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
  • Pediatrics and Neonatology
  • Pediatrics and Neonatology News
  • Hydroxyurea dose...

Hydroxyurea dose escalation effective in children with sickle cell anemia

Written By : Hina Zahid |Medically Reviewed By : Dr. Kamal Kant Kohli Published On 2020-06-29T18:00:01+05:30  |  Updated On 30 Jun 2020 10:50 AM IST
Hydroxyurea dose escalation  effective in children with sickle cell anemia
  • facebook
  • twitter
  • linkedin
  • whatsapp
  • Telegram
  • Email

A team of international researchers has learned that dose escalation of Hydroxyurea for treatment for children in Uganda with sickle cell anemia is more effective and has similar side effects than a lower fixed dose of the same drug.

The study, known as NOHARM MTD (Novel use Of Hydroxyurea in an African Region with Malaria - Maximum Tolerated Dose), focused on children in Uganda, but the results could impact use of hydroxyurea worldwide, including the United States and Europe. The findings have been published in the New England Journal of Medicine.

This clinical research milestone removes a major barrier to broadly expand the use of hydroxyurea in low-resource regions like sub-Saharan Africa, according to the physicians who led the study at Makerere University in Kampala, Uganda, Cincinnati Children's Hospital Medical Center, and the Indiana University (IU) School of Medicine.

For this study, 187 children with sickle cell anemia living in Uganda received hydroxyurea. About half received a fixed-dose of 20 mg per kilogram of body weight per day. The other half received an escalating dose, which started at 25 mg per kilogram of body weight per day and increased up to 35 mg per kilogram of body weight per day, if tolerated. Doctors evaluated the children every 2-3 months for laboratory and clinical benefits, as well as potential side effects.

While the study team was planning to keep the children on these separate treatment arms for two years, an independent data review panel changed the course about 18 months into the study, due to clear benefits of the higher dose.

"Our study's data safety and monitoring board noted a highly significant difference between the treatment groups, with the children on escalated dosing having superior clinical results, but the same number of side effects, so at their recommendation we halted the trial and moved all of the children to that escalated dosing strategy," said Robert Opoka, MMed, who oversaw the study at Makerere University and Mulago Hospital in Uganda.

Sickle cell anemia is a life-threatening blood disorder that distorts red blood cells into a sickle or crescent shape and leads to anemia, recurrent pain, organ damage, and early death. It is also a serious global health issue: in the United States, about 100,000 individuals are affected, but worldwide more than 300,000 children are born with the disease each year, with more than 80% born in sub-Saharan Africa. Yet most therapeutic developments for sickle cell have not been available to children in Africa, including hydroxyurea, which is FDA-approved and effectively reduces the acute and chronic disease manifestations.

Earlier studies in sub-Saharan Africa showed hydroxyurea to be safe, feasible to use, and effective for treating sickle cell anemia, according to Russell Ware, MD, PhD, a hematologist at Cincinnati Children's who led those studies and is senior investigator on the current NEJM paper. Ware said the drug boosts fetal hemoglobin, which reduces sickling of the red blood cells and improves anemia, lowers pain and other sickle-related events, and reduces clinical interventions such as transfusions and hospitalizations.

According to lead study co-investigator Chandy John, MD, a physician scientist at the Ryan White Center for Pediatric Infectious Diseases and Global Health at the IU School of Medicine, the optimal dosing and monitoring plan for hydroxyurea was unknown when the study started. Particularly for low-resource countries like Uganda, determining the hydroxyurea optimal dosing and monitoring plan was crucial.

John said the new study confirms that the dose escalation regimen is better than fixed dose, but expanding hydroxyurea treatment will require affordable drug costs, education of healthcare providers, and an increased drug supply. In addition, newborn screening for sickle cell anemia is needed to help identify those who will benefit from treatment as early as possible.

"There will be some additional costs associated with screening and increasing access to the drug, but they will be more than offset by benefits to patients," John explained. "Our data make it clear that children on a higher daily dose had substantially better clinical outcomes, with fewer adverse events."

"The study shows clearly that the optimized dosing strategy for hydroxyurea, though it requires more effort than a fixed-dose treatment regimen, results in far better outcomes for children with sickle cell anemia," Ware concluded. "We think this study can substantially benefit children with sickle cell anemia in Africa and throughout the world, by choosing the optimal hydroxyurea dose to decrease the disease complications."

"In Africa and other low-resource settings, children with sickle cell anemia have been a neglected population" said Opoka. "We are glad that our work together as African and US researchers has resulted in findings that will improve the health of all children with sickle cell anemia."

For more details click on the link: http://dx.doi.org/10.1056/NEJMoa2000146

sickle cell anemiahydroxyurea treatment
Source : New England Journal of Medicine
Hina Zahid
Hina Zahid

    Hina Zahid Joined Medical Dialogue in 2017 with a passion to work as a Reporter. She coordinates with various national and international journals and association and covers all the stories related to Medical guidelines, Medical Journals, rare medical surgeries as well as all the updates in the medical field. Email: editorial@medicaldialogues.in. Contact no. 011-43720751

    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

    Intensive Blood Pressure Control Reduces Dementia Risk: Study Finds

    Intensive Blood Pressure Control Reduces Dementia Risk: Study Finds

    View All

    Health News Today

    Health Bulletin 02/June/2025

    Health Bulletin 02/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