Latest news with #sicklecell
WebMD
01-08-2025
- Health
- WebMD
Post-Crisis Coping Strategies: Learning to Heal With Intention
After my most recent sickle cell crisis, I found myself grappling not only with physical recovery but also with the emotional and mental toll it left behind. In hindsight, I thought I fully recovered in a couple of weeks. Still, three months later, I only recognize that I have fully recovered. While finding my rhythm, I asked myself: What does healthy coping look like after a crisis? And, honestly, I didn't have the answer right away. In the early stages of recovery, my default coping mechanism was video games. Now, I enjoy playing video games without a crisis or recovery from a sickle cell crisis. However, during this period, I was playing these games excessively. They gave me an escape from the constant pain and fatigue. For a while, they helped. But soon, they became all I was doing – not just to manage pain but to avoid life. Even after the physical symptoms eased, I found myself still stuck in that virtual world, disconnected from everything else that made me who I am. And that's when I realized it wasn't just about how I was coping but about what I was neglecting. Getting back to a healthy routine was hard. The structure I'd once built of therapy, regular gym sessions, and social engagement had quietly slipped away. Without it, I didn't feel like myself. It took a lot of honest conversations and encouragement from my support system to help me start putting the pieces back together. I know I'm not alone in this. Many others struggle to reconnect with themselves post-crisis, unsure how to begin again. For me, the return to "normal" felt overwhelming. I was involved in my community, constantly giving of myself. So, when I stopped functioning at that level, it felt like I was letting people down. I retreated into gaming to silence those feelings and to silence myself. But silence isn't always healing. Sometimes it's avoidance. This reflection, in part, is a form of accountability. I need to remind myself of what truly helps me heal, not just physically but mentally and emotionally. That includes: One of the most complex parts was realizing I hadn't been honest, not even with myself. When people asked if I was OK, I defaulted to "I'm fine." But beneath the surface was a tangle of self-blame, doubt, and frustration that I hadn't fully processed. Things were happening emotionally that I didn't recognize until much later. Recovery from a crisis isn't just about the body. It's also about reclaiming the mind. And sometimes, when you're too deep in it to be aware, it takes the people around you to reflect on yourself: your strengths, your purpose, your joy. As much as I understand the fundamentals of healthily dealing with crises, I still have periods of succumbing to them. One important realization I had through therapy was that the problem wasn't gaming – it was the absence of boundaries. It wasn't the activity but how I let it consume the time and space meant for other parts of my healing. So now, I'm trying to be more intentional. Structure has always helped me, and I've started writing things down again – something I'd let slip. Setting time limits for rest, work, and even play helps me maintain balance. Slowly, I'm rebuilding routines, understanding that progress isn't about speed but direction. Some days, it's still hard. But I'm learning to give myself grace. Recovery doesn't follow a straight line, and coping isn't one-size-fits-all. What matters is that I keep learning, adapting, and growing. I do feel more hopeful about things and have improved my boundaries. Right now, I'm in a better place. I feel more grounded. I'm reconnecting with my mentoring, my faith, and the relationships that give me joy. While I'm still on this journey, I'm deeply grateful for the awareness, support, and tools that are helping me move forward.
