- Design
- Phase 3, double-blind, randomised, placebo-controlled trial
- Population
- 258 adults with transfusion-dependent alpha- or beta-thalassaemia in 19 countries
- Primary outcome
- Transfusion reduction response over 48 weeks
- Effect
- 30% vs 13%; adjusted difference 18 percentage points (95% CI 8 to 27)
ENERGIZE-T randomised 258 adults with transfusion-dependent alpha- or beta-thalassaemia across 19 countries, 2:1, to mitapivat 100 mg twice daily or placebo for 48 weeks. Mitapivat is an oral activator of red cell pyruvate kinase. The paper appeared in The Lancet on 12 September 2026.
A transfusion reduction response, at least a 50% fall in units with at least two fewer units over any 12 weeks, occurred in 30% on mitapivat and 13% on placebo (adjusted difference 18 points, 95% CI 8 to 27). Serious adverse events were similar (11% vs 15%), but 6% stopped mitapivat for adverse events against 1% on placebo. Headache, insomnia and diarrhoea were common.
Thalassaemia is a large burden in India, where lifelong transfusion and iron chelation shape daily life for many families. An oral drug that reduces transfusions would matter, but most patients did not reach the response threshold, and the drug's regulatory status and cost in India are unknown.
- Mitapivat is an oral drug with phase 3 evidence of cutting transfusions in both alpha- and beta-thalassaemia
- Expect a meaningful response in a minority: about 30% versus 13% on placebo
- Its availability and cost in India are not yet known
- Iron chelation and transfusion schedules remain the standard of care
Why it matters
It opens an oral route to fewer transfusions in a disease that has relied on blood, chelation and transplant.
The statistics, in plain English
An 18-point difference means roughly one extra responder for every six patients treated. The confidence interval (8 to 27) is well clear of zero.
Read the rest in the app
You have read your two free briefings this month. The app carries all 27 specialties, every morning, free — and this finding is waiting in it.
Scan to keep reading on your phone. No account needed to start.
Tomorrow morning, before your first patient
One edition a day for top clinical updates, written by the desk, every claim tied to its paper. Six minutes.
Get the app — free