Cochrane

Review question We reviewed the evidence for the effectiveness and safety of statins in children with inherited high blood cholesterol. Background Familial hypercholesterolemia is an inherited disease in which the blood cholesterol level is high. Vascular disease, i.e. furring up of the blood vessels, often occurs at an earlier age than usual, especially ...

Review question We reviewed the evidence available to see if rituximab is effective and safe when treating clotting factor inhibitors in people with severe hemophilia. This is an update of a previously published Cochrane Review. Background Hemophilia A and B are inherited conditions in which there is either reduced levels (or none at all) of factor ...

Review question We wanted to determine if there were any safe and effective interventions that prevent or reduce kidney complications in people with sickle cell disease (SCD). Background SCD is a serious inherited blood disorder where the red blood cells, which carry oxygen around the body, develop abnormally. Normal red blood cells are flexible ...

Cystic Fibrosis and Genetic Disorders Group Karla Mendonça Title Thursday, November 13, 2008 ...

Review question We reviewed the evidence about the effect of testing antibiotics in combination for acute airway infections in people with long-term (chronic) infection with Pseudomonas aeruginosa. Background The main cause of death in people with cystic fibrosis is chronic lung infection. People with cystic fibrosis now live longer due to the aggressive ...

Review question We wanted to determine if there were any safe and effective interventions that prevent silent strokes (also known as silent cerebral infarcts) in people with sickle cell disease (SCD). Background SCD is a serious inherited blood disorder where the red blood cells, which carry oxygen around the body, develop abnormally. Normal red ...

We reviewed the evidence for the use of oral supplements to increase calorie intake in people with cystic fibrosis. Background Cystic fibrosis affects many organs, including the digestive system, and can lead to food not being absorbed as it should be, which in turn leads to growth problems. Children with cystic fibrosis need more energy than other ...

Review question We reviewed the evidence about the effect of vibrating devices (e.g. Flutter, acapella, cornet, Quake®, intrapulmonary percussive ventilation, high frequency chest wall oscillators (e.g. Vest®), VibraLung® and MetaNeb®) to help people with cystic fibrosis clear their airways of mucus. This is an update of a previously published review. Background People ...

Review question What is the best way of using antibiotics to eliminate lung infections caused by a germ called Pseudomonas aeruginosa in people with cystic fibrosis? Background Cystic fibrosis is an inherited condition where the airways often become blocked with mucus. It is associated with chest infections, which can lead to progressive breathing ...

Review question What is the effect of hydroxyurea on clinical outcomes (changes in pain crises, life-threatening illnesses, survival, haemoglobin levels, quality of life and side effects) in people with sickle cell disease (SCD) of any genotype? Background SCD is an inherited genetic disorder that creates problems with haemoglobin (the substance ...