Materiały źródłowe

• #1 Sickle cell anemia – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/sickle-cell-anemia/diagnosis-treatment/drc-20355882

  Management of sickle cell anemia is usually aimed at avoiding pain episodes, relieving symptoms and preventing complications. Treatments might include medicines and blood transfusions. For some children and teenagers, a stem cell transplant might cure the disease. Gene therapies also are being developed that may offer cures for people with sickle cell disease. […] Hydroxyurea reduces the frequency of pain crises and might reduce the need for blood transfusions and hospital stays. […] L-glutamine oral powder helps in reducing the frequency of pain crises. […] Crizanlizumab can help reduce the frequency of pain crises in adults and in children older than 16 years. […] Voxelotor is used to treat sickle cell disease in adults and in children older than 12 years. […] Pain-relieving medicines might be prescribed to help relieve pain during sickle cell pain crises.

• #2 Get Sickle Cell Disease Treatment | Cleveland Clinic

  https://my.clevelandclinic.org/services/sickle-cell-disease-treatment

  Left untreated, sickle cell disease can damage vital organs. Thats why getting care early is so important. Even though SCD is chronic, with the right treatment, it can be effectively managed. Cleveland Clinics experts provide comprehensive and personalized treatment for SCD that relieves symptoms and helps prevent serious complications. […] Theres no single treatment for sickle cell disease. Its a chronic condition. And each person will experience it differently. Thats why we work with you or your child to make sure care matches your exact needs. Starting treatment right away can help lessen pain, manage symptoms and reduce the risk of complications. […] Most often, we use one or more medications to treat the symptoms of SCD. Not all of these drugs are approved for children. Your childs pediatric provider will recommend the best medications for your child. They may include: Disease-modifying agents like hydroxyurea, Oxbryta or Adakveo. Pain medications like nonsteroidal anti-inflammatory drugs (NSAIDs) (Motrin or Advil) or prescription pain relievers. Antibiotics like penicillin to prevent or treat infections. Vitamins and supplements like folic acid to help red blood cells form correctly.

• #3

  https://www.nhs.uk/conditions/sickle-cell-disease/

  People with sickle cell disease need treatment throughout their lives. This is usually delivered by different health professionals in a specialist sickle cell centre. […] A number of treatments for sickle cell disease are available. […] For example: drinking plenty of fluids and staying warm to prevent painful episodes […] painkillers, such as paracetamol or ibuprofen (sometimes treatment with stronger painkillers in hospital may be necessary) […] daily antibiotics and having regular vaccinations to reduce your chances of getting an infection […] a medicine called hydroxycarbamide (hydroxyurea) to reduce symptoms […] regular blood transfusions if symptoms continue or get worse, or there are signs of damage caused by sickle cell disease […] an emergency blood transfusion if severe anaemia develops. […] The only cure for sickle cell disease is a stem cell or bone marrow transplant, but they’re not done very often because of the risks involved.

• #4 Sickle Cell Disease (SCD) Treatment & Management: Approach Considerations, Hydroxyurea Therapy, Transfusion

  https://emedicine.medscape.com/article/205926-treatment

  The National Institutes of Health advises that optimal care for patients with sickle cell disease (SCD), including preventive care, is best achieved through treatment in clinics that specialize in the care of SCD. All patients with SCD should have a principal health care provider, who should either be a hematologist or be in frequent consultation with one. […] An expert panel has released evidence-based guidelines for the treatment of SCD, including a strong recommendation that hydroxyurea and long-term, periodic blood transfusions should be used more often to treat patients. […] In 2017, the US Food Drug Administration (FDA) approved L-glutamine oral powder (Endari) for patients age 5 years and older to reduce severe complications of SCD. […] Crizanlizumab, a P-selectin inhibitor, was approved by the FDA in 2019 to reduce the frequency of vaso-occlusive crisis (VOC) in adults with SCD.

• #5 Sickle Cell Disease – Treatment | NHLBI, NIH

  https://www.nhlbi.nih.gov/health/sickle-cell-disease/treatment

  The U.S. Food and Drug Administration (FDA) has approved four medicines to treat sickle cell disease. In 1998, hydroxyurea was approved. Later, L-glutamine and crizanlizumabtmca were approved. They treat different aspects of the disease. […] Hydroxyurea is an oral medicine that can reduce sickling of red blood cells and help prevent serious symptoms of sickle cell disease, including pain crises. […] Hydroxyurea helps reduce serious symptoms such as pain crises and acute chest syndrome. It can also improve anemia and reduce the need for blood transfusions and hospitalizations. […] Hydroxyurea can help prevent pain crises and hospitalizations for children with sickle cell disease. […] L-glutamine is approved for people ages 5 years and older. Taking L-glutamine may lead to fewer hospital admissions, fewer pain crises, less need for blood transfusions, and a lower risk of acute chest syndrome.

• #6 Sickle Cell Disease (SCD) Treatment & Management: Approach Considerations, Hydroxyurea Therapy, Transfusion

  https://emedicine.medscape.com/article/205926-treatment

  Allogeneic hematopoietic stem cell transplantation (HSCT) can cure SCD, but it has many risks, so the risk-to-benefit ratio must be assessed carefully. […] In 2023, the FDA approved the first 2 gene-editing therapies for severe SCD in patients aged 12 years and older. […] Hydroxyurea (hydroxycarbamide) has an established role as a safe and effective treatment for SCD. […] Hydroxyurea increases total and fetal hemoglobin in children with SCD. […] Hydroxyurea was first approved for use in adults with SCD, in 1998. […] In 2017, the FDA approved Siklos (hydroxyurea) to reduce the frequency of painful crises and the need for blood transfusions in children 2 years of age and older and adolescents with SCD who have recurrent moderate to severe painful crises. […] Blood transfusions are not needed for the usual anemia or episodes of pain associated with SCD.

• #7 New Therapeutic Options for the Treatment of Sickle Cell Disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6328043/

  Hydroxyurea or hydroxycarbamide (HU) is the key therapeutic tool for SCD approved by Food and Drug Administration (FDA) and European Medical Agency (EMEA). US and European guidelines highlighted that HU should be available for all SCD patients from pediatric to adult populations. […] Studies in SCD show a multimodal action of HU, which (i) increases HbF production, resulting in delayed HbS polymerization; (ii) reduces hemolysis and increase NO availability targeting cGMP production; (iii) modulates endothelial activation and reduces neutrophil counts, contributing to the reduction of chronic inflammation. […] Long-term use of HU has been shown to be safe and well-tolerated in large cohorts of children and adults with SCD, reducing mortality and morbidity of both children and adult patients.

• #8 Treatment of Sickle Cell Disease: There’s More Than Just Pain Medications | Texas Children’s

  https://www.texaschildrens.org/content/wellness/treatment-sickle-cell-disease-theres-more-than-just-pain-medications

  Sickle cell disease (SCD) affects nearly 100,000 Americans currently. This chronic debilitating disease of the red blood cells, can cause a multitude of complications – pain, stroke, kidney disease, etc. For many years the only thing available to patients was the treatment of pain episodes with medications. Unfortunately, this only treats a symptom and not the disease itself. However, now we have hydroxyurea as a treatment option. […] Hydroxyurea (sometimes referred to as HU) is a medication taken by mouth that can help children and adults with sickle cell disease. Currently, it is the only U.S. Food and Drug Administration (FDA) approved medication to treat SCD in adults. Multiple studies have shown that it is safe and effective in children as well. Hydroxyurea helps red blood cells stay round and flexible instead of the sickle or banana shape seen in SCD. These round red blood cells are able to flow more easily through the blood vessels, reducing SCD complications. Specifically, hydroxyurea has been shown to reduce the number of pain crises, reduce the number of acute chest syndrome (lung complication) events, reduce the need for blood transfusions and reduce number of hospitalizations. Additionally, most patients become less anemic while taking hydroxyurea.

