Carrier screening
Routine obstetric practice in the US has included prepregnancy and antenatal carrier screening for CF since 2001.[37]Watson MS, Cutting GR, Desnick RJ, et al. Cystic fibrosis population carrier screening: 2004 revision of American College of Medical Genetics mutation panel. Genet Med. 2004 Sep-Oct;6(5):387-91.
https://www.doi.org/10.1097/01.gim.0000139506.11694.7c
http://www.ncbi.nlm.nih.gov/pubmed/15371902?tool=bestpractice.com
Therefore, every pregnant woman or woman considering pregnancy should receive information about genetic carrier screening and be free to make an informed decision after appropriate counselling.[35]American College of Obstetricians and Gynecologists. Committee opinion no. 691: carrier screening for genetic conditions. Mar 2017 [internet publication].
https://www.acog.org/clinical/clinical-guidance/committee-opinion/articles/2017/03/carrier-screening-for-genetic-conditions
http://www.ncbi.nlm.nih.gov/pubmed/28225426?tool=bestpractice.com
[38]Hopkins MK, Dugoff L, Kuller JA. Guidelines for cystic fibrosis carrier screening in the prenatal/preconception period. Obstet Gynecol Surv. 2022 Oct;77(10):606-10.
http://www.ncbi.nlm.nih.gov/pubmed/36242530?tool=bestpractice.com
Although ethnicity affects the sensitivity of current screening tests, resulting in variation from <50% with Asian ancestry to 94% for the Ashkenazi Jewish population, aim to screen every pregnant woman for cystic fibrosis once during her lifetime, irrespective of their ethnic background.[39]American College of Medical Genetics. Standards and guidelines for clinical genetics laboratories, 2008 edition. March 2011 [internet publication].
https://www.acmg.net//docs/CFTR_Mutation_Testing_2011.pdf
[40]American College of Obstetricians and Gynecologists. Committee opinion no. 690: carrier screening in the age of genomic medicine. Mar 2017 [internet publication].
https://www.acog.org/clinical/clinical-guidance/committee-opinion/articles/2017/03/carrier-screening-in-the-age-of-genomic-medicine
http://www.ncbi.nlm.nih.gov/pubmed/28225425?tool=bestpractice.com
Expanded panels can improve CF carrier detection rates within specific populations. Routine carrier screening should not include a complete analysis of the CFTR gene by DNA sequencing.[35]American College of Obstetricians and Gynecologists. Committee opinion no. 691: carrier screening for genetic conditions. Mar 2017 [internet publication].
https://www.acog.org/clinical/clinical-guidance/committee-opinion/articles/2017/03/carrier-screening-for-genetic-conditions
http://www.ncbi.nlm.nih.gov/pubmed/28225426?tool=bestpractice.com
If a woman receives a positive screening result, offer her partner a screening test. If neither is affected, but one or both has a relevant family history, conduct genetic counselling and a medical record review to determine if there has been a CFTR mutation analysis. If a woman’s reproductive partner has CF or apparently isolated congenital bilateral absence of the vas deferens, offer follow-up genetic counselling by a healthcare provider with genetics expertise for mutation analysis and consultation.[35]American College of Obstetricians and Gynecologists. Committee opinion no. 691: carrier screening for genetic conditions. Mar 2017 [internet publication].
https://www.acog.org/clinical/clinical-guidance/committee-opinion/articles/2017/03/carrier-screening-for-genetic-conditions
http://www.ncbi.nlm.nih.gov/pubmed/28225426?tool=bestpractice.com
Newborn screening
The US Cystic Fibrosis Foundation recommends appropriately performed newborn screening for all infants.[34]Comeau AM, Accurso FJ, White TB, et al. Guidelines for implementation of cystic fibrosis newborn screening programs: Cystic Fibrosis Foundation workshop report. Pediatrics. 2007 Feb;119(2):e495-518.
http://www.ncbi.nlm.nih.gov/pubmed/17272609?tool=bestpractice.com
This should not replace prepregnancy or antenatal carrier screening.[35]American College of Obstetricians and Gynecologists. Committee opinion no. 691: carrier screening for genetic conditions. Mar 2017 [internet publication].
https://www.acog.org/clinical/clinical-guidance/committee-opinion/articles/2017/03/carrier-screening-for-genetic-conditions
http://www.ncbi.nlm.nih.gov/pubmed/28225426?tool=bestpractice.com
Newborn screening ensures early diagnosis and treatment, providing significant clinical benefits (e.g., improved nutrition and cognition). This approach may decrease lung disease and the amount of hospital admissions. It is performed by quantifying serum immunoreactive trypsinogen (IRT) from a dried heel-stick blood spot. Test results only indicate risk, not a diagnosis, with each laboratory defining a positive IRT test result differently.[29]Farrell PM, White TB, Ren CL, et al. Diagnosis of cystic fibrosis: consensus guidelines from the Cystic Fibrosis Foundation. J Pediatr. 2017 Feb;181S:S4-15.e1.
