Andrew J. McArdle (United Kingdom)
Imperial College London Infectious DiseasePresenter of 1 Presentation
IMMUNOMODULATORY TREATMENT EFFECTIVENESS IN A LARGE INTERNATIONAL COHORT OF MIS-C/PIMS-TS PATIENTS FROM THE BEST AVAILABLE TREATMENT STUDY (BATS) (ID 1323)
Abstract
Background
From April 2020, clinicians in multiple countries reported a novel and unusual inflammatory syndrome sharing features with Kawasaki Disease (KD), though occurring more in school-aged children, and with shock and abdominal symptoms common.
Treatment has been inspired by KD, with use of steroids and IVIg common.
Randomised trials are underway, but there is an urgent need to understand treatment efficacy.
Methods
In May 2020 we commenced the BATS study (bestavailabletreatmentstudy.co.uk), collecting data from paediatric units around the world on children presenting with PIMS-TS/MIS-C.
Data is collected in Redcap on demographics, presentation, exposure history, microbiological/serological results, outcomes and complications, with timecourse data on inflammatory markers, level of care, cardiological findings and treatments.
Exported data undergoes post-processing and QC. Patients are categorised by criteria for PIMS-TS/MIS-C and descriptive data on demographics, severity and treatment patterns is produced.
Propensity-score based methods will be used to compare effectiveness of initial treatments.
Results
At time of submission, 394 patient entries with admission/discharge dates are available from 49 hospitals in 25 countries. The majority (61%) of admissions come from the UK, Russia, Panama and USA.
Of 347 records where patients were previously untreated, initial immunomodulator treatment (two-day window) was steroids in 49 (14%), IVIG in 99 (29%) and both in 157 (45%). 15 (4%) had other treatment combinations and 27 (8%) received no immunomodulator.
Comparative analysis by treatment is underway.
Conclusions
Whilst randomised trials reduce bias, a low proportion of children globally are being recruited into trials. Low recruitment may lead to imprecise estimates of efficacy and uncertainty.
The depth, growing size and global reach of our study potentially offers the best available evidence of efficacy of primary treatments, as well as insight into treatment patterns, outcomes and complications.