“When I was 10, I was flogged by a teacher because I didn’t do an assignment. I fainted… That episode became the beginning of the worst years of my life”. More Sickle cell Warriors like Timi Edwin are telling their stories and, reshaping the sickle cell narratives. Read this sickle cell survival story from Timi Edwin, Founder of Crimson bow. How Did You Discover You Had Sickle Cell? My parents discovered that I had sickle cell when I was 1 year old, but I didn’t really know what it meant and how it impacted my life till I was 10 years old. This does not mean that I didn’t have pain episodes and illnesses before that age, I did, I only felt and realized the full impact (pain, stigma, depression) from that age. Related story

Welcome to the oneSCDvoice Blog launch! To thank the growing oneSCDvoice community and in anticipation of our second anniversary, we are excited to launch the new oneSCDvoice Blog. In keeping with the tradition of providing the oneSCDvoice community members with trusted resources and support, our goal is to maintain a positive and helpful platform where individuals impacted by sickle cell disease gather to share their life experiences, support each other, and access valuable, trustworthy tools and information. Each week, we will publish a new blog featuring guest writers from across the oneSCDvoice community. Until now, oneSCDvoice has provided our community members with trusted resources by linking out to high quality content on the internet. The blog allows us to share our own unique voice with the sickle cell community. Here’s what you can look forward to when you read the blog in coming weeks:

• We'll introduce you to the Working Group members, the Community Managers, and oneSCDvoice community members—the people who make oneSCDvoice what it is.
• We'll share with you information and resources about the latest developments in sickle cell treatment including success stories, new research studies, and other highlights from the world of sickle cell disease.
• We'll respond to your questions on sickle cell disease.

We encourage questions and feedback and want to ensure you that we are listening to the community. Please contact us with ideas or topics you would like to suggest we write about—or let us know if you would like to submit a blog for consideration. Please reach out to us at contact@onescdvoice.com. We are kicking off the oneSCDvoice Blog with a poll: [yop_poll id=31]

What is Sickle Cell Disease? Sickle Cell Disease (SCD) is a group of disorders that affects hemoglobin. It is an atypical hemoglobin molecule (hemoglobin S) which can distort red blood cells into a sickle or crescent shape. How Does SCD Affect the Oral Health of Children? Children with SCD may present anemia, repeated infections, and shortness of breath. Also, they might experience episodes of pain. These manifestations of the condition need to be taken into consideration when treating children with SCD. Dental Characteristics of Sickle Cell Disease: • Delayed teeth eruption • Enamel Hypoplasia (Softening of the outer-most layer of the teeth) • Malocclusion (abnormal alignment of the teeth) • Periodontitis (condition that affects the gingiva and bone around the teeth) Abstract source

When it comes to taking care of yourself away from home, a lot depends on how severe your sickle cell symptoms can be and where you go to college. If you're going to college where the climate may make symptoms worse (like a high-altitude location or a region with very hot or cold weather), you may need more care for pain or other symptoms. If your college is near a big city, it may be easier to find doctors familiar with treating sickle cell disease than if your college is in a small town. Regardless of where you go to school, you'll need to plan your care around home and school. Here are some tips: Keep in touch with your at-home hematology team: Doctors and nurses who have been caring for you over the years are the best people to manage your overall health. Arrange in advance to get regular checkups during school breaks. Let your at-home care team know whenever you get medical care at school and send or bring a copy of any lab results or health center records to your hematologist. Have your hematologist back home make a copy of your records: This way you can share your health records with your new doctors and you have a copy ready in case of an emergency. Abstract source

