Situational Ethics of Study Designs for Orthopaedic Surgery Interventions in Children With Cerebral Palsy

Tamer A. El-Sobky, MD; Mohamed El-Sayed, MD

Disclosures

Curr Orthop Pract. 2019;30(2):178-180. 

In This Article

Ethics Versus Evidence

Well-conducted RCTs provide a gold standard by which the clinical research validity is evaluated despite ethical and practical limitations (Figure 1). The ethical limitations of RCTs are multifold. First, there should be an ultimate degree of uncertainty as to which treatment group is better for a child with CP. That applies to both the intervention group and the control group. This ultimate status of uncertainty is known as clinical equipoise or counterbalance.[4] The presence of any clinical or academic clues to favor one group - intervention or control - over the other will eventually compromise the ethical integrity of the study, at least in theory, and this will put the patient group allocated to receive the potentially inferior treatment at health risks.[4] Needless to say, achieving clinical equipoise or equilibrium is mandatory in RCTs involving human participants. Nonetheless, an important lingering question remains. Is it realistic to expect strictly implemented RCTs to fully address the clinical equipoise and at the same time satisfy the clinical evidence aspects? In fact, finding a straightforward answer to this question seems exceedingly difficult. Nevertheless, RCTs with the aim of assessing the short-term outcomes of nonsurgical orthopaedic interventions,[5,6] have been implemented on children with CP without remarkable ethical barriers. Single-blinded[7] and double-blinded[8,9] RCTs have been proposed to assess the effectiveness of nonsurgical interventions in children with CP, such as goal-oriented motor or cognitive training and enrichment intervention programs. Furthermore, these protocols have been designed to study CP in children as young as 12 wk old.[9] It is noteworthy that short-term studies (1 yr) do not really put the matched control (non-intervention) group at risk of functional deterioration in contrast to the treatment-intervention group.[5–8,10] This is because some children with CP are by default on the waiting list for other major interventions or are receiving the regular physiotherapy or neurorehabilitation protocols. Likewise, RCTs with the aim of assessing the short-term outcomes of orthopaedic interventions[1,2] have been implemented in children with CP without remarkable ethical barriers. However, these short-term studies usually have limited generalizability of conclusions and do little to guide clinical decision making, especially when applied to major orthopaedic surgical interventions, such as hip reconstruction and multilevel surgery. For example, in CP dislocation the emergence of redislocation after bony hip reconstruction is most appropriately evaluated long-term. Optimally these children should be followed to skeletal maturity.[11] Short-term follow-up RCTs can entirely overlook important complications and treatment end points. Consequently, they cannot demonstrate the maintainability of the results in the long-term. This undermines the ability of these short-term RCTs to produce credible evidence and generalizable conclusions. Contrasting, long-term follow-up RCTs can produce strong evidence and generalizable conclusions. Nonetheless, these long-term RCTs represent a serious breach of ethical foundations and patients' rights (Figure 2). This is because such study designs put the control group that receives no or regular treatment at risk of functional deterioration.[10,12,13] Hence, reaching the optimal state of clinical equipoise is enormously challenging at best, especially in regard to long-term studies involving major surgical orthopaedic interventions in the treatment group and no treatment in the control group. This is of great relevance to the control group as the orthopaedic manifestations of CP are progressive. Not surprisingly, the use of RCTs in major orthopaedic surgery interventions is limited.[3]

Figure 1.

Hierarchy of evidence-based medicine.

Figure 2.

Randomized controlled trials for major orthopaedic surgery interventions in children with cerebral palsy.

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