Check here regularly for news from the North Shore-LIJ/Cohen Children's Medical Center Cystic Fibrosis Center team. This is where you will find out about new CF treatments, clinical studies, and news about the Center and our staff.

Important new Infection Control Policies!!! Learn more on the CFF Webcast Wednesday, May 27 at 2pm. Click here to register. Read the CFF's current guidelines here.

Responding to our patients: Adult self administration program

Self Administration Program: If admitted to LIJ, you'll find yourself on 5 North. The CF team has developed a self administration program where, if you so wish, you will be able to self-administer your daily CF maintenance medications in the hospital. The need for this program arose from CF patients' complaints that hospital's schedules did not coordinate with their specific needs, often resulting in missed doses especially if people with CF were asleep or out of their rooms.

Presently, CF patients are given the option self administer some or all routine daily medications. Medications for self administration include their short and long acting beta agonists (such as albuterol, Advair) either via hand held puffer or nebulizer, pancreatic enzymes, CF vitamins, TOBI®, Pulmozyme ®, and hypertonic saline.

If you choose to self administer, the nurse manager picks up a lock box from the pharmacy containing the CF medications and delivers to bedside nurse (RN) within 4 hours of your arrival to 5 North. The RN will explain the use of box, refilling procedure, and proper documentation of self administration. A respiratory therapist will supply you with any respiratory equipment necessary for self- administration.

This way, you become an integral part of this process by participating and documenting administration of medication. Adherence to regimen and proper administration techniques are monitored by medical, nursing and respiratory therapy staff.

Confusion about payment for Cayston and its nebulizer (Altera)

There is confusion about Medicare coverage for Cayston and the Altera nebulizer. In December, the Centers for Medicare & Medicaid Services (CMS) made the determination that the drug would be covered under Medicare Part D, a decision which excludes the Altera nebulizer from Medicare coverage. The CF Foundation (CFF) met with CMS officials numerous times in the latter half of 2010 to articulate its position that CF patients expect and require timely and affordable access to both Cayston and the Altera nebulizer. CFF continues to work with CMS to determine a pathway which enables Medicare beneficiaries with CF patients to obtain both the medication and the required delivery device, without undue financial burden including high out-of-pocket costs.

Pediatric

Parenting Children with Cystic Fibrosis
CF parents face a unique set of challenges - keeping up with treatments, clinic visits and daily activities can be frustrating, tiresome and isolating. Parenting Children with Health Issues is based on the Love and Logic parenting program developed by child psychologists and parents of children with CF. Click here for more information.

Cystic Fibrosis and Constipation in Children: An Underestimated Medical Condition
A recent investigation of 214 pediatric CF patients revealed that 47 percent of the CF children studied have constipation. Furthermore, 106 abdominal radiographs were independently assessed utilizing the Barr and Leech scores to determine the test's diagnostic value. The study concluded that constipation is a significant medical issue in CF and was associated with low total fat absorption and a history of meconium ileus; and that abdominal radiography seems of little value in the regular follow-up of CF patients.
Source: Constipation in pediatric cystic fibrosis patients: an underestimated medical condition. J Cyst Fibros 2010 Jan; 9(1):59-63.

Adult

Adult Guide to Cystic Fibrosis at CFF.org
The Adult Guide to Cystic Fibrosis — is a one-stop source for accurate information and practical advice on all aspects of living with CF, with pages packed with answers, tips, tricks, and tons of resources related to your health and well-being, all compiled from advice from medical experts and others living with CF. The specific challenges of being an adult with CF - finances, relationships, work — are covered as well. http://www.cff.org/adults/

Status of Gene Therapy in CF
The CF gene was discovered in 1989; and it was thought that a cure, especially a genetic one, would be close behind. Since then many attempts have been made, in the laboratory and in clinical trial settings, to correct the CF defect with gene therapy. In fact, more than 20 clinical trials have been conducted with a variety of viral and non viral gene transfer agents. Unfortunately, there have been many problems with CF gene transfer. The lung itself presents a barrier as it tries to protect itself from anything foreign, and the body’s immune system recognizes the transfer agent as foreign and attacks it. The use of a virus to transfer the gene also results in a mild infection. There have also been problems with transferring enough normal genetic material and in many cases several applications (via bronchoscopy) had to be done. Major questions still remain: how much correction of the defective gene must be achieved? and how would this translate into clinical benefit for people with CF? Research is now centered on the use of non-viral agents that can be 1) manufactured in large quantities, 2) nebulized into the lungs, and 3) are safe for inhalation. This is still in early stages of research, but progress has been made to make these agents more “humanized” so there will be less chance of them causing an infection or being destroyed by the body’s own defenses.
Source: Jane C. Davies and Eric W. F. W. Alton : Gene Therapy for Cystic Fibrosis. Proc Am Thorac Soc Vol 7. pp 408–414, 2010,

