How are children’s lives affected now?
Children with conditions that severely weaken their immune system – such as primary immunodeficiencies (PIDs*) or those undergoing chemotherapy – are particularly vulnerable to infections.
“Even common lung infections, such as influenza, can be extremely difficult for their bodies to fight, increasing the risk of serious complications,” says Dr Smith. “Unfortunately, current anti-viral treatment options for these children are extremely limited – and many will lose their lives.”
Each year, around 400 children a year receive HSCT as a potential curative treatment for problems affecting their immune system – including around 70 with severe PIDs, as well as children with blood cancers that have not responded well to chemotherapy or other rare blood disorders.1 The procedure works by using powerful drugs to wipe out their existing immune system and then rebuilding it using healthy cells from a donor.
“Unfortunately, these children are at increased risk of developing a life-threatening viral lung infection before or after their transplant, reducing the likelihood of a successful outcome,” says Dr Smith.
*PIDs are a group of conditions that affect how well a child’s immune system works.
How could this research help?
“Our goal is to identify safe and effective new treatments for children with severely weakened immune systems facing viral lung infections, particularly those awaiting or recovering from HSCT,” says Dr Smith.
Early results from a small study of seriously ill children with viral lung infections showed that most improved after receiving a combination of existing anti-viral drugs.
“Building on these promising results, we now plan to test the safety and effectiveness of potential new drug combinations – from laboratory screening through to clinical testing – to identify the best treatment strategies,” says Dr Smith.
The researchers will also look for key indicators of drug resistance within respiratory viruses, which could help personalise treatment for each child. They will also establish a national database to track how well anti-viral drugs work in routine care across the UK.
Our findings will help shape future clinical trials and inform guidelines for treating life-threatening lung infections in vulnerable children – giving them the best chance of recovery in both the short and long term.
References
- British Society of Blood and Marrow Transplantation and Cellular Therapy (BSBMTCT) Registry, 2022, Table 2 (Paed totals): https://bsbmtct.org/activity/2022/.
Research table
Project details
| Project Leader | Dr Claire Smith |
| Location | UCL Great Ormond Street Institute of Child Health |
| Project Team |
Professor Judith Breuer MD FRCPath FMEdSci
Professor Joseph Standing MPharm MSc PhD Dr Samuel Ellis PhD, AFHEA Dr Seilesh Kadambari MBBS, PhD, FRCPCH, FHEA Dr Austen Worth MA BMBCh MRCPCH PhD Dr Alasdair Bamford MBBS MA (Cantab) FRCPCH DTM+H PhD Dr Andrew Gennery MD, MRCP, FRCPCH Dr Kanchan Rao MBBS, MD, MNAMS, MRCPCH |
| Other Locations |
Great Ormond Street Hospital
Great North Children’s Hospital |
| Grant Amount | £199,442 |
| Duration | 36 months |
| Grant Code (GN number) | GN4003 |
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