The edition · Top Clinical Updates
An oral drug cuts transfusions in thalassaemia, and physiology beats the eye in STEMI
Mitapivat reduces transfusion burden in transfusion-dependent thalassaemia; liposuction outperforms decongestive therapy for lipoedema pain; fifth-dose yellow fever vaccine holds up in toddlers; and a fuel additive explains a cluster of near-fatal methaemoglobinaemia.
The edition in brief
A phase 3 trial of mitapivat, an oral pyruvate kinase activator, reduced transfusion burden in adults with transfusion-dependent alpha or beta thalassaemia: 30% of patients met the transfusion reduction endpoint against 13% on placebo, an 18 percentage point difference. It is the first oral disease-modifying option in a condition that in India is managed almost entirely by transfusion and chelation. A German randomised trial found liposuction far better than conservative decongestive therapy for leg pain in lipoedema — 68% versus 8% achieving a two-point pain reduction at 12 months — at the cost of serious adverse events in 8% of those operated on. In Uganda, one-fifth and one-half doses of 17DD yellow fever vaccine were non-inferior to a full dose for seroconversion in 1,784 children aged 9 to 23 months, supporting dose-sparing during outbreak supply shortages. An Italian investigation traced 82 cases of severe acquired methaemoglobinaemia in migrants arriving by sea to dermal absorption of N-methylaniline, a petrol additive, from fuel-contaminated water in the vessels; median methaemoglobin was 44.6% and two patients died. A pearl covers the inter-arm blood pressure difference. The edition closes with AIR-STEMI, which randomised 1,823 patients with ST-elevation myocardial infarction and multivessel disease to complete revascularisation guided by coronary physiology or by angiographic appearance: the physiology-guided strategy reduced the composite of death, infarction, stroke and ischaemia-driven revascularisation from 13.7% to 8.9%, with fewer contrast-associated kidney injury and bleeding events as well.
An oral pyruvate kinase activator reduced transfusion burden in thalassaemia
There is now an oral option with randomised evidence in transfusion-dependent thalassaemia, but it reduces transfusion burden rather than removing it.
Liposuction relieved lipoedema pain where decongestive therapy did not
Name the diagnosis rather than attributing it to weight — lipoedema now has a randomised trial showing large pain relief from surgery.
One-fifth doses of yellow fever vaccine held up in children under two
Fractional yellow fever dosing is now supported down to nine months of age, for outbreak response during supply shortage.
A petrol additive explained 82 cases of near-fatal methaemoglobinaemia
Cyanosis that will not correct with oxygen and a normal arterial oxygen tension is methaemoglobinaemia until co-oximetry says otherwise.
Measure the blood pressure in both arms once, then always use the higher one
Record the arm as well as the reading, and make sure the home machine is used on the same one.
In STEMI with multivessel disease, physiology beat the angiographic eye
Assess non-culprit lesions physiologically rather than by appearance — it reduced events and contrast-associated harm at the same time.
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