Improving Outpatient CF Care at the Adult Center
Our adult CF Center was selected by The Cystic Fibrosis Foundation (CFF) as one of ten adult centers in the nation to participate in an adult Quality Improvement initiative to provide our people living with CF the opportunity to partner with us in education/re-education of airway clearance techniques (ACT) to stabilize and improve lung health.

Interestingly, we discovered, through surveys and from speaking to our patients, that many are not performing ACT effectively and/or adequately.

This is very important because if we do nothing but improve adherence to CF care by properly using ACT and other available drugs; we will increase life expectancy in people living with CF by five years!

We are asking all our adult patients to please make an appointment to meet with Donna Tsang, our CF respiratory therapist and director of the PFT lab. To make this appointment, call Maria, Donna›s secretary at 516 465-5420.

This session will last 60 minutes. Patients who already did this, found the session very helpful. This can be done at same time when you come to see our doctors. Please bring your ACT devices (Acapella, Flutter, Vest garment {not machine}, Precursor) with you so Donna can check your technique and give you instant feedback and suggest any improvement (if any is needed). Katherine will also go over CF physiology and the importance of daily treatment.

Patients are a very important part of our CF team and we need your help and input to improve your lung health.

Hope to see you soon!

Pharmacy

FDA approves VERTEX 770 drug : Kalydeco®, (Ivacaftor) in patients with CF and the G551D mutation
Kalydeco™ (generic name, ivacaftor; previously known as VX-770) is a new oral medication for the treatment of cystic fibrosis, approved by the U.S. Food and Drug Administration (FDA) in January 2012. It is the first drug available that targets the underlying cause of CF — a faulty gene and its protein product, CFTR. The FDA approved Kalydeco (kuh-LYE-deh-koh) for people ages 6 and older with the G551D mutation of CF.

Click here for more information.

PHARMAXIS ANNOUNCES MAJOR MILESTONE: POSITIVE BRONCHITOL OPINION FOR EUROPE
Pharmaceutical company Pharmaxis today announced the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending the granting of a Marketing Authorisation for Bronchitol, clearing the way for the product to be used in Europe "for the treatment of cystic fibrosis in adults as an add-on therapy to best standard of care.Pharmaxis expects the European Commission to confirm this opinion and grant the Marketing Authorisation for Bronchitol in January 2012. SOURCE: Pharmaxis Ltd, Sydney, Australia Bronchitol has been developed to help clear mucus (a major source of lung infections), improve lung function and reduce exacerbations in patients with cystic fibrosis. Bronchitol is a proprietary formulation of mannitol administered as a dry powder in a convenient hand-held inhaler. Bronchitol hydrates the lungs, helps restore normal lung clearance, and allows patients to clear mucus more effectively. Clinical studies have shown Bronchitol to be safe, effective, and well tolerated in treating patients cystic fibrosis.

Two Potential Drugs for CF Show Disappointing Results
Denufosol,
Denufosol, a drug being tested in mild CF lung disease does not appear to be effective, according to the newly-released results of the phase III TIGER-2 trial. Denufosol is a novel channel regulator that increases the movement of the salt chloride while inhibiting the movement of sodium; this would make the very thick CF mucus more hydrated and less sticky. TIGER-2 (Transport of Ions to Generate Epithelial Rehydration) was a phase III study of CF patients with mild lung disease (baseline FEV1 >75% to < 110% predicted). The study was a 48 weeks study that enrolled a total of 466 people with CF. "The analysis of the primary endpoint, key secondary endpoints and select subgroup populations in TIGER-2 indicates an absence of meaningful treatment benefit in this patient population," said Dr. Charles Johnson, Chief Medical Officer, during the teleconference. Dr. Johnson is the former director of the CF Center at Washington University, St. Louis.

