Multicenter Prospective Cohort Study on Current Treatments of Legg-Calvé-Perthes Disease

Study Overview
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Brief Summary
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Legg-Calvé-Perthes disease is a childhood hip disorder which is common enough to be a significant public health problem (affects 1 in 740 boys between ages 0-14), but uncommon enough to have a sufficient number of patients from a single institution to perform a definitive prospective study comparing the results of current treatments. The present study will establish a database of prospectively identified patients with Legg-Calvé-Perthes (LCP) Disease and collect information regarding their presentation, treatment, and outcomes in the course of receiving currently available treatments. This study seeks to compare the outcomes of current treatments in the management of different age groups (ages 1-6, 6-8, 8-11, >11) of patients with Perthes disease at two- and five-year followup and at skeletal maturity. For each age group, two to three common treatment regimens currently used by practicing pediatric orthopaedic surgeons will be compared. The intervention a patient receives is determined through physician treatment expertise, and is not pre-determined by the study.

Detailed Description
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Approximately 50 pediatric orthopaedic surgeons from pediatric centers in the US and other countries have agreed to participate in this database as members of the International Perthes Study Group (IPSG). TSRH will be the lead center. Sites who agree to participate will seek IRB approval from their own institutions. Data will be collected prospectively and entered into REDCap (Research Electronic Data Capture), a browser-based research database. 1-6 Cohort: For the 1-6 age patient group, patients involved in any treatment will be asked to participate in the study. 6-8 Cohort: For the 6-8 age patient group, patients presenting in an early stage of the disease (stage I or IIa), the surgeons who treat their patients with one of the following three treatment regimens currently used in practice will be asked to participate in the study : Non-operative management (i.e., no osteotomy but can include soft tissue release); Operative containment treatment (femoral or pelvic osteotomy or Shelf acetabuloplasty). 8-11 Cohort: For the 8-11 patient age group, patients presenting in an early stage of the disease (stage I or IIa), the surgeons who treat their patients with one of the following treatment regiments currently used in practice will be asked to participate in the study: Non-operative management (i.e., no osteotomy); Operative containment treatment (femoral or pelvic osteotomy or Shelf acetabuloplasty) followed by 6 weeks of postoperative non-weight bearing; and, Operative containment treatment (femoral or pelvic osteotomy or Shelf acetabuloplasty) followed by 6 months of postoperative non-weight bearing. >11 Cohort Registry: For the >11 patient age group, we will collect prospective data from surgeons who are currently treating their patients with any treatment regimens. Patients who present to an IPSG member or their group during the late stages of the disease (stage IIb or stage III) will also be arranged into four separate age cohorts, identical to the list above. We will collect prospective data for these cases, as well. Regardless of the patient's stage at enrollment, the patient's age at diagnosis or assigned cohort, the following data will be collected: information regarding patient characteristics at presentation, physical exam findings, responses to Perthes patient and/or outcomes questionnaires, and results of radiographic and MR imaging. Participants will be assigned a unique study number. A secure web application, REDCap, will be used to capture and store research information including radiographic and MR images, clinical information, and de-identified outcomes questionnaire responses. Data will be transmitted and stored on a secure and dedicated server for the purpose of this study.

Official Title
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Multicenter Prospective Cohort Study on Current Treatments of Legg-Calvé-Perthes Disease

Conditions
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Legg Calve Perthes Disease

Intervention / Treatment
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* Procedure: Osteotomy + Long Term Non-Weight Bearing
* Procedure: Osteotomy + Short Term Non-Weight Bearing
* Procedure: Nonoperative Observation
* Procedure: Multiple Epiphyseal Drilling


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosed with Legg-Calvé-Perthes disease Between age 1-18 Patients with possible secondary femoral osteonecrosis if over the age of 11 due to trauma or corticosteroid therapy are also eligible. Exclusion Criteria: Patients with previous surgical treatment on the affected hip

