Primary Versus Delayed Surgical Therapy for Pediatric Spontaneous Pneumothorax

Study Overview
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Brief Summary
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The purpose of this study is to compare the effectiveness of two treatment strategies--delayed versus immediate surgery-- for children with primary spontaneous pneumothorax (collapse of the lung). Currently, both treatment modalities are used and there is no clear evidence that either option is superior. The investigators hypothesize that immediate surgery will have better outcomes with lower recurrence rates than delayed surgery.

Detailed Description
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All children presenting to Texas Children's Hospital with a primary spontaneous pneumothorax will be potentially eligible for the study. After the diagnosis has been made and initial medical therapy has been initiated, the patients will be identified and parents or legal guardian approached by the staff surgeon for consent to participate in the study. The attending pediatric surgeon or surgical fellow will discuss the trial with all eligible patients and their families. Written, informed consent will be obtained by the research nurse coordinator, a pediatric surgical fellow, or an attending pediatric surgeon. Once enrolled, the patients will be randomized to primary surgery: Video-Assisted Thoracoscopic Surgery (VATS), blebectomy and mechanical pleurodesis, versus initial nonoperative treatment. Prior to initiating the study, the randomization sequence will be computer-generated by an independent statistician using a permuted block random allocation scheme with a block size of 4,6. The randomization sequence will be maintained securely within the randomization module in REDCap (Research electronic data capture), and it will be accessed only after enrollment has occurred and inclusion criteria are verified by the admitting staff surgeon. The participants and the surgeons will not be blinded to group assignment. Primary Surgery Group. Patients randomized to the primary surgical intervention group will undergo VATS, apical blebectomy and mechanical pleurodesis during the initial hospital admission by the admitting staff surgeon. The general principles of the surgical technique consist of a 3-port thoracoscopic approach, stapled blebectomy, apical mechanical pleurodesis, and placement of chest tube . Variations of this technique will be at the discretion of the surgeon. Initial non-operative management group. Those randomized to the control group will be admitted and their chest tube or percutaneous drainage catheter managed according to standard protocol. This consists of a minimum of 48 hours of Pleur-Evac suction and daily chest radiographs. The drainage tube is then placed to water seal when resolution of the pneumothorax is documented by x-ray, as well as absence of an air leak. If there are no clinical or radiographic changes after a water seal period, the chest tube is then removed. A post-removal chest radiograph is obtained and the patient is discharged if clinical and radiographic criteria are met. Rescue VATS/ blebectomy/pleurodesis will be performed in those managed initially nonoperatively who meet criteria for surgery thereafter (i.e. develop a persistent air leak after drainage tube placement). A persistent air leak will be defined as bubbling of air in the Pleur-Evac water chamber upon eliciting positive pressure lasting >4 days, noted and documented in progress note by attending physician. If recurrence is identified in the control group, it will be managed operatively, as per current clinical practice, with VATS/ blebectomy/pleurodesis. Discharge Criteria. Discharge criteria include being afebrile for 24 hours, normal oxygen saturation with no oxygen requirement, benign physical exam, unchanged or improved post-removal chest radiograph, ability to tolerate regular diet and ambulate, and pain well controlled on oral pain medication. Discharge Instructions. A discussion will take place between the surgical team and the family prior to discharge in order to review standardized discharge instructions. These will address an understanding by the patient and family of the signs and symptoms of recurrent pneumothorax as well as specific follow up appointments and the scheduled phone follow-up thereafter. For patients who have had primary surgery, specific postoperative instructions will be given according to current clinical practice. Follow-up. Follow-up will be the same for both groups and will take place at 2-3 weeks after hospital discharge (per standard of care), with subsequent telephone interviews at 3 months, 6 months and 12 months. The primary outcome regarding effectiveness of primary VATS will be the overall recurrence rate at 12 months. Secondary outcomes include surgical complication rate, cumulative hospital length of stay (total number of hospital days during study period), time to resumption of usual activities, and direct variable hospital costs. The recurrence rate is defined as the proportion of patients with recurrent ipsilateral pneumothorax during the follow-up period. Complications to be assessed include surgical site infection, persistent air leak (> 4 days), and prolonged need for chest tube (as quantified by number of chest tube days). Standard National Surgical Quality Improvement Program (NSQIP) definitions of these outcomes will be utilized. The time to resumption of usual activities will be adjudicated at the time of the post-op follow up visit or by means of a phone interview at 2-3 weeks postoperatively. The longest time period to resumption all usual activities will be ascertained from patient questionnaires addressing return to school date, level of pre- and post-illness activity, and daily activity log kept by the patient or their parent. Post-operative quality of life will be assessed at 3 months, 6 months, and 12 months by telephone administration of The PedsQL Measurement Model for the Pediatric Quality of Life Inventory, a widely-used and validated tool.