Liprotamase
An advisory panel of the U.S. Food & Drug Administration (FDA) said it did not have sufficient clinical trial data to recommend approval of liprotamase, a non-porcine pancreatic enzyme therapy for the treatment of pancreatic enzyme insufficiency in CF. The FDA panel was not convinced that liprotamase was any better than current FDA-approved porcine-derived pancreatic enzyme products.
Source: Cystic Fibrosis News Network. January 13, 2011

ARIKACE™ Demonstrates Sustained Benefit
Transave, Inc., today reported interim results from a multi-cycle Phase II open label clinical trial in 49 CF patients on its lead investigational drug, ARIKACE™ (liposomal amikacin for inhalation). The data indicated that ARIKACE™, delivered once daily for 28 consecutive days followed by 56 days off-treatment for four cycles demonstrated statistically significant improvement in lung function that was sustained during the 56 days off study drug. ARIKACE™ was well-tolerated during the four cycles. According to the company, preparations are underway to launch Phase III studies. ARIKACE demonstrated statistically significant reduction in Pseudomonas density which was sustained of the treatment period of four cycles for 12 months.
Source: ARIKACE™ Demonstrates Sustained Benefit in the Treatment of Cystic Fibrosis Patients Who Have PSEUDOMONAS Lung Infections

ARIKACE^® trial on hold
The U.S. Food and Drug Administration (FDA) has notified Insmed, the manufacturers of ARIKACE^®, that the agency has placed a clinical hold on Insmed's phase 3 clinical trials for ARIKACE^® (liposomal amikacin for inhalation) in Cystic Fibrosis (CF) patients with Pseudomonas lung infections and patients with non-tuberculous mycobacterial (NTM) lung disease. A clinical hold is a notification issued by FDA to the sponsor to delay a proposed clinical trial or suspend an ongoing clinical trial. The Company has been informed by FDA that this decision was based on an initial review of the interim results of a long-term rat inhalation carcinogenicity study, recently reported to the agency by Insmed, with ARIKACE. In this study, rats received daily doses of ARIKACE by inhalation for up to two years.

PARI Pharma’s Altera Delivers Gilead’s Cayston in Head-to-Head Study with Tobramycin Inhalation Solution
Phase III clinical trials of Cayston delivered by the Altera Nebulizer System versus tobramycin inhalation solution in CF patients with Pseudomonas aeruginosa delivered a mean increase in FEV (1) percent predicted from baseline to day 28 of 8.35 percent, meeting statistical superiority for the Cayston/Altera combination over the tobramycin solution. Safety results were similar across both arms of the study of 268 randomized patients, with lower incidence of cough in patients receiving Cayston. Altera delivers Cayston in two to three minutes – “a significant reduction in delivery time compared to other inhalation therapies,” according to the president of PARI Pharma GmbH.
Source: PARI Pharma’s Altera Delivers Gilead's Cayston in Head-to-Head Study with Tobramycin Inhalation Solution

Social Work

To help you navigate the complex world of health insurance and healthcare coverage, the CFF Patient Assistance Resource Library (http://www.cff.org/LivingWithCF/AssistanceResources/Library/) offers a range of useful information, including:

• Sample letters of medical necessity and prior authorization;
• How-to guides to navigating common insurance obstacles;
• Templates for insurance appeals, exceptions and reconsiderations;
• CF care guidelines;
• Evidence-based medical journal articles; and
• White papers for medical professionals.

Mother of CF Patient Receives Prestigious Recognition
Maryann O'Mahony is the recipient of 2010 Long Island MVP award from the Greater New York Chapter of the Cystic Fibrosis Foundation. Many thanks to Maryann for her tremendous work in CF, and congratulations on this well-deserved recognition.

Team Announcements

FDA approves TOBI Podhaler to treat a type of bacterial lung infection in cystic fibrosis patients
The U.S. Food and Drug Administration today approved TOBI Podhaler (tobramycin inhalation powder) for the management of cystic fibrosis patients with Pseudomonas aeruginosa, a bacterium that causes lung infections.

Cystic fibrosis is a genetic disease that affects about 30,000 pediatric and adult patients in the United States. Cystic fibrosis causes the body to produce thick, sticky mucus that builds up in the lungs and blocks airways. The buildup of mucus makes it easy for bacteria like P. aeruginosa to grow and cause a chronic lung infection that, over time, can severely damage the lungs. Many patients with cystic fibrosis are treated with antibiotics using a nebulizer machine.

TOBI Podhaler, a plastic, handheld inhaler device, contains a dry powder formulation of tobramycin, an antibiotic used to treat P. aeruginosa infection. The powder is inhaled twice daily using the Podhaler device for 28 days. Patients should then stop TOBI Podhaler therapy for 28 days before resuming again.

