children s health issues
Cystic Fibrosis (CF)
Treatment of Cystic Fibrosis
Routine immunizations Antibiotics, inhaled drugs to thin airway secretions, and airway clearance techniques to remove airway secretions Medications that help prevent airways from narrowing (bronchodilators) and sometimes corticosteroids Pancreatic enzyme and vitamin supplements High-calorie diet In people with specific variants, CFTR modulators A person with cystic fibrosis should have a comprehensive program of therapy directed by a doctor experienced with cystic fibrosis care—usually a pediatrician or an internist—along with a team of other doctors, nurses, a dietitian, a respiratory or physical therapist, and ideally a social worker, genetics counselor, pharmacist, and a mental health professional. The goals of therapy include long-term prevention and treatment of lung and digestive problems and other complications, maintenance of good nutrition, and encouragement of physical activity. Children with cystic fibrosis need mental health and social support because they may be unable to participate in normal childhood activities and may feel isolated. Much of the burden of treating a child with cystic fibrosis falls on the parents, who should receive adequate information, training, and support so they can understand the disorder and the reasons for the treatments. Adolescents need guidance and education as they transition to independence and assume responsibility for their care. Adults need support as they deal with issues related to employment, relationships, health insurance, and deteriorating health. The treatment of lung problems is focused on Preventing airway blockage Controlling infection The person should receive all Childhood Vaccination Schedules , particularly for infections that can cause respiratory problems such as which include postural drainage, chest percussion, hand vibration over the chest wall, and encouragement of coughing (see Chest Physical Therapy ), are started when cystic fibrosis is first diagnosed. Parents of a young child can learn these techniques and carry them out at home every day. Older children and adults can carry out airway clearance techniques independently by using special breathing devices, an inflatable vest that vibrates at a high frequency (a high-frequency oscillation vest), or special breathing maneuvers. Aerobic exercise, done regularly, can also help keep the airways clear. Bronchodilatorsare medications that help prevent the airways from narrowing. People usually take bronchodilators by inhaling them. People with severe lung problems and a low level of oxygen in the blood may need supplemental oxygen therapy. In general, people with chronic respiratory failure do not benefit from using a ventilator (breathing machine). However, occasional, short periods of mechanical ventilation in the hospital may help during an acute infection, after a surgical procedure, or while waiting for a lung transplant. Medications that help thin the thick mucus in the airways,such asdornase alfaor hypertonic saline (a highly concentrated salt solution), are widely used. These medications are inhaled through a nebulizer. They make it easier to cough up sputum, improve lung function, and may also decrease the frequency of serious respiratory tract infections. , given by mouth can relieve symptoms in infants with severe bronchial inflammation, in people who have narrowed airways that cannot be opened with bronchodilators, and in people who have an allergic lung reaction to a type of fungus ( Allergic Bronchopulmonary Aspergillosis ). Allergic bronchopulmonary aspergillosis is also treated with an antifungal medication given by mouth, by vein, or both. a nonsteroidal anti-inflammatory drug ( Nonsteroidal Anti-Inflammatory Drugs ), is sometimes used to slow the deterioration of lung function. Antibiotics are often used to treat both acute and chronic respiratory tract infections in people with cystic fibrosis. A sample of coughed-up sputum or a swab of a sample from the back of the throat and tonsils is collected and tested at regular intervals during stable health, and also during periods of increasing symptoms, so that the infecting organism can be identified and the doctor can choose the medications most likely to control it.Staphylococcus aureus, including methicillin-resistant or methicillin-sensitive strains, andPseudomonasspecies are commonly found. Many different antibiotics can be given by mouth to treat staphylococcal infections. To treat a newPseudomonasinfection, people are given the inhaled form oftobramycin,aztreonam, or colistin for 4 weeks. However, if the infection is severe, or results in significant worsening of symptoms or lung function, antibiotics given by vein (intravenously) may be needed. For this treatment, the Aminoglycosides Taking the inhaled form oftobramycinoraztreonamevery other month for a long time and also continuously taking an oral form of the antibioticazithromycin3 times each week may help control chronicPseudomonasinfection and slow the decline in lung function. CFTR modulators are oral medications taken chronically that improve the function of the defective protein made by variants in theCFTRgene. There are 4 CFTR modulators or combinations for people who have specific variants:ivacaftor,lumacaftor/ivacaftor, tezacaftor/ivacaftor, andelexacaftor/tezacaftor/ivacaftor. These medications can be used to treat about 90% of people who have cystic fibrosis. Doctors give these medications to people based on their age and the variants they have. Ivacaftorcan be given to people 1 month of age and older who have at least 1 copy of a specific cystic fibrosis variant. Thecombination of lumacaftor andivacaftorcan be given to people 1 years of age and older who carry 2 copies of the F508del variant. Thecombination of tezacaftor andivacaftorcan be given to people 6 years of age and older who carry 2 copies of the F508del variant or other specified variants. Thecombination of elexacaftor, tezacaftor, andivacaftorcan be given to people 2 years of age and older