Preschool-Aged Care Clinical Care Guidelines

These preschool guidelines provide clinical care guidelines for people with cystic fibrosis between 2 and 5 years. It includes 53 recommendations intended to help make informed decisions in the care of preschool-aged children with CF.
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Preschool-Aged Care Clinical Care Guidelines: Executive Summary

Lahiri T, Hempstead SE, Brady C, Cannon CL, Clark K, Condren ME, Guill MF, Guillerman RP, Leone CG, Maguiness K, Monchil L, Powers SW, Rosenfeld M, Schwarzenberg SJ, Tompkins CL, Zemanick ET, Davis SD. Clinical Practice Guidelines From the Cystic Fibrosis Foundation for Preschoolers With Cystic Fibrosis. Pediatrics. 2016;137(4):e20151784. Epub 2016 mar 16. PMID: 27009033

In 2014, a multidisciplinary committee convened to develop preschool guidelines to fill in the gap between existing clinical care guidelines for infants 2 years and younger and individuals 6 years and older. Fifty-three recommendations were included on the following topics:

  • Health maintenance.
  • Caregiver engagement.
  • Screening and monitoring.
  • Therapeutics.
  • Nutrition, behavior, and gastrointestinal (GI).

The purpose of these guidelines is to help make informed decisions in the care of preschool-aged children with CF. Research is needed to improve the strength of many of the recommendations in the preschool guidelines.

Since these guidelines were published, guidelines for enteral tube feeding in individuals with CF have been published that provide more detailed recommendations on the process of enteral tube placement and feeding.

Methodology

Fifty-three recommendations were approved by 80 percent or more of the committee. Reflecting the limited evidence in this age group, the majority of recommendations were made based on consensus only, and only three recommendations met the U.S. Preventive Services Task Force (USPSTF) Grade A criteria. Find additional information about USPSTF grading definitions.

Recommendations

The guidelines recommend the following for children with CF, ages 2 to 5 years:

Health Maintenance
Recommendations Evaluation of the Evidence
1. Routine well-child care according to American Academy of Pediatrics (AAP) guidelines, including routine immunizations. Consensus
2. Children, family members, and caregivers should receive annual influenza vaccinations. Consensus
3. The first dose of pneumococcal polysaccharide vaccine (PPSV23) should be administered. Consensus

Screening and Monitoring
Recommendations Evaluation of the Evidence
4. Parents and CF health care professionals should review treatment goals and care plans quarterly. Consensus
5. Spirometry should be attempted as early as 3 years old and be used to monitor for exacerbations and response to therapy. Consensus
6. Chest X-rays should be obtained at least every other year. Consensus
7. A chest computed tomography (CT) scan may be considered every two to three years, to replace chest X-rays, using the lowest possible radiation dose. Consensus
8. Oropharyngeal cultures should be performed at least quarterly to monitor airway microbiology. Consensus

Therapeutics
Recommendations Evaluation of the Evidence
9. Daily airway clearance is recommended. Consensus
10. For children who are pancreatic insufficient, the maximum pancreatic enzyme replacement therapy dose is 2,500 lipase units/kilogram (kg)/meal and 10,000 lipase units/kg/day. Consensus
11. Hypertonic saline and dornase alfa may be selectively offered based on individual circumstances. Grade: C  
Certainty: Moderate 
Benefit: Moderate
12. Children persistently infected with Pseudomonas aeruginosa should be treated chronically with alternate-month inhaled antipseudomonal antibiotics. Grade: C  
Certainty: Moderate 
Benefit: Moderate
13. Ivacaftor should be used for children with specific gating mutations and considered for children with a confirmed diagnosis of CF and an R117H mutation. Consensus
14. For pulmonary exacerbations:
  1. Increase frequency and/or duration of airway clearance
  2. Use oral, inhaled, and/or intravenous antibiotics to treat
 Consensus

