Cystic Fibrosis Care at the University of Florida
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1 Cystic Fibrosis Care at the University of Florida
2 Objectives To introduce you to the University of Florida CF Center To review center specific data for the UF pediatric CF Center To review current status of the CF Foundation Drug Pipeline To review CF related research currently in progress nationally and locally
3 The CF Team Physicians Nurse Practitioners Pharmacologists Family Partner YOU Nurses Social Worker Office Staff Dietitians
4 Your CF Center Doctors Dr. Mutasim Abu-Hasan Dr. Abby Wagner Dr. Pamela M. Schuler Dr. Sreekala Prabhakaran Dr. Satya Hegde Dr Sarah Chesrown, Professor Emertius Dr Mai El Mallah (fellow) Dr. Hendeles (Pharmacology)
5 Nursing/Nurse Elizabeth LeFave Dawn Baker Rhoda Reed Cindy Capen Practitioners
6 Social Work, Dieticians and Family Partner Susan Horky Ellen Bowser Jennifer Hillan Angela Miney
7 In the Office Brenda Lisa Autumn Leeann Joanna
8 National Recognition Ellen Bowser served on the Vitamin D Evidence Based Guidelines Committee Cindy Capen currently is in her second term on the Cystic Fibrosis education committee Susan Horkey She participates in CFF mentoring program & has several CF related research projects. Dawn Baker research nurse extraordinaire Angela Miney along with several other UF professionals is working on the Interdisciplinary Collaborative on Education and Healthcare Transition Dr Abu Hasan has served as a CF center director and currently serves as the UF CF center co-director Dr. Schuler presented a case at the Physicians Grand Rounds at the NACFC last year. 2 CF related research projects
9 So What s New? Newborn screening began in the state of FL in 2007 and as of Jan 2010 all 50 states have a newborn screening program for CF Almost 60% of CF patients are now dx by newborn screening Advantages-babies diagnosed by newborn screening have better weight & healthier lungs later in life
10 The progression of knowledge about Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) *Identification of the CF gene in 1989 on chromosome 7 at q31.2 *Gene codes for a protein CFTR which is a chloride channel *Over 1800 variations of the CF gene have been identified *The type of malfunction in CFTR can be different depending on the structure of the gene *This has led to options for treatment
11
12 Kalydeco (previously known as VX-770) is a potentiator. It is a new oral medication that was approved by the FDA on January 31, 2012 for people with CF ages 6 and older with the G551D mutation of CF. It is the first drug available that targets the underlying cause of CF a faulty gene and its protein product, CFTR.
13 Nonsense mutations & Ataluren (PTC 124) In nonsense mutations the abnormality in the CFTR protein is a premature stop codon which prevents the whole protein from being made W1282X is an example resulting in absent functional protein and typically a severe CF phenotype Ataluren helps create a read through the premature stop hopefully creating a functional CFTR Phase III trials are complete and the results are pending
14 VX-809 VX-809 is a "corrector," designed to move defective CFTR protein to the proper place in the airway cell membrane and improve its function as a chloride channel. A Phase 2a trial with VX-809 alone in people with CF who have the F508del mutation was completed in There were no safety concerns and the treatment group demonstrated an small drop in sweat chloride but no change in clinical endpoints such as lung function. Laboratory experiments demonstrated that the effect of VX-809 can be potentiated by adding Kalydeco (formerly known as VX-770).
15 Most common mutations % Over 1800 known mutations Delta F508 G542X G551D R117H N1303K W1282X R553X
16 UF We are in the process of enrolling all of our eligible pts with at least one copy of G551D for treatment with Kalydeco Secondary to the need to know individuals gene mutations we are pushing to have all of our children and adults with CF genotyped. In the CF registry 92% have had mutations identified Of the 98 we follow so far all but 3 have been genotyped
17 Bronchitol is an inhaled dry powder form of mannitol that acts by drawing water into the airway to help rehydrate CF secretions, thereby improving airway clearance. Gilead GS9411 is a compound that blocks sodium absorption. Excessive sodium absorption from the airway is thought to contribute to airway dehydration.
18 Oral N-acetylcysteine is an antioxidant that replenishes glutathione levels. Docosahexaenoic acid (DHA) is an omega-3 fatty acid with antiinflammatory properties. KB001 is a humanized monoclonal Fab fragment that targets a Pseudomonas aeruginosa virulence factor (Type III secretion system). GSK SB is a once daily oral anti-inflammatory agent for maintenance treatment of CF. Sildenafil is a phosphodiesterase inhibitor. Researchers are looking to see if Sildenafil can lower markers of airway inflammation in people with CF.