who carry at least 1 copy of the F508del variant or 1 copy of certain rare variants. CFTR modulators can improve lung function, pancreas function, and quality of life; increase weight; and decrease the salt concentration in sweat and the frequency of lung infections and hospitalizations. Although all of these drugs can be helpful, onlyivacaftorand the combination of elexacaftor, tezacaftor, andivacaftorare considered to be highly effective therapy. Doctors are working to develop drugs that will help people with other variants that cause cystic fibrosis. Newborns who have blocked intestines may be treated with special Enemas but often require surgery. Older children and adults who have Constipation in Adults or partially blocked intestines may be treated with The diet should provide enough calories and protein for normal growth. Because digestion and absorption can be abnormal even when pancreatic enzyme supplements are used, most children need to consume 30 to 50% more calories than the usually recommended amount to ensure adequate growth. The proportion of fat should be normal to high. High-calorie oral supplements can provide extra calories for children and adults. People who cannot absorb enough nutrients from food may need supplemental Tube Feeding inserted into the stomach or small intestine. People with cystic fibrosis should take double the usual recommended daily amount of Overview of Vitamins (A, D, E, and K) in a special formulation that is more easily absorbed. Medications that stimulate the appetite may be helpful (although many people with cystic fibrosis have a strong appetite without any such medications). When people exercise, have a fever, or are exposed to hot weather, they should increase their fluid and salt intake. People whose pancreas has been affected by cystic fibrosis must take capsules of pancreatic enzyme supplements with all meals and snacks. For infants, parents open the capsules and mix the contents with an acidic food such as applesauce so that the special coating on the pancreatic enzyme supplement does not dissolve before reaching the intestines. For some people, Medications for the Treatment of Stomach Acid , such as a histamine-2 blocker or a proton pump inhibitor, can improve the effectiveness of the pancreatic enzymes. Special milk formulas containing protein and fats that are easy to digest may help infants who have pancreatic problems and poor growth. People with cystic fibrosis who have Diabetes Mellitus (DM) in Children and Adolescents need to take In addition toinsulin, management includes nutrition counseling, a diabetes self-management program, and monitoring for eye and kidney complications. People also need special nutrition counseling because standard dietary recommendations for people with only diabetes or only cystic fibrosis are not adequate. At times, surgery may be needed to treat a collapsed lung, chronic sinus infections, severe chronic infection restricted to one area of the lung, bleeding from blood vessels in the esophagus, gallbladder disease, or obstruction of the intestine. Massive or recurring bleeding in the lung can be treated by a procedure called embolization, which blocks off the bleeding artery. Liver Transplantation has been successful for people who have bleeding resulting from esophageal varices or severe liver damage. Double Lung and Heart-Lung Transplantation for severe lung disease is becoming more routine and more successful with experience and improved techniques. For adults who have cystic fibrosis, average survival after a double lung transplant is about 9 years. Medications to treat chronic inflammation of the sinuses(sinusitis) are needed because this problem is very common. Treatment options include flushing a saltwater solution through the nose (nasal saline irrigation), inhalingdornase alfainto the nose using a nebulizer, and irrigating the nose and sinuses with antibiotics. A corticosteroid nasal spray is recommended to treat inflammation and swelling of the mucous membranes of the nose (allergic rhinitis). People who have Heart Failure (HF) are given medications (diuretics) to reduce the amount of fluid they retain. Diuretics help by increasing the amount of water the kidneys can get rid of from the body. People should also limit their intake of table salt and salty foods. Injections ofhuman growth hormonemay improve lung function, increase height and weight, and reduce the rate of hospitalization. However, this medication is costly and inconvenient for people to receive, so doctors do not commonly prescribe it. Some people with low oxygen levels in their blood may need supplemental oxygen that is usually given through a two-pronged nasal tube (cannula). Some people are treated with a tight-fitting mask placed over the nose or nose and mouth. A mixture of oxygen and air is delivered under pressure through the mask. This technique, called Alternatives (BiPAP) or People who have cystic fibrosis and their family members need to have discussions with their doctor and care team about their prognosis and what types of treatment they want to receive. These discussions are especially important for people whose lung function is worsening. People need to be prepared for what is to come and know what treatments may be done to extend life. With advanced cystic fibrosis, people and their families need to discuss the potential benefits and burdens of lung transplantation. Doctors should give people who have cystic fibrosis the information they need to make care choices and should help people determine how and when to accept dying and how to talk about dying. When aggressive treatments are no longer helpful, doctors may begin giving people treatments that aim only to relieve symptoms (called Hospice Care ). People usually do best when they make such decisions for end-of-life care well in advance of needing to. Such early discussions are very important because, later on, the illness often prevents people from explaining their wishes. This process of making decisions in advance for end-of-life care is called 