The Independent
24-07-2025
- Health
- The Independent
How do I donate blood? NHS call for donors with rare blood types
An initiative to help individuals with rare blood types secure a "precision-matched" donation to avoid the risk of severe reactions has been launched by the NHS. Scientists have embarked on a large-scale research project, DNA -testing the blood of 77,000 donors to date. Blood collected from these rare donors will be frozen, creating a vital reserve for others with the same specific blood type. Crucially, this stored blood will also be available to the donor themselves, should they ever require a transfusion. The reserve of rare blood donors will help avoid severe transfusion reactions, where the body sees the transfused blood as 'foreign' and rejects it. People who are particularly at risk of these reactions are those who regularly receive blood donations, including those with sickle cell disorder and thalassaemia. It comes after the NHS raised the alarm over low blood stocks last month. The NHS said it needed 200,000 more regular blood donors to sustain a safe and sufficient supply. Low blood stocks in 2024 prompted an "amber alert', but blood stocks have remained low ever since. NHS Blood and Transplant (NHSBT) is now urging more people to donate to avoid a "red alert", which is a critical situation where public safety is threatened. It described the past year as "challenging" for blood stocks, noting that just under 800,000 people are supporting the entire blood supply in England. How do I donate blood? The whole process of giving blood takes just one hour. When arriving at a blood donation centre, you are asked to complete a safety check to make sure you can give blood. You will be given 500ml of fluid to drink – drinking this will help the body maintain blood pressure, prevent dizziness and help the body replenish the donated blood. Then, to ensure it is safe to donate blood, medics will confirm your identity and information during a health check. In some cases, a registered nurse will follow it up. A drop of blood from your finger is then checked for iron levels. If these levels are too low, the appointment will be rescheduled. Those able to donate will be seated in the waiting area before being called to a donation chair. Here, a cuff will be placed on your arm to maintain a small amount of pressure during the donation before a suitable vein is found and the area is cleaned. Then, a needle will be inserted into your arm to collect your blood into a blood bag, which has your unique donor number on it. A scale weighs the blood and stops when you have donated 470ml - that's just under a pint. The needle is then removed from your arm, and a dressing is applied. The NHS says the process of giving blood should not hurt and should only take about 10 minutes. Who can donate blood? Although more donors are needed, some health, travel and lifestyle reasons may stop you from donating blood. Donors need to be fit and well, aged between 17 and 65, weigh between 7 stone 12 Ibs (50kg) and 25 stone (158kg) and have suitable veins. However, you can't donate blood if you have cancer, some heart conditions, have tested positive for HIV or are a hepatitis B or C carrier. If you have received blood platelets, plasma or any other blood products after January 1980, or if you have injected non-prescribed drugs including body-building and injectable tanning agents, you also cannot donate. If you've had anal sex with a new partner in the last three months, you may have to postpone your donation. If you feel unwell, are pregnant or have had a baby in the last six months, have had a tattoo or piercing, or have recently travelled to certain countries outside the UK, you may also have to wait before you can donate blood. Where can I donate blood? There are thousands of blood donation venues across England, some are permanent and others are pop-ups. Church halls, sports centres, mosques, football stadiums and hotels are all used as pop-up venues. To check where your closest one is, visit the Give Blood website. You will need to book an appointment ahead of your donation, which can be done online as soon as you have signed up as a blood donor. What type of blood is needed? There is a 'critical' need for more donors who have the so-called universal blood type, O negative blood, which is needed for treatment in emergencies. The NHS said there is also a need for more Black donors, who are more likely to have specific blood types which can help treat people with sickle cell disease.
BBC News
13-07-2025
- Health
- BBC News
Ipswich teen's memory to live on through sickle cell foundation
The grieving sister of a 15-year-old girl who died with sickle cell disease says she wants to honour her memory by raising awareness of the blood from Ipswich, was born with the life-threatening condition which causes intense pain when blood vessels become blocked by misshapen red Chantry Academy pupil appeared to be rallying after a bone marrow transplant from sister Chelsea, but she deteriorated last month and died on 1 July. "We were really hopeful. We thought the transplant went really well, but it went downhill, which has been a huge shock," said Chelsea. "She fought it for a long time and was always able to get back up."It was always very difficult to watch, because as an older sister you want to protect your younger sibling, but I was always encouraged by her strength." About 15,000 people in England live with the inherited condition, which affects people mainly of Black African and Black Caribbean blood cells are produced in a sickle shape and are stiff and sticky rather than flexible smooth discs, and they do not live as long as healthy red blood cells can clump as they travel around blood vessels – reducing oxygen to vital parts of the body, putting people at risk of organ damage, stroke and heart failure, and leading to a greatly reduced quality of had received care at Ipswich Hospital, Addenbrooke's Hospital in Cambridge, and St Mary's Hospital in London, with the family encountering some clinicians who did not fully understand the is one of the reasons why Chelsea and her parents want to honour Vivica with a foundation called LLV (which stands for Long Live Vivica).The acronym was created by friends who were deeply affected by her death, with some of them scrawling her name or LLV on the back of their school shirts, Chelsea said."We have always discussed shining a light on sickle cell disease," she said."It's the biggest growing blood condition in the UK but I feel it's really under-represented."It was really traumatic experience for us, and we wouldn't want anyone to go through this."The foundation would support children and family members affected by the condition and raise its profile, including with policymakers and in schools. "I know it's something she would have wanted," Chelsea 25, said she and Vivica would spend "countless hours" together having fun, and would chat late into the evenings. "Having her as my sister was an absolute joy for me; we were really close," she added."She adored being around other people. She really came out when she was around her close friends, especially planning things, doing make-up – she was really girly." Follow Suffolk news on BBC Sounds, Facebook, Instagram and X.
