Hussnain Javaid: Thalassaemia 2026 New Therapies Transforming UK Practice
Hussnain Javaid, Consultant Haematologist at NHS UK, Founder of ClinHaem, shared a post on LinkedIn about recent articles, adding:
“2026 Update: Thalassaemia — What’s changing for UK practice?
Some exciting developments in disease-modifying therapies with key updates below:
Gene editing — Exa-cel (Casgevy)
A major milestone for transfusion-dependent β-thalassaemia (TDT)!
- CRISPR-Cas9 gene editing to reactivate fetal Hb production.
- NICE-recommended for eligible patients (now from) ages higher 5 years, where HSCT is suitable but no HLA-matched related donor is available.
Luspatercept — BELIEVE trial
An erythroid maturation agent that reduces transfusion requirements.
- 21.4 percent vs 4.5 percent achieved higher 33 percent reduction in transfusion burden (with higher 2 fewer units) during weeks 13–24, compared with placebo.
- Approved for adults with TDT in the US and Europe plus European authorisation for NTDT.
- UK routine access remains pending, NICE appraisal seems underway
Mitapivat — ENERGIZE / ENERGIZE-T
Another exciting addition to the therapeutic landscape! Oral pyruvate kinase activator.
- ENERGIZE: Improved Hb responses in NTDT.
- ENERGIZE-T: Reduced transfusion requirements in TDT.
- Approved: adults with α- and β-TDT in the US (2025), Europe (2026), Saudi Arabia (2025), and the UAE (2026). Not reached NICE yet…
Key clinical takeaway:
Thalassaemia management is moving beyond regular transfusions and iron chelation towards therapies that address ineffective erythropoiesis and, in selected patients, offer the possibility of a functional cure.
Recognising eligible patients early and liaising with specialist haemoglobinopathy centres remains crucial.”
Title 1: A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia
Authors: Domenica Cappellini, Vip Viprakasit, Ali T. Taher, Pencho Georgiev, Kevin H.M. Kuo, Thomas Coates, Ersi Voskaridou, Hong-Keng Liew, Idit Pazgal-Kobrowski, G.L. Forni, Silverio Perrotta, Abderrahim Khelif, Ashutosh Lal, Antonis Kattamis, Efthymia Vlachaki, Raffaella Origa, Yesim Aydinok, Mohamed Bejaoui, P. Joy Ho, Lee-Ping Chew, Ping-Chong Bee, Soo-Min Lim, Meng-Yao Lu, Adisak Tantiworawit, Penka Ganeva, Liana Gercheva, Farrukh Shah, Ellis J. Neufeld, Alexis Thompson, Abderrahmane Laadem, Jeevan K. Shetty, Jun Zou, Jennie Zhang, Dimana Miteva, Tatiana Zinger, Peter G. Linde, Matthew L. Sherman, Olivier Hermine, John Porter, Antonio Piga
Read the Full Article on NEJM
Title 2: Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
Authors: Franco Locatelli, Peter Lang, Donna Wall, Roland Meisel, Selim Corbacioglu, Amanda M. Li, Josu de la Fuente, Ami J. Shah, Ben Carpenter, Janet L. Kwiatkowski, Markus Mapara, Robert I. Liem, Maria Domenica Cappellini, Mattia Algeri, Antonis Kattamis, Sujit Sheth, Stephan Grupp, Rupert Handgretinger, Puja Kohli, Daoyuan Shi, Leorah Ross, Yael Bobruff, Christopher Simard, Lanju Zhang, Phuong Khanh Morrow, William E. Hobbs, Haydar Frangoul
Read the Full Article on NEJM
Title 3: ENERGIZE-T: A Global, Phase 3, Double-Blind, Randomized, Placebo-Controlled Study of Mitapivat in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia
Authors: Maria Domenica Cappellini, Sujit Sheth, Ali T. Taher, Hanny Al-Samkari, Ali Bülent Antmen, David Beneitez, Giovanna Cannas, Thomas D. Coates, Lauren Czapla, Jayme L. Dahlin, Jeremie H. Estepp, Elizabeth Feenstra, Pencho Georgiev, Sarah Gheuens, Keely S Gilroy, Andreas Glenthøj, Khaled Musallam, Kareem Osman, John B. Porter, Hui Shao, Katrin Uhlig, Eduard J. Van Beers, Vip Viprakasit, Kevin H.M. Kuo, Antonis Kattamis
Read the Full Article on Blood
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