Clinical trial NCT04614974: Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia
Official title: Initiation of Acid Suppression Therapy Prospective Outcomes for Laryngomalacia
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Laryngomalacia
Gastro Esophageal Reflux
Keywords: Laryngomalacia
Symptom improvement
Famotidine
Speech Language Therapy
Lead sponsor: University of Pittsburgh
Sponsor class: OTHER
First posted: 2020-11-04
Enrollment: 65
Sex: ALL
Minimum age (years): 0
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Experimental, open-label randomized control trial
Primary purpose: TREATMENT

Brief summary: Laryngomalacia (LM) is the most common cause of stridor in infants. Symptoms of gastroesophageal reflux (GER) are often seen in the setting of LM; therefore, acid suppression therapy (AST) has been empirically used in the management of this disorder. The investigators recently performed a retrospective chart review assessing improvement of airway and dysphagia symptoms, weight gain, and need for surgery with AST. It was found that there was a similar improvement between LM severity groups and most patients received AST (96.6%). It is unclear if these improvements are due to AST or natural resolution of the disease. With heightened concerns of side effects related to AST in infants, particularly among those born prematurely, judicious use of these medications is needed. The investigators are now performing a prospective study looking at the outcome differences in patients with laryngomalacia who are evaluated by speech language therapy (SLP) alone versus those with SLP evaluation and acid suppression therapy (famotidine).

Detailed description: Purpose: To determine the outcome differences in patients 6 months and younger with laryngomalacia and dysphagia who are evaluated by speech language therapy (SLP) alone versus those with both SLP evaluation and acid suppression therapy (AST) (famotidine)

Hypothesis: There will be no differences in outcomes between those that had SLP alone versus those that had both SLP and AST.

Laryngomalacia (LM) is the most common cause of stridor in infants. Symptoms of gastroesophageal reflux (GER) are often seen in the setting of LM; therefore, acid suppression therapy (AST) has been empirically used in the management of this disorder. However, there is no gold standard in treating mild and moderate LM patients and therefore this study will help establish guidelines for treatment.

A medical chart review will be performed to assess airway and dysphagia symptom improvement from consult to the 3-month follow up appointment and then up to a year. The Pittsburgh Airway Symptom Score (PASS) questionnaire and the Infant Gastroesophageal Reflux Questionnaire (I-GERQ-R) will be given to families at the consult and at the 3-month follow up appointment for caregiver assessment. The physician will then perform the standard procedures at the clinic appointment. The Flexible Laryngoscopy Findings sheet will be filled out in conjunction with the clinic procedures for objective data. The surveys will then be scored to determine true GERD (score \>=16) and severe laryngomalacia (LM) (RED questions on the PASS), which would further exclude these patients. There are 12 total questions on the I-GERQ-R. On the PASS, questions 1 \& 2 are in the GREEN category and signifies mild LM, YELLOW signifies moderate LM, and RED is severe LM. On the PASS questionnaire, "Yes" to either #1 or #2 and nothing else is mild LM, "yes" to at least one #3-5 and none of #6-10 is moderate LM, and "yes" to any of the #6-10" indicates severe LM. There are 10 total questions on the PASS. Mild and moderate LM patients will be block randomized the day before the appointment to receive speech language therapy alone or speech language therapy with famotidine (Pepcid). Both treatments are standard of care in these patients. Speech language therapy (feeding therapy) is part of the normal clinic visit for LM patients. These patients will then be re-evaluated at their follow up appointment in 3 months (+/- 1 month). The families will take the PASS and I-GERQ-R surveys again to determine LM severity.

Eligibility criteria: Inclusion Criteria:

* Pediatric patients ages 0 to 6 months who do need meet the criteria at the initial appointment for supraglottoplasty
* Seen in University of Pittsburgh Medical Center (UPMC) Children's Hospital of Pittsburgh (CHP) Otolaryngology Department
* Laryngomalacia without prolonged (\>20 seconds) cyanosis, apnea, nor failure to thrive.

Exclusion Criteria:

* Children over the age of 6 months old will be excluded from participation.
* Premature infants (\<37 weeks gestation)
* Patients with lung disease.
* Laryngomalacia with prolonged (\>20 seconds) cyanosis, apnea, and failure to thrive
* Sleep induced laryngomalacia
* Patients with craniofacial abnormalities
* Patients with a syndrome
* Patients with additional airway abnormalities, seen before or at consult
* Patients with symptoms that necessitate surgery
* Patients with a prior cardiac surgery
* Patients with AST prescribed prior to the initial otolaryngology consult.

Primary outcomes:
measure: Airway symptom score change from consult (baseline) to 3 month follow-up appointment
timeFrame: 3 months
measure: Dysphagia symptom score change from consult (baseline) to 3 month follow-up appointment
timeFrame: 3 months
measure: Change in incidence of airway symptoms from consult up to 1 year assessed via electronic medical chart review
timeFrame: 1 year
measure: Change in incidence of dysphagia symptoms from consult up to 1 year assessed via electronic medical chart
timeFrame: 1 year
Secondary outcomes:
measure: Weight (kg) from consult up to 1 year
timeFrame: 1 year
measure: Infant Gastroesophageal Reflux Questionnaire (I-GERQ-R) score change from consult (baseline) to 3 month follow-up appointment
timeFrame: 3 months
measure: Number of participants with the need for supraglottoplasty surgery (escalation of treatment) up to 1 year
timeFrame: 1 year
measure: Number of participants with the need for acid suppression therapy medication (famotidine) from speech language therapy alone group from the day after the consult up to the 3 month follow up appointment
timeFrame: 3 months
measure: Number of participants with each type of laryngomalacia (Types 1-3) found on the Flexible Laryngoscopy procedure at the consult
timeFrame: At initial consult
measure: Number of participants with the need for a different acid suppression therapy medication (other than famotidine) from the day after the consult up to the 3 month follow up appointment
timeFrame: 3 months
Other outcomes:
Locations:
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
Study officials:
affiliation: Division of Pediatric Otolaryngology, Children's Hospital of Pittsburgh
name: Reema Padia, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Rosbe KW, Kenna MA, Auerbach AD. Extraesophageal reflux in pediatric patients with upper respiratory symptoms. Arch Otolaryngol Head Neck Surg. 2003 Nov;129(11):1213-20. doi: 10.1001/archotol.129.11.1213.
citation: Hartl TT, Chadha NK. A systematic review of laryngomalacia and acid reflux. Otolaryngol Head Neck Surg. 2012 Oct;147(4):619-26. doi: 10.1177/0194599812452833. Epub 2012 Jun 27.
citation: Bibi H, Khvolis E, Shoseyov D, Ohaly M, Ben Dor D, London D, Ater D. The prevalence of gastroesophageal reflux in children with tracheomalacia and laryngomalacia. Chest. 2001 Feb;119(2):409-13. doi: 10.1378/chest.119.2.409.
citation: Landry AM, Thompson DM. Laryngomalacia: disease presentation, spectrum, and management. Int J Pediatr. 2012;2012:753526. doi: 10.1155/2012/753526. Epub 2012 Feb 27.
citation: Thompson DM. Laryngomalacia: factors that influence disease severity and outcomes of management. Curr Opin Otolaryngol Head Neck Surg. 2010 Dec;18(6):564-70. doi: 10.1097/MOO.0b013e3283405e48.