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Efficacy and safety of Mitapivat in sickle cell disease: a systematic review
Shalu R Chaudhary1, Komal Sharma2, Deepak B Saxena3
1Department of Public Health Program, Indian Institute of Public Health Gandhinagar (IIPHG), Gandhinagar, India.
Background:
Sickle cell disease (SCD) is a chronic hemoglobinopathy marked by hemolytic anemia, VOEs, and multiorgan complications. Mitapivat, an oral Pyruvate Kinase (PK) activator, has emerged as a potential disease-modifying therapy. This systematic review assessed the efficacy and safety of Mitapivat in individuals with SCD.
Methods:
A systematic literature search was conducted from different search engines from inception. The review followed PRISMA 2020 guidelines, and the protocol was registered in PROSPERO [CRD420251000061]. RCTs and NRSIs evaluating Mitapivat monotherapy were included. ROB was assessed. A narrative synthesis was undertaken due to heterogeneity.
Results:
One RCT and two NRSIs involving 156 participants were included. Reduced annualized Vaso-occlusive episodes (VOE) rates compared to placebo by 51.6% (50 mg) and 70.0% (100 mg). Statistically significant hemoglobin response was observed in 46.2%-56% of participants. Increased markers of hemolysis and reduced transfusion requirements in Mitapivat groups were reported. However, SAEs were low, in 8%-19% of Mitapivat recipients.
Conclusions:
Preliminary evidence suggests Mitapivat demonstrates potential to reduce VOEs, improve hemoglobin levels, and decrease hemolysis with an acceptable safety profile. However, small sample sizes, lack of functional outcomes, and limited follow-up warrant cautious interpretation. Further large-scale RCTs are needed to validate these findings and establish a long-term benefit-risk balance.
Insights
Mitapivat shows promise in reducing vaso-occlusive episodes and improving hemoglobin in sickle cell disease (SCD) patients. Further trials are needed to confirm long-term benefits and safety.
Area of Science:
- Hematology
- Pharmacology
Background:
- Sickle cell disease (SCD) is a chronic hemoglobinopathy causing hemolytic anemia and organ complications.
- Mitapivat, an oral pyruvate kinase (PK) activator, is being investigated as a disease-modifying therapy for SCD.
Approach:
- A systematic review adhering to PRISMA 2020 guidelines was conducted.
- Included randomized controlled trials (RCTs) and non-randomized studies of intervention (NRSIs) evaluating Mitapivat monotherapy.
- Data synthesis was performed using a narrative approach due to study heterogeneity.
Key Points:
- Mitapivat significantly reduced annualized vaso-occlusive episode (VOE) rates by 51.6% (50 mg) and 70.0% (100 mg) compared to placebo.
- Hemoglobin response was observed in 46.2%-56% of participants, with increased hemolysis markers and reduced transfusion needs.
- Serious adverse events (SAEs) were reported in 8%-19% of Mitapivat recipients, indicating a generally acceptable safety profile.
Conclusions:
- Mitapivat shows potential in reducing VOEs, improving hemoglobin, and decreasing hemolysis in SCD.
- Current evidence is based on small sample sizes and limited follow-up, necessitating cautious interpretation.
- Larger RCTs are required to validate these findings and establish the long-term benefit-risk profile of Mitapivat for SCD.
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