Approval of the new drug Trikafta
October 22, 2019
Dear Patients and Families,
We hope this letter finds everyone well and enjoying a good Fall. As you may already be aware, the FDA approved yesterday the drug Trikafta (a triple combination of the CFTR modulators Elexacaftor, Tezacaftor and Ivacaftor) for the treatment of CF patients 12 years of age and older who carry at least one delta F508 mutation in their CF genes. The approval was based on the impressive effects noted in patients that participated in the clinical trials that evaluated the drug. This represents a significant advancement in CF care, since close to 90% of CF patients carry at least one copy of delta F508 mutation. This expands substantially our ability to offer to CF patients access to highly effective CFTR modulator therapy. At the same time, we recognize the importance of continued research for the remaining 10% of CF patients who may not have access to this type of therapy and remain committed to meaningfully contribute to ongoing research efforts in this regard.
At the Stanford Cystic Fibrosis Center, we have compiled a list of patients who qualify for the new modulator therapy. We are working closely with the manufacturer and specialty pharmacy teams to determine first availability of Trikafta for those that qualify. We will be reaching out to those patients and families on an individual basis to discuss the new treatment option and plan start of therapy.
Please always keep in mind that in our model of care, you are the most important member of the care team. We value very much the trust you placed on us and hope to continue building on the partnership that is established through our Center to serve you and the CF community at large. If you have any qeustions or concerns, please do not hesitate to call the RN phone line at 650-736-1359.
With best regards,
Dr. Carlos Milla
The Stanford Cystic Fibrosis Center
Stanford Children's Health
Cystic Fibrosis (CF) is one of the most common genetic (inherited) diseases in America. It is also one of the most serious. It mainly affects the lungs and the digestive systems in the body, causing breathing problems and problems digesting foods. It is a chronic disease that currently has no cure.
The CF Center at Stanford is an integrated disease management program that follows patients from diagnosis through adulthood.
With the current longer life expectancy for patients with Cystic Fibrosis, our clinic population includes patients of all ages. More than half the patients followed by the Stanford CF Center are adults aged 18 years or older.
770 Welch Road, Suite 380, 3rd Floor
Monday, Tuesday, Wednesday, Friday
For New Pediatric Patients
Pediatric Routine Appointments
Please call: (650)724-4788
Nurse Coordinators/Clinic Nurse:
Please call: (650)736-1359
Bone Density (DEXA) (650)723-6855
PFT (full PFT's) (650)497-8655
SAVE THE DATE
CF Education Day
March 14th, 2020
Pediatric Clinic News
Make sure you bring your CF Passport with you! Use the PASSPORT around the hospital wherever you have an appointment, test, or procedure. Remember Parents/Patients to carry your child's CF PASSPORT in your wallet. If for some reason you do not have one or tossed it, please ask for one at your next clinic visit. We now have them in English and Spanish
Have A Question?
Do you drive away from clinic thinking, "oh no, I forgot to ask a question?" You can now fill out this form ahead of time and bring it to your clinic appointment. This form is designed to help you get all your questions answered. This is not mandatory, but a tool to assist you in jogging your memory in preparation for your clinic visit.
Did you know that you can get assistance with your PG&E bill? All you need to do is visit the website, print and complete the form with all your medical devices listed. Then bring your form to your next CF clinic visit, and your provider will sign it. Finally, mail the completed form to PG&E.
CF and Dental Health
Oral hygiene (caring for your teeth and gums) can impact CF health. Here are some guidelines and tips for how to teach and encourage your children to practice good habits to stay happy and healthy.
Adult Clinic News
Adult Support Group
CF Caregivers Support Group
A CFRI CF Quality of Life Program
Group Meets Third Tuesday of Every Month.
Two Groups to Choose from:
Parents of children with CF: 5:00 pm - 6:00 pm PST
Parents/Spouses/Partners of Adults with CF: 6:00 pm - 7:00 pm PST
Location: 1st Floor Conference Room, 730 Welch Road, Palo Alto, CA
or Participate by Phone: 1-650-736-4444
Facilitated by Meg Dvorak, LCSW
For more information about this program or other CF Quality of LIfe programs, please call CFRI at 650-665-7576
Herbs and Oils: Applications in CF, Russell Wise, PharmD, BCPPS
Cystic Fibrosis Research
Research is being conducted on the cause, biological mechanisms, new and better form of therapy and, ultimately, the cure for CF is an important part of our CF Center activities. You can be an important part of that research!
Research Participation: A Families Perspective
Richard and Rohini along with their rambunctious one-year-old daughter Ria live in Palo Alto, CA. Richard is originally from Belfast, Northern Ireland and Rohini is from Topeka, Kansas. Work brought them to the Bay Area and now they cannot imagine living anywhere else. On any given weekend you can find this family hiking the Dish, picking up goodies at the local farmers market, or running around Stanford.
Learning of Ria's condition through the newborn testing process Rohini and Richard experienced a whole variety of emotions from confusion to disbelief to anger and eventual acceptance. They are forever grateful to their family, friends, care team at Stanford, and pediatrician for their love, support, and patience.
Questions and Answers regarding the Research Process
1. How did you hear about the study?
Our incredible care team including Jackie Zirbes, Julie Matel, and Zach Sellers all introduced us to this opportunity
2. What were your concerns regarding enrolling your child in a study?
Our primary concern was the potential side effects that our daughter may face. It was a matter of understanding the anticipated benefits versus potential risks that helped us to decide to participate in this study.
3. What would you share with parents of young children who are considering enrolling their child in a study?
No question is too small or insignificant, ask every question that comes to mind. It can be directly or even peripherally related to the study, all of your questions matter. And my experience is that the team welcomes any and all questions at any time of day or night.
4. How would you describe your research experience?
Emotional. There have been some high highs as we see her stats improve but there have also been some low lows while we try to calm our nearly inconsolable daughter during blood draws. We would not have been able to get through those low moments without the support of our friends, family, pediatrician, trial care team, and clinic care team. We feel incredibly grateful for this opportunity and are hopeful that our daughter will be an even stronger, smarter, sassier version of herself at the end of it.