• #9 Sickle Cell Disease Treatments: Blood Transfusions and Stem Cell Transplant

  https://www.webmd.com/a-to-z-guides/sickle-cell-disease-treatment

  If you have sickle cell disease (SCD), your red blood cells arent round and bendable or pliable like they should be. Instead, theyre stiff and sticky. […] The medicine hydroxyurea (Droxia, Hydrea, Sikos) prevents abnormal red blood cells from forming. This cuts down on episodes of sickle cell crises. Hydroxyurea is a mainstay in the overall management of individuals with SCD but it can cause serious side effects, like a plunge in the number of white blood cells that help your body fight infections. […] Your doctor may prescribe a drug called L-glutamine oral powder (Endari), which can be used by children as young as 5 years old. It can cut down on your trips to the hospital for pain and also guard against a dangerous condition called acute chest syndrome. […] The most recent medication approved by the FDA is a monoclonal antibody called crizanlizumab-tmca (Adakveo). It’s the first targeted therapy for SCD. It prevents red blood cell clumping and lowers vaso-occlusive crises (VOC).

• #10 Management of Sickle Cell Disease: Recommendations from the 2014 Expert Panel Report | AAFP

  https://www.aafp.org/pubs/afp/issues/2015/1215/p1069.html

  Family physicians are the primary and sometimes only health care resource for families affected by sickle cell disease. Recently published guidelines provide important recommendations for health maintenance, acute care, and monitoring of disease-modifying therapy in persons with this condition. […] There is strong evidence to support the promotion and use of hydroxyurea therapy in patients nine months and older who have sickle cell anemia because its use can decrease the frequency of vasoocclusive crises and acute chest syndrome with limited adverse effects. […] Offer hydroxyurea therapy to infants, children, and adolescents with sickle cell anemia regardless of clinical severity to reduce sickle cell disease-related complications. […] Initiate hydroxyurea therapy in adults with sickle cell anemia who have one or more of the following: three or more moderate to severe pain crises in a 12-month period; daily sickle cell pain that affects quality of life; severe or recurrent acute chest syndrome; or severe symptomatic chronic anemia.

• #11 Treatments To Manage—And Even Cure—Sickle Cell Disease | Henry Ford Health – Detroit, MI

  https://www.henryford.com/blog/2023/04/treatments-to-manage-and-cure-sickle-cell-disease

  L-glutamine is a powder thats taken daily. It has been shown to reduce pain and acute chest syndrome, which occurs when someone develops sickling in the lungs, causing symptoms of pneumonia. […] Voxelotor is a pill thats taken daily. It prevents red blood cells from sickling and improves hemoglobin (the protein in red blood cells that transports oxygen throughout the body). […] Crizanlizumab is an IV medication thats given monthly. It prevents red blood cells from sticking to blood vessels, reducing pain crises. […] Two treatments have been shown to cure sickle cell disease: stem cell transplant and gene therapy, the latter of which is still in clinical trials: An allogeneic stem cell transplant replaces sickle-forming stem cells with a donors healthy stem cells. […] After a successful allogeneic stem cell transplant, your body will no longer produce sickled red blood cells.

• #12 Sickle Cell Disease: Current Treatment and Emerging Therapies

  https://www.ajmc.com/view/sickle-cell-disease-current-treatment-and-emerging-therapies

  Hydroxyurea was the only approved treatment for SCD for nearly 2 decades; in 2017, L-glutamine oral powder was approved for the prevention of the acute complications of SCD. […] Few options are currently available for the management of SCD. Hydroxyurea, which until recently was the only FDA-approved drug for adults with severe SCD genotypes (and is also used off-label for adults with less severe genotypes and children ages 9 months to 2 years), improves the course of SCD and results in substantial cost savings. […] Recently, L-glutamine became the second drug approved for SCD in the United States. […] Long-term RBC transfusion therapy is commonly given to patients with SCD for the management of both acute and chronic complications. […] Nearly 2 decades after the approval of hydroxyurea, L-glutamine was approved by the FDA in 2017 to reduce acute complications of homozygous SCD in adult and pediatric patients aged 5 years.

• #13 Newer Modalities and Updates in the Management of Sickle Cell Disease: | JBM

  https://www.dovepress.com/newer-modalities-and-updates-in-the-management-of-sickle-cell-disease–peer-reviewed-fulltext-article-JBM

  L-glutamine aims to lower the burdens associated with this disease by acting through various pathways. L-glutamine operates by replenishing cellular antioxidants. Thus by enhancing the antioxidant defenses within cells, it helps to overcome the damaging effects of reactive oxygen species. L-glutamine also enhances the production of nitric oxide, a pivotal molecule involved in vasodilation. Treatment with L-glutamine has shown positive outcomes in the control of SCD. According to studies, giving L-glutamine therapy results in a lesser number of hospital admissions, fewer red blood cell transfusions, a lesser number of pain crises, and longer intervals between first and second crises. […] The mechanism of action of Voxelotor involves blocking the polymerization of hemoglobin. This is achieved by binding to sickle hemoglobin (HbS) and stabilizing it in the oxygenated state leading to an overall decrease in the formation of HbS polymer, thereby reducing sickling of red blood cells. Furthermore, the reduced viscosity and lesser deformability of the red blood cells improves blood flow and it results in fewer incidences of vaso-occlusive crises.

• #14 Complications & Treatments – Sickle Cell Disease Association of America Inc.

  https://www.sicklecelldisease.org/treatments/

  L-glutamine, an antioxidant used in glutathione production, was approved for patients five years old and above by the FDA in 2017. […] An infusional medication FDA-approved for patients 16 and above in 2019. […] A bone marrow transplant is one of the few methods that can cure sickle cell disease; however, for most members of the SCD community, it is not a viable option. […] Two gene therapies were approved in Dec. 2023 by the Food and Drug Administration (FDA) to treat sickle cell disease: exagamglogene autotemcel and lovotibeglogene autotemcel. […] Blood transfusions help benefit sickle cell disease patients by reducing recurrent pain crises, risk of stroke and other complications. […] Patients with sickle-cell disease (SCD) receiving chronic transfusions of red blood cells are at risk of developing transfusional iron overload over time. […] Preventative care and screening can help you stay healthy and keep you out of the hospital. […] Individuals with SCD who wish to have children may need extra support as they consider starting a family and/or navigate a pregnancy. […] Living with a chronic disease can be stressful.

• #15 Sickle Cell Disease – Treatment | NHLBI, NIH

  https://www.nhlbi.nih.gov/health/sickle-cell-disease/treatment

  Crizanlizumab-tmca is approved for adults and children ages 16 years and older who have sickle cell disease. The medicine helps prevent blood cells from sticking to blood vessel walls and blocking blood flow. […] Daily treatment with an antibiotic called penicillin helps lower children’s chance of having a serious infection in the bloodstream. […] Your healthcare provider may recommend a blood transfusion to treat and prevent certain sickle cell disease complications. […] A blood and bone marrow transplant is a potential curative therapy for some people with sickle cell disease. […] Gene therapies aim to treat conditions by adding new DNA or changing existing DNA. In December 2023, the Food and Drug Administration approved two gene therapies to treat sickle cell disease: Exagamglogene autotemcel makes changes to a gene that is already in the body and Lovotibeglogene autotemcel adds a modified gene to the body. […] Blood and bone marrow transplants and gene therapies require weeks in a hospital, specialized care, and costly medicines.

• #16 Sickle Cell Anemia: Symptoms, Causes & Treatment

  https://my.clevelandclinic.org/health/diseases/4579-sickle-cell-anemia

  Treatment for sickle cell anemia depends on your symptoms and your overall health. For example, if you have severe complications like acute chest syndrome, frequent acute pain crises or stroke, your provider may recommend an allogeneic stem cell transplant. This procedure is the only cure for sickle cell anemia. […] Other sickle cell anemia treatments are blood transfusions, antibiotics to treat infections and medications that ease specific symptoms. Those medications include: […] Hydroxyurea is an anticancer drug. Its treatment for infants aged 6 to 9 months, children and adults. It may reduce how often you have serious complications and ease anemia symptoms. […] Voxelotor keeps red blood cells from becoming sickled cells. Its treatment for children age 4 and older. […] L-glutamine therapy (Endari): This medication, which is a treatment for children age 5 and older and adults, keeps sickled cells from becoming even more abnormal. […] Crizanlizumab-tmca (Adakveo): This reduces how often you may have VOC/acute pain crisis. Its treatment for people age 16 and older.

