Having PK deficiency or caring for someone with a rare disease can feel isolating.
This is why a growing community of support and resources is available for you through our website.
TIF is excited to be participating in the 26th EHA Annual Congress, held this year virtually on 09 – 17 June 2021.
17 June 2021 Satellite Symposium
Ms Lily Cannon, TIF Operations Manager, attended a satellite symposium entitled Pyruvate Kinase Activation: Update on Clinical Trial Results across Hemolytic Anaemias discussed by Dr EJ van Beers (Netherlands), Dr Hanny Al-Samkari (USA) and Dr Kevin Kuo (Canada).
The results of the clinical trials of Mitapivat in pyruvate kinase deficiency were presented, demonstrating that the activation of pyruvate kinase with Mitapivat represents a potential therapeutic mechanism that may improve clinical outcomes.
More specifically, Mitapivat in two Phase 3 clinical studies (one with patients who are not regularly transfused and one for patients who are regularly transfused) showed a sustained increase in haemoglobin (40% and 37% respectively), thus reducing their transfusion burden. 22% of regularly transfused patients becoming transfusion – free, and half of those achieving normal haemoglobin levels.
The esteemed panel discussed the potential therapeutic value of Mitapivat, remarking that the patient-reported outcome tools developed for the studies demonstrated that even a modest improvement in haemoglobin levels (>1g/dl) went a long way to improve quality of life markers. Concluding the panel indicated the promising results of all the clinical studies, across disease areas will continue to be evaluated.
11 June 2021 Oral Presentations
Ms Lily Cannon attended the oral presentation session with Dr Hanny Al-Samkari (USA) and Dr Andreas Glenthøj (Denmark) who presented the results of the clinical trials of mitapivat: ACTIVATE for non regularly transfused patients and ACTIVATE-T for regularly transfused patients.
On 20 September 2019, pyruvate kinase deficiency patients, caregivers and advocates came together with healthcare providers, industry representatives and government officials for an externally-led, Patient-Focused Drug Development meeting hosted by the National Organization for Rare Disorders (NORD) and the Foundation for Rare Blood Diseases (SZB).
This report constitutes the summary composed the host organisations’ account of the perspectives of patients and caregivers who took part in the public meeting.
Click on the picture to access the report.