Administering complex pediatric IV therapies in the home
keeps the child close to family
keeps the child close to family
Common symptoms of infantile-onset Pompe disease include severe and rapidly progressive muscle weakness, delayed motor development, cardiomegaly/cardiomyopathy, respiratory distress and feeding difficulties.
http://www.genzyme.com/corp/media/Pompe_backgrounder.pdf
While to some the diagnosis of Pompe may feel like a reason to lose hope, Ira’s team of caregivers chose not to let him live his young life waiting to die. Instead, they researched opportunities to let Ira live life as a normal infant should.
When Ira was just 6-weeks old, his doctors started biweekly infusions of Myozyme®, an enzyme replacement therapy (ERT) which just received FDA approval in April, 2006. Administered intravenously, Myozyme is the first and only approved drug in the United States and Europe for the treatment of Pompe disease. ERT is intended to replace the deficient enzyme that causes Pompe disease.
http://www.genzyme.com/corp/media/Pompe_backgrounder.pdf
The severity of symptoms associated with Pompe’s disease can vary greatly among patients. Ira required a ventilator to assist with breathing and feeding pump for his nutritional intake. PHS’ staff of clinicians worked with his mother and team of caregivers at the hospital to ensure that Ira would be able to get the care he needed at home instead of the hospital.
Even though he was now living at home, Ira still required medical transport to go back to the hospital every other week for his Myozyme treatments. There is a risk for severe infusion reactions to Myozyme infusions and appropriate medical support measures need to be readily available when Myozyme is administered. Because of this warning, the decision was made that the hospital was the safest place for Ira to receive the infusions.
After about a year of the trips to the hospital, Ira’s family and medical team began researching the possibility of providing the Myozyme infusions in the home. Ira was tolerating the drug, but the costs and risks involved with transporting a medically fragile child to the hospital were adding up.
In September 2007, Ira’s medical team approached PHS about the possibility of providing the complex infusion therapy in Ira’s home. Before making a decision, PHS’ Pharmacist in Charge, Jill Liebers, PharmD, and Nursing Supervisor, Jean Stumpf, RN conducted extensive research into the drug and requirements for administering it safely.
According to Stumpf, “The primary question we had to address was, can this infusion be done safely in the home? We worked with the patient’s doctors, the inpatient infusion center and the drug manufacturer, Genzyme, to answer that question. Once we were able to develop a safe plan of care, we agreed to provide the service.”
As a safe plan of care was being developed by PHS clinicians and Ira’s team of caregivers, PHS billers worked to ensure that insurance coverage would not be an issue for the family and preparation began to not only provide ventilator and food pump support to Ira, but also the complex Myozyme and other IV treatments.
On November 21, 2007, the first home Myozyme infusion was administered by a PHS nurse, and while Ira has had to be readmitted to the hospital for short stays due to various complications related to Pompe, he is living his life at home as a strong, resilient toddler with his mother, brother and sister. PHS clinicians continue to monitor Ira’s progress as his treatment continues, adjusting his therapy when necessary to give Ira the greatest opportunity to live a long, prosperous life.
One-way medical transport to |
| *Cost Benefit Analysis of Providing Non-Emergency Medical Transportation, October 2005 http://trb.org/publications/tcrp/tcrp_webdoc_29 **Specialty Pharmacy News, Volume 4, Number 5 May 2007 |