• #17 Newer Modalities and Updates in the Management of Sickle Cell Disease: | JBM

  https://www.dovepress.com/newer-modalities-and-updates-in-the-management-of-sickle-cell-disease–peer-reviewed-fulltext-article-JBM

  Crizanlizumab works as a P-selectin inhibitor, targeting important mechanisms of vaso-occlusive events. P-selectin is an adhesion molecule found on endothelial cells and platelets, it is responsible for the adhesion of red blood cells to the vascular endothelium. Crizanlizumab binds to P-selectin and covers its active site thereby preventing the interaction between RBCs, platelets, and endothelial cells. This effectively lowers the frequency and severity of vaso-occlusive crises. […] Even with the introduction of disease-modifying medications such as L-glutamine oral powder, Voxelotor, and Crizanlizumab and the use of stem cell transplantation in certain situations, issues like restricted availability, related hazards, and insufficient effectiveness highlighted the necessity for more thorough and easily accessible therapeutic methods.

• #18 Sickle Cell Disease Treatment

  https://www.froedtert.com/sickle-cell-disease/treatments

  Other medications include: Endari, which is a naturally occurring amino acid called L-glutamine. Taken orally, it helps prevent damage to red blood cells and reduce complications of sickle cell disease. […] Crizanlizumab-tmca (Adakveo), which is a targeted biologic medication that works by attaching to P-selectin, a protein related to stickiness in your blood. The drug works by making certain blood cells and blood vessels less sticky. It improves blood flow and reduces the number of pain crises you have. […] Voxtlotor (Oxbryta) is a drug that can reduce sickling and breakdown of red blood cells, allowing more healthy red blood cells to carry oxygen throughout the body. This reduces anemia, pain crises and other damage caused by sickle cells in your blood. […] Sickle cell disease can affect many organ systems. Therefore, it can cause a variety of symptoms and complications. Many times, a patient will need different types of therapies to treat these symptoms and complications.

• #19 Sickle Cell Disease (SCD) Treatment & Management: Approach Considerations, Hydroxyurea Therapy, Transfusion

  https://emedicine.medscape.com/article/205926-treatment

  Regular blood transfusions are used for primary and secondary stroke prevention in children with SCD. […] Erythrocytapheresis is an automated red cell exchange procedure that removes blood that contains HbS from the patient while simultaneously replacing that same volume with packed red cells free of HbS. […] Hydroxyurea may decrease the frequency and severity of pain episodes. […] Voxelotor, a hemoglobin S (HbS) polymerization inhibitor that increases the affinity of Hb for oxygen, was approved by the FDA in 2019 for treatment of SCD. However, in September 2024 the manufacturer voluntarily withdrew voxelotor from all markets, citing an imbalance in vaso-occlusive crises and fatal events that outweighs the benefits of the drug for SCD.

• #20 Treatments To Manage—And Even Cure—Sickle Cell Disease | Henry Ford Health – Detroit, MI

  https://www.henryford.com/blog/2023/04/treatments-to-manage-and-cure-sickle-cell-disease

  L-glutamine is a powder thats taken daily. It has been shown to reduce pain and acute chest syndrome, which occurs when someone develops sickling in the lungs, causing symptoms of pneumonia. […] Voxelotor is a pill thats taken daily. It prevents red blood cells from sickling and improves hemoglobin (the protein in red blood cells that transports oxygen throughout the body). […] Crizanlizumab is an IV medication thats given monthly. It prevents red blood cells from sticking to blood vessels, reducing pain crises. […] Two treatments have been shown to cure sickle cell disease: stem cell transplant and gene therapy, the latter of which is still in clinical trials: An allogeneic stem cell transplant replaces sickle-forming stem cells with a donors healthy stem cells. […] After a successful allogeneic stem cell transplant, your body will no longer produce sickled red blood cells.

• #21 NHS England » NHS rolls out ‘life-changing’ treatment for thousands with sickle cell disease

  https://www.england.nhs.uk/2024/05/nhs-rolls-out-life-changing-treatment-for-thousands-with-sickle-cell-disease/

  Thousands of patients with sickle cell disorder are to be offered a new life-changing treatment on the NHS from today (3 May), following the approval and roll-out of a new drug that could significantly improve their quality of life. […] Up to 4,000 people with sickle cell disease in England could benefit from the drug Voxelotor if recommended by their clinician, which will offer an additional treatment option from today, following its approval by the National Institute for Health and Care Excellence (NICE). […] Voxelotor (Oxbryta) will be administered in the form of a tablet taken once a day for eligible patients and could help reduce the need for blood transfusions and lead to fewer hospital appointments. […] Voxelotor will be used to treat sickle cell anaemia in people aged 12 and older after it was recommended for use on the NHS in England by NICE.

• #22 Sickle Cell Disease (SCD) Treatment & Management: Approach Considerations, Hydroxyurea Therapy, Transfusion

  https://emedicine.medscape.com/article/205926-treatment

  Allogeneic hematopoietic stem cell transplantation (HSCT) can cure SCD, but it has many risks, so the risk-to-benefit ratio must be assessed carefully. […] In 2023, the FDA approved the first 2 gene-editing therapies for severe SCD in patients aged 12 years and older. […] Hydroxyurea (hydroxycarbamide) has an established role as a safe and effective treatment for SCD. […] Hydroxyurea increases total and fetal hemoglobin in children with SCD. […] Hydroxyurea was first approved for use in adults with SCD, in 1998. […] In 2017, the FDA approved Siklos (hydroxyurea) to reduce the frequency of painful crises and the need for blood transfusions in children 2 years of age and older and adolescents with SCD who have recurrent moderate to severe painful crises. […] Blood transfusions are not needed for the usual anemia or episodes of pain associated with SCD.

• #23 Sickle Cell Disease – Treatment | NHLBI, NIH

  https://www.nhlbi.nih.gov/health/sickle-cell-disease/treatment

  Crizanlizumab-tmca is approved for adults and children ages 16 years and older who have sickle cell disease. The medicine helps prevent blood cells from sticking to blood vessel walls and blocking blood flow. […] Daily treatment with an antibiotic called penicillin helps lower children’s chance of having a serious infection in the bloodstream. […] Your healthcare provider may recommend a blood transfusion to treat and prevent certain sickle cell disease complications. […] A blood and bone marrow transplant is a potential curative therapy for some people with sickle cell disease. […] Gene therapies aim to treat conditions by adding new DNA or changing existing DNA. In December 2023, the Food and Drug Administration approved two gene therapies to treat sickle cell disease: Exagamglogene autotemcel makes changes to a gene that is already in the body and Lovotibeglogene autotemcel adds a modified gene to the body. […] Blood and bone marrow transplants and gene therapies require weeks in a hospital, specialized care, and costly medicines.

• #24 Sickle cell anemia – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/sickle-cell-anemia/diagnosis-treatment/drc-20355882

  Blood transfusions are used to treat and prevent complications, such as stroke, in people with sickle cell disease. […] A stem cell transplant can cure sickle cell anemia. […] Stem cell gene addition therapy may be a cure for people with sickle cell disease who do not have a well-matched donor. […] Gene editing therapy works by making changes to the DNA in a person’s stem cells.

• #25 Blood Treatment for Sickle Cell Disease | NYU Langone Health

  https://nyulangone.org/conditions/sickle-cell-disease-in-children/treatments/blood-treatment-for-sickle-cell-disease

  At Hassenfeld Childrens Hospital at NYU Langone, doctors may recommend blood transfusions to prevent or treat certain complications in children with sickle cell disease. Blood transfusions are given as an intravenous (IV) infusion through a catheter thats inserted into a vein in your childs arm or leg. Our experts can identify children who are likely to benefit from this treatment. […] For a small number of children with sickle cell disease, our doctors may recommend a stem cell transplant. This treatment, also given as an IV infusion, is a one-time procedure that can cure sickle cell disease. […] Doctors may use blood transfusions to boost the level of normal red blood cells in children with sickle cell disease when they have episodes of more severe anemia. […] Because transfusions increase the number of normal red blood cells, they can help to alleviate complications, such as acute chest syndromea reduction in oxygen that can cause coughing, chest pain, and shortness of breath. When used as a long-term therapy, blood transfusions can also prevent the occurrence of a stroke by reducing the number of abnormal red blood cells, which can narrow blood vessels and cause a stroke.

