Clinical trial NCT02449980: Primary Versus Delayed Surgical Therapy for Pediatric Spontaneous Pneumothorax
Official title: Primary Versus Delayed Surgical Therapy for Pediatric Spontaneous Pneumothorax: A Randomized Controlled Trial
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Pneumothorax
Keywords: Spontaneous Pneumothorax
Video Assisted Thoracoscopic Surgery
Tube Thoracostomy
Pediatric Surgery
Lead sponsor: Baylor College of Medicine
Sponsor class: OTHER
First posted: 2015-05-21
Enrollment: 3
Sex: ALL
Minimum age (years): 5
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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: 1. All children presenting to Texas Children's Hospital with a primary spontaneous pneumothorax will be potentially eligible for the study.
2. 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.
3. Written, informed consent will be obtained by the research nurse coordinator, a pediatric surgical fellow, or an attending pediatric surgeon.
4. Once enrolled, the patients will be randomized to primary surgery: Video-Assisted Thoracoscopic Surgery (VATS), blebectomy and mechanical pleurodesis, versus initial nonoperative treatment.
5. 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.
6. 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.
7. 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.
8. 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.
9. 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.
10. 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.
11. 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.

Eligibility criteria: 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

Primary outcomes:
measure: Recurrence rate
timeFrame: 1 month
measure: Recurrence rate
timeFrame: 3 months
measure: Recurrence rate
timeFrame: 6 months
measure: Recurrence rate
timeFrame: 12 months
Secondary outcomes:
measure: Postoperative complications
timeFrame: 30 days
measure: Hospital Length of stay
timeFrame: 1 month
measure: Time to return to normal activities
timeFrame: 1 month, 3 months, 6 months, 12 months
measure: Hospital Direct Variable Costs (in US dollars, from hospital cost-accounting department)
timeFrame: 1 month
Other outcomes:
Locations:
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
Study officials:
affiliation: Baylor College of Medicine
name: Monica E Lopez, MD
role: PRINCIPAL_INVESTIGATOR
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