Cystic Fibrosis Expert Update 2017

During one of our recent group clinical meetings, one of my partners (Edith Pilzer, MD) presented an update on cystic fibrosis.

Here is a link to her slides:Link: cf-presentation

Here are a few of my notes:

There has been a great improvement in survival of cystic fibrosis patients..  From the Cystic Fibrosis Foundation:

  • ” Today the median predicted survival age is close to 40. This is a dramatic improvement from the 1950s, when a child with CF rarely lived long enough to attend elementary school.”

cftr2org  —website provide information on specific genotypes, including whether genotype is associated with pancreatic insufficiency

From the website:

  • This website provides information for members of the general public, including cystic fibrosis patients and their family members, about what is currently known about specific genetic variants related to cystic fibrosis.
  • Patients and their family members are encouraged to visit the section, “For patients and family members” first.
  • This website also provides more in-depth research-related information for health care professionals and researchers

Pancreatic enzyme replacement therapy (PERT) (see slides)

  • Creon 3000 beads are small enough to go through Gastrostomy tube
  • Pertzye has bicarbonate; thus, additional acid blocker administration is usually not needed
  • Viokase is hard to obtain
  • Relizorb –external lipase cartiledge.  This allows formula, delivered by NG, to run through column and obviates the need for additional PERT dosing.  One cartridge designed for 500 cc but several cartridges can be ‘piggy-backed.’ Here is website:  Relizorb intent is to eliminate enzymes for night feeds, though it only has lipase; yet, there still could be a need additional PERT for protease and amylase.  Potentially PERT could be administered before or after and hopefully avoid awakening at night for enzymes..

Cystic Fibrosis Related Diabetes (CFRD)

  • Frequent reason for poor growth
  • Now, with increased survival, ~35% of Cystic Fibrosis patients develop CFRD

Distal Intestinal Obstruction Syndrome (DIOS)

  • If mild, treatment with miralax is reasonable
  • If vomiting, consider surgery consult
  • If more than mild, consider water-soluble enema with 10% mucomyst

My take: Great update.  Edith has been taking care of children with cystic fibrosis for more than 30 years and has witnessed/participated in the improvement in the survival of these patients.

Related blog posts:

Disclaimer: These blog posts are for educational purposes only. Specific dosing of medications (along with potential adverse effects) should be confirmed by prescribing physician.  This content is not a substitute for medical advice, diagnosis or treatment provided by a qualified healthcare provider. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a condition.


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