A bone marrow transplant is the only way to cure sickle cell disease, but it’s not a simple process. If you’re thinking about a transplant for yourself or a child, here are some things to know. A bone marrow transplant replaces the cells in your body that make red blood cells, called hematopoietic stem cells, with new ones. That means your body will stop making the sickle-shaped cells that cause the disease. In the procedure, a doctor takes healthy stem cells from the bone marrow of a donor and injects them into your body, usually through an IV tube into one of your veins.   Abstract source

Hydroxyurea (Hydrea) is used alone or with other medications or radiation therapy to treat a certain type of chronic myelogenous leukemia (CML; a type of cancer of the white blood cells) and certain types of head and neck cancer (including cancer of the mouth, cheek, tongue, throat, tonsils, and sinuses). Hydroxyurea (Droxia) is used to reduce the frequency of painful crises and reduce the need for blood transfusions in patients with sickle cell anemia (an inherited blood disorder in which the red blood cells are abnormally shaped [shaped like a sickle] and cannot bring enough oxygen to all parts of the body). Hydroxyurea is in a class of medications called antimetabolites. Hydroxyurea treats cancer by slowing or stopping the growth of cancer cells in your body. Hydroxyurea treats sickle cell anemia by helping to prevent formation of sickle-shaped red blood cells. Abstract source

Babies with sickle cell disease (SCD) should be referred to a doctor or provider group that has experience taking care of people with this disease. The doctor might be a hematologist (a doctor with special training in blood diseases) or an experienced general pediatrician, internist, or family practitioner. For infants, the first SCD visit should take place before 8 weeks of age. If someone was born in a country that doesn’t perform newborn SCD screening, he or she might be diagnosed with SCD later in childhood. These people should also be referred as soon as possible for special SCD care. All people who have SCD should see their SCD care providers regularly. Regularly means every 3 to 12 months, depending on the person’s age. The SCD doctor or team can help to prevent problems by: Examining the person Giving medicines and immunizations Performing tests Educating families about the disease and what to watch out for Abstract source

Usually, red blood cells (RBCs) are shaped like round discs. People who have sickle cell disease, though, have RBCs that are shaped like sickles, or crescent moons. This defect can cause painful episodes, serious infections, chronic anemia, and damage to body organs. These complications can vary from person to person depending on which type of sickle cell disease each has. Some people are relatively healthy and others are hospitalized often. But thanks to advancements in early diagnosis and treatment, most kids born with this inherited disorder grow up to live active, healthy, and productive lives. Abstract source

If you or your child has sickle cell disease (SCD), you should learn as much as you can about the disease. Your health care providers are there to help you, and you should feel comfortable asking questions. Pursue a Healthy Lifestyle Like all people, you or your child should strive to maintain a healthy lifestyle that includes: A nourishing diet Enough sleep Regular physical activity People with SCD often tire easily, so be careful to pace yourself and to avoid very strenuous activities. Don’t smoke and try to avoid second-hand smoke. If you drink alcohol, do so in moderation and drink extra water to avoid dehydration.   Abstract source

People who do not know whether they make sickle hemoglobin or another abnormal hemoglobin, such as SC, Sβ thalassemia, and SE, can find out by having their blood tested. This way, they can learn whether they carry a gene—or have the trait—for an abnormal hemoglobin that they could pass on to a child. With this information, parents can be better informed about the chances of having a child with some type of sickle cell disease, such as hemoglobin SS or SC, Sβ thalassemia, or others. Abstract source

Sickle cell disease changes the shape of the oxygen-carrying molecules (called hemoglobin) found inside red blood cells. Red blood cells are usually round and flexible. This allows them to glide smoothly through your bloodstream. But when you have sickle cell disease, the abnormal hemoglobin creates stiff, sickle-shaped red blood cells. If you have this disease, these abnormally shaped red blood cells can cause a number of health problems. For example: • The cells build up and block blood flow to organs and tissues. • The cells die sooner. Sickle cells live about 10 to 20 days. Healthy red blood cells live from 90 to 120 days. Red blood cells may burst open (hemolyze). Even though your body constantly makes new red blood cells to replace the old ones, sickle cell disease may prevent your body from keeping up with the demand.   Abstract source

During a blood transfusion, a person (the recipient) receives healthy blood from another person (the donor). The donated blood is carefully screened for diseases before it is used. Before receiving a blood transfusion, the recipient's blood is analyzed closely (using blood type) to make sure the donor blood is a close match to the recipient's. Blood is transfused into an arm vein slowly over 1 to 4 hours (except in an emergency when blood is transfused more quickly). Blood transfusions can: -Treat a sudden or short-term condition related to sickle cell disease. -Treat severe complications of sickle cell disease. -Prevent complications of sickle cell disease. -Lower the risk of stroke in infants and children. -Blood transfusions aren't usually used to treat uncomplicated painful events or mild to moderate anemia. Abstract source

SCD is a group of inherited red blood cell disorders. Healthy red blood cells are round, and they move through small blood vessels to carry oxygen to all parts of the body. In someone who has SCD, the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”. The sickle cells die early, which causes a constant shortage of red blood cells. Also, when they travel through small blood vessels, they get stuck and clog the blood flow. This can cause pain and other serious problems such infection, acute chest syndrome and stroke. Abstract source

Over the past century, great advances have been made in the understanding and treatment of sickle cell disease (SCD). This first “molecular disease,” caused by a single gene mutation, has advanced the field of modern human molecular biology. Many important discoveries have been made, and some treatments developed. These discoveries have identified innumerable questions and opportunities to better understand and treat this complex disease. Yet, many basic scientific processes are still not fully understood, too few treatments have been developed, and most of the people who have SCD do not have access to these treatments which could improve the duration and quality of their lives. With profound concern about the unmet scientific opportunities and lack of access to high-quality care, a group of SCD stakeholders has convened and is eager to find remedies. This document outlines their analysis and details a comprehensive plan to address these issues. The plan encompasses four priority areas – Access to Care in the United States, Training and Professional Education, Research and Clinical Trials, and Global Issues Related to SCD – which will be the focus of our collective efforts toward ultimately advancing SCD care, early diagnosis, treatment, and research. Abstract source