Materiały źródłowe

• #1 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. […] The goals of treatment are symptom control and management of disease complications. Treatment strategies include the following seven goals: Management of vaso-occlusive crisis, Management of chronic pain syndromes, Management of chronic hemolytic anemia, Prevention and treatment of infections, Management of the complications and the various organ damage syndromes associated with the disease, Prevention of stroke, Detection and treatment of pulmonary hypertension.

• #2 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.

• #3 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.

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

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

  Hydroxyurea (hydroxycarbamide) has an established role as a safe and effective treatment for 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. […] 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.

• #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 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.

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

  https://medlineplus.gov/sicklecelldisease.html

  What are the treatments for sickle cell disease (SCD)? There are many ways to manage sickle cell disease. Your medical team will probably include a hematologist, a doctor who specializes in blood diseases. You will work with your medical team to set up a treatment plan. Possible treatment options may include: […] Treatments that can help relieve symptoms and lessen complications, including: Hydroxyurea, a medicine to reduce sickling of red blood cells. This can help prevent serious symptoms of sickle cell disease. This medicine can be used in adults and in children as young as 9 months old. But this medicine is not safe during pregnancy. Voxelotor, another medicine to prevent the sickling of red blood cells. It can be used in adults and children ages 4 years and older. Pain relievers for acute or chronic pain. Antibiotics to try to prevent infections in younger children. Blood transfusions for severe anemia. If you have had some serious complications, such as a stroke, you may have transfusions to prevent more complications. Other treatments for specific complications, such as medicines to lower blood pressure and vitamins to treat a vitamin deficiency.

• #8 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.

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

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

  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. […] 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.

• #10 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. It also helps prevent pain crises, lowers the need for transfusions, and calms inflammation. […] 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. […] Regular or ongoing blood transfusions may help lower the chances of another stroke in people who have already had one stroke. […] A blood and bone marrow transplant is a potential curative therapy for some people with sickle cell disease.

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

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

  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). […] One way to treat anemia is with a blood transfusion. You’ll get healthy red blood cells from a donor whose blood has been screened for infections and matched to your type and needs. […] Transfusions can lower the odds of another stroke by about 90%. […] Theyre the only way to cure sickle cell disease. Stem cells are cells in your bone marrow that grow into new red or white blood cells and platelets (cells that help form blood clots). Using this treatment is risky and relatively new for treating SCD.

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

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

  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.

• #13 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. It also helps prevent pain crises, lowers the need for transfusions, and calms inflammation. […] 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. […] Regular or ongoing blood transfusions may help lower the chances of another stