Medical Dialogues
  • Dermatology
Login Register
This site is intended for healthcare professionals only
Login Register
  • MD Brand Connect
  • Vaccine Hub
  • MDTV
    • Breaking News
    • Medical News Today
    • Health News Today
    • Latest
    • Journal Club
    • Medico Legal Update
    • Latest Webinars
    • MD Shorts
    • Health Dialogues
  • Fact Check
  • Health Dialogues
Medical Dialogues
  • Medical News & Guidelines
      • Anesthesiology
      • Cardiology and CTVS
      • Critical Care
      • Dentistry
      • Dermatology
      • Diabetes and Endocrinology
      • ENT
      • Gastroenterology
      • Medicine
      • Nephrology
      • Neurology
      • Obstretics-Gynaecology
      • Oncology
      • Ophthalmology
      • Orthopaedics
      • Pediatrics-Neonatology
      • Psychiatry
      • Pulmonology
      • Radiology
      • Surgery
      • Urology
      • Laboratory Medicine
      • Diet
      • Nursing
      • Paramedical
      • Physiotherapy
  • Health news
      • Doctor News
      • Government Policies
      • Hospital & Diagnostics
      • International Health News
      • Medical Organization News
      • Medico Legal News
      • NBE News
      • NMC News
  • Fact Check
      • Bone Health Fact Check
      • Brain Health Fact Check
      • Cancer Related Fact Check
      • Child Care Fact Check
      • Dental and oral health fact check
      • Diabetes and metabolic health fact check
      • Diet and Nutrition Fact Check
      • Eye and ENT Care Fact Check
      • Fitness fact check
      • Gut health fact check
      • Heart health fact check
      • Kidney health fact check
      • Medical education fact check
      • Men's health fact check
      • Respiratory fact check
      • Skin and hair care fact check
      • Vaccine and Immunization fact check
      • Women's health fact check
  • AYUSH
    • Ayurveda
    • Homeopathy
    • Siddha
    • Unani
    • Yoga
  • State News
      • Andaman and Nicobar Islands
      • Andhra Pradesh
      • Arunachal Pradesh
      • Assam
      • Bihar
      • Chandigarh
      • Chattisgarh
      • Dadra and Nagar Haveli
      • Daman and Diu
      • Delhi
      • Goa
      • Gujarat
      • Haryana
      • Himachal Pradesh
      • Jammu & Kashmir
      • Jharkhand
      • Karnataka
      • Kerala
      • Ladakh
      • Lakshadweep
      • Madhya Pradesh
      • Maharashtra
      • Manipur
      • Meghalaya
      • Mizoram
      • Nagaland
      • Odisha
      • Puducherry
      • Punjab
      • Rajasthan
      • Sikkim
      • Tamil Nadu
      • Telangana
      • Tripura
      • Uttar Pradesh
      • Uttrakhand
      • West Bengal
  • Medical Education
      • Ayush Education News
      • Dentistry Education News
      • Medical Admission News
      • Medical Colleges News
      • Medical Courses News
      • Medical Universities News
      • Nursing education News
      • Paramedical Education News
      • Study Abroad
  • Industry
      • Health Investment News
      • Health Startup News
      • Medical Devices News
      • Pharma News
      • Pharmacy Education News
      • Industry Perspective
  • MDTV
      • Health Dialogues MDTV
      • Health News today MDTV
      • Latest Videos MDTV
      • Latest Webinars MDTV
      • MD shorts MDTV
      • Medical News Today MDTV
      • Medico Legal Update MDTV
      • Top Videos MDTV
      • Health Perspectives MDTV
      • Journal Club MDTV
      • Medical Dialogues Show
This site is intended for healthcare professionals only
LoginRegister
Medical Dialogues
LoginRegister
  • Home
  • Medical news & Guidelines
    • Anesthesiology
    • Cardiology and CTVS
    • Critical Care
    • Dentistry
    • Dermatology
    • Diabetes and Endocrinology
    • ENT
    • Gastroenterology
    • Medicine
    • Nephrology
    • Neurology
    • Obstretics-Gynaecology
    • Oncology
    • Ophthalmology
    • Orthopaedics
    • Pediatrics-Neonatology
    • Psychiatry
    • Pulmonology
    • Radiology
    • Surgery
    • Urology
    • Laboratory Medicine
    • Diet
    • Nursing
    • Paramedical
    • Physiotherapy
  • Health news
    • Doctor News
    • Government Policies
    • Hospital & Diagnostics
    • International Health News
    • Medical Organization News
    • Medico Legal News
    • NBE News
    • NMC News
  • Fact Check
    • Bone Health Fact Check
    • Brain Health Fact Check
    • Cancer Related Fact Check
    • Child Care Fact Check
    • Dental and oral health fact check
    • Diabetes and metabolic health fact check
    • Diet and Nutrition Fact Check
    • Eye and ENT Care Fact Check
    • Fitness fact check
    • Gut health fact check
    • Heart health fact check
    • Kidney health fact check
    • Medical education fact check
    • Men's health fact check
    • Respiratory fact check
    • Skin and hair care fact check
    • Vaccine and Immunization fact check
    • Women's health fact check
  • AYUSH
    • Ayurveda
      • Ayurveda Giuidelines
      • Ayurveda News
    • Homeopathy
      • Homeopathy Guidelines
      • Homeopathy News
    • Siddha
      • Siddha Guidelines
      • Siddha News
    • Unani
      • Unani Guidelines
      • Unani News
    • Yoga
      • Yoga Guidelines
      • Yoga News
  • State News
    • Andaman and Nicobar Islands
    • Andhra Pradesh
    • Arunachal Pradesh
    • Assam
    • Bihar
    • Chandigarh
    • Chattisgarh
    • Dadra and Nagar Haveli
    • Daman and Diu
    • Delhi
    • Goa
    • Gujarat
    • Haryana
    • Himachal Pradesh
    • Jammu & Kashmir
    • Jharkhand
    • Karnataka
    • Kerala
    • Ladakh
    • Lakshadweep
    • Madhya Pradesh
    • Maharashtra
    • Manipur
    • Meghalaya
    • Mizoram
    • Nagaland
    • Odisha
    • Puducherry
    • Punjab
    • Rajasthan
    • Sikkim
    • Tamil Nadu
    • Telangana
    • Tripura
    • Uttar Pradesh
    • Uttrakhand
    • West Bengal
  • Medical Education
    • Ayush Education News
    • Dentistry Education News
    • Medical Admission News
    • Medical Colleges News
    • Medical Courses News
    • Medical Universities News
    • Nursing education News
    • Paramedical Education News
    • Study Abroad
  • Industry
    • Health Investment News
    • Health Startup News
    • Medical Devices News
    • Pharma News
      • CDSCO (Central Drugs Standard Control Organisation) News
    • Pharmacy Education News
    • Industry Perspective
  • Home
  • Medicine
  • Medicine News
  • Mitapivat improves Hb,...