"Today's approval broadens the available delivery mechanism options for patients with cystic fibrosis who require treatment for P. aeruginosa," said Edward Cox, M.D., M.P.H, director of the Office of Antimicrobial Products in the FDA's Center for Drug Evaluation and Research. "This product is the first dry powder antibacterial drug delivered with a handheld dry powder inhaler."

TOBI Podhaler's effectiveness was established in a study of 95 pediatric and adult patients with cystic fibrosis. All patients were 6 years of age or older and infected with P. aeruginosa. Patients were randomly assigned to receive TOBI Podhaler or a placebo for the first 28 days of the study. All patients then received treatment with TOBI Podhaler for the remainder of the study.

The study was designed to determine the improvement in lung function by measuring the change in forced expiratory volume in one second (FEV1). Patients treated with TOBI Podhaler experienced a statistically significant increase of 12.5 percent in FEV1 compared to 0.09 percent in patients treated with placebo. Additional data supporting safety and effectiveness were available from other studies enrolling 487 patients.

Common side effects reported in patients treated with TOBI Podhaler during the clinical studies included cough, including a cough that produces phlegm or mucus; coughing up of blood (hemoptysis); lung disorder; shortness of breath; fever; mouth and throat pain; changes in voice volume or quality (dysphonia); and headache.

TOBI Podhaler is marketed by East Hanover, N.J.-based Novartis.

For more information:

FDA Approved Drugs: Questions and Answers

NIH: Cystic Fibrosis



Join us at the annual "Great Strides" Walk on June 9, 2013!

Join us at the annual "Great Strides" Walk on June 9 the at the New York Institute of Technology Campus. Walk starts at 9am. Click here to register, and enter "NSLIJ CF Team" in the "Find a Team" field to become part of our team!

If you can't join us on June 9, please participate at one of the other regional Great Strides walks:
May 11th - Great Strides Smithtown (Blydenburgh Park)
May 18th - Great Strides Westhampton Beach (Village Gazebo)
June 2nd - Great Strides Holtsville (Holtsville Park & Zoo)

Register for any of these events here, or make your donation now.



Listen to Dr. Germana on "Island Outlook"
Dr. Joan Germana was interviewed on Island Outlook for the Long Island Radio Group. The interview aired on WHLI, WKJY, WBZO and WIGZ on April 29, 2012.





Steven and Alexandra Cohen Children’s Center/Long Island Jewish Medical Center honored at the North American Cystic Fibrosis Conference.
Steven and Alexandra Cohen Children's Center/Long Island Jewish Medical Center was honored at the North American Cystic Fibrosis Conference, being named one of eleven centers to earn the 2010-2011 Quality Care Award. These awards honor centers that excel at the following quality improvement performance standards:

• Actively uses clinical outcomes data to identify opportunities for improvement and documents results of improvement efforts.
• Aligns improvement efforts to result in measurable improvement in important clinical outcomes.
• Consistently and actively involves patients and families in identifying, designing and/or implementing improvement efforts.
• Employs innovative strategies to improve care processes and outcomes.
• Implements system changes that result in high reliability of care processes.

Learn more at the News and Events section on the CF Foundation's website. You can read the full article in the CFF Winter Highlights newsletter, here.

Insurance Information

CF Foundation launches new Patient Assistance Resource Library
The CF Foundation recently launched the Patient Assistance Resource Library on its website. The CF community can now use this unique library’s online resources when faced with health care coverage and reimbursement challenge, including sample letters, how-to guides, templates for insurance appeals, CF care guidelines and other valuable information. Visit the library at www.cff.org/LivingWithCF/AssistanceResources/Library. You can read the full article in the CFF Winter Highlights newsletter, here.

Important Information on Health Care Reform
According to the Department of Health and Human Services, the following changes will apply to all insurance plans which begin on or renew after September 23, 2010:

• No pre-existing condition exclusions for children
• No withdrawal of coverage based on an unintentional mistake on an application
• No lifetime limits on health coverage
• Restrictions on insurer use of annual limits of coverage (must be at least $750,000), phasing in to no limits by 2014
• Right to see an OB-GYN without a referral
• Right to use emergency rooms without prior approval.

Click here for more information.

Research

Stay Informed about CF Clinical Trials
To help keep the CF community informed about CF clinical trials, the Cystic Fibrosis Foundation recently launched a new online tool that generates an email alert whenever new CF clinical trials and trial results are posted. Sign up today at www.cff.org/research/ClinicalResearch/Find/ClinicalTrialAlerts/.