Spontaneous Pneumothorax in Children
Condition: Spontaneous Pneumothorax
MeSH terms: Pneumothorax
Study type: Interventional

Summary: 
      The objective of this multi-center, non-randomized, prospective pilot study is to examine the
      rate of successful primary spontaneous pneumothorax (PSP) resolution using the simple
      aspiration technique.

      In this study, eligible subjects diagnosed with PSP at eleven participating large children's
      hospitals (members of the Midwest Pediatric Surgical Clinical Research Consortium) will be
      enrolled and offered a choice of management with either the simple aspiration protocol or
      management according to their surgeon's preference, which may include simple aspiration,
      chest tube placement, or rarely, an operation.
    

Detailed description: 
      This is a prospective, non-randomized, multi-center pilot study involving children (age 12-17
      years old) who present with a first episode of primary spontaneous pneumothorax (PSP) that
      requires treatment. Treatment with simple aspiration has been researched in adults but it has
      not been investigated in children. For this reason, this will be a pilot study design with
      two non-randomized groups. Since some pediatric surgeons have started to offer this treatment
      in children and some of the adult studies include adolescent patients, simple aspiration is
      considered a standard of care treatment at many institutions, and is performed as part of
      standard care at UW. Patient and Provider communication regarding treatment options and the
      Patient and Provider choice choice of treatment play a large role both in the standard of
      care for PSP and this research study.

      Potential subjects will be identified by a consulting surgeon (who will be a study team
      member as all pediatric surgeons are on this application) when surgical consultation is
      requested for acute management of a child with spontaneous pneumothorax. This most commonly
      occurs in the emergency department after a chest X-ray is obtained as part of the usual
      evaluation for the associated symptoms of pain and shortness of breath. Medical records may
      be reviewed to confirm eligibility prior to approaching potential subjects and their
      parents/guardians for assent/consent. The consulting surgeon will also assess patient
      eligibility by screening for inclusion and exclusion criteria. If all eligibility criteria
      are met, the consulting surgeon will notify a member of the study staff about the potential
      subject.

      A member of the research team will explain the study rationale and procedures in detail with
      potential subjects and their legal guardian upon surgical consultation. All written
      information will be reviewed and all questions answered. A study physician will be available
      at all times for any consent-related questions. The potential subject and legal guardian will
      have up to one hour to consider if they would like to participate in the study. The child's
      legal guardian will sign the informed consent, and the subject will sign assent.

      Once consent/assent is obtained subjects and their parents/guardians will have the choice of
      consenting to initial management with either the simple aspiration protocol (aspiration
      group) or according to the preferences of the treating surgeon (preference group). All
      patients will be properly informed of the options for each arm during the consent discussion,
      most of the information is conveyed during the clinical treatment discussion with the
      patients which is then incorporated into the consent discussion. Patient preference and
      choice of treatment plays a large role in the standard of care methods for treating PSP and
      subject preference and choice will play just as strong a role in this research study.

      As a pilot study, we aim to enroll 32 subjects in the aspiration arm across all participating
      institutions and 48 subjects will enroll into the preference arm. Since all medical
      treatments performed during the study are considered standard of care, the only research
      components of this study include the subject's self-allocation to a particular study arm
      (even though patient choice is just as strong a role in standard of care, there are defined
      arms for this research study) and the recording of medical and billing data for each subject,
      as well as a follow-up phone call to the subject.

      Aspiration Arm:

      Subjects in this arm will undergo initial management of their pneumothorax with a simple
      aspiration procedure according to standard care described here. The procedure will involve
      placement of a small catheter into the chest cavity and applying negative pressure to
      manually aspirate the air out of the chest cavity, which will allow the lung to re-expand.
      The aspiration catheter will be less than or equal to 12 French in diameter. Further details
      of the type of device and size of catheter will be determined by the surgeon and will depend
      on the devices available for standard practice at each participating institution. The
      procedure may be performed with local anesthetic, with or without sedation, depending on
      surgeon judgment and available resources or standard practice at each participating
      institution. The procedure may be performed with or without image guidance (i.e. ultrasound
      or fluoroscopy) during the aspiration, but a chest X-ray must be obtained at the conclusion
      of the procedure, with the catheter still in place, to verify successful expansion of the
      lung and for comparison with subsequent chest X-rays. A small residual pneumothorax after the
      initial aspiration procedure may still be deemed a success. Successful aspiration at the time
      of the initial aspiration procedure will be defined as: expansion of the lung apex to the top
      of the 4th rib or higher and expansion at the lateral chest wall to the 6th rib or higher. A
      failure will be defined as a residual pneumothorax that is not limited to the apex and
      extends laterally down the chest wall below the 6th rib. If the first post-procedure chest
      X-ray demonstrates failure of adequate lung expansion, the existing catheter may be used for
      one additional aspiration attempt, with application of negative pressure followed by another
      chest X-ray.

      After the aspiration procedure, the subject will be admitted to the hospital for a minimum
      observation period of 6 hours. The small catheter will be capped and left in place and the
      insertion site covered by an occlusive dressing, such that this same catheter could be
      utilized in the event of failure of the aspiration procedure. After 6 hours, a second chest
      X-ray will be obtained to assess for recurrent pneumothorax or enlargement of a small
      residual pneumothorax. If the pneumothorax is stable or smaller in size after 6 hours of
      observation, then the catheter will be removed and the patient discharged to home. However,
      if the pneumothorax recurs or enlarges after 6 hours, then the aspiration procedure has
      failed. The existing catheter could be uncapped and used as an indwelling chest tube for a
      more prolonged period of chest drainage, but all management is according to the surgeon's
      preference and standard practice. At any time, if the aspiration procedure is determined to
      be a failure, by either inadequate lung expansion or recurrent pneumothorax, the patient will
      be managed according to the judgment and standard practice of the treating surgeon.

      Preference Arm:

      Subjects that choose the surgeon preference arm of the study are enrolling for prospective
      data collection only. These subjects will not have any portion their care directed by the
      study protocol. The decision to proceed with any treatment or intervention will be made
      jointly by the surgeon and the patient and his or her legal guardian. Any standard treatment
      option may be utilized, including simple aspiration, chest tube placement, or an operation,
      video-assisted thoracoscopic surgery (VATS). If the surgeon's preference and standard
      treatment is simple aspiration but the patient does not want the aspiration procedure, then
      the surgeon will perform an alternative treatment option, such as chest tube placement or
      VATS.

      Management strategies in the preference group may include placement of an indwelling chest
      tube, with or without thoracoscopic resection of blebs (VATS), and may also include a simple
      aspiration procedure if recommended by the treating surgeon.

      For both arms, a telephone survey will be conducted at approximately one year from
      enrollment. The primary purpose is to ascertain whether the subject has sought additional
      medical treatment at another facility for unresolved or recurrent pneumothorax and time to
      recurrence. Additionally, we wish to know if the subject has ongoing or intermittent episodes
      of symptoms attributed to pneumothorax for which he/she has not sought medical treatment. A
      yearly chart audit will continue for 5 additional years to determine if any further treatment
      was needed for the initial pneumothorax.
    

Intervention name(s): Simple Aspiration; Surgeon Preference
Intervention type(s): Procedure; Procedure
Intervention description(s): Aspiration of pneumothorax with a small-bore catheter; Any standard treatment option may be utilized for the treatment of the pneumothorax, including simple aspiration, chest tube placement, or an operation (VATS).
Other intervention name(s): Thoracentesis
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment