What're the treatments for cystic fibrosis?
Treatment for cystic fibrosis depends on the severity of the disease and which parts of the body are affected. Regular monitoring is essential. Comprehensive and intensive therapy should be directed by an experienced physician in conjunction with other physicians, nurses, nutritionists, physical and respiratory therapists, counselors, and social workers. The goals of therapy are maintenance of adequate nutritional status, prevention or aggressive therapy of pulmonary and
other complications, encouragement of physical activity, and provision of adequate psychosocial support.
Treatment usually consists of the intake of digestion enzymes, nutritional supplements, percussion and postural drainage of the lungs, improved antibiotics and inhalation of aerosols containing medication.A few attempts at gene therapy were initially successful, but failed to produce acceptable long-term results. Also, medications are being tested to correct a defective protein, called the CFTR protein, which is made at the command of the cystic fibrosis gene. The defective CFTR protein is responsible for the abnormalities in salt and water regulation that lead to thickened secretions. Due to advances in medical treatment, the median life expectancy of a newborn with cystic fibrosis increased from 4 years (in the 1960s) to 32 years today.
Cystic fibrosis patients are typically hospitalized somewhat regularly, often every 6 months depending on the severity of the case. Patients often have IV antibiotics through a PICC line or chest port for IV Antibiotics. Lung disease can be treated with antibiotics, mucus-thinning drugs and chest physical therapy, a form of physical therapy in which another person drums the patient's chest and back with cupped hands to help clear mucus from the lungs. Earlier approaches to diabetes treatment among cystic fibrosis patients generally did not address long-term effects because of the short cystic fibrosis life expectancy. However due to improving treatment of cystic fibrosis patients and their resulting longer lifespan, it is increasingly common to address diabetes symptoms that are not immediately harmful.
Daily chest physiotherapy and aerosol breathing treatments are very commonly prescribed for cystic fibrosis treatment. Typical physical therapy involves manual chest precussion (pounding), or possibly using a device such as the ThAIRapy Vest or the Intrapulmonary Percussive Ventilator (IPV) to achieve the same effect: loosening of the thick mucus. Drugs that help break up the mucus, such as dornase alfa, may help clear the lungs. Anti-inflammatory medication like ibuprofen may reduce the inflammation in the lungs. Inhaled antibiotics are used to prevent lung infections that may lead to hospitalization.
Digestive problems are treated with high-calorie diets, pancreatic enzymes and vitamin supplements. Pancreatic enzyme replacement as powder (in infants) or capsules should be given with all meals and snacks. The most effective enzyme preparations contain pancrelipase in pH-sensitive, enteric-coated microspheres or microtablets. The diet calls for a high-caloric content (twice what is considered normal for the child's age), which is typically low in fat and high in protein. As maintaining body weight is important for cystic fibrosis patients, a typical diabetic diet is not feasible and therefore insulin doses are instead adjusted to fit the typical high-calorie/high-fat cystic fibrosis diet.
Lung transplantation has become increasingly common for people with cystic fibrosis. Liver transplants have been performed successfully in patients with end-stage liver disease. Heart-lung and bilateral lung transplants have been performed successfully in patients with advanced cardiopulmonary disease. Transplantation is not a cure, however, and has been likened to trading one disease for another. Long-term immunosuppression is required, increasing the likelihood of other types of infection. About 50% of adults and more than 80% of children who receive lung transplants live longer than two years. Liver transplants are also done for cystic fibrosis patients whose livers have been damaged by fibrosis.
Gene therapy involves using an inhaled spray to deliver normal copies of the cystic fibrosis gene to the lungs. Gene therapy is being tested in several medical centers nationwide, but has not been approved for general use. Already scientists have transferred normal genes from chromosome 7 into laboratory animals and seen promising results.