It is very important that every person or family with a young child with sickle cell disease have a plan for how to get help immediately, at any hour, if there’s a problem. Be sure to find a place that will have access to your medical records or bring a copy.   Go to an emergency room or urgent-care facility right away for: • Fever above 101° F • Difficulty breathing • Chest pain • Abdominal (belly) swelling • Severe headache • Sudden weakness or loss of feeling and movement • Seizure • Painful erection of the penis that lasts more than 4 hours   Call a doctor right away for: • Pain anywhere in the body that will not go away with treatment at home • Any sudden problem with vision   Abstract source

People with sickle cell disease can live full lives and enjoy most of the activities that other people do. The following tips will help you, or someone you know with sickle cell disease, stay as healthy as possible.  • Find good medical care. Sickle cell disease is a complex disease. Good quality medical care from doctors and nurses who know a lot about the disease can help prevent some serious problems. Often the best choice is a hematologist (a doctor who specializes in blood diseases) working with a team of specialists. • Prevent infections. Common illnesses, like the flu, can quickly become dangerous for a child with sickle cell disease. The best defense is to take simple steps to help prevent infections. • Learn healthy habits. People with sickle cell disease should drink 8 to 10 glasses of water every day and eat healthy food. They also should try not to get too hot, too cold, or too tired.   Abstract source

Children and adults with sickle cell disease (SCD) often require care in the emergency department (ED) of hospitals and clinics for health issues related to SCD. The ED may be a patient’s only option for health care when symptoms, such as pain crises, cannot be managed at home or when a patient does not have access to a healthcare provider who specializes in treating SCD. The Sickle Cell Data Collection (SCDC) program found that in California, people with SCD seek care in the ED an average of three times a year from their late teens to their late 50s.   Tips for ED Health Providers • Take complaints of pain from patients with SCD seriously and treat promptly with appropriate fluids and pain medication. • Work with the SCD team at your hospital or clinic to develop individualized care plans for patients with SCD, especially those with frequent ED use. When possible, make these plans available in the electronic medical record.   Abstract source

What Is Sickle Cell Disease? Sickle cell disease (SCD) is a group of inherited red blood cell disorders. • Healthy red blood cells are round and they move through small blood vessels carrying oxygen to all parts of the body. • In SCD, the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”. • Sickle cells die early, which causes a constant shortage of red blood cells. • Sickle cells can get stuck in small blood vessels and block the flow of blood and oxygen to organs in the body. These blockages cause repeated episodes of severe pain, organ damage, serious infections, or even stroke.   What Causes Sickle Cell Disease? SCD is inherited in the same way that people get the color of their eyes, skin, and hair. • A person with SCD is born with it. • People cannot catch SCD from being around a person who has it.   Abstract source

Introduction: Hydroxyurea decreases many complications of sickle cell anemia (SCA) but is underused in treatment-eligible patients. Barriers to hydroxyurea initiation occur on the health care system, provider, and patient level. Novel strategies to increase hydroxyurea use in patients with SCA are needed. To address this challenge at our center, we implemented the Quick Start Hydroxyurea Initiation Project (Q-SHIP).   Methods: Patients with SCA were eligible to participate in Q-SHIP if they presented to the Children’s National Health System (CNHS) emergency department (ED) for pain or acute chest syndrome and were not taking hydroxyurea. Patients <9 months old, on chronic transfusions, pregnant, or not followed by CNHS hematology were excluded. Eligible patients were referred to a weekly Q-SHIP clinic visit focused on hydroxyurea education and were offered initiating treatment at the visit’s conclusion. Participants completed a pre-session questionnaire, discussed hydroxyurea with a hematologist using a handbook developed by CNHS, and watched videos featuring patients and parents of children with SCA sharing their experience with hydroxyurea. Subjects were classified as starting hydroxyurea if they had a clinic visit for hydroxyurea monitoring within 3 months of participation in a Q-SHIP session.   Results: Over 13 months (2/1/2016 – 3/31/2017) 65 eligible patients participated in Q-SHIP a median of 5 days (IQR 2, 20 days) after ED or hospital discharge. Although 44% (28/64) of participants reported no previous hydroxyurea offer, provider clinic documentation indicated that 61% (17/28) of these families had declined a previous hydroxyurea offer. After Q-SHIP, 55% (36/65) of participants started hydroxyurea. Subjects who started hydroxyurea after Q-SHIP were similar to those who did not, except subjects who started were more likely have a history of an intensive care unit admission (Table 1). After a median follow-up of 11 months, 81% (29/36) of participants who started hydroxyurea after Q-SHIP continued on therapy. Among Q-SHIP participants continuing treatment, mean corpuscular volume increased by a median of 8.6 fL (IQR +5.4, +17.7, p<0.0001) and hemoglobin F increased by a median of 5.8% (IQR +3.0, +11.3, p<0.001). One year after implementation of Q-SHIP, the proportion of treatment-eligible patients with SCA who presented to the ED with pain or ACS who were receiving hydroxyurea increased; February 2016: 56% (32/57) vs. February 2017: 73% (43/59), p=0.059. Abstract source