There is a large therapeutic gap in sickle cell disease (SCD). Hydroxy-urea reduces acute sickle cell-related events, but does not appear to protect patients from cardiopulmonary complications, which are the major causes of death in SCD patients. The long-term effects of newer agents such as crizanlizumab and L-glutamine on the disease course remain to be determined. The only cure currently available for SCD is allogeneic stem cell transplant (allo-SCT); however, many barriers prevent allo-SCT from being readily offered to these patients. Investigations into other therapeutic approaches are, therefore, appropriate. Activated and circulating aged neutrophils (CANs) adhere to vascular endothelium and are pivotal for the pathogenesis of sickle cell vaso-occlusive crisis (VOC). CANs in mice are regulated by intestinal micro biota. Treatment of SCD mice with a cocktail of ampicillin, neomycin, metronidazole, and vancomycin (ANMV) induced reduction in CANs and protected the mice from fatal tumor necrosis factor-α-induced VOC. Therefore, antibiotic therapy might be a potential therapeutic approach for SCD. Abstract source Full text

Sickle cell disease is one of the most common autosomal recessive genetic diseases. It gives rise to abnormally shaped red blood cells with altered function, the primary clinical features being haemolytic anaemia and vascular occlusion. Acute complications are frequent and variable and include chest syndrome, stroke, infection mainly due to asplenia, bone pain and priapism. Other chronic complications which can occur are bone necrosis, nephropathy and heart, lung and skin disorders. Oral lesions are also very common and include aseptic pulp necrosis, mucosal damage due to anaemia, fungal infections due to numerous antibiotic therapies, dental eruption delays, bone pain and osteomyelitis of the maxilla, and oral neuropathies, including of the mental nerve of the chin. The oral care of sickle cell patients requires specific precautions such as good management of local anaesthetics, rigorous anti-infective prophylaxis as well as controlled prescription of analgesics. Regular oral follow-up of sickle cell patients is necessary. Abstract source Full text

Purpose: Genome editing holds both tremendous therapeutic promise and significant potential risk. Sickle cell disease (SCD), the most commonly inherited blood disorder, is a frontline candidate for the clinical applications of this tool. However, there is limited knowledge of patient community values and concerns regarding this new technology. This study aims to investigate the perspectives of three key decision-makers (patients, parents, and physicians) toward participation in future CRISPR-mediated somatic genome editing clinical trials. Methods: We utilized a mixed-methods approach, involving an educational video tool, two-part survey, and 15 moderated, audio-recorded focus groups, which were conducted in seven U.S. cities. Results: Study participants expressed hope that genome editing technology would rechart the course for SCD, but concerns related to involvement burden, uncertainty of clinical outcomes, and equity in access were identified. Major themes emerged from the focus groups: facilitators of, and barriers to, participation in future somatic genome editing clinical trials; information pertinent to the decision-making process; persons from whom participants would seek counsel before making a decision; and recommendations for the research community on meaningful engagement as clinical trials are designed and approved. Conclusion: The advent of genome editing has renewed hope for the SCD community, but caution tempers this optimism. Abstract source Full text

Background: Deoxygenated sickle hemoglobin (HbS) polymerization drives the pathophysiology of sickle cell disease. Therefore, direct inhibition of HbS polymerization has potential to favorably modify disease outcomes. Voxelotor is an HbS polymerization inhibitor. Methods: In a multicenter, phase 3, double-blind, randomized, placebo-controlled trial, we compared the efficacy and safety of two dose levels of voxelotor (1500 mg and 900 mg, administered orally once daily) with placebo in persons with sickle cell disease. The primary end point was the percentage of participants who had a hemoglobin response, which was defined as an increase of more than 1.0 g per deciliter from baseline at week 24 in the intention-to-treat analysis. Results: A total of 274 participants were randomly assigned in a 1:1:1 ratio to receive a once-daily oral dose of 1500 mg of voxelotor, 900 mg of voxelotor, or placebo. Most participants had sickle cell anemia (homozygous hemoglobin S or hemoglobin Sβ0-thalassemia), and approximately two thirds were receiving hydroxyurea at baseline. In the intention-to-treat analysis, a significantly higher percentage of participants had a hemoglobin response in the 1500-mg voxelotor group (51%; 95% confidence interval [CI], 41 to 61) than in the placebo group (7%; 95% CI, 1 to 12). Anemia worsened between baseline and week 24 in fewer participants in each voxelotor dose group than in those receiving placebo. At week 24, the 1500-mg voxelotor group had significantly greater reductions from baseline in the indirect bilirubin level and percentage of reticulocytes than the placebo group. The percentage of participants with an adverse event that occurred or worsened during the treatment period was similar across the trial groups. Adverse events of at least grade 3 occurred in 26% of the participants in the 1500-mg voxelotor group, 23% in the 900-mg voxelotor group, and 26% in the placebo group. Most adverse events were not related to the trial drug or placebo, as determined by the investigators. Conclusions: In this phase 3 randomized, placebo-controlled trial involving participants with sickle cell disease, voxelotor significantly increased hemoglobin levels and reduced markers of hemolysis. These findings are consistent with inhibition of HbS polymerization and indicate a disease-modifying potential. (Funded by Global Blood Therapeutics; HOPE ClinicalTrials.gov number, NCT03036813.). Abstract source