• #26 Sickle cell disease – Wikipedia

  https://en.wikipedia.org/wiki/Sickle_cell_disease

  An exchange transfusion is a procedure in which blood is removed from the body, then processed to extract sickled cells, which are replaced by healthy red blood cells from a donor. […] In children, preventive RBC transfusion therapy has been shown to reduce the risk of first stroke or silent stroke when transcranial Doppler ultrasonography shows abnormal cerebral blood flow. […] Most people with sickle cell disease have intensely painful episodes called vaso-occlusive crises (VOC). […] Acute chest syndrome is caused by vaso-occlusion occurring in the lungs. […] When treating avascular necrosis of the bone in people with sickle cell disease, the aim of treatment is to reduce or stop the pain and maintain joint mobility. […] Psychological therapies such as patient education, cognitive therapy, behavioural therapy, and psychodynamic psychotherapy, that aim to complement current medical treatments, require further research to determine their effectiveness.

• #27

  https://www.nhs.uk/conditions/sickle-cell-disease/treatment/

  Sickle cell disease usually requires lifelong treatment. […] If you continue to have episodes of pain, a medicine called hydroxycarbamide (hydroxyurea) may be recommended. You usually take it as a capsule once a day. […] If anaemia is particularly severe or persistent, treatment with blood transfusions or hydroxycarbamide may be necessary. […] Stem cell or bone marrow transplants are the only cure for sickle cell disease, but they’re not done very often because of the significant risks involved. […] A stem cell transplant is an intensive treatment that carries a number of risks. […] People who need a lot of blood transfusions may also need to take medicine called chelation therapy. This reduces the amount of iron in their blood to safe levels.

• #28 Sickle Cell Disease – Treatment | NHLBI, NIH

  https://www.nhlbi.nih.gov/health/sickle-cell-disease/treatment

  Crizanlizumab-tmca is approved for adults and children ages 16 years and older who have sickle cell disease. The medicine helps prevent blood cells from sticking to blood vessel walls and blocking blood flow. […] Daily treatment with an antibiotic called penicillin helps lower children’s chance of having a serious infection in the bloodstream. […] Your healthcare provider may recommend a blood transfusion to treat and prevent certain sickle cell disease complications. […] A blood and bone marrow transplant is a potential curative therapy for some people with sickle cell disease. […] Gene therapies aim to treat conditions by adding new DNA or changing existing DNA. In December 2023, the Food and Drug Administration approved two gene therapies to treat sickle cell disease: Exagamglogene autotemcel makes changes to a gene that is already in the body and Lovotibeglogene autotemcel adds a modified gene to the body. […] Blood and bone marrow transplants and gene therapies require weeks in a hospital, specialized care, and costly medicines.

• #29 Sickle cell anemia – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/sickle-cell-anemia/diagnosis-treatment/drc-20355882

  Blood transfusions are used to treat and prevent complications, such as stroke, in people with sickle cell disease. […] A stem cell transplant can cure sickle cell anemia. […] Stem cell gene addition therapy may be a cure for people with sickle cell disease who do not have a well-matched donor. […] Gene editing therapy works by making changes to the DNA in a person’s stem cells.

• #30 Sickle Cell Disease | Boston Children’s Hospital

  https://www.childrenshospital.org/conditions/sickle-cell-disease

  Hydroxyurea is an oral medication that causes the body to produce fetal hemoglobin (HbF), a type of hemoglobin normally found only in fetuses and very young children. Increasing the healthy fetal hemoglobin can significantly reduce the side effects and complications of sickle cell disease. […] Blood transfusions may be given acutely in order to treat severe anemia, acute chest syndrome, and other life-threatening complications of sickle cell disease. […] Right now, the only cure for sickle cell disease is stem cell transplant (also called bone marrow transplant) the transplantation of normal blood stem cells from another person (the donor) to your child. […] Through our Sickle Cell Disease Program, children with sickle cell disease have access to world-renown pediatric hematologists, top-rated nursing care, and the latest treatment options including hydroxyurea and stem cell transplantation.

• #31 Advances in the diagnosis and treatment of sickle cell disease | Journal of Hematology & Oncology | Full Text

  https://jhoonline.biomedcentral.com/articles/10.1186/s13045-022-01237-z

  The landscape of disease-modifying therapies, however, has improved with the recent FDA approval of 3 other treatments l-glutamine and crizanlizumab for reducing acute complications (e.g., pain), and voxelotor for improving anemia. […] For individuals with SCD undergoing hematopoietic stem cell transplantation (HSCT) using HLA-matched sibling donors and either myeloablative or reduced-intensity conditioning regimens, the five-year event-free and overall survival is high at 91% and 93%, respectively. […] Hydroxyurea may be associated with improvements in glomerular hyperfiltration and urine concentrating ability in children with SCD. […] ACE-I or ARB therapy reduces microalbuminuria in patients with SCD. […] Renal transplant should be considered for individuals with SCD and ESRD because of recent improvements in renal graft survival and post-transplant mortality.

• #32 Cure for Sickle Cell Disease in Chicago | UI Health

  https://hospital.uillinois.edu/primary-and-specialty-care/sickle-cell/a-cure-for-sickle-cell-disease

  UI Health now provides hope for a cure to patients with sickle cell disease through stem cell transplantation and without the need for chemotherapy. […] The procedure developed at the National Institutes of Health in Bethesda, Maryland, and validated at UI Health relies on transplanted cells from a healthy, tissue-matched, full sibling. Patients receive immunosuppressive drugs just before the transplant, along with a very low dose of total body irradiation, a treatment much less harsh and with fewer potentially serious side effects than chemotherapy. […] In 2011, Chicagoan Iesha Thomas became the first patient in the Midwest to successfully receive a stem cell transplant to cure sickle cell. […] The following year, brothers Julius and Desmond Means were cured of sickle at UI Health following a stem cell transplantation from their older brother, Clifford.

• #33 Sickle cell disease – Wikipedia

  https://en.wikipedia.org/wiki/Sickle_cell_disease

  Hematopoietic stem cells (HSC) are cells in the bone marrow that can develop into all types of blood cells, including red blood cells, white blood cells, and platelets. […] Since 1991, a small number of patients have received bone marrow transplants from healthy matched donors, although this procedure has a high level of risk. […] More recently, it has become possible to use CRISPR gene editing technology to modify the patient’s own HSCs in a way that reduces or eliminates the production of sickle hemoglobin HbS and replaces it with a non-sickling form of hemoglobin. […] In 2023, both exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) were approved for the treatment of sickle cell disease.

• #34 Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease > News > Yale Medicine

  https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease

  Essentially, Casgevy works by reducing the number of sickle cells in the body to a negligible amount, which may permanently ease symptoms. […] Both are trying to dilute the sickle hemoglobin that the body would otherwise continue to make. […] For patients, the process to receive either of these two gene therapies is similar, in that they both require multiple hospital visits and chemotherapy, and they are delivered over a period that ranges from eight to 12 months from start to finish. […] While bone marrow transplant remains an option, Dr. Krishnamurti says the gene therapies are less burdensome. […] Casgevy was studied in a multicenter trial involving adult and adolescent patients with a history of sickle cell crises during each of the past two years. […] Trials are ongoing. […] Lyfgenia was studied in a two-year multicenter trial of adults and adolescents with a history of sickle cell crises.

• #35 VUMC part of new study validating curative therapy for sickle cell disease – VUMC News

  https://news.vumc.org/2025/02/27/vumc-part-of-new-study-validating-curative-therapy-for-sickle-cell-disease/

  Of 38 adults with severe SCD who participated in the study, more than 97% no longer required immunosuppressive therapy one year after the transplant. […] A modified bone marrow transplant procedure for sickle cell disease (SCD) co-developed at Vanderbilt University Medical Center, Johns Hopkins University School of Medicine and the University of California, San Francisco, can cure the disease, a new study has found. […] Current curative treatment options for SCD include allogeneic hematopoietic stem cell transplants (when a patient receives health stem cells most often from a related donor) and autologous genome-modification therapies (when a patients own cells are modified). […] The haploidentical transplant protocol developed at Johns Hopkins requires only a half HLA match from a related family member.