The NCAA Sport Science Institute is committed to the health, safety and well-being of student-athletes with sickle cell trait. Sickle cell trait is not a disease; it is an inherited red blood cell condition that can affect athletes at all levels. While sickle cell trait is not a barrier to playing competitive sports, athletes with sickle cell trait have experienced significant physical distress, including collapse and death during intense exercise. Heat, dehydration, inadequate acclimatization, altitude and asthma can increase the risk for medical complications in athletes with sickle cell trait.   Abstract source

Background: Sickle nephropathy (SN) is a common and progressive manifestation of sickle cell anemia (SCA). Albuminuria indicates glomerular injury and portends renal failure. In a murine model of SCA, we found that increased TGF-β signaling underlies SN, and its downregulation by losartan, an angiotensin receptor blocker, reduced albuminuria and SN progression. The effect of losartan in humans with SCA is unknown.   Objectives: Determine the effect of losartan on SN in a phase-2, open-label study to inform the design of a phase-3 randomized trial.   Design/Method: Oral losartan was administered for 6 months to individuals with SCA (HbSS or S/β0-thalassemia) >=6 years of age. Main exclusion criteria: chronic transfusions, GFR<60 mL/min/1.73m2, hyperkalemia, and contraindications to losartan. Enrollment was in 3 groups defined by urinary albumin-to-creatinine ratio (UACR): no albuminuria (NoA: <30mg/g); microalbuminuria (MicroA: 30-300); and macroalbuminuria (MacroA: >300). The primary endpoint was a >=25% reduction UACR from baseline. Sample size was calculated to detect the primary endpoint in >=30% of the MicroA group with >80% power. Secondary endpoints: urine osmolality, GFR, UACR classification and toxicity.   Results: There were 36 participants (mean age 24.1y; 53% female) in 3 groups (NoA=15; MicroA=13; MacroA=8). Four were non-evaluable (marked non-compliance and/or early termination). The primary endpoint (>=25% reduction in UACR) was met in 41% (13/32) overall [83% (5/6) of MacroA; 58% (7/12) of MicroA; 7% (1/14) of NoA]. Among MacroA and MicroA participants, 67% (12/18) met the primary endpoint. Overall, UACR classification improved in 28% but worsened in 9%. In MacroA and MicroA participants, UACR classification improved in 50% but worsened in 11%. Mean UACR was 202 mg/g (S.E.M. 59, median 50) before and 137 (S.E.M. 47, median 16) after losartan (P=0.29). Urine osmolality and GFR did not change significantly (393 vs 370 mosm/kg•H20, P=0.11; 147 vs 139 mL/min/1.73m2, P=0.43). Losartan was discontinued for leg cramps (N=1); GFR decline >25% (N=1); and serum creatinine rise >50% (N=1).   Abstract source

