Khaled Musallam: No Better Conclusion For a Clinical Trial than For It to Be Published in the LANCET
Khaled Musallam, Director of Thalassemia & Sickle Cell Center at Burjeel Medical City, shared a post on LinkedIn:
“This is how we do it in the UAE!
No better conclusion for a clinical trial than for it to be published in the LANCET (impact factor 98, no.1 Gen Med Journal).
Data from our ENERGIZE phase 3 global RCT showed the efficacy (across all primary and key secondary endpoints) and safety of mitapivat in non-transfusion-dependent thalassemia (NTDT) and is now being used for regulatory filings globally. If approved, mitapivat will be the first oral disease-modifying therapy in beta-NTDT and first ever therapy in alpha-NTDT to be available for patients.
We have been sounding the alarm in the past few years on the harms of un/subtreated anemia (ineffective erythropoiesis/hemolysis) in these patients with significantly increased risks of morbidity and mortality. With this new advance, we hope to transform the prospect of NTDT patients and give them better quality of life.
Big thanks go to all those who believed in this trial, from patients who didn’t hesitate to partake in a placebo-controlled trial, to my research team at BMC | Burjeel Medical City, my sub-investigators at Burjeel Cancer Institute | BCI, and the Department of Health Abu Dhabi for all the support throughout the past two years. Congrats to all my global co-authors, this is BIG.”
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