19 Antibiotics
20 Nutritional Support
21 Nutrition UF PEDIATRIC DATA
22 Mean BMI Percentile ages 2-19, 2010
23 Mean BMI Percentile for Patients
24 Glucose screening in non-diabetic ages 10 and older
25 What are we doing to improve? Regular clinic visits with RD Anticipatory guidance given during clinic visits Encourage use of nutrition supplements Yearly evaluation for diabetes over age 10 Placement of feeding tubes encouraged if unable to meet caloric needs Weekly clinic preview and discuss nutrition status RDs attend CF conferences regularly Vitamin D QI study
26 Importance of Growth Pulmonary function and nutritional status are highly correlated Nutr Hosp Brazil Dec;26(6): a. BMI below the 10th percentile was associated with a 25.58% drop in FEV1, b. plasma albumin levels equal to or lower than 4.1 mg/dl was associated with 18.6% FEV1 reduction J Pediatr Dec;153(6): Cincinnati Slower decline in lung function associated with a. Higher baseline BMI % b. Slower rate BMI decline
27 Mean FEV1 % predicted ages
28 Mean FEV1 % predicted ages
29 P. aeruginosa Infection Rate in ages 18 and under National average 35.3 UF 44
30 Pseudomonas aeruginosa infection rate in under age 18
31 Multidrug-resistant P. aeruginosa infection rate
32 Multidrug-resistant P. aeruginosa
33 Prevention of Chronic P aeruginosa lung infections Eradication Dornase alfa TOBI Regular monitoring with sputum cultures Bronchoscopy Aggressive airway clearance/exercise programs Good hand washing Immunizations Aggressive treatment of exacerbations
34 MRSA infection rate 2010
35 MRSA infection rate
36 MSSA MRSA Staphylococcus aureus is one of the first bacteria cultured from infants and children with CF Since 1960 approx. 80% isolates resistant to PCN Within 2 years of the introduction of Methicillin in 1959 resistance was reported Rate of lung function decline greater in those with CF and MRSA compared to non-mrsa (FEV1 decline of 2.06% predicted per year vs 1.44%) MRSA risk factor for failure to recover lung function after acute pulm exacerbation
37 MRSA ERADICATION No conclusive studies if eradication is possible, prevents chronic infection or improves outcomes Risk of increased resistance Increased risk of acquiring Pseudomonas a Attempts to eradicate MRSA in non-cf patients with variable success Treatment TMP-SMX all ages, minocyline >8 yr, fusidic acid + rifampin (not available in US), linezolid, vancomycin but avoid fluoroquinolones
38 Our Goals as a CF Center To improve the understanding of patients and families of the multisystem involvement in cystic fibrosis To help families understand that to slow the process of CF is a team effort and they are the most important members of the team To further improve nutrition To decrease the incidence of MRSA and P aeruginosa To have all pts genotyped To continue to participate in CF research to help advance the science of CF toward a cure To have children and their families involved in the research process
39 Current research Let me introduce you to Dawn Baker C-PNP, CCRC
40 THE END
41 Sildenafil is a phosphodiesterase inhibitor. Researchers at National Jewish Health in Denver are examining whether sildenafil can lower markers of airway inflammation in people with CF. A Phase 2 trial is currently enrolling. This single center trial will look at the effect of Sildenafil (Revatio ), a drug already used in other types of lung disease, on symptoms related to lung inflammation and infection in people with CF who are 12 years of age and older.
42 GSK SB is a once daily oral antiinflammatory agent for maintenance treatment of CF. A Phase 2 trial was completed in Results of the Phase 2 trial are expected in 2012.
43 KB001 is a humanized monoclonal Fab fragment that targets a Pseudomonas aeruginosa virulence factor (Type III secretion system). The mechanism by which KB001 is predicted to provide clinical benefit for people with CF is by reducing local inflammation in the lung associated with this virulence factor. Although KB001 has been shown to reduce bacterial load in some animal model systems, it is not predicted to kill or suppress the growth of Pseudomonas aeruginosa in vivo. For these reasons, KB001 will be developed as an antiinflammatory therapy for people with CF who are chronically infected with Pseudomonas aeruginosa. A Phase 2a trial was completed in 2009.
44 mucomyst Oral N-acetylcysteine is an antioxidant that replenishes glutathione levels. Anti-Inflammatory A placebo-controlled 12-week Phase 2 trial at Stanford University demonstrated a decrease in inflammatory cells in the lung and improvement in pulmonary function. 01/31/2012 Results from a Phase 2b multi-center trial did not reproduce these findings but an improvement in lung function was noted.
45 GS9411 is a compound that acts by blocking sodium absorption. Excessive sodium absorption from the airway is thought to contribute to airway dehydration.
46 Regular clinic visits with RD CFF requires yearly nutrition visits, but we usually see more often, especially if high nutrition risk review growth charts, nutrition goals, calorie intake, maximize enzyme dose, review vitamin status, etc Coordinate care between outpatient and inpatient nutrition services as needed Anticipatory guidance given during clinic visits preparing for developmental issues related to nutrition toddler feeding issues, school-aged issues, teens Encourage use of nutrition supplements or high-calorie shakes and smoothies as needed; feeding tubes discussed if unable to meet calorie needs po Weekly clinic preview and discuss nutrition status RDs attend CF conferences yearly (or every other year) RDs on CF nutrition list-serv up to date on latest research and clinical issues Vitamin D QI study Dietetic interns complete case studies on CF patients at high nutrition risk which are attended by the team
47 FEV1 vs BMI % ages each dot is a CF center
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