METHODS
The literature on the respiratory complications of TEF is based on small, single institution, retrospective case series and case reports. There are no systematic reviews or randomized controlled trials to formulate evidence-based guidelines on diagnosis and treatment. To address this deficiency the RCWG chose a modification of the RAND Appropriateness Method (RAM)21 developed by the RAND Corporation–University of California Los Angeles, that helps synthesize the available empirical evidence using the collective experience of a panel of experts. The RAM approach has been widely used in the development of clinical practice guidelines, including in adult respiratory care22, in primary pediatric care23 and in rare pediatric conditions such as Duchenne Muscular Dystrophy24–26.
The RAM rates diagnostic and therapeutic interventions on the basis of their “appropriateness” and “necessity”. The appropriateness of an intervention is based on its known or expected medical benefit without consideration of any logistical or financial obstacles. Appropriateness does not automatically imply necessity (e.g. bronchoalveolar lavage is an appropriate diagnostic modality for the evaluation of pneumonia, but it is not necessary for every case of pneumonia). The “necessity” is based on whether a) the specific intervention has been deemed appropriate; b) there is reasonable expectation it will benefit the patient in a substantial way; c) according to prevailing standards of care, it would be inappropriate not to offer this intervention to the patient. The RAM enables individual expert opinion to be independently and anonymously expressed and identifies areas of agreement as well as of disagreement or uncertainty that are in need of further study.
The development of the recommendations consisted of the following steps (Figure 1):
Step 1 . Review of the literature andformulation of statements . Questions relating to the domains ofRespiratory Physiology (RP), Diagnostic Methods (DM) andTherapeutic Interventions (TI) for the management of respiratory complications in EA-TEF were generated by literature reviews limited to the English language. Each question was subsequently discussed and reformulated into specific statements during a face-to-face meeting. The agreed upon statements were entered into matrices containing clinical scenarios with a list of potential interventions in 3 areas: Respiratory Pathophysiology (RP) Diagnostic Methods (DM) and Therapeutic Interventions (TI).
Step 2. Rating of statements for “appropriateness” . All statements were individually rated for appropriateness on an ordinal scale of 1-9 as follows: INAPPROPRIATE: ratings 1-3; UNCERTAIN: ratings 4-6; and APPROPRIATE: ratings 7-9. Pathophysiologic mechanisms were rated as “appropriate” according to the degree to which the statement was supported by generally acknowledged pulmonary physiology, currently available literature, and/or by the clinical experience of the responders. Statements in which all responses were in agreement i.e. within the same category were set aside whilst the remaining statements underwent a second and third round of rating. The results of each round were shared within the RCWG without identifying the responders.
Step 3. Rating of statements for “necessity” . Necessity ratings were applied only to statements pertaining to Diagnostic Methods and Therapeutic Interventions and were rated on an ordinal scale 1-9 as follows: UNNECESSARY: 1-3; UNCERTAIN: 4-6; NECESSARY: 7-9. The median, range and the mean (±SD) were calculated. In the final collation, the responses were classified as follows:
Step 4 . Formulation of the recommendations. The recommendations were classified as Level A (based on strong agreement), B (moderate agreement) and C (weak agreement). The ratings on Necessity were formulated into specific recommendations presented in 10 sections (6 for Diagnostic Methods and 4 for Therapeutic Interventions) with their supportive evidence.