Official Title
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Primary Versus Delayed Surgical Therapy for Pediatric Spontaneous Pneumothorax: A Randomized Controlled Trial

Conditions
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Pneumothorax

Intervention / Treatment
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* Procedure: Primary Surgery Group
* Procedure: Initial Non-operative management


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children ages 5 to 18 with clinical diagnosis of primary spontaneous pneumothorax (symptoms of chest pain and/or shortness of breath and pneumothorax demonstrated on chest radiograph) and no prior history of pneumothorax Exclusion Criteria: Blunt or penetrating trauma Cystic fibrosis Pneumonia Uncontrolled asthma with hospitalization for exacerbation within previous 30 days Congenital cystic adenomatoid malformation or congenital lobar emphysema History of previous cardiac or pulmonary surgery

Ages Eligible for Study
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Minimum Age: 5 Years
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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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Primary Surgery Group<br>Patients randomized to the primary surgical intervention group will undergo VATS (video-assisted thoracoscopic surgery), apical blebectomy and mechanical pleurodesis during the initial hospital admission by the admitting staff surgeon. The general principles of the surgical technique consist of a 3-port thoracoscopic approach, stapled blebectomy, apical mechanical pleurodesis, and placement of chest tube . Variations of this technique will be at the discretion of the surgeon. | Procedure: Primary Surgery Group<br>* Patients will receive surgical intervention during the initial hospital admission, as previously described, for the spontaneous pneumothorax. Patients will then be admitted postoperatively and monitored until discharge criteria are met<br>|
| Active Comparator: Initial Non-operative management<br>Those randomized to the control group will be admitted and their chest tube or percutaneous drainage catheter managed according to standard protocol. This consists of a minimum of 48 hours of Pleur-Evac suction and daily chest radiographs. The drainage tube is then placed to water seal when resolution of the pneumothorax is documented by x-ray, as well as absence of an air leak. If there are no clinical or radiographic changes after a water seal period, the chest tube is then removed. A post-removal chest radiograph is obtained and the patient is discharged if clinical and radiographic criteria are met. | Procedure: Initial Non-operative management<br>* Patients will receive percutaneous drainage or chest tube placement as initial management for spontaneous pneumothorax. This will be followed by an observation period until discharge criteria are met.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Recurrence rate | The recurrence rate is defined as the proportion of patients with recurrent ipsilateral pneumothorax during the follow-up period. | 1 month | 
| Recurrence rate | The recurrence rate is defined as the proportion of patients with recurrent ipsilateral pneumothorax during the follow-up period. | 3 months | 
| Recurrence rate | The recurrence rate is defined as the proportion of patients with recurrent ipsilateral pneumothorax during the follow-up period. | 6 months | 
| Recurrence rate |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Postoperative complications | Surgical site infection (NSQIP definition), persistent air leak (> 4 days), and prolonged need for chest tube (as quantified by number of chest tube days) | 30 days | 
| Hospital Length of stay |  | 1 month | 
| Time to return to normal activities |  | 1 month, 3 months, 6 months, 12 months | 
| Hospital Direct Variable Costs (in US dollars, from hospital cost-accounting department) |  | 1 month | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Spontaneous Pneumothorax, Video Assisted Thoracoscopic Surgery, Tube Thoracostomy, Pediatric Surgery