Endari is an amino acid, approved by Food and Drug Administration (FDA) to treat sickle cell disease. It is available in powder form to be taken orally.  The exact mechanism of action of Endari is not fully understood but it increases the amount of free glutamine in the blood.  Glutamine is used to make anti-oxidant molecules which reduce the oxidative stress in sickle red blood cells resulting in increased flexibility of red blood cells while they travel through the body.  Safety profile of Endari is not available for children less than 5 years of age and risk of taking Endari in pregnancy is not known yet. Abstract source

Bone marrow transplantation is the only cure for sickle cell disease. Bone marrow is the fatty spongy tissue inside the bone where new blood cells are made. During transplantation, unhealthy bone marrow is removed with chemotherapy medicine and replaced with healthy donor stem cells which are the most immature form of cells in our body from bone marrow or umbilical cord blood.  After successful bone marrow transplantation, the donor bone marrow will begin to make new blood cells which will not sickle. It may take one year to recover or get better after your transplant.  There are two different types of bone marrow transplant depending upon who is donating the stem cells - Autologous Transplant and Allogeneic Transplant. Donors may come from your family or a member not related to you. Usually unrelated transplants have a higher risk of complications than match related transplant. Abstract source

Hydroxyurea is a chemotherapy drug used to treat cancer and blood diseases particularly in the conditions such as polycythemia vera (increased number of red blood cells), essential thrombocytosis (increased number of platelets) and sickle cell disease. It is the only drug approved by the Food and Drug Administration (FDA) to treat sickle cell disease. It works by increasing the levels of fetal hemoglobin (HbF) in Red Blood Cells (RBC) which allows the RBCs to be more flexible and slippery and makes it harder for RBCs to form the sickle shape. Hydroxyurea is available in pill and liquid form. It is usually available in pills of 200 mg, 300 mg, 400 mg and 500 mg dose strength under different brand names. Generic capsules of dose strength 250 mg and 500 mg of Hydroxyurea are also available in market. It is usually covered by insurance. Abstract source

Miracle kid Jordin Jackson speaks to living with sickle cell disease, raising money for her children’s hospital, and how to stay positive through pain. Jordin Jackson has a genetic blood disorder called sickle cell, which causes severe and unmanageable pain. The body makes sickle-shaped red blood cells instead of round cells that gather together and prevent oxygen from getting to the most important parts of her body.  Jordin is the 2017 Children’s Miracles Network Hospitals Champion from California. Related story

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When you meet 2-year-old Evelyn Islam, the first thing you notice is her grin. And her friendliness. And her curiosity.You can't help but smile as she mischievously steals phones to play with. You can't help but be drawn into a conversation as she explains her surroundings to you in earnest toddler-speak. And you can't help but play right along with her as she shows you her collection of beads from Beads of Courage, an organization that supports children who have serious illnesses.Although Evelyn's sickle cell anemia is a serious chronic medical condition, the fact that it isn't obvious from the outside makes it easy to see Evelyn for the person she is. It's easy for her to be defined by something other than her illness. Related story

Importance:  African Americans and individuals of African ancestry have a higher risk of stroke compared with non-Hispanic white individuals. Identifying the source of this disparity could provide an opportunity for clinical stroke risk stratification and more targeted therapy. Whether sickle cell trait (SCT) is an indicator of increased risk of ischemic stroke among African Americans is still unclear.Objective: To examine whether SCT is associated with a higher risk of incident ischemic stroke among African Americans.Design, Setting, and Participants:  This meta-analysis assessed the association of SCT with the risk of incident ischemic stroke. Four large, prospective, population-based studies with African American cohorts were assessed: Jackson Heart Study (September 1, 2005, through December 31, 2012), Multi-Ethnic Study of Atherosclerosis (July 1, 2002, through December 31, 2012), Reasons for Geographic and Racial Differences in Stroke (January 1, 2003, through December 31, 2014), and Women’s Health Initiative (October 1, 1998, through December 31, 2012). Using a Cox proportional hazards regression model adjusted for major stroke risk factors, this study estimated the hazard ratio for incident ischemic stroke associated with SCT. Data analysis was performed from July 10, 2016, to February 2, 2017.Interventions or Exposures:  Participants’ SCT status determined by polymerase chain reaction assay genotyping or a combination of whole-exome sequencing and imputation.Main Outcomes and Measures:  Incident ischemic stroke.Results:  This meta-analysis included 19 464 African American individuals (1520 with SCT, 17 944 without SCT, and 620 with ischemic stroke) from 4 studies, with a mean (SD) age of 60.0 (13.0) years (5257 [27.0%] men and 14 207 [73.0%] women). No differences were found in the distribution of risk factors for ischemic stroke comparing participants with and those without SCT at study visit 1 in each cohort. The crude incidence of ischemic stroke was 2.9 per 1000 person-years (95% CI, 2.2-4.0 per 1000 person-years) among those with SCT and 3.2 per 1000 person-years (95% CI, 2.7-3.8 per 1000 person-years) among those without SCT. After stroke risk factors were adjusted for, the hazard ratio of incident ischemic stroke independently associated with SCT in the meta-analysis of all 4 cohorts was 0.80 (95% CI, 0.47-1.35; P = .82). The results of the meta-analysis were similar to those of individual cohorts, in which the results were also similar.Conclusions and Relevance:  Sickle cell trait may not be associated with incidence of ischemic stroke among African Americans. The results of this study suggest performing a more thorough clinical evaluation of a stroke patient with SCT rather than assuming that SCT is the etiologic factor for the stroke. Abstract source Full text

This leaflet explains about priapism associated with sickle cell disease. If you have any further questions or concerns, please do not hesitate to contact the doctor or nurse caring for you. What is a priapism? Priapism is an unusual erection of the penis that is frequently painful and distressing. These are not associated with normal sexual function or desire. The majority of males with sickle cell anaemia (HbSS) are likely to experience a priapism in their life time. Priapism is less common in boys and men with sickle cell disease (HbSC) and sickle beta thalassaemia (HbSB). There are two common types of priapism: -Stuttering -Acute (or fulminant)   Abstract source