ESPN 51th Annual Meeting

ESPN 2018


 
Patient-reported fatigue amongst children enrolled in the Global aHUS Registry
CHRISTOPH LICHT 1 LISA SARTZ 2 IMAD AL-DAKKAK 3 SPERO R. CATALAND 4 HAE IL CHEONG 5 LARRY A. GREENBAUM 6 MASAYO OGAWA 3

1- THE HOSPITAL FOR SICK CHILDREN, TORONTO, ONTARIO, CANADA
2- DEPARTMENT OF PEDIATRICS, CLINICAL SCIENCES LUND, LUND UNIVERSITY, LUND, SWEDEN
3- ALEXION PHARMACEUTICALS, INC., NEW HAVEN, CT, USA
4- THE OHIO STATE UNIVERSITY, WEXNER MEDICAL CENTER, COLUMBUS, OH, USA
5- SEOUL NATIONAL UNIVERSITY CHILDRENS HOSPITAL, SEOUL, KOREA
6- EMORY UNIVERSITY SCHOOL OF MEDICINE AND CHILDRENS HEALTHCARE OF ATLANTA, ATLANTA, GA, USA
 
Introduction:

Fatigue in children suffering from atypical haemolytic uraemic syndrome (aHUS) is not well-investigated. The objective of this analysis was to evaluate fatigue in paediatric patients enrolled to the Global aHUS Registry.

Material and methods:

The Global aHUS Registry is an observational longitudinal, multicentre registry (NCT01522183) that collects demographic data, medical/disease history, and treatment/outcomes data. Registry enrolment can occur at any stage of the disease. The Paediatric Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale was used to assess fatigue, whereby higher scores reflect better quality of life. Patients aged 5–17 years with evaluable data for enrolment, date of birth and FACIT-Fatigue score, as of May 5, 2017, were included. Data were stratified according to gender, age, disease history, dialysis/transplantation status, hospitalisation, plasma exchange/infusion (PE/PI), and treatment with eculizumab.

Results:

A total of 193 paediatric patients were included in the analysis (Table 1). The median (inter-quartile range) age was 10.1 (7.4–13.3), and 101 (52%) of these patients were male. At the time of Registry enrolment, 58 (30%) patients had an ongoing thrombotic microangiopathy, 8 (4%) were receiving dialysis, and 47 (24%) had recently been hospitalised. Fifteen (8%) were currently receiving eculizumab, 63 (33%) had previously received eculizumab, and 22 (11%) had recently (within 6 months of Registry enrolment) received PE/PI. The mean (standard deviation) FACIT-Fatigue score across the cohort was 41.6 (11.0). The largest differences in mean score occurred within the following subgroups: dialysis status (17.0, acute; 42.3, none); recent hospitalisation (36.3, yes; 43.5, no); and recent PE/PI treatment (36.3, yes; 42.5, no).

Conclusions:

Acute dialysis, recent hospitalisation, and recent PE/PI treatment had the most detrimental impact on FACIT-Fatigue score. Longitudinal changes in FACIT-Fatigue score will be evaluated in future analyses from the Registry.

 

Table 1. Descriptive data and FACIT-Fatigue score for paediatric patients upon enrolment in the Global aHUS Registry

 a Data presented as n (%), unless otherwise specified. b dialysis that started on or prior to Registry enrolment and lasted ≤3. c dialysis that started on or prior to Registry enrolment and lasted >3 months. d dialysis that started on or prior to Registry enrolment is currently ongoing. FACIT, Functional Assessment of Chronic Illness Therapy; IQR, interquartile range; PE, plasma exchange; PI, plasma infusion; SD, standard deviation; TMA, thrombotic microangiopathy.