Nutritional, Behavior, and GI
Recommendations Evaluation of the Evidence
15. Weight-for-age ≥10th percentile should be maintained. Grade: A
Certainty: High
Benefit: Substantial
16. Use body mass index (BMI) percentile to assess weight-for-stature and maintain BMI ≥50th percentile. Grade: B
Certainty: High
Benefit: Moderate
17. CF team members should work with the family to set energy-intake goals and assess progress regularly. Grade: B
Certainty: Moderate
Benefit: Substantial
18. For those meeting optimal nutrition thresholds, calorie intake should be ≥90-110 kcal/kg/day, and protein intake should be ≥13g protein/day in 2- to 3-year olds and ≥19 g protein/day in 4- to 5-year olds. Grade: A
Certainty: High
Benefit: Substantial
19. Children at nutritional risk include those with BMI <50th percentile, weight gain <50th percentile expected for age, weight-for-age <10th percentile, or inappropriate weight loss. These children should be evaluated and receive more intensive management. Grade: B
Certainty: High
Benefit: Moderate
20. For children at nutritional risk, evaluate and intensify management:
  1. Re-evaluate within eight weeks for medical, behavioral, and nutritional assessments and education, and implement interventions aimed at achieving the patient's target goal for both weight-for-age and BMI
  2. Increase energy intake incrementally by 10-20 percent, as needed up to 200 percent of baseline
  3. Add oral nutrition supplements in addition to usual dietary intake
  4. Introduce the concept of enteral feedings early as a component of CF care
  1. Consensus
  2. Grade: B
    Certainty: Moderate
    Benefit: Moderate
  3. Grade: B
    Certainty: Moderate
    Benefit: Moderate
  4. Grade: B
    Certainty: Moderate
    Benefit: Moderate
21. For children at nutritional risk who are not responding: 
  1. Evaluate causes of poor growth, including GI, endocrine, behavioral, and social causes
  2. If this evaluation does not lead to improvements, use enteral nutritional supplements
  1. Consensus
  2. Grade: B
    Certainty: Moderate
    Benefit: Moderate
22. Vitamin supplementation:
  1. Standard non-fat-soluble vitamins, and fat-soluble vitamins formulated for children with CF, are recommended
  2. Measure blood levels of fat-soluble vitamins annually and more frequently if abnormal
  3. Additional supplementation of vitamins A, D, E, and K is recommended if indicated, based on levels
  4. Manage vitamin D deficiency according to the Cystic Fibrosis Foundation vitamin D deficiency guidelines
 Consensus
23. Add salt to meals and snacks, especially during summer months and in warm climates. Consensus
24. Regularly assess for mealtime behavior challenges and provide proactive assistance when needed. Grade: A 
Certainty: High 
Benefit: Substantial
25. Provide behavioral therapy for children at nutritional risk or for children who exhibit challenging mealtime behaviors. Grade: A 
Certainty: High 
Benefit: Substantial
26. Be aware of the presenting symptoms of constipation, gastroesophageal reflux disease, small bowel overgrowth, distal intestinal obstruction syndrome, and celiac disease. Consensus
27. Evaluate for abdominal pain at each visit, and investigate if persistent or recurrent. Consensus
28. For children who are pancreatic sufficient:
  1. Obtain fecal elastase yearly, especially if mutations are associated with pancreatic insufficiency
  2. Measure lipase and amylase for severe abdominal pain, especially associated with vomiting
 Consensus
29. With terminal ileal bowel resection, annually measure serum vitamin B12 concentration. Consensus

Insufficient Evidence to Recommend For or Against
Recommendations Evaluation of the Evidence
30. Routine pulse oximetry, assessment of bronchodilator responsiveness, or multiple breath washout Grade: I
Certainty: low 
31. Chronic inhaled bronchodilators, high-dose ibuprofen, leukotriene modifiers, or azithromycin Grade: I
Certainty: low 
32. Attempts to eradicate Staphylococcus aureus (S. aureus), including methicillin-resistant S. aureus (MRSA), in asymptomatic patients Grade: I
Certainty: low 
33. Chronic use of oral antistaphylococcal antibiotics in children persistently infected with S. aureus Grade: I
Certainty: low 