• #36 VUMC part of new study validating curative therapy for sickle cell disease – VUMC News

  https://news.vumc.org/2025/02/27/vumc-part-of-new-study-validating-curative-therapy-for-sickle-cell-disease/

  Both trials validate a new standard of care for curative therapy for adults with SCD, said Kassim and DeBaun. Unlike gene therapy, these results will not require FDA approval. […] The therapy, haploidentical bone marrow transplant with thiotepa and posttransplant cyclophosphamide, is as safe and more affordable than the recently FDA-approved myeloablative gene therapy and gene editing treatments. […] The Vanderbilt Stem Cell Transplant and Cellular Therapy Symposium offers clinicians detailed information on the latest developments for hematological diseases, ranging from immunotherapies for blood cancers to new, curative therapies for sickle cell disease.

• #37 FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease | FDA

  https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease

  Today, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older. […] Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited. […] Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. […] Lyfgenia is a cell-based gene therapy. Lyfgenia uses a lentiviral vector (gene delivery vehicle) for genetic modification and is approved for the treatment of patients 12 years of age and older with sickle cell disease and a history of vaso-occlusive events.

• #38 Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease > News > Yale Medicine

  https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease

  Until recently, the only possible cure for sickle cell disease, an inherited genetic blood disorder most common in people with African ancestry, was a bone marrow transplant, which has its own set of challenges. […] Now, people with sickle cell disease (SCD) which affects an estimated 100,000 Americans and can cause chronic pain, organ damage, strokes, and shortened life expectancy have additional, potentially curative options. In early December, the Food and Drug Administration (FDA) approved two gene therapies for SCD, one of which is the first approved medication that uses the gene-editing tool CRISPR. […] Both treatments Casgevy, which is made by Vertex Pharmaceuticals and CRISPR Therapeutics, and Lyfgenia, by Bluebird Bio are for people 12 and older. […] With Casgevy, an edit (or cut) is made in a particular gene to reactivate the production of fetal hemoglobin, which dilutes the faulty red blood cells caused by sickle cell disease. Lyfgenia, on the other hand, uses a viral envelope to deliver a healthy hemoglobin-producing gene.

• #39 Patient Information | CASGEVY® (exagamglogene autotemcel)

  https://www.casgevy.com/sickle-cell-disease

  CASGEVY is a one-time gene therapy that may help people 12 years and older with sickle cell disease (SCD) and frequent vaso-occlusive crises (VOCs) live severe VOC-free. […] CASGEVY uses your own blood stem cells to help your body make working red blood cells, which can eliminate VOCs. […] In the clinical study, 93.5% (29 out of 31 people) did not have a severe VOC for at least 12 months in a row after receiving CASGEVY. […] CASGEVY is a one-time therapy used to treat people aged 12 years and older with sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs. […] CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with SCD.

• #40 FDA Approves Two Gene Therapies for Sickle Cell Disease | Children’s Hospital of Philadelphia

  https://www.chop.edu/news/fda-approves-two-gene-therapies-sickle-cell-disease

  In the case of sickle cell disease, the newly approved therapy edits DNA within the patients own cells and enables the patient to produce a different form of hemoglobin in their red blood cells. […] In the case of LYFGENIA, the gene therapy is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene that enables production of adult hemoglobin that does not form into the crescent shape associated with the disease. […] In clinical trials with CASGEVY, we have seen elimination of painful vaso-occlusive crisis in more than 90% of patients and a complete elimination of VOC-related hospitalizations in all patients, with a safety profile generally consistent with autologous stem cell transplantation, said Stephan A. Grupp, MD, PhD, Section Chief of the Cellular Therapy and Transplant Section, Inaugural Director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy, and Medical Director of the Cell and Gene Therapy Laboratory at CHOP.

• #41 Sickle Cell Disease

  https://patienteducation.asgct.org/disease-treatments/sickle-cell-disease

  Chemotherapy is administered before gene therapy, as it eliminates existing stem cells that are still carrying the faulty gene. […] In one approach, referred to as gene addition, the HSCs can be instructed to produce more healthy adult hemoglobin by delivering (or adding) a working HBB gene. […] In another approach, referred to as gene silencing, a gene would be delivered that silences the BCL11A gene (or prevents it from working). […] Gene editing is a type of gene therapy that removes, disrupts, or corrects faulty elements of DNA within a gene by using an enzyme that cuts the DNA at one specific location. […] The edited cells are then infused back into the patient as part of a stem cell transplant, helping them produce normal adult hemoglobin or fetal hemoglobin. […] CASGEVY is a CRISPR/Cas 9 based one-time cell-based gene therapy given for the treatment of individuals 12 years of age and older with sickle cell disease who have recurrent vaso-occlusive events (pain crisis).

• #42 Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease > News > Yale Medicine

  https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease

  The therapies are hailed as groundbreaking as they represent the first-ever gene therapies to potentially cure a hereditary condition. […] This new technology uses gene therapy to allow patients to be their own match. […] If we can intervene and prevent these complications and let these patients live full lives, that is huge, Dr. Calhoun says. […] This is a big step forward. […] However, the gene therapies are time-intensive taking about a year to complete the process and grueling. […] The gene therapies will be available only at large, authorized medical centers because they require advanced care. […] Dr. Krishnamurti says both treatments will be available at Yale and that anyone interested in learning more should speak to their physician. […] The goal of genetic therapies for SCD is to treat or cure the disease by changing DNA or adding new DNA.

• #43 Patient Cured of Sickle Cell Anemia With Innovative Gene Therapy

  https://www.pharmacytimes.com/view/patient-cured-of-sickle-cell-anemia-with-innovative-gene-therapy

  Lovotibeglogene autotemcel offers a potential cure for sickle cell disease, marking a significant advancement in treatment options. […] The therapy involves modifying blood stem cells to produce HbAT87Q, reducing sickling and improving blood flow. […] Until the development of novel gene therapies, there were no curative options for patients with SCD. […] Lovotibeglogene autotemcel is a cell-based gene therapy that was approved by the FDA on December 8, 2023, for treatment of patients 12 years of age and older with SCD and a history of vaso-occlusive events. […] Lovotibeglogene autotemcel involves collecting a patient’s blood stem cells and genetically modifying them to produce HbAT87Q, a gene-therapy-derived hemoglobin that functions similarly to hemoglobin A, the adult hemoglobin produced in individuals unaffected by SCD.

• #44 FDA Approves Two Gene Therapies for Sickle Cell Disease | Children’s Hospital of Philadelphia

  https://www.chop.edu/news/fda-approves-two-gene-therapies-sickle-cell-disease

  In the case of sickle cell disease, the newly approved therapy edits DNA within the patients own cells and enables the patient to produce a different form of hemoglobin in their red blood cells. […] In the case of LYFGENIA, the gene therapy is specifically designed to treat the underlying cause of sickle cell disease by adding a functional gene that enables production of adult hemoglobin that does not form into the crescent shape associated with the disease. […] In clinical trials with CASGEVY, we have seen elimination of painful vaso-occlusive crisis in more than 90% of patients and a complete elimination of VOC-related hospitalizations in all patients, with a safety profile generally consistent with autologous stem cell transplantation, said Stephan A. Grupp, MD, PhD, Section Chief of the Cellular Therapy and Transplant Section, Inaugural Director of the Susan S. and Stephen P. Kelly Center for Cancer Immunotherapy, and Medical Director of the Cell and Gene Therapy Laboratory at CHOP.

• #45 Sickle Cell Disease

  https://patienteducation.asgct.org/disease-treatments/sickle-cell-disease

  LYFGENIA is a one-time cell-based gene therapy utilizing a lentiviral vector for the treatment of individuals 12 years and older with sickle cell disease who have a history of vaso-occlusive events (pain crisis). […] Other treatment options: A bone marrow transplant is a one-time, potentially curative option for sickle cell disease. […] Gene therapy aims to be administered only once and has the potential to be curative. […] Further gene therapy and gene editing approaches are being researched in trials for SCD. […] Clinical trials may differ on various aspects of their design. […] Gene therapy aims to be a one-time treatment with lasting positive effects that slow or stop disease progression for a lifetime. […] At this time, we do not know if or when more gene therapies will be approved by the FDA and commercially available for people living with Sickle Cell Disease.

