Clinical trial NCT02260856: A Randomized Controlled Trial of Leg Length Discrepancy Techniques
Official title: PEDS Trial: Percutaneous Epiphysiodesis, Drill Versus Screws for Leg Length Discrepancy a Randomized Clinical Trial
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Leg Length Discrepancy
Keywords: RCT
Epiphysiodesis
Tantalum beads
Patient centered outcome data
Cost effective analysis
Lead sponsor: Hospital for Special Surgery, New York
Sponsor class: OTHER
Collaborators: name: Boston Children's Hospital
name: The Hospital for Sick Children
First posted: 2014-10-09
Enrollment: 170
Sex: ALL
Minimum age (years): 7
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a randomized clinical trial of epiphysiodesis techniques: percutaneous transphyseal screw epiphysiodesis versus percutaneous drill epiphysiodesis for the correction of leg length discrepancy. Failed epiphysiodesis was selected as the primary outcome. Failed epiphysiodesis was defined as one or more of the following: development of angular deformity \> 5°, revision epiphysiodesis, or growth inhibition \< 70% of expected. EOS, low dose biplanar X-Ray, will be used to make all length measurements. Secondary outcomes assess mean growth at the physis following epiphysiodesis, in the subset of patients with bead implantation, fluoroscopy and operative times, length of stay, return to full weight bearing, as well as functional and quality of life outcomes. The following outcome scales will be used to measure pain (VAS), quality of life (PROMIS Pediatric Pain Interference Scale), and function (PROMIS Pediatric Mobility Scale, Pedi-FABS, and UCLA Activity Score). Each of these scales has been validated for use in children (Beyer, Denyes, \& Villarruel, 1992; DeWitt et al., 2011; Fabricant et al., 2013; Novais et al., 2014; Varni et al., 2010a). We will also determine the cost associated with each technique and perform a cost-effectiveness analysis to establish which technique is preferred from a societal perspective.

Detailed description: Drill and screw epiphysiodesis are the two most common techniques for surgical correction of predicted limb length discrepancies 2-5cm. Both procedures require minimal incisions, less than 1cm, (Canale \& Christian, 1990; Metaizeau et al., 1998). Previous studies have demonstrated that both drill and screw epiphysiodesis result in improved outcomes compared to open techniques (Alzahrani, Behairy, Alhossan, Arab, \& Alammari, 2003; Canale \& Christian, 1990). Moreover, alternative approaches such as medial and lateral 8 plates may not sufficiently tether growth, or cause peripheral but not central growth arrest (Stewart et al., 2013). We selected drill vs. screw epiphysiodesis as the two treatment groups as they are both minimally invasive, relatively effective, in common usage, and are thought to differ in costs and other characteristics.

Although outcomes of drill and screw epiphysiodesis exist{{10 Campens,C. 2010; 11 Ghanem,I. 2011}} , the assessments are retrospective non-randomized series, which may be at risk for selection bias, and may not adequately capture all of the outcomes of interest, depending on what data is routinely collected and documented in the medical chart. To our knowledge, no prospective randomized comparison of epiphysiodesis techniques and clinical outcomes has been published accurately assessing how effective each technique is in disabling growth at the physis, or taking into account patient-centered outcomes or cost. Operative measures such as the surgical time and radiation exposure from intra-operative fluoroscopy have not previously been compared. Patient-centered outcomes such as level of pain, activity, and function by measures such PROMIS and Ped-FABS, have not been previously assessed in this population. The proposed research study aims to fill this gap.

This study was designed as a multicenter randomized trial to answer an important clinical question and to do so with a clearly defined objective and validated outcomes. This trial can be executed on a relatively small budget with simple outcome measures, and recruitment of a moderate number of patients at each of a few centers well equipped for research. By involving multiple surgeons and patients from various geographies, we improve the generalizability of this study. Our institution and collaborating institutions have been successful in completing randomized clinical trials in the past. This study will answer a clinical question that is important and current, providing orthopedic surgeons with an evidence-based identification of the ideal technique for treating predicted limb length discrepancies 2-7cm.

