapies, patients with sickle cell disease experience multiple pain crisis episodes leading to hospitalizations and end-organ damage. The US Food and Drug Administration has approved three new drugs-L-glutamine, voxelotor, and crizanlizumab-for the prophylaxis and treatment of complications related to sickle cell disease. This review was aimed at assessing the efficacy and safety of recently approved drugs for the treatment of sickle cell disease. A comprehensive search was made on PubMed and clinicaltrials.gov to look for clinical trials reporting the efficacy and safety of recently approved drugs for sickle cell disease. Based on the results of clinical trials, L-glutamine, voxelotor, and crizanlizumab were well tolerated by sickle cell disease patients. L-Glutamine and crizanlizumab reduced the number of sickle cell crisis episodes, while voxelotor improved the level of hemoglobin in sickle cell disease patients. These drugs were effective alone and in combination with hydroxyurea.
Indexed as
Identifiers
What OpenQuestion holds
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.