WebMD
03-07-2025
- Health
- WebMD
Mastering the Art of Medication Management in Sickle Cell Care
In the beginning of my journey as a caregiver for my daughter, I struggled with one of the most important aspects of her care: managing her medications. It was overwhelming at first. One day, my mom noticed that I had forgotten to give my daughter her folic acid. She looked at me, disappointed, and asked why I hadn't given it to her. I admitted that I had forgotten. She gently reminded me how important it was and took the time to explain why folic acid is crucial for sickle cell patients. It was early in my caregiving journey, and there was so much to learn. I will always be grateful for that moment, as my mom's guidance became a cornerstone in my caregiving approach. That simple conversation with my mom opened my eyes to the importance of staying on top of medications. It also taught me that medication management isn't just about remembering to give pills; it's about understanding the reasons behind them and how they help keep my loved one healthy. Understanding Treatment Plans One of the first things that helped me with managing my daughter's medications was learning about the treatments she needed. There are many different medications used in the treatment of sickle cell disease, and understanding their roles can make the process much easier to navigate. Some of the most common medications prescribed for sickle cell patients include: Folic acid. As I learned, folic acid helps the body produce healthy red blood cells. This is especially important for people with sickle cell, as they need extra support to prevent anemia, which is a common complication of the disease. Vitamins B12 and D3. These vitamins support overall health and help manage some of the side effects of sickle cell disease. For example, vitamin B12 is important for nerve function, and vitamin D3 supports bone health. Pain medication. Pain is one of the most common and challenging symptoms of sickle cell disease. Pain crises – or episodes of severe pain – are frequent for many sickle cell patients. That's why pain management is a key part of treatment. Doctors often prescribe medications like acetaminophen, ibuprofen, or stronger opioids for more severe pain. Vitamin C. This vitamin is often recommended to support the immune system and to help the body absorb iron, which is another common issue for people with sickle cell. In my case, one of the most important lessons I learned was about pain medication. I remember a pediatric specialist sitting down with me and explaining how important it is to give pain meds regularly when my daughter is in pain. I had always thought that I should only give her medicine when she was in obvious pain, but the doctor explained that pain can build up slowly, and it's better to stay ahead of it. That advice was a game-changer for us. After that conversation, I began setting reminders to give her pain medications around the clock. It helped reduce her discomfort and improved her quality of life. Staying Organized Staying organized is a huge part of managing medications effectively. Early on, I found that keeping track of all the pills, doses, and schedules was a real challenge. The more medications we added, the harder it became to remember what was due and when. But over time, I figured out strategies to stay on top of it. Here are some tips that helped me: Create a medication chart. A simple chart with each medication's name, dosage, and time of day it needs to be taken can be a lifesaver. You can hang it on the refrigerator or keep it in a binder for easy reference. This way, you can track what's been taken and when the next dose is due. Set alarms or reminders. Use your phone to set reminders for each medication dose. I set alarms for morning, afternoon, and evening doses to ensure we didn't miss anything. Pill organizers. A pill organizer with separate sections for each day of the week can be helpful. It allows you to prep all the medications for the week in advance, so you don't have to worry about sorting them out every day. Apps to help track medications. Several medication tracking apps are available, which can send you reminders and let you know when to give the next dose. I've found these tools incredibly helpful. Communication With Health Care Providers Regular communication with health care providers is key to effective medication management. It's important to have check-ins to make sure the treatment plan is working and to adjust medications as needed. Sometimes, a medication might not be as effective as hoped, or side effects might arise. In those cases, talking to your doctor can lead to adjustments that will improve care. For example, when my daughter's pain wasn't being controlled effectively, we worked with her doctors to adjust her pain medication plan. They recommended adding more regular doses of pain relief to prevent flare-ups. By working together with the health care team, we found a solution that worked for her. I also learned that when you're managing a chronic condition like sickle cell, it's important to advocate for your loved one. Keep a detailed record of their symptoms, medication effectiveness, and any side effects they have. This helps your doctor make informed decisions and ensures that the treatment plan is always aligned with the patient's needs. Mastering medication management is a journey, but it is one that can make a world of difference for sickle cell patients and their caregivers. The key is to stay organized, communicate with health care providers, and understand why each medication is needed. By doing so, we can ensure that our loved ones get the best possible care and live the healthiest, most comfortable lives they can. As a caregiver, it's normal to feel overwhelmed at first. But with time, patience, and support, you can become confident in managing medications and providing the best care for your loved one. Remember, you're not alone in this journey. There are many resources, tools, and communities available to help. Be proactive and never hesitate to ask for help when you need it. Together, we can make a real difference in the lives of those living with sickle cell disease.