This study will also be the first to accurately measure mean growth following both epiphysiodesis techniques, through implantation of tantalum beads in consenting participants. These beads will provide stationary landmarks by which growth can be measured directly, and not inferred. Previous methods of judging epiphysiodesis success have primarily relied on assessing efficacy by estimating growth inhibition through calculation. Tantalum beads have been successfully used in adults and children (Lauge-Pedersen,H. 2006), and although it requires implantation of small radio-opaque beads, it is considered the gold standard when making detailed radiographic measures, and the optimal technique for physeal growth measurements.(Lauge-Pedersen,H. 2006)(Haugan,K. 2012). Over 300 000 beads have been inserted in vivo without significant complications.

Eligibility criteria: Inclusion Criteria:

* Open growth plates
* Skeletally immature requiring isolated complete epiphysiodesis of the distal femur and/or proximal tibia
* At least one year of predicted growth remaining
* Less than 18 years of age
* Predicted limb length discrepancy 2-7 cm

Exclusion Criteria:

* Patients undergoing additional orthopedic procedures at time of epiphysiodesis
* Metabolic bone disease or "sick physis" syndrome, that may cause bone to grow in an unpredictable manner.
* Pregnancy

Primary outcomes:
measure: Failure of Epiphysiodesis
timeFrame: 2-6 weeks, 5-7 months, 11-13 months, 23-25 months, 59-61 months
Secondary outcomes:
measure: Change in Physeal Growth
timeFrame: Baseline, 2-6 weeks, 5-7 months, 11-13 months, 23-25 months, 59-61 months
measure: Time to Baseline Pain
timeFrame: Baseline, 2-6 weeks, 5-7 months, 11-13 months, 23-25 months, 59-61 months
measure: Quality of Life
timeFrame: Baseline, 2-6 weeks, 5-7 months, 11-13 months, 23-25 months, 59-61 months
measure: Length of Hospital Stay
timeFrame: An expected average of 2 days
measure: Time to full weight-bearing
timeFrame: 2- 6 weeks
measure: Need for Splint or Cast
timeFrame: 2- 6 weeks
measure: Days Until Return to Sports
timeFrame: 2-6 weeks
measure: Need for Secondary Surgery
timeFrame: 2-6 weeks, 5-7 months, 11-13 months, 23-25 months, 59-61 months
measure: Physical Activity Level
timeFrame: Baseline, 2-6 weeks, 5-7 months, 11-13 months, 23-25 months, 59-61 months
measure: Physical Activity Level
timeFrame: Baseline, 2-6 weeks, 6 months, 12 months, 24 months, and 60 months
measure: Complications
timeFrame: 2-6 weeks, 6 months, 12 months, 24 months, and 60 months
measure: Medical Costs
timeFrame: 5 years (end of study)
measure: Operative Outcomes
timeFrame: intra-operative
measure: Radiographic Assessments
timeFrame: Baseline, 2-6 weeks, 5-7 months, 11-13 months, 23-25 months, 59-61 months
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: Toronto
country: Canada
facility: Hospital for Sick Children
state: Ontario
Study officials:
affiliation: Hospital for Special Surgery, New York
name: Emily R Dodwell, MD MPH FRCSC
role: PRINCIPAL_INVESTIGATOR
References:
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citation: Lauge-Pedersen H, Hagglund G, Johnsson R. Radiostereometric analysis for monitoring percutaneous physiodesis. A preliminary study. J Bone Joint Surg Br. 2006 Nov;88(11):1502-7. doi: 10.1302/0301-620X.88B11.17730.
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citation: Canale ST, Christian CA. Techniques for epiphysiodesis about the knee. Clin Orthop Relat Res. 1990 Jun;(255):81-5.
citation: Metaizeau JP, Wong-Chung J, Bertrand H, Pasquier P. Percutaneous epiphysiodesis using transphyseal screws (PETS). J Pediatr Orthop. 1998 May-Jun;18(3):363-9.
citation: Stewart D, Cheema A, Szalay EA. Dual 8-plate technique is not as effective as ablation for epiphysiodesis about the knee. J Pediatr Orthop. 2013 Dec;33(8):843-6. doi: 10.1097/BPO.0b013e3182a11d23.
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