Tag: Uganda

Hydroxyurea for Children with Sickle Cell Anaemia Significantly Reduces Infections

Sickle cell disease. Credit: National Institutes of Health

A clinical trial in Uganda has revealed that hydroxyurea significantly reduces infections in children with sickle cell anaemia. Their latest findings enhance strong evidence of hydroxyurea’s effectiveness and could ultimately reduce death in children in Africa, the continent most burdened by the disease.

The group’s research, appearing in the journal Blood, revealed that hydroxyurea treatment resulted in a remarkable 60% reduction in severe or invasive infections, including malaria, bacteraemia, respiratory tract infections and gastroenteritis, among Ugandan children with sickle cell anaemia.

“Our investigation provides powerful justifications for hydroxyurea’s use in children with sickle cell anaemia in Africa,” said Dr Chandy John, paediatrics professor at IU School of Medicine and co-lead investigator of the latest study.

“Given the high rates of infection in this region, we hope our evidence will encourage ministries of health to continue supporting and expanding access to hydroxyurea for young patients who can greatly benefit from the treatment.”

Sickle cell anaemia is a genetic blood disorder that alters the structure of red blood cells and affects oxygen distribution throughout the body, increasing susceptibility to serious health complications and life-threatening infections.

According to the World Health Organization, more than 300 000 children worldwide are born with sickle cell disease each year, with a high prevalence found in African countries.

While hydroxyurea has had U.S. Food and Drug Administration approval as a sickle cell disease treatment for children since 2017, its accessibility and acceptance in Africa have been comparatively limited.

As hydroxyurea has become more recognised in African countries for its effectiveness in treating sickle-cell-related complications, John and his colleagues noticed a knowledge gap about the treatment’s effect on infections.

This led the research group to incorporate hydroxyurea treatment and analysis into their established clinical trial, Zinc for Infection Prevention in Sickle Cell Anemia, led by Indiana University School of Medicine and collaborators in Uganda.

During the study, the researchers examined the effects of hydroxyurea on 117 children in Uganda and focused on a range of infections. After hydroxyurea treatment, results showed a substantial decrease in the incidence of these infections.

Additionally, eight of the nine deaths that occurred in the trial were children whose parents declined hydroxyurea treatment. The only death in a child on hydroxyurea treatment occurred four days after starting treatment, providing insufficient time for hydroxyurea to have an effect.

Of the five children for whom a cause of death was known, all five died of infectious causes.

The high death rate in the study, despite expert clinical care by study personnel, provides further evidence of the urgent need for additional interventions to decrease mortality in children with sickle cell disease in Africa.

“Infections commonly precede other complications related to sickle cell anaemia and often result in hospitalizations that can lead to death,” said Dr Ruth Namazzi, site principal investigator, first author and a lecturer in the Department of Pediatrics and Child Health at Makerere University in Uganda.

“We believe incorporating hydroxyurea treatment as the standard of care for sickle cell anaemia across Africa will not only reduce infections but will more importantly save countless lives.”

Source: Indiana University

Penicillin Reduces Rheumatic Heart Disease Progression in Kids

Photo by Roman Nguyen on Unsplash

In a new study, penicillin significantly reduces the risk of underlying rheumatic heart disease progression in children and adolescents.  

The research also showed that early screening was critical for preventing serious rheumatic heart disease progression and death in young children. Rheumatic heart disease affects 40.5 million people globally, causing 306 000 or more deaths every year. The chronic disease results from damage to the valves of the heart after a case of Strep throat. It’s considered a disease of poverty and disadvantage.

Associate Professor Andrea Beaton of Cincinnati Children’s Hospital Medical Center said that prior to this study, it was unknown if antibiotics were effective at preventing the progression of latent rheumatic heart disease.

“The trial is the first contemporary randomised controlled trial in rheumatic heart disease. The results are incredibly important on their own, but also demonstrate that high-quality clinical trials are feasible to address this neglected cardiovascular disease,” she said.

The trial involved 818 Ugandan children aged 5 to 17 years with latent rheumatic heart disease, who received either four-weekly injections of penicillin for two years or no treatment. All underwent echocardiography screening at the beginning and end of the trial.

The findings from the screenings, published in the New England Journal of Medicine, reported just three (0.8%) participants who received penicillin experienced latent rheumatic heart disease progression, compared to 33 (8.3%) who didn’t receive the treatment.

Dr Daniel Engelman of Murdoch Children’s Research Institute (MCRI) said the results showed a significant and greater than expected reduction in disease development.  

MCRI Professor Andrew Steer said screening for latent rheumatic heart disease was critical to stop progression because heart valve damage was largely untreatable.

“Children with latent rheumatic heart disease have no symptoms and we cannot detect the mild heart valve changes clinically,” he said.

“Currently, most patients are diagnosed when the disease is advanced, and complications have already developed. This late diagnosis is associated with a high death rate at a young age, in part due to the missed opportunity to benefit from preventative antibiotic treatment. If patients can be identified early, there is an opportunity for intervention and improved health outcomes.”

Uganda Heart Institute Dr Emmy Okello said the Ugandan government should strengthen programs that promote screening of rheumatic heart disease and the availability of penicillin.

“Our study found a cheap and easily available penicillin can prevent progression of latent rheumatic heart disease into more severe, irreversible valve damage that is commonly seen in our hospitals with little or no access to valve surgery,” Dr Okello said.

Source: Murdoch Childrens Research Institute