Not To Be Used Routinely
Recommendations Evaluation of the Evidence
34. Bronchoscopy Grade: D
Certainty: Moderate
Benefit: Negative
35. For children without asthma or recurrent wheezing, inhaled corticosteroids Grade: D
Certainty: High
Benefit: Low
36. For children without allergic bronchopulmonary aspergillosis (ABPA), chronic use of systemic corticosteroids Grade: D
Certainty: High
Benefit: Low
37. Oral antistaphylococcal antibiotics Grade: D
Certainty: Moderate
Benefit: Negative

Unanswered Questions

The authors note that additional research is needed in the following areas for preschool-aged children with CF:

  • The optimal approach to monitoring and detecting lung disease (e.g., with magnetic resonance imaging [MRI], multiple breath washout)
  • Efficacy of chronic respiratory medications, including any newly developed CFTR modulators
  • Nutritional, behavioral, and GI studies to optimize nutrition and overcome behavioral challenges
  • Timing of nutritional interventions and conversion from pancreatic sufficiency to pancreatic insufficiency
  • Determining the prevalence of key GI disorders (e.g., constipation, reflux, bacterial overgrowth)

Further Reading

  • Schwarzenberg SJ, Hempstead SE, McDonald CM, et al. Enteral tube feeding for individuals with cystic fibrosis: Cystic Fibrosis Foundation evidence-informed guidelines. J Cyst Fibros. 2016 Nov;15(6):724-35. Epub2016 Sep 3. PMID: 27599607. This article provides more guidance on the process of enteral tube placement and enteral feeding in individuals with CF.
  • Mayer-Hamblett N, Retsch-Bogart G, Kloster M, et al. Azithromycin for Early Pseudomonas Infection in Cystic Fibrosis. The OPTIMIZE Randomized Trial. Am J Respir Crit Care Med. 2018 Nov 1;198(9):1177-1187. doi: 10.1164/rccm.201802-0215OC
  • McNamara JJ, McColley SA, Marigowda G, et al. Safety, pharmacokinetics, and pharmacodynamics of lumacaftor and ivacaftor combination therapy in children aged 2-5 years with cystic fibrosis homozygous for F508del-CFTR: an open-label phase 3 study. Lancet Respir Med. 2019;7(4):325-335. doi:10.1016/S2213-2600(18)30460-0. PMID: 30686767.
  • Ratjen F, Davis SD, Stanojevic S, et al. Inhaled hypertonic saline in preschool children with cystic fibrosis (SHIP): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Respir Med. 2019;7(9):802-809. doi:10.1016/S2213-2600(19)30187-0. PMID: 31178421.
  • Voldby C, Green K, Philipsen L, et al. Withdrawal of dornase alfa increases ventilation inhomogeneity in children with cystic fibrosis.J Cyst Fibros. 2021;20(6):949-956. doi:10.1016/j.jcf.2021.02.004. PMID: 33619014.
  • Goralski JL, Hoppe JE, Mall MA, et al. Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Allele. Am J Respir Crit Care Med. 2023;208(1):59-67. doi:10.1164/rccm.202301-0084OC. PMID: 36921081.

Use of These Guidelines

The CF Foundation intends for this executive summary of its guideline to summarize the published guideline. The published guideline summarizes evidence, and provides reasonable clinical recommendations based on that evidence, to clinicians, patients, and other stakeholders. Care decisions regarding individual patients should be made using a combination of these recommendations, the associated benefit-risk assessment of treatment options from the clinical team, the patient's individual and unique circumstances, as well as the goals and preferences of the patients and families that the team serves, as a part of shared decision-making between the patient and clinician.

This executive summary was prepared by:
Don B. Sanders, MD, MS, and Stephanie D. Davis, MD, (Indiana University School of Medicine)

The guidelines were published in March 2016, they were reviewed in July 2021 and it was determined that no update is needed at this time.

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