• #46 Experimental Gene Therapy Reverses Sickle Cell Disease for Years | Columbia University Irving Medical Center

  https://www.cuimc.columbia.edu/news/experimental-gene-therapy-reverses-sickle-cell-disease-years

  A study of an investigational gene therapy for sickle cell disease has found that a single dose restored blood cells to their normal shape and eliminated the most serious complication of the disease for at least three years in some patients. […] The single-dose therapy, tested on 35 adults and adolescents with sickle cell disease, essentially corrected the shape of the patients red blood cells, but also completely eliminated episodes of severe pain, caused when rigid, crescent-shaped red blood cells clump together and block blood vessels. […] You cannot overstate the potential impact of this new therapy, says Markus Y. Mapara, MD, PhD, professor of medicine at Columbia University Vagelos College of Physicians and Surgeons and a co-author of the study. […] With the new gene therapy, called LentiGlobin, blood-forming stem cells are collected from the patients blood. Harmless lentiviruses are then used to deliver a modified copy of the beta-globin gene into the stem cells. When the cells are later reinfused into the patient, they take up residence in the bone marrow and start making healthy new red blood cells.

• #47 Gene Therapy for Sickle Cell Disease

  https://www.sparksicklecellchange.com/treatment/sickle-cell-gene-therapy

  Gene editing modifies a person’s DNA by either changing a mutated gene directly or by changing a related gene to compensate for the mutated one. […] In addition to gene editing and gene addition, there are other types of gene therapy being studied as part of the greater sickle cell disease treatment landscape, like gene correction and gene silencing. […] The treatment process for gene therapy relies on close collaboration between a patient and their primary physician, their treatment team, and their caregivers. […] In general, there are 4 steps to the treatment process: consultation, preparation, treatment, and recovery. […] Gene therapy treatment that was prepared using your (if you’re a caregiver, your loved one’s) own cells is infused back in. […] After gene therapy is administered, there is a critical process of recovery and a follow-up monitoring period that can last for at least 15 years.

• #48 Gene Therapy for Sickle Cell Disease | Duke Health

  https://www.dukehealth.org/treatments/gene-therapy-sickle-cell-disease

  Duke Health is one of the few centers in North Carolina to offer a new, FDA-approved gene therapy that can help alleviate severe pain and other serious complications associated with sickle cell disease. […] Gene therapy replaces or modifies abnormal genes to treat or cure disease. During gene therapy for sickle cell disease, cells are removed from your blood, re-engineered in a special facility, and returned to your body through an infusion. The treatment changes parts of your DNA so your body can create healthy, normally shaped red blood cells. This allows your blood to flow freely, mitigating the pain and organ damage that often occur in people with sickle cell disease. […] Once we receive your gene therapy treatment, you will undergo chemotherapy to make room in your blood and bone marrow for your manufactured cells. This treatment will take place in the hospital where you will stay until you have recovered from any side effects of chemotherapy.

• #49 Patient Information | CASGEVY® (exagamglogene autotemcel)

  https://www.casgevy.com/sickle-cell-disease

  CASGEVY is a one-time gene therapy that may help people 12 years and older with sickle cell disease (SCD) and frequent vaso-occlusive crises (VOCs) live severe VOC-free. […] CASGEVY uses your own blood stem cells to help your body make working red blood cells, which can eliminate VOCs. […] In the clinical study, 93.5% (29 out of 31 people) did not have a severe VOC for at least 12 months in a row after receiving CASGEVY. […] CASGEVY is a one-time therapy used to treat people aged 12 years and older with sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs. […] CASGEVY is made specifically for each patient, using the patient’s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with SCD.

• #50 Patient Cured of Sickle Cell Anemia With Innovative Gene Therapy

  https://www.pharmacytimes.com/view/patient-cured-of-sickle-cell-anemia-with-innovative-gene-therapy

  According to data from the clinical trial, the therapy was very successful, resulting in a complete resolution of symptoms within 6 to 18 months in 88% of the trial participants. […] Despite the promise of this treatment, the cost of lovotibeglogene autotemcel raises questions about its accessibility.

• #51 Cures for Sickle Cell Disease Arrive After a Painful Journey | Scientific American

  https://www.scientificamerican.com/article/new-sickle-cell-treatments-highlight-the-power-of-patient-perspectives/

  Sickle cell experts hope more treatments and cures are coming. What we currently have approved for drug therapies is not adequate, says hematologist Modupe Idowu, medical director of the UT Physicians Comprehensive Adult Sickle Cell Center in Houston, which treats about 1,300 adults with sickle cell disease. Hydroxyurea remains the gold standard, but some patients really have reservations about it. […] Until now, the only true cure for sickle cell has been a bone marrow transplant from a family member who is a perfect immunological match. But only a few patientsbetween 10 and 20 percentcan identify one. […] The new gene therapies are an extraordinary scientific achievement, but they are difficult to obtain. The two recent treatments, Casgevy and Lyfgenia, became two of the most expensive drugs on the U.S. market when they were approved, costing $2.2 million and $3.1 million, respectively.

• #52 Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease > News > Yale Medicine

  https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease

  The therapies are hailed as groundbreaking as they represent the first-ever gene therapies to potentially cure a hereditary condition. […] This new technology uses gene therapy to allow patients to be their own match. […] If we can intervene and prevent these complications and let these patients live full lives, that is huge, Dr. Calhoun says. […] This is a big step forward. […] However, the gene therapies are time-intensive taking about a year to complete the process and grueling. […] The gene therapies will be available only at large, authorized medical centers because they require advanced care. […] Dr. Krishnamurti says both treatments will be available at Yale and that anyone interested in learning more should speak to their physician. […] The goal of genetic therapies for SCD is to treat or cure the disease by changing DNA or adding new DNA.

• #53 Gene Therapy: What You Need to Know (Warrior FAQs) – Sickle Cell Disease Association of America Inc.

  https://www.sicklecelldisease.org/2023/12/08/gene-therapy-what-you-need-to-know/

  Only a few centers across the USA offer gene therapy for sickle cell disease. […] Gene therapy is expensive, and FDA-approved high-cost medications can come with barriers. Exagamglogene autotemcel is estimated to cost $2.2 million, and lovotibeglogene autotemcel is estimated to cost $3.1 million. […] As far as we know, yes. It is designed to be able to help raise fetal hemoglobin (HbF), which should work for all different kinds of sickle cell disease. […] For many people, the benefits of this new treatment outweigh the risks. Your doctors will help you determine whether this is a good option for you.

• #54 Sickle cell anemia – Diagnosis & treatment – Mayo Clinic

  https://www.mayoclinic.org/diseases-conditions/sickle-cell-anemia/diagnosis-treatment/drc-20355882

  Management of sickle cell anemia is usually aimed at avoiding pain episodes, relieving symptoms and preventing complications. Treatments might include medicines and blood transfusions. For some children and teenagers, a stem cell transplant might cure the disease. Gene therapies also are being developed that may offer cures for people with sickle cell disease. […] Hydroxyurea reduces the frequency of pain crises and might reduce the need for blood transfusions and hospital stays. […] L-glutamine oral powder helps in reducing the frequency of pain crises. […] Crizanlizumab can help reduce the frequency of pain crises in adults and in children older than 16 years. […] Voxelotor is used to treat sickle cell disease in adults and in children older than 12 years. […] Pain-relieving medicines might be prescribed to help relieve pain during sickle cell pain crises.

• #55 How is Sickle Cell Disease Treated?

  https://www.seattlechildrens.org/conditions/sickle-cell-disease/treatment/

  We watch your childs health closely and recommend the best treatments for them. […] Regular check-ups are an important way to keep your child healthy. […] We help our patients, families and their schools know about their disease and how to take care of it. […] Pain is a common problem, caused by sickle cells blocking the blood flow to an organ or bones. […] We work with you and your child to plan how to deal with pain. […] Most young children will need to take the antibiotic penicillin daily to help prevent infection. […] Your child may need a transfusion of red blood cells from a healthy donor. […] If sickle cells clog blood vessels in the lungs, it can damage the lungs. […] Sickle cell disease increases a childs chance of having a stroke. […] For certain types of sickle cell, the doctor will recommend a medicine to reduce or prevent some of the complications of the disease.

• #56

  https://www.nhs.uk/conditions/sickle-cell-disease/

  People with sickle cell disease need treatment throughout their lives. This is usually delivered by different health professionals in a specialist sickle cell centre. […] A number of treatments for sickle cell disease are available. […] For example: drinking plenty of fluids and staying warm to prevent painful episodes […] painkillers, such as paracetamol or ibuprofen (sometimes treatment with stronger painkillers in hospital may be necessary) […] daily antibiotics and having regular vaccinations to reduce your chances of getting an infection […] a medicine called hydroxycarbamide (hydroxyurea) to reduce symptoms […] regular blood transfusions if symptoms continue or get worse, or there are signs of damage caused by sickle cell disease […] an emergency blood transfusion if severe anaemia develops. […] The only cure for sickle cell disease is a stem cell or bone marrow transplant, but they’re not done very often because of the risks involved.

