New & emerging medicines

Beyond transfusion and chelation, new medicines target the root problem — the imbalance that makes red cells mature poorly. Luspatercept (Reblozyl) is a newer injection that helps red cells mature, reducing how much blood some transfusion-dependent patients need; possible effects include bone pain and joint aches.

An older, widely available tablet — hydroxyurea (hydroxycarbamide) — can raise fetal haemoglobin and helps some people (more so in NTDT and certain genetic types); its benefit in transfusion-dependent beta-thalassaemia is more limited.

Other agents are in development, including more drugs that boost fetal haemoglobin or improve red-cell metabolism (for example, pyruvate-kinase activators). Not everyone will be suitable, and availability varies by country.

If you’re curious whether a newer treatment could fit you, ask your haematologist — it’s a fast-moving area.