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Mitapivat effective for Hemolytic Anemia in patients with non-transfusion dependent Thalassemia
![Mitapivat effective for Hemolytic Anemia in patients with non-transfusion dependent Thalassemia Mitapivat effective for Hemolytic Anemia in patients with non-transfusion dependent Thalassemia](https://medicaldialogues.in/h-upload/2022/04/26/750x450_175083-world-thalassemia-day-2022-2.webp)
Mitapivat was found to be effective in treating hemolytic anemia in patients with α-thalassemia and β-thalassemia not dependent on transfusions, finds a new study. The study was published in the journal, The Lancet.
Non-transfusion-dependent thalassemia (NTDT) patients do not require regular blood transfusions for survival. But can still acquire many comorbidities. There are no approved disease-modifying therapies for this. Hence researchers investigated the safety and efficacy of Mitapivat which is a pyruvate kinase activator in adults with non-transfusion-dependent (NTD) α-thalassemia or NTD β-thalassaemia. They conducted an open-label, multicentre, phase 2 study by recruiting patients aged 18 years and older from four academic clinical study sites in Oakland, CA, and Boston, MA, USA; Toronto, ON, Canada; and London, UK. Patients with NTDT including β-thalassemia with or without α-globin gene mutations, hemoglobin E β-thalassemia, or α-thalassemia, and a baseline hemoglobin concentration of 10·0 g/dL or lower were included. Mitapivat was administered orally at 50 mg twice daily for the first 6 weeks followed by an escalation to 100 mg twice daily for 18 weeks thereafter during a 24-week core period. The primary endpoint was hemoglobin response which is a≥1·0 g/dL increase in hemoglobin concentration from baseline at one or more assessments between weeks 4 and 12. Efficacy and safety were assessed in the full analysis set in patients who received at least one dose of the study drug.
Findings:
- 27 patients were screened, of whom 20 were enrolled and received Mitapivat between Dec 28, 2018, and Feb 6, 2020. Of these, 15 were with β-thalassemia and five with α-thalassemia.
- The median age of patients was 44 years. 15 of 20 patients were female, five were male, and ten were identified as Asian.
- 16 of 20 patients had a hemoglobin response. Of these five were with α-thalassemia and 11 with β-thalassemia.
- 17 (85%) patients had a treatment-emergent adverse event, and 13 had a treatment-emergent event that was considered to be treatment-related.
- One serious treatment-emergent adverse event occurred (grade 3 renal impairment), which was considered unrelated to the study drug, resulting in the discontinuation of treatment.
- The most commonly reported treatment-emergent adverse events were initial insomnia, dizziness, and headache.
- No patients died during the 24-week core period.
Thus, researchers concluded that Mitapivat was found to be effective in treating hemolytic anemia in patients with α-thalassemia and β-thalassemia not dependent on transfusions and increased hemoglobin in 80% of non-transfusion-dependent patients.
For the full article, click here: https://doi.org/10.1016/S0140-6736(22)01337-X
Kuo KHM, Layton DM, Lal A, et al. Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study. Lancet. 2022;400(10351):493-501.
BDS, MDS
Dr.Niharika Harsha B (BDS,MDS) completed her BDS from Govt Dental College, Hyderabad and MDS from Dr.NTR University of health sciences(Now Kaloji Rao University). She has 4 years of private dental practice and worked for 2 years as Consultant Oral Radiologist at a Dental Imaging Centre in Hyderabad. She worked as Research Assistant and scientific writer in the development of Oral Anti cancer screening device with her seniors. She has a deep intriguing wish in writing highly engaging, captivating and informative medical content for a wider audience. She can be contacted at editorial@medicaldialogues.in.
Dr Kamal Kant Kohli-MBBS, DTCD- a chest specialist with more than 30 years of practice and a flair for writing clinical articles, Dr Kamal Kant Kohli joined Medical Dialogues as a Chief Editor of Medical News. Besides writing articles, as an editor, he proofreads and verifies all the medical content published on Medical Dialogues including those coming from journals, studies,medical conferences,guidelines etc. Email: editorial@medicaldialogues.in. Contact no. 011-43720751