Researchers studied 130 children with PFAPA—a condition causing repeated fevers, mouth sores, sore throats, and swollen neck glands. They compared two treatments: colchicine (a medicine) and probiotics (helpful bacteria supplements with vitamins). Both treatments helped reduce how often and severe the symptoms were. Colchicine worked slightly faster at preventing the first fever attack, but probiotics also showed real improvement. This study is important because it’s one of the first to carefully measure how well probiotics work for this condition, giving families more information to choose the best treatment with their doctor.

The Quick Take

  • What they studied: Whether probiotics (good bacteria) or colchicine (a medicine) work better at preventing fever attacks in children with PFAPA syndrome
  • Who participated: 130 children with PFAPA who took either colchicine (97 kids) or probiotics with vitamins (33 kids) for at least 3 months
  • Key finding: Both treatments significantly reduced disease activity. Colchicine delayed the first fever attack longer than probiotics, but both showed meaningful improvement in symptoms
  • What it means for you: If your child has PFAPA, both treatments may help reduce fever episodes. Talk with your doctor about which option fits best for your family, considering how quickly you need relief and possible side effects

The Research Details

This was a retrospective study, meaning researchers looked back at medical records of children who had already received treatment. They compared disease activity scores from the 3 months before treatment started to the 3 months after treatment began. The researchers used a special scoring system called AIDAI (Auto-Inflammatory Disease Activity Index) to measure how severe the symptoms were each month.

One group of 97 children took colchicine, a medicine that reduces inflammation. The other group of 33 children took probiotics—which are beneficial bacteria—combined with vitamin D, vitamin B12, and folic acid. The researchers tracked when the first fever attack happened after starting treatment and how the disease activity scores changed over time.

This approach allowed doctors to see real-world results from children already being treated, rather than doing a new experiment. However, because it wasn’t a controlled experiment where some kids got fake treatment, the results need to be confirmed with more research.

Using an objective scoring system (AIDAI) instead of just asking families if their child felt better makes the results more reliable. This study is important because very little research has tested whether probiotics actually work for PFAPA. By measuring real improvements in both groups, this research helps families and doctors make informed decisions together about which treatment to try.

Strengths: The study used an objective measurement tool (AIDAI scores) rather than just asking families about improvement, and it included a decent number of patients. Weaknesses: This was a retrospective study looking at past records rather than a controlled experiment, so we can’t be completely sure the treatments caused the improvement. The probiotic group was smaller (33 kids) than the colchicine group (97 kids), which could affect comparisons. The study didn’t randomly assign kids to treatments, so the groups might have been different in ways that affected results.

What the Results Show

Both treatments led to significant improvement in disease activity. In the colchicine group, the average disease activity score dropped noticeably from the month before treatment to the month after (p < 0.001, meaning this result is very unlikely to be due to chance). The probiotic group also showed the same level of significant improvement (p < 0.001).

When looking at how long it took for the first fever attack to happen after starting treatment, colchicine performed better. Children taking colchicine stayed fever-free longer than children taking probiotics (p = 0.048). This suggests colchicine may work faster at preventing the first attack.

However, the short-term improvements in disease activity were similar between the two groups. This means that after a few months, both treatments helped reduce how often and severe the fever episodes were. The main difference was that colchicine delayed the first attack longer, but both treatments eventually helped control symptoms.

The study noted that some children had gastrointestinal (stomach and digestive) symptoms, and these symptoms appeared to affect how well colchicine worked. This suggests that doctors might need to consider each child’s individual symptoms when choosing treatment. The researchers also mentioned that the two treatments have different side effect profiles, which is important for families to discuss with their doctors when deciding which option to try.

This is one of the first studies to carefully measure how well probiotics work for PFAPA using an objective scoring system. Previous research on PFAPA treatment has mostly focused on colchicine. By showing that probiotics also produce meaningful improvement, this study adds important new information to the limited research available. It suggests probiotics could be a real option for families who want to try something other than traditional medicine, though more research is needed to fully understand how they compare long-term.

The biggest limitation is that this wasn’t a controlled experiment—researchers looked at past medical records rather than randomly assigning children to treatments. This means we can’t be completely sure the treatments caused the improvement; other factors might have helped. The probiotic group was much smaller (33 kids) than the colchicine group (97 kids), making direct comparisons harder. The study only looked at short-term results (3 months before and after), so we don’t know how well these treatments work over years. Finally, the study didn’t track side effects systematically, so we don’t have complete information about which treatment causes fewer problems.

The Bottom Line

If your child has PFAPA: Both colchicine and probiotics appear to help reduce fever episodes (moderate confidence). Colchicine may work faster at preventing the first attack (moderate confidence). Consider discussing both options with your pediatrician, including potential side effects and your family’s preferences. If your child has stomach problems, mention this to your doctor as it may affect which treatment works best (low to moderate confidence).

This research is most relevant for: Families with children diagnosed with PFAPA syndrome; Pediatricians treating PFAPA; Parents looking for alternatives to traditional medicine. This research is NOT a substitute for medical advice—always work with your child’s doctor to choose the right treatment.

Based on this study, you should see improvement in disease activity within the first month of starting either treatment. However, colchicine may delay the first fever attack longer than probiotics. It typically takes 3 months to fully evaluate how well a treatment is working for PFAPA.

Want to Apply This Research?

  • Track fever episodes weekly: Record the date, duration (how long it lasted), and severity (mild, moderate, severe) of each fever attack. This creates a clear picture of whether your chosen treatment is reducing how often fevers happen.
  • Set a daily reminder to take the prescribed treatment at the same time each day. Consistent use is important for probiotics and colchicine to work effectively. Use the app to log when you take your dose to ensure you don’t miss days.
  • Calculate your monthly disease activity score by tracking: number of fever episodes, duration of each episode, presence of mouth sores, sore throat severity, and swollen glands. Compare scores month-to-month to see if your treatment is working. Share these trends with your doctor at each visit to adjust treatment if needed.

This summary is for educational purposes only and should not replace professional medical advice. PFAPA is a medical condition that requires diagnosis and treatment by a qualified healthcare provider. Do not start, stop, or change any treatment without consulting your child’s pediatrician. The findings in this study are preliminary and should be discussed with your doctor to determine the best treatment approach for your child’s specific situation. Individual responses to treatment vary, and what works for one child may not work the same way for another.