Background: Patients with sickle cell anemia (SCA) experience recurrent painful crises that affect quality of life. Studies using patient diaries have shown that SCA pain is more prevalent and severe than previously reported based on healthcare facility utilization alone. DOVE was a multinational, study that assessed the efficacy and safety of prasugrel in reducing the rate of vaso-occlusive events in children with SCA (NCT01794000). DOVE incorporated an electronic patient reported outcome (ePRO) diary to assess daily sickle cell pain.   Objectives: To determine the feasibility of an ePRO diary to assess the frequency and intensity of daily sickle-cell pain and its impact on daily function in a large global study of children and adolescents with SCA.   Design/Method: DOVE utilized an hand-held ePRO device to collect daily data related to sickle cell pain, use of analgesics for pain and study drug adherence for up to 9 months in children >= 4 years of age. The diary content was translated into 11 languages and comprised of subjective (pain intensity, activity interference) and objective (analgesics used, school attendance) components. Pain intensity was measured using a modified version of the Faces Pain Scale-Revised. Data were transferred daily via cellular network to a central database that was accessible by study personnel to monitor compliance. Rates of patient compliance, as defined by a diary completion rate of at least 80%, were calculated. Completion was calculated as the number of daily diary entries divided by the total number of expected diary entries.   Results: A total of 311 patients received a diary and 268 patients provided up to 9 months of diary entries. Rates of diary compliance and completion were high throughout the diary collection period. For the subjective components, 92.6% of patients were compliant and the overall completion rate was 94.4%. For the objective components, 89.7% of patients were compliant and the overall completion rate was 93.3%. Data from the diary have been analyzed and reported (Heeney et al NEJM 2015).   Conclusion: With careful design, patient support and appropriate monitoring, an ePRO diary can successfully measure the impact of daily sickle cell pain in large global studies. Study sponsors Daiichi Sankyo and Lilly.   Abstract source

Objective: The study objective was to identify the perceptions of children with sickle cell disease (SCD) in the school environment. Methods: Semistructured interviews (N = 14) were conducted with pediatric patients ages 6 to 10 who attended Metro Nashville Public Schools. These participants were recruited through the Vanderbilt Sickle Cell Disease Clinic. Participants were asked about the perceived efficacy of their teachers to (1) understand SCD; (2) communicate with students regarding SCD; (3) handle an SCD-related pain episode in school; and (4) identify methods to compensate for school absenteeism associated with an SCD diagnosis. Content analysis identified underlying themes. Results: Five themes emerged that highlighted the perceptions and recommendations of children with SCD in the school environment: (1) perceptions that allow students to prevent SCD from limiting their school experience; (2) administrator actions to alleviate challenges associated with SCD; (3) communication about SCD; (4) how SCD interferes with school activities; and (5) ways students advocate for themselves. Students also provided four areas of recommendations for school personnel: (1) ways teachers can help with school activities; (2) make-up work for school absences; (3) empowering students with SCD; and (4) helping with SCD episodes at school. Conclusions: Students with SCD advocated strongly for their needs at school to attain their education. However, they perceived school personnel to lack knowledge about SCD management. This could be overcome with a handbook specific for teachers of students with SCD that could address each of the five themes. Abstract source Full text

The cell adhesion molecule P-selectin plays a key role in the pathogenesis of a vaso-occlusive crisis (VOC) in patients with sickle cell disease (SCD). In the double-blind, placebo-controlled phase 2 SUSTAIN study, crizanlizumab (humanized, anti-P-selectin monoclonal antibody) 5 mg/kg significantly lowered the rate of VOC in patients with SCD by 45% vs placebo. In SUSTAIN, patients with SCD were randomized to crizanlizumab 2.5 mg/kg, crizanlizumab 5 mg/kg, or placebo intravenously 14 times over 52 weeks. The primary endpoint was the annual rate of VOC with crizanlizumab vs placebo. This post hoc descriptive analysis evaluated the proportion of patients who did not experience a VOC during the study in the following subgroups: VOCs in the year prior to study entry (2-4/5-10), SCD genotype (HbSS/non-HbSS), and concomitant hydroxyurea use (yes/no). More patients were VOC event-free in the crizanlizumab 5 mg/kg arm than in the placebo arm, including those with more frequent prior VOCs (ie, 5-10; 28.0% vs 4.2%), the HbSS genotype (31.9% vs 17.0%) and/or using concomitant hydroxyurea (33.3% vs 17.5%). Further analyses of secondary endpoints demonstrated that crizanlizumab treatment significantly increased time-to-first VOC vs placebo in these subgroups. The rates of treatment-emergent adverse events were similar between treatment arms across all subgroups. This post hoc analysis of SUSTAIN shows that in patients with a high number of prior VOCs, on concomitant hydroxyurea and/or with the HbSS genotype, crizanlizumab treatment increases the likelihood of patients being VOC event-free and delays time-to-first VOC. Abstract source