Associated Press
03-07-2025
- Business
- Associated Press
Vertex Presents Longer-Term Data at the 2025 European Hematology Association (EHA) Congress Demonstrating Durability of CASGEVY® and Provides Update on Expanding Global Access to CASGEVY
- Data from longer-term follow-up of patients in ongoing clinical trials further demonstrate durability of the clinical benefits of CASGEVY® - - Multiple reimbursement agreements secured, expanding access to CASGEVY to more patients around the world - TORONTO, July 3, 2025 /CNW/ - Vertex Pharmaceuticals (Nasdaq: VRTX) recently announced positive longer-term data for PrCASGEVY® (exagamglogene autotemcel) from global ongoing pivotal clinical trials in people with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, presented at the European Hematology Association (EHA) Congress, continue to demonstrate the durable clinical benefits of CASGEVY. The longest follow up in SCD patients now extends more than 5.5 years and in TDT patients more than 6 years, with a mean of 39.4 months and 43.5 months, respectively. CASGEVY is the first authorized CRISPR/Cas9 gene-edited therapy. 'This longer-term data reinforces CASGEVY's durable clinical benefits for eligible people living with sickle cell disease or transfusion-dependent beta thalassemia,' said Kevin Kuo, M.D., Hematologist and Associate Professor in the Division of Hematology, University of Toronto, Clinician Investigator in the Red Blood Cell Disorders Clinic at University Health Network, and Principal Investigator for the CLIMB-131 clinical program. 'These results are a reminder of what science can achieve, especially for patients and communities with significant unmet need.' New longer-term follow-up data presented from the CASGEVY trials Progress in bringing CASGEVY to patients Through reimbursement agreements, Vertex has secured access for eligible SCD or TDT patients in multiple countries including Austria, Bahrain, England, Denmark, the Kingdom of Saudi Arabia, Northern Ireland, Scotland, the United Arab Emirates, the United States and Wales. In Canada, CASGEVY received positive recommendations for reimbursement from both Canadian health technology agencies between December 2024 and January 2025; however, a Letter of Engagement from the pan-Canadian Pharmaceutical Alliance (pCPA) is pending. Vertex is continuing to work with government and reimbursement authorities globally to secure sustainable access for additional eligible patients. About Sickle Cell Disease (SCD) SCD is a debilitating, progressive, life-shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or 'sickled' red blood cells. The clinical hallmark of SCD is vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. About Transfusion-Dependent Beta Thalassemia (TDT) TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and significant health care resource utilization. TDT requires frequent blood transfusions and iron chelation therapy throughout a person's life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. About PrCASGEVY® (exagamglogene autotemcel) PrCASGEVY® is an autologous genome edited hematopoietic stem cell-based therapy for eligible patients with SCD or TDT, in which a patient's own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate vaso-occlusive crises (VOCs) for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. About the CLIMB Trials The ongoing Phase 1/2/3 open-label trials, CLIMB-111 and CLIMB-121, are designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12 to 35 years with TDT or with SCD and recurrent VOCs. The trials are closed for enrollment. Patients will be followed for approximately two years after CASGEVY infusion in these trials. Each patient will be asked to participate in the ongoing long-term, open-label trial, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients who received CASGEVY, including those in other CLIMB trials. The trial is designed to follow patients for up to 15 years after CASGEVY infusion. About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies in select regions for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1. Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit Vertex Special Note Regarding Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Kevin Kuo, M.D., in this press release, and statements regarding expectations for the anticipated durable clinical benefits of CASGEVY, expectations for the safety profile of CASGEVY, expectations for the Letter of Engagement from the pCPA, plans to continue working with government and reimbursement authorities globally to secure sustainable access for patients, and our plans for and design of the CLIMB studies. While we believe the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that eligible patient access to CASGEVY may not be achieved on the anticipated timeline, or at all, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy, and other reasons, and other risks listed under the heading 'Risk Factors' in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available through the company's website at You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available. Vertex Pharmaceuticals Incorporated Investors: [email protected] Media: Canada: +1 647-790-1600 or U.S.: 617-341-6992 SOURCE Vertex Pharmaceuticals (Canada) Inc.