Individuals with sickle cell disease (SCD) experience cognitive deficits; however, it remains unclear whether medical treatments for SCD improve cognition. Given that executive abilities are typically impaired in individuals with SCD, they were the focus of the current study. Our primary hypothesis was that executive abilities would be higher acutely soon after a blood transfusion in children and young adults with SCD. We used tests from the NIH Toolbox to assess executive abilities in 27 participants with SCD receiving chronic transfusion in comparison to 34 participants with SCD receiving hydroxyurea (HU) and 41 non-SCD demographically matched controls, all of whom were tested at two time points. Participants in the transfusion group completed cognitive testing within 3 days after a transfusion (soon after transfusion) and then within 3 days before their next transfusion (long after transfusion) over an interval of 3-7 weeks. We found that executive abilities were significantly poorer for the transfusion and HU groups than for the control group. In support of our primary hypothesis, executive abilities for the transfusion group were significantly better soon after a transfusion compared to long after a transfusion, χ2 (1) = 17.8, P < .0001. Our results demonstrate that executive abilities were higher acutely following a blood transfusion. These findings have implications for daily functioning, medical decision making, and academic achievement in children and young adults with SCD. Abstract source

Background: Pain is a major complication of sickle cell disease (SCD), spanning vaso-occlusive crises and persistent pain. Although it is known that persistent pain is associated with considerable impairment in youth without SCD, little is known about the functional effects of persistent pain in SCD. The current study aimed to (a) characterize persistent pain in youth with SCD and (b) determine the extent to which youth with SCD and persistent pain differ in disease morbidity, functional impairment, and neurocognitive and psychological functioning. Procedure: Eighty-nine participants (ages 7-16) and caregivers completed questionnaires (BRIEF [Behavior Rating Inventory of Executive Function], Conners-3 [Conners-third edition], and PedsQL™-SCD Module, where PedsQL is Pediatric Quality of Life Inventory). Participants completed neurocognitive tests WISC-V [Wechsler Intelligence Scale for Children-fifth edition], WJ-III [Woodcock Johnson Tests of Achievement-third edition], and WIAT-III [Wechsler Individual Achievement Test-third edition]). Youth were classified as having persistent pain if they reported daily pain for 7 days. Chi-square and independent sample t-test analyses were used to assess group differences (those with vs without persistent pain). Results: Patients with persistent pain (n = 18) reported lower health-related quality of life (P = .000). Caregivers were more likely to rate youth with persistent pain as having lower planning/organization abilities (P = .011) and clinically elevated symptoms of defiance/aggression and oppositional defiance (Ps = .00; .01). Patients with persistent pain demonstrated poorer working memory (P = .023) and processing speed (P = .027), and fewer demonstrating reading fluency abilities in the average or above range (P = .026). Conclusions: Youth with SCD and persistent pain are at risk for psychosocial and neurocognitive impairments, suggesting that persistent pain may be an important indicator of disease burden. Furthermore, disease management may be enhanced by assessing cognitive and psychosocial functioning and incorporating interdisciplinary treatments addressing impairment associated with persistent pain. Abstract source