• #57 Sickle Cell Disease – Treatment | NHLBI, NIH

  https://www.nhlbi.nih.gov/health/sickle-cell-disease/treatment

  Crizanlizumab-tmca is approved for adults and children ages 16 years and older who have sickle cell disease. The medicine helps prevent blood cells from sticking to blood vessel walls and blocking blood flow. […] Daily treatment with an antibiotic called penicillin helps lower children’s chance of having a serious infection in the bloodstream. […] Your healthcare provider may recommend a blood transfusion to treat and prevent certain sickle cell disease complications. […] A blood and bone marrow transplant is a potential curative therapy for some people with sickle cell disease. […] Gene therapies aim to treat conditions by adding new DNA or changing existing DNA. In December 2023, the Food and Drug Administration approved two gene therapies to treat sickle cell disease: Exagamglogene autotemcel makes changes to a gene that is already in the body and Lovotibeglogene autotemcel adds a modified gene to the body. […] Blood and bone marrow transplants and gene therapies require weeks in a hospital, specialized care, and costly medicines.

• #58 How is Sickle Cell Disease Treated?

  https://www.seattlechildrens.org/conditions/sickle-cell-disease/treatment/

  We watch your childs health closely and recommend the best treatments for them. […] Regular check-ups are an important way to keep your child healthy. […] We help our patients, families and their schools know about their disease and how to take care of it. […] Pain is a common problem, caused by sickle cells blocking the blood flow to an organ or bones. […] We work with you and your child to plan how to deal with pain. […] Most young children will need to take the antibiotic penicillin daily to help prevent infection. […] Your child may need a transfusion of red blood cells from a healthy donor. […] If sickle cells clog blood vessels in the lungs, it can damage the lungs. […] Sickle cell disease increases a childs chance of having a stroke. […] For certain types of sickle cell, the doctor will recommend a medicine to reduce or prevent some of the complications of the disease.

• #59 Sickle Cell Disease | Sickle Cell Anemia | MedlinePlus

  https://medlineplus.gov/sicklecelldisease.html

  Complementary and alternative medicine (CAM) seems to help some people deal with pain caused by SCD. These types of CAM may lower your pain, especially if it is not well managed with medicines: Cognitive behavioral therapy (a type of counseling) […] Acupuncture […] Exercise or movement programs, such as yoga […] Massage […] Meditation and mindfulness practices […] Virtual reality, a computer-generated 3D environment you can see with special goggles. […] It’s also important to take steps to keep yourself as healthy as possible: Get regular medical care […] Get your routine vaccinations […] Live a healthy lifestyle […] Avoid situations that may set off a pain crisis.

• #60 Sickle cell disease – Wikipedia

  https://en.wikipedia.org/wiki/Sickle_cell_disease

  Vaccination, antibiotics, high fluid intake, folic acid supplementation, pain medication, blood transfusions […] The care of people with sickle cell disease may include infection prevention with vaccination and antibiotics, high fluid intake, folic acid supplementation, and pain medication. Other measures may include blood transfusion and the medication hydroxycarbamide (hydroxyurea). […] Hydroxyurea was the first approved drug for the treatment of SCD, which has been shown to decrease the number and severity of attacks and possibly increase survival time. […] Voxelotor was received accelerated approval as a treatment for SCD in the United States in 2019, and was approved by the European Medicines Agency (EMA) in 2021. […] A simple blood transfusion can be used to treat SCD when hemoglobin levels drop too low, or to prepare for an operation or pregnancy.

• #61

  https://www.beaumont.org/treatments/sickle-cell-anemia-diagnosis-treatment

  Folic acid. Folic acid will help prevent severe anemia. […] Hydroxyurea. Hydroxyurea is an oral medication that has recently been found to help reduce the frequency of pain crises and acute chest syndrome. It may also help decrease the need for frequent blood transfusions. The long-term effects of the medication, however, are unknown. […] Regular eye exams. These are done to screen for retina damage. […] Bone marrow transplant. Bone marrow transplants have been effective in curing some people with sickle cell disease; the decision to undergo this procedure is based on the severity of the disease and ability to find a suitable bone marrow donor. These decisions need to be discussed with your doctor and are only done at centers that specialize in stem cell transplantation.

• #62

• #63 Sickle Cell Disease (SCD) Treatment & Management: Approach Considerations, Hydroxyurea Therapy, Transfusion

  https://emedicine.medscape.com/article/205926-treatment

  The National Institutes of Health advises that optimal care for patients with sickle cell disease (SCD), including preventive care, is best achieved through treatment in clinics that specialize in the care of SCD. All patients with SCD should have a principal health care provider, who should either be a hematologist or be in frequent consultation with one. […] An expert panel has released evidence-based guidelines for the treatment of SCD, including a strong recommendation that hydroxyurea and long-term, periodic blood transfusions should be used more often to treat patients. […] In 2017, the US Food Drug Administration (FDA) approved L-glutamine oral powder (Endari) for patients age 5 years and older to reduce severe complications of SCD. […] Crizanlizumab, a P-selectin inhibitor, was approved by the FDA in 2019 to reduce the frequency of vaso-occlusive crisis (VOC) in adults with SCD.

• #64 Sickle Cell Disease Care | UCLA Health

  https://www.uclahealth.org/medical-services/sickle-cell-disease-care

  Our comprehensive Sickle Cell Disease Program offers you convenient access to highly specialized providers who deliver a broad spectrum of sickle cell disease treatments. […] The UCLA Health Sickle Cell Disease Program is the only dedicated, subspecialized program for patients with sickle cell disease in Los Angeles County. […] This dedicated-care model significantly reduces the risk of hospitalization and complications for patients with this complex condition. […] Our sickle cell disease treatment team includes primary care providers (PCPs) with specific training in sickle cell disease in children and adults, hematologists, pain management specialists, neurologists, orthopaedic specialists, infectious disease specialists, transfusion medicine specialists, pulmonologists, cardiologists, nephrologists, ophthalmologists, dentists, psychologists, physical therapists and more.

• #65 Get Sickle Cell Disease Treatment | Cleveland Clinic

  https://my.clevelandclinic.org/services/sickle-cell-disease-treatment

  We may recommend a blood transfusion or red blood cell exchange (replacing abnormal cells with healthy donor cells). These treatments wont cure SCD, but they may prevent or treat serious complications like strokes, acute chest syndrome and organ failure. […] Cleveland Clinic experts have the necessary experience to treat people with sickle cell disease who need a bone marrow transplant and gene therapy. We are now a qualified treatment center (QTC) for sickle cell gene therapy and can offer this clinically. […] Starting treatment early and having ongoing care is key to effectively managing this chronic condition. The SCD experts at Cleveland Clinic are here to help. Working as a team, our specialists will confirm your or your childs diagnosis and craft a treatment plan to help manage symptoms, get pain relief and help prevent complications.

• #66 Complications & Treatments – Sickle Cell Disease Association of America Inc.

  https://www.sicklecelldisease.org/treatments/

  L-glutamine, an antioxidant used in glutathione production, was approved for patients five years old and above by the FDA in 2017. […] An infusional medication FDA-approved for patients 16 and above in 2019. […] A bone marrow transplant is one of the few methods that can cure sickle cell disease; however, for most members of the SCD community, it is not a viable option. […] Two gene therapies were approved in Dec. 2023 by the Food and Drug Administration (FDA) to treat sickle cell disease: exagamglogene autotemcel and lovotibeglogene autotemcel. […] Blood transfusions help benefit sickle cell disease patients by reducing recurrent pain crises, risk of stroke and other complications. […] Patients with sickle-cell disease (SCD) receiving chronic transfusions of red blood cells are at risk of developing transfusional iron overload over time. […] Preventative care and screening can help you stay healthy and keep you out of the hospital. […] Individuals with SCD who wish to have children may need extra support as they consider starting a family and/or navigate a pregnancy. […] Living with a chronic disease can be stressful.