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Nonoperative management between ages 6-8 in early stage<br>The choice of non-osteotomy management without containment or surgical containment would be solely governed by the current practice of the participating surgeons (i.e. the current practice of the individual surgeon would be followed within the broad framework of the study). | Procedure: Nonoperative Observation<br>* Group will not undergo any surgical or invasive procedures during course of treatment.<br>|
| Operative management between age 6-8 in early stage<br>Operative containment treatment (femoral or pelvic osteotomy or Shelf acetabuloplasty) rendered in the early stage of the disease process | Procedure: Osteotomy + Long Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that requires patient to maintain non-weight bearing for 6 months.<br>Procedure: Osteotomy + Short Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that allows early return to function after 6 weeks of non-weight bearing.<br>|
| Nonoperative management between age 8-11 in early stage<br>Patients who do not undergo some form of containment surgery because of medical, social, or other reasons will receive no surgical treatment. | Procedure: Nonoperative Observation<br>* Group will not undergo any surgical or invasive procedures during course of treatment.<br>|
| Operative containment with short-term non-weightbearing in early stage<br>As per the current standard practice for patients between age 8-11 in developed countries, all patients will undergo some form of containment surgery (pelvic or femoral osteotomy) in order to better contain the femoral head underneath the acetabulum. Post-operative treatment will include 6 weeks of non-weight bearing on the operated leg. | Procedure: Osteotomy + Short Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that allows early return to function after 6 weeks of non-weight bearing.<br>|
| Operative containment with prolonged non-weightbearing in early stage<br>As per the current standard practice for patients between age 8-11, all patients will undergo some form of containment surgery (pelvic or femoral osteotomy) in order to better contain the femoral head underneath the acetabulum. Post-operative treatment will include 6 months of non-weight bearing on the operated leg. | Procedure: Osteotomy + Long Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that requires patient to maintain non-weight bearing for 6 months.<br>|
| Operative containment for over 11 age group<br>Patients presenting over age 11 may receive multiple drilling and be non weight bearing for up to 6 months according to the treating physician's preference. Patients with application of fixator (arthrodiastasis) will be studied in this cohort arm as well, which typically involved 3-4 months followed by 8-12 weeks of non-weight bearing after fixator removal. Other surgical techniques may be included, however these are the most common for this age group. | Procedure: Osteotomy + Long Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that requires patient to maintain non-weight bearing for 6 months.<br>Procedure: Osteotomy + Short Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that allows early return to function after 6 weeks of non-weight bearing.<br>Procedure: Multiple Epiphyseal Drilling<br>* Multiple epiphyseal drilling is a procedure that creates small holes in the femoral head growth plate to increase blood flow into the femoral head.<br>|
| Nonoperative management in over 11 age group<br>Patients will be non-weight bearing and receive physical therapy according to the physician preferences. | Procedure: Nonoperative Observation<br>* Group will not undergo any surgical or invasive procedures during course of treatment.<br>|
| Nonoperative management in 1-6 age group<br>The choice of non-osteotomy management without containment or surgical containment would be solely governed by the current practice of the participating surgeons (i.e. the current practice of the individual surgeon would be followed within the broad framework of the study). | Procedure: Nonoperative Observation<br>* Group will not undergo any surgical or invasive procedures during course of treatment.<br>|
| Operative management in 1-6 age group<br>The choice of osteotomy management with containment would be solely governed by the current practice of the participating surgeons (i.e. the current practice of the individual surgeon would be followed within the broad framework of the study). | Procedure: Osteotomy + Long Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that requires patient to maintain non-weight bearing for 6 months.<br>Procedure: Osteotomy + Short Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that allows early return to function after 6 weeks of non-weight bearing.<br>Procedure: Multiple Epiphyseal Drilling<br>* Multiple epiphyseal drilling is a procedure that creates small holes in the femoral head growth plate to increase blood flow into the femoral head.<br>|
| Late Stage Bracing group<br>Patients presenting with <= 20 degrees of abduction on a maximum abduction x-ray treated with bracing who also present in the late stages of the disease (Waldenstrom Stage IIb or IIIa).The choice of management with or without containment or surgical containment would be solely governed by the current practice of the participating surgeons (i.e. the current practice of the individual surgeon would be followed within the broad framework of the study). | Procedure: Nonoperative Observation<br>* Group will not undergo any surgical or invasive procedures during course of treatment.<br>|
| Late Stage Symptomatic treatment group<br>Patients presenting in the late stages of the disease (defined as Waldenstrom stage IIb or IIIa) who are treated symptomatically. The choice of management with or without containment or surgical containment would be solely governed by the current practice of the participating surgeons (i.e. the current practice of the individual surgeon would be followed within the broad framework of the study). | Procedure: Nonoperative Observation<br>* Group will not undergo any surgical or invasive procedures during course of treatment.<br>|
| Late Stage Surgical Containment group<br>Patients presenting in the late stages of the disease (defined as Waldenstrom stage IIb or IIIa) who are treated with surgical containment. The choice of management with or without containment or surgical containment would be solely governed by the current practice of the participating surgeons (i.e. the current practice of the individual surgeon would be followed within the broad framework of the study). | Procedure: Osteotomy + Long Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that requires patient to maintain non-weight bearing for 6 months.<br>Procedure: Osteotomy + Short Term Non-Weight Bearing<br>* Surgical procedure that improves femoral head containment, plus a post-operative protocol that allows early return to function after 6 weeks of non-weight bearing.<br>Procedure: Multiple Epiphyseal Drilling<br>* Multiple epiphyseal drilling is a procedure that creates small holes in the femoral head growth plate to increase blood flow into the femoral head.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Sphericity deviation score of the femoral head | The primary outcome for each age group will be a quantitative estimate of the sphericity of the femoral head as a measure of femoral head deformity. A more deformed head will be less spherical. Greater deformity leads to an increased risk of arthritis early in life. | 5 years post intervention | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Perfusion percentage | The perfusion percentage is a measure of the amount of blood flow in the femoral head relative to the volume of the whole head. This measure is calculated from the perfusion MRI images that are collected just prior to the application of the intervention. | Just after diagnosis of Perthes disease | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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femur head necrosis, hip, pediatric orthopedics, MRI, Osteonecrosis, Bone diseases, Legg Calve Perthes Syndrome