Mitapivat improves Hb, decreases hemolysis in pyruvate kinase deficiency: NEJM

Written By : Medha Baranwal |Medically Reviewed By : Dr. Kamal Kant Kohli Published On 2022-04-15T09:45:53+05:30  |  Updated On 15 April 2022 9:50 AM IST
Mitapivat improves Hb, decreases hemolysis in pyruvate kinase deficiency: NEJM
  • facebook
  • twitter
  • linkedin
  • whatsapp
  • Telegram
  • Email

USA: Mitapivat significantly increases the hemoglobin level, improves patient-reported outcomes, and decreases hemolysis in patients with pyruvate kinase deficiency, a recent study has found. The researchers identified no new safety signals in the patients who received mitapivat. The study appears in the New England Journal of Medicine.


"The lifelong anemia associated with pyruvate kinase deficiency results in chronic fatigue, reduced exercise tolerance, and a reduced ability to concentrate at work or school, which can make it a challenge to get through even a normal day," says lead author Hanny Al-Samkari, MD, a hematologist and clinical investigator at MGH and an assistant professor of Medicine at Harvard Medical School. "Moreover, most patients develop other potentially serious complications, like iron overload in the liver and/or heart (which can cause cancer or death), osteoporosis, gallbladder disease, blood clots, and other issues."

