Once-daily cystic fibrosis drug offers simpler home treatment and fewer side effects for children

Nottingham researchers have found a way to treat children with cystic fibrosis (CF), which could reduce their side effects and see more young patients treated at home.

Experts at The University of Nottingham organised the largest successful clinical trial in the UK looking at cystic fibrosis in an attempt to discover whether a common antibiotic used to treat the condition could be administered just once a day instead of the usual three daily doses.

The recommendations from the study, which included CF patients from Nottingham City Hospital and will be published in The Lancet this week, could lead to simpler home treatment with less disruption to patients and their families.

The study was funded with £404,464 from the Cystic Fibrosis Trust, as part of their UK-wide work to improve clinical support for people with cystic fibrosis and help find a cure for this disease.

One of the patients who took part in the study was 17-year-old Kayleigh Boden. Kayleigh, an A-level student from Welton near Lincoln, found that her side effects reduced and she liked being able to administer her drugs just once a day at 4pm rather than the usual three times a day, including a midnight shift.

“The drugs usually lead to tinnitus and headaches and my eyes are affected, but I definitely suffered fewer side effects during the trial,” she added.

Kayleigh hopes that administering her drugs just once a day will help her to effectively manage her condition while allowing her to lead an active life that will include sitting her exams in the summer and, hopefully, achieving her ambition of going to university to train as a quantity surveyor.

Cystic fibrosis is the UK’s most common life-threatening inherited disease. Sufferers have difficulty in digesting food and are vulnerable to chronic chest infections. Every week five babies are born with CF and three people die from the condition — 90 per cent from the lung damage, which often results from chronic chest infections.

Patients are frequently given prolonged courses of intravenous antibiotics to treat chronic chest infections, often for two weeks or more. This treatment may be needed every few months and can interfere with children’s schooling or adults studying or getting work. Sometimes intravenous treatment is given at home rather than in hospital to make this easier. However, administering this treatment puts an extra strain on parents, partners or the patient themselves.

In an effort to make home treatment easier, the team in the University’s Division of Respiratory Medicine, led by Professor Alan Knox and Dr Alan Smyth, organised the TOPIC (Tobramycin Once-daily Prescribing In Cystic fibrosis) national trial.

The researchers are based at the study’s co-ordinating centre, Nottingham City Hospital, where they care for 80 children with CF and travel out to see another 80 children at five other hospitals. The hospital also has a busy adult clinic treating more than 100 patients.

The study looked at tobramycin, a commonly-used antibiotic, to see if it would be effective when given once a day rather than the usual three times daily treatment. The drug can potentially lead to reduced kidney function and hearing loss. However, the TOPIC team found that once daily treatment worked just as well and seemed to have fewer side effects in children.

Rosie Barnes, Chief Executive of the Cystic Fibrosis Trust, said: “This is a very encouraging and exciting piece of research not least because of the immediate impact on patients. It will help relieve what is very time-consuming care and reduce the amount of time spent taking the required dose of antibiotics.”

The team is now looking at new ways to help people with CF. Current projects are looking at the role of garlic capsules against chest infection and whether home treatment can be simplified further by giving a second antibiotic as a 24-hour infusion through a small pump device.