These issues more commonly occurs while asleep, when muscles are more relaxed. Swallowing muscles in the pharynx can be affected, leading to aspiration coupled with a poor coughing mechanism increases the likelihood of infection/pneumonia. Mobilizing and clearing secretions involve manual or mechanical chest physiotherapy with postural drainage, and manual or mechanical cough assistance device. To assist in breathing, Non-invasive ventilation (BiPAP) is frequently used and tracheostomy may be sometimes performed in more severe cases; both methods of ventilation prolong survival to a comparable degree, although tracheostomy prevents speech development. Nutrition
The more severe the type of SMA, the more likely to have nutrition related health issues. Health issues can include difficulty in feeding, jaw opening, chewing and swallowing. Individuals with such difficulties can be at increase risk of over or undernutrition, failure to thrive and aspiration. Other nutritional issues, especially in individuals that are non-ambulatory (more severe types of SMA), include food not passing through the stomach quickly enough, gastric reflux, constipation, vomiting and bloating. Therein, it could be necessary in SMA type I and people with more severe type II to have a feeding tube or gastrostomy. Additionally, metabolic abnormalities resulting from SMA impair β-oxidation of fatty acids in muscles and can lead to organic acidemia and consequent muscle damage, especially when fasting. It is suggested that people with SMA, especially those with more severe forms of the disease, reduce intake of fat and avoid prolonged fasting (i.e., eat more frequently than healthy people) as well as choosing softer foods to avoid aspiration.
Sargramostim (tradename Leukine) is a recombinant granulocyte macrophage colony-stimulating factor (GM-CSF) that functions as an immunostimulator. Medical uses
Sargramostim is primarily used for myeloid reconstitution after autologous or allogeneic bone marrow transplantation. It is also used to treat neutropenia induced by chemotherapy during the treatment of acute myeloid leukemia. It also used as a medical countermeasure for treating people who have been exposed to sufficient radiation to suppress bone marrow myelogenesis.It is administered via intravenous infusion. Contraindications
Sargramostim should not be used in people with known hypersensitivity to GM-CSF, yeast-derived products or any component of the product and for concomitant use with chemotherapy and radiotherapy.There is a formulation with benzyl alcohol, which is toxic to babies; other formulations should be used. Sargramostim has not been tested in pregnant women but appears to be toxic to fetuses. There is no data as to whether sargramostim is expressed in breast milk. Adverse effects
Some people have experienced anaphylaxis when given the drug; and infusion reactions have occurred as well, including edema, capillary leak syndrome, a build up of fluid around the lungs and around the heart. Irregular heart rhythms have occurred, especially in people with a history of that problem. It suppresses some white blood cells, and may promote tumor growth. Pharmacology
Sargramostim is a version of GM-CSF, which has a normal role in human biology, causing progenitor cells to differentiate into neutrophils, monocytes, macrophages, and, myeloid-derived dendritic cells; it can also activate mature granulocytes and macrophages, and can contribute to the differentiation of megakaryocytic progenitors and erythroid progenitor cells.