Background/Purpose:

It is an ethical requirement for setting up a randomized controlled trial (RCT) that the physician-investigator must have genuine uncertainty about the therapeutic options. This is called equipoise. Ideally patient-participants should also be in equipoise. In pediatric rheumatology, there are no data on whether this uncertainty also consists among parents and patients or if they have a specific preference for a particular treatment strategy. We conducted an interview study on the preferences of parents and children and the influence of the informed consent procedure on preferences in the setting of a randomized clinical trial in Juvenile Idiopathic Arthritis patients.

Methods:

Semi-structured interviews with parents (n = 23, 1 father and 22 mothers) and patients aged 12 and older (n = 7) participating in the BeSt for Kids study, a randomized clinical trial with three treatment strategies (arm 1: initial monotherapy with sulfasalazine or methotrexate, arm 2 : initial combination therapy with methotrexate and prednisone and arm 3: initial combination therapy with etanercept and methotrexate) in selected categories of newly diagnosed juvenile idiopathic arthritis patients.                                                                                                                                                 

Results:

All parents had a preference for a particular treatment strategy, 65% had a preference for the combination therapy with etanercept and methotrexate (arm 3). Five parents and two patients participated in the study to have a chance to be initially treated with etanercept, as initial treatment with etanercept in daily practice is currently neither possible nor reimbursed. The preference of parents and patients for arm 3 was based on their idea that etanercept is the best treatment for juvenile arthritis. The parents indicated that these beliefs were mainly based on knowledge they had gained through the internet and from experiences from people in their environment. Four parents had a preference for a non-prednisone arm. Aversion for prednisone was primary due to the fear for side-effects, such as weight gain. According to four parents, the physician-investigator had a preference for arm 3, but the vast majority of parents (n = 19) stated that the physician-investigator had no preferred strategy. Similar results emerged from the interviews with children.                                                  

Conclusion:

A large part of parents and children in the BeSt for Kids study are not in equipoise. In this study this is not caused by an assumed preference of the physician-investigator but by information on the various treatment possibilities obtained from other sources. The question is whether the absence of equipoise in parents and patients indicates that randomization is unethical or that this equipoise is not feasible in medical research.

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ACR Meeting Abstracts - https://acrabstracts.org/abstract/randomized-clinical-trial-in-pediatric-rheumatology-are-parents-and-patients-in-equipoise/