https://www.jpeds.com/article/S0022-3476(16)31048-4/fulltext
http://www.ncbi.nlm.nih.gov/pubmed/28129811?tool=bestpractice.com
Protocols also differ for the follow-up offered after a positive IRT test, but this usually includes confirmatory testing with a positive sweat test and the identification of two disease-causing mutations on genetic testing. All patients with a positive IRT test should be referred to a CF centre.[41]Castellani C, Southern KW, Brownlee K, et al. European best practice guidelines for cystic fibrosis neonatal screening. J Cyst Fibros. 2009 May;8(3):153-73.
http://www.ncbi.nlm.nih.gov/pubmed/19246252?tool=bestpractice.com
Expanded genetics can be considered when there is more ethnic diversity, depending on the local population (i.e., state or region).
Unintended consequence of CF newborn screening
A positive NBS test may be accompanied by inconclusive diagnostic testing.[29]Farrell PM, White TB, Ren CL, et al. Diagnosis of cystic fibrosis: consensus guidelines from the Cystic Fibrosis Foundation. J Pediatr. 2017 Feb;181S:S4-15.e1.
https://www.jpeds.com/article/S0022-3476(16)31048-4/fulltext
http://www.ncbi.nlm.nih.gov/pubmed/28129811?tool=bestpractice.com
[42]Ren CL, Borowitz DS, Gonska T, et al. Cystic fibrosis transmembrane conductance regulator-related metabolic syndrome and cystic fibrosis screen positive, inconclusive diagnosis. J Pediatr. 2017 Feb;181S:S45-S51.e1.
https://www.doi.org/10.1016/j.jpeds.2016.09.066
http://www.ncbi.nlm.nih.gov/pubmed/28129812?tool=bestpractice.com
CFTR-related metabolic syndrome (CRMS), also known as a CF screen-positive inconclusive diagnosis (CFSPID) in Europe, refers to infants with a positive newborn screening test for CF and either of the following:[29]Farrell PM, White TB, Ren CL, et al. Diagnosis of cystic fibrosis: consensus guidelines from the Cystic Fibrosis Foundation. J Pediatr. 2017 Feb;181S:S4-15.e1.
https://www.jpeds.com/article/S0022-3476(16)31048-4/fulltext
http://www.ncbi.nlm.nih.gov/pubmed/28129811?tool=bestpractice.com
A sweat chloride value <30 mmol/L (<30 mEq/L) and two CFTR mutations, at least one of which does not cause any physical symptoms, or
An intermediate sweat chloride value (30-59 mmol/L [30-59 mEq/L]) and one or no CF-causing mutations.
It is important to follow these patients closely as the prognosis of CRMS/CFSPID currently remains unclear.[43]Barben J, Castellani C, Munck A, et al. Updated guidance on the management of children with cystic fibrosis transmembrane conductance regulator-related metabolic syndrome/cystic fibrosis screen positive, inconclusive diagnosis (CRMS/CFSPID). J Cyst Fibros. 2021 Sep;20(5):810-9.
http://www.ncbi.nlm.nih.gov/pubmed/33257262?tool=bestpractice.com
Certain individuals with CRMS/CFSPID have a higher risk of developing related respiratory, sinus, intestinal, pancreatic, or reproductive pathology similar to CF. In some cases, evolving signs and symptoms, new information about disease-causing CFTR mutations, or changes in sweat chloride concentration levels may ultimately lead to a CF diagnosis.[42]Ren CL, Borowitz DS, Gonska T, et al. Cystic fibrosis transmembrane conductance regulator-related metabolic syndrome and cystic fibrosis screen positive, inconclusive diagnosis. J Pediatr. 2017 Feb;181S:S45-S51.e1.
https://www.doi.org/10.1016/j.jpeds.2016.09.066
http://www.ncbi.nlm.nih.gov/pubmed/28129812?tool=bestpractice.com
[43]Barben J, Castellani C, Munck A, et al. Updated guidance on the management of children with cystic fibrosis transmembrane conductance regulator-related metabolic syndrome/cystic fibrosis screen positive, inconclusive diagnosis (CRMS/CFSPID). J Cyst Fibros. 2021 Sep;20(5):810-9.
http://www.ncbi.nlm.nih.gov/pubmed/33257262?tool=bestpractice.com