Pyruvate kinase deficiency is a rare, hereditary, chronic condition that is tied to hemolytic anemia. In a phase 2 study, mitapivat, an oral, first-in-class activator of erythrocyte pyruvate kinase was shown to increase the hemoglobin level in patients with pyruvate kinase deficiency.

Hanny Al-Samkari and colleagues evaluated the safety and efficacy of mitapivat in adults with pyruvate kinase deficiency who were not receiving regular red-cell transfusions. They were assigned to receive either mitapivat (5 mg twice daily, with potential escalation to 20 or 50 mg twice daily) or a placebo for 24 weeks.

A hemoglobin response (an increase from baseline of ≥1.5 g per deciliter in the hemoglobin level) that was sustained at two or more scheduled assessments at weeks 16, 20, and 24 was the primary endpoint.

Salient findings of the study include:

  • Sixteen of the 40 patients (40%) in the mitapivat group had a hemoglobin response, as compared with none of the 40 patients in the placebo group (adjusted difference, 39.3 percentage points).
  • Patients who received mitapivat had a greater response than those who received placebo with respect to each secondary end point, including the average change from baseline in the hemoglobin level.
  • The most common adverse events were nausea (in 7 patients [18%] in the mitapivat group and 9 patients [23%] in the placebo group) and headache (in 6 patients [15%] and 13 patients [33%], respectively). Adverse events of grade 3 or higher occurred in 10 patients (25%) who received mitapivat and 5 patients (13%) who received placebo.

The findings confirm that through its novel mechanism of up-regulating the enzymatic activity of pyruvate kinase and thus increasing ATP levels in red cells, mitapivat successfully addressed the underlying cause of chronic hemolysis and hemolytic anemia in some patients with pyruvate kinase deficiency. The clinical efficacy of mitapivat and its benefit with respect to health-related quality of life is further supported by improvements in the pyruvate kinase deficiency–specific patient-reported outcome measures.

"Patients who received mitapivat had substantially greater decreases in symptom severity and disease effects at 24 weeks compared to those who received placebo," wrote the authors. "A longer follow-up is warranted to assess the durability of these improvements."

Reference:

The study titled, "Mitapivat versus Placebo for Pyruvate Kinase Deficiency," was published in the New England Journal of Medicine.

DOI: 10.1056/NEJMoa2116634


New England Journal of Medicinepyruvate kinase deficiencymitapivathemolytic anemia
Source : New England Journal of Medicine
Medha Baranwal
Medha Baranwal

    MSc. Biotechnology

    Dr. Kamal Kant Kohli
    Dr. Kamal Kant Kohli

    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: drkohli@medicaldialogues.in. Contact no. 011-43720751

    Show Full Article
    Next Story

    Editorial

    Aspirin Use in Male Smokers Aged 40-45 Years with Hypertension: Identifying the Ideal Candidates

    Aspirin Use in Male Smokers Aged 40-45 Years with Hypertension: Identifying the Ideal Candidates

    National Consensus 2025 on Role of Bisoprolol Across CV Continuum in Women: Top 10 Takeaways

    National Consensus 2025 on Role of Bisoprolol Across CV Continuum in Women: Top 10 Takeaways

    Relevance of Pioglitazone in Indian T2DM Care Continuum

    Relevance of Pioglitazone in Indian T2DM Care Continuum

    Azmarda Outperforms Generic Sacubitril/Valsartan in HFrEF Management, says new study

    Azmarda Outperforms Generic Sacubitril/Valsartan in HFrEF Management, says new study

    First Indian Consensus on Managing Refractory Gastroesophageal Reflux Disease Recommends Vonoprazan

    First Indian Consensus on Managing Refractory Gastroesophageal Reflux Disease Recommends Vonoprazan

    View All

    Journal Club Today

    New Nasal Deformity Classification System May Enhance Standardization in Cleft Lip and Palate Assessment: Research Shows

    New Nasal Deformity Classification System May Enhance Standardization in Cleft Lip and Palate...

    View All

    Health News Today

    Health Bulletin 03/June/2025

    Health Bulletin 03/June/2025

    View All
    © 2022 All Rights Reserved.
    Powered By: Hocalwire
    X
    We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok