Preliminary core sets of measures for disease activity and damage assessment in juvenile systemic lupus erythematosus and juvenile dermatomyositis.
OBJECTIVE
To identify preliminary core sets of outcome variables for disease activity and damage assessment in juvenile systemic lupus erythematosus (JSLE) and juvenile dermatomyositis (JDM).


METHODS
Two questionnaire surveys were mailed to 267 physicians from 46 different countries asking each member to select and rank the response variables used when assessing clinical response in patients with JSLE or JDM. Next, 40 paediatric rheumatologists from 34 countries met and, using the nominal group technique, selected the domains to be included in the disease activity and damage core sets for JSLE and JDM.


RESULTS
A total of 41 response variables for JSLE and 37 response variables for JDM were selected and ranked through the questionnaire surveys. In the consensus conference, domains selected for both JSLE and JDM activity or damage core sets included the physician and parent/patient subjective assessments and a global score tool. Domains specific for JSLE activity were the immunological tests and the kidney function parameters. Concerning JDM, functional ability and muscle strength assessments were indicated for both activity and damage core sets, whereas serum muscle enzymes were included only in the activity core set. A specific paediatric domain called 'growth and development' was introduced in the disease damage core set for both diseases and the evaluation of health-related quality of life was advised in order to capture the influence of the disease on the patient lifestyle.


CONCLUSIONS
We developed preliminary core sets of measures for disease activity and damage assessment in JSLE and JDM. The prospective validation of the core sets is in progress.
Ruperto
Nicolino
N
IRCCS G. Gasalini, Pediatria II, Genova, Italy. nicolaruperto@ospedale-gaslini.ge.it
Ravelli
Angelo
A
Murray
Kevin J
KJ
Lovell
Daniel J
DJ
Andersson-Gare
Boel
B
Feldman
Brian M
BM
Garay
Stella
S
Kuis
Wietse
W
Machado
Claudia
C
Pachman
Lauren
L
Prieur
Anne-Marie
AM
Rider
Lisa G
LG
Silverman
Earl
E
Tsitsami
Elena
E
Woo
Pat
P
Giannini
Edward H
EH
Martini
Alberto
A
D002648:Child; D002986:Clinical Trials as Topic / Q000379:methods; D003697:Delphi Technique; D003882:Dermatomyositis / Q000175:diagnosis* / Q000628:therapy; D006801:Humans; D008180:Lupus Erythematosus, Systemic / Q000175:diagnosis* / Q000628:therapy; D011446:Prospective Studies; D012720:Severity of Illness Index; D011795:Surveys and Questionnaires; D016896:Treatment Outcome