• #67 Sickle Cell Disease Care | UCLA Health

  https://www.uclahealth.org/medical-services/sickle-cell-disease-care

  Clinical trials provide patients with improved treatment options that can impact patient outcomes. […] Through our program, you have easy access to a sickle cell disease-trained PCP. […] Our coordinated approach helps ensure you receive the ongoing care you need without visiting the hospital or emergency department for pain or other complications. […] Our dedicated medical social workers can help you connect to support groups, find transportation or fill housing or food needs. […] Sickle cell disease management typically focuses on reducing symptoms and lowering your risk of complications. Your personalized treatment plan may include: […] Your provider may prescribe oral or intravenous (IV) medications to reduce the frequency of pain episodes, increase blood flow, lower your risk of anemia or relieve pain during severe flare-ups.

• #68 How is Sickle Cell Disease Treated?

  https://www.seattlechildrens.org/conditions/sickle-cell-disease/treatment/

  Transfusing blood means giving red blood cells from a healthy donor. […] Some children may be cured of sickle cell disease by a transplant of blood-forming stem cells from a healthy donor. […] Our Non-Malignant Transplant Program specializes in stem cell transplants for children with blood disorders like sickle cell disease. […] We work to support your child at home, at school and in the hospital. […] We help our patients keep getting the care they need as they become adults.

• #69 New Therapeutic Options for the Treatment of Sickle Cell Disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6328043/

  In the last two decades, the availability of mouse models for SCD has allowed both characterization of the pathogenesis of sickle cell related organ damage(s) and identification of pathophysiology-based new therapeutic options in addition to HU. […] Recent reports indicate GBT440, an oral direct anti-sickling agent, to be beneficial in SCD. GBT440 (or voxelotor) blocks HbS intermolecular contacts, preventing the generation of HbS fibers and red cell sickling. […] In the last two decades, progresses on hematopoietic stem cell transplantation (HSCT) strategies have allowed to offer a new curative option to patients with SCD. The major limitation in diffusion of HSCT is (i) the availability of leukocyte antigen (HLA)-matched sibling donor; (ii) the toxicities associated with myeloablative conditioning; and (iii) inflammatory vasculopathy.

• #70 New Therapeutic Options for the Treatment of Sickle Cell Disease

  https://pmc.ncbi.nlm.nih.gov/articles/PMC6328043/

  Recently, lentiviral gene therapy has been reported to be safe and to positively impact hematologic phenotype in a child with SCD. Different clinical trials on gene therapy in SCD are on-going in various countries. […] Finally, the development of CRISPR/Cas9 genome editing (GE) strategy has been reported to represent a new potential therapeutic tool for genetic correction of SCD.

• #71 CRISPR Sickle Cell Gene Therapy: Approaches, Challenges, and Progress

  https://www.synthego.com/crispr-sickle-cell-disease

  One of the main approaches to CRISPR sickle cell gene therapy is to repair the mutation in the adult hemoglobin gene responsible for sickle cell disease, causing the healthy, normal form of adult hemoglobin (hemoglobin S) to be produced. […] The second approach to CRISPR sickle cell gene therapy involves a gene knockout, switching off the gene that suppresses fetal hemoglobin. […] CTX001, also referred to Casgevy, is one such investigational new drug application, co-sponsored by CRISPR Therapeutics and Vertex Pharmaceuticals, to treat sickle cell disease. […] The RUBY trial, spearheaded by Editas, is a phase I/II trial of an autologous CD34+ hematopoietic stem cell therapy called EDIT-301. […] Graphite Bio is taking a different approach to treating sickle cell anemia, hoping to cure the condition by restoring the expression of adult hemoglobin rather than replacing it with fetal hemoglobin.

• #72 Casgevy and Lyfgenia: Two Gene Therapies Approved for Sickle Cell Disease > News > Yale Medicine

  https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease

  Patients interested in knowing more about gene therapy should have a conversation with their primary hematologist, Dr. Calhoun says. […] There is more in the pipeline. Today, someone might not be a candidate because they have organ dysfunction, but in the next few years, we will be further along with the science, and there may be a new treatment for that person, he says.

• #73 Cures for Sickle Cell Disease Arrive After a Painful Journey | Scientific American

  https://www.scientificamerican.com/article/new-sickle-cell-treatments-highlight-the-power-of-patient-perspectives/

  The gene therapies are built on the back of a stem cell transplant. A patient must receive transfusions to force down the number of sickled cells in their bloodstream, then growth factors to make sure they produce enough new stem cells; they need other drugs to move the cells out from their bone marrow. […] Sickle cell medicine now has two goals. One is to imagine the next genetic therapies, perhaps developing gene edits that can be inserted into a patient without disrupting their immune system and blood cells first. The other, even more important, is to envision fair, thorough and accessible care. Patients and researchers agree that is whats most essential now.

• #74 Advances in the diagnosis and treatment of sickle cell disease | Journal of Hematology & Oncology | Full Text

  https://jhoonline.biomedcentral.com/articles/10.1186/s13045-022-01237-z

  Sickle cell disease (SCD) is characterized by chronic hemolytic anemia, severe acute and chronic pain as well as end-organ damage that occurs across the lifespan. Treatment requires early diagnosis, prevention of complications and management of end-organ damage. […] Updates in disease-modifying and curative therapies for SCD are also discussed. […] The recent availability of l-glutamine, crizanlizumab and voxelotor provides an alternative or supplement to hydroxyurea, which remains the mainstay for disease-modifying therapy. […] Despite progress in the field, additional longitudinal studies, clinical trials as well as dissemination and implementation studies are needed to optimize outcomes in SCD. […] Hydroxyurea continues to represent a mainstay of disease-modifying therapy for SCD.

• #75 Newer Modalities and Updates in the Management of Sickle Cell Disease: | JBM

  https://www.dovepress.com/newer-modalities-and-updates-in-the-management-of-sickle-cell-disease–peer-reviewed-fulltext-article-JBM

  Sickle cell disease (SCD) mainly affects people of African, Mediterranean, Middle Eastern, and South Asian descent, while it can strike any ethnic group. The primary intervention for anemia is blood transfusion, which increases the oxygen-carrying capacity of the blood and reduces the percentage of sickled cells. However, for vaso-occlusive crises (VOCs), the interventions include pain relief (typically with analgesics), hydration (to reduce blood viscosity), and, in some cases, blood transfusions to reduce the concentration of sickle cells and prevent further sickling. […] While gene editing and bone marrow transplantation (BMT) represent significant advancements in the treatment of SCD, they are not universally accessible or without significant risks. The availability of these treatments is limited by various factors, including cost, healthcare infrastructure, and patient eligibility. Moreover, while these treatments offer the potential for a cure, they come with substantial risks of morbidity and mortality, particularly in the context of BMT.

• #76 NHS England » NHS rolls out ‘life-changing’ treatment for thousands with sickle cell disease

  https://www.england.nhs.uk/2024/05/nhs-rolls-out-life-changing-treatment-for-thousands-with-sickle-cell-disease/

  Professor Bola Owolabi, Director of the National Healthcare Inequalities Improvement Programme at NHS England, said: We are proud to make this new treatment available on the NHS it could help thousands of patients living with sickle cell disease across the country to have a higher quality of life and experience fewer side effects, and represents a significant step forward in addressing the healthcare inequalities experienced by some of our communities. […] It is vital that we continue to get new drugs into the hands of NHS clinicians to improve the lives of people living with sickle cell disorder, like Jennifer. […] Dr Jennifer OConnor, who was diagnosed with sickle cell disease as a child and treated at Whittington Health NHS Trust, said: My life was a constant treadmill of both acute and chronic pain, but in 2022, I accessed a trial of Voxelotor, through the Early Access Medicine Scheme and, within weeks, I felt a very positive impact.

• #77 NHS England » NHS rolls out ‘life-changing’ treatment for thousands with sickle cell disease

  https://www.england.nhs.uk/2024/05/nhs-rolls-out-life-changing-treatment-for-thousands-with-sickle-cell-disease/

  Helen Knight, director of medicines evaluation at NICE, said: This is great news for people with sickle cell disease, particularly given the health inequalities experienced by people with the condition. […] Throughout our appraisal of Voxelotor the appraisal committee has always considered that it has the potential to address the need for effective treatments for sickle cell disease, as well as address NICEs aim of reducing health inequalities.

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