Clinical Trial: Probiotics and Corticosteroids for Treating Periodic Fever, Aphthous Stomatitis, Pharyngitis, Cervical Adenitis (PFAPA)

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Effects of Probiotic Addition to Corticosteroid Treatment for PFAPA

Brief Summary: The purpose of study is to see if adding probiotics to corticosteroid treatment for children with PFAPA could improve the health and daily of patients through reduction in febrile period frequency and length, along with concomitant reduction of associated symptoms. Current standard of care incorporates the administration of corticosteroids; however, while limiting the symptoms associated with PFAPA, corticosteroid use has been shown to increase the frequency at which these symptoms occur. Investigators hypothesize that administration of probiotics along with corticosteroids will work to decrease the frequency at which the febrile episodes occur. Additionally, probiotics may decrease the maximal fever experienced during these episodes, amount of corticosteroid needed to control the symptoms, average length of the episodes, and the number of patients who ultimately undergo tonsillectomy due to unsuccessful treatment with medication.

Detailed Summary:

Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) was first described in 1987 in a study of 12 pediatric patients. PFAPA is predominately considered a pediatric illness with onset typically occurring under the age of 5. This syndrome is characterized by periodic febrile episodes (39°C - 42°C) lasting an average of 4 days with recurrence approximately every 21-30 days. Patients classically exhibit at least one of three major associated symptoms; aphthous stomatitis, pharyngitis, or cervical adenitis. Between periods of fever, patients are asymptomatic and do not experience long-term deleterious effects on growth or development. Currently, the etiology of this syndrome is unknown but does not appear to be heritable or target specific populations.

Due to a lack of applicable diagnostic tests, the identification of PFAPA is predominantly dependent on patient history and physical examination. This methodology relies heavily upon clinical presentation of the patient's illness to rule out other causes of periodic fever. As a result of the unknown cause of PFAPA, there are several proposed treatment approaches available. Corticosteroids are the current standard of care; typically a single dose of prednisolone (1 mg/kg), given upon first signs of fever with a subsequent dose during the febrile period if necessary. Several studies have shown that administration of prednisolone was sufficient to abort the febrile episode. However, this treatment method typically results in a decrease in the healthy interval between fever reoccurrence. The reduction in fever allows for children to attend school and participate in other activates, but the increased frequency provides additional complications for children and parents.

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Sponsor: Connecticut Children's Medical Center

Current Primary Outcome:

• Effects of the investigational treatment [ Time Frame: Time to event: number of fever-free days will be calculated, as well as the average number of days between fever cycles from baseline to end of study which is 12 months. ]
  Increased healthy intervals between cyclic febrile episodes in patients with PFAPA compared to patients being solely treated with corticosteroids.
• Safety of the investigational treatment - reported descriptively as percentages of patients experiencing adverse and serious adverse events. [ Time Frame: Time to event: number of fever-free days will be calculated, as well as the average number of days between fever cycles from baseline to end of study which is 12 months. ]
  Safety will be reported descriptively as percentages of patients experiencing adverse and serious adverse events.

Original Primary Outcome:

• Effects of the investigational treatment [ Time Frame: one year time frame from study enrollment ]
  Increased healthy intervals between cyclic febrile episodes in patients with PFAPA compared to patients being solely treated with corticosteroids.
• Safety of the investigational treatment - reported descriptively as percentages of patients experiencing adverse and serious adverse events. [ Time Frame: one year time frame from study enrollment ]
  Safety will be reported descriptively as percentages of patients experiencing adverse and serious adverse events.

Current Secondary Outcome:

• Maximal fever experienced during a PFAPA febrile episode will be recorded as an average of the peak temperature recorded during each episode throughout the year and compared between the study groups [ Time Frame: Maximal fever experienced during a PFAPA febrile episode will be recorded as an average of the peak temperature recorded during each episode throughout the year ]
  Decreased maximal fever experienced during a PFAPA febrile episode.
• Number of administrations of corticosteroid necessary for treatment of PFAPA syndrome, recorded as the average number of administrations per fever episode throughout the year [ Time Frame: Number of administrations of corticosteroid necessary for treatment of PFAPA syndrome, recorded as the average number of administrations per fever episode throughout the year ]
  Decreased administrations of corticosteroid necessary for treatment of PFAPA syndrome.
• The average duration of the individual cyclic febrile episodes [ Time Frame: average duration of the individual cyclic febrile episodes in patients with PFAPA will be assessed from baseline to the end of study which is 1 year ]
  Decrease duration of the individual cyclic febrile episodes in patients with PFAPA.
• The number of patients undergoing tonsillectomy will be compared between the study groups [ Time Frame: At any time during the study from baseline to end of study, which is 12 months this will recorded ]
  Fewer patients undergoing tonsillectomy as a treatment for PFAPA syndrome

Original Secondary Outcome:

• Maximal fever experienced during a PFAPA febrile episode will be recorded as an average of the peak temperature recorded during each episode throughout the year and compared between the study groups [ Time Frame: one year from study enrollment ]
  Decreased maximal fever experienced during a PFAPA febrile episode.
• Number of administrations of corticosteroid necessary for treatment of PFAPA syndrome, recorded as the average number of administrations per fever episode throughout the year [ Time Frame: one year from study enrollment ]
  Decreased administrations of corticosteroid necessary for treatment of PFAPA syndrome.
• The average duration of the individual cyclic febrile episodes [ Time Frame: one year from study enrollment ]
  Decrease duration of the individual cyclic febrile episodes in patients with PFAPA.
• The number of patients undergoing tonsillectomy will be compared between the study groups [ Time Frame: one year from study enrollment ]
  Fewer patients undergoing tonsillectomy as a treatment for PFAPA syndrome

Information By: Connecticut Children's Medical Center

Dates:
Date Received: August 20, 2015
Date Started: October 2016
Date Completion: September 2019
Last Updated: September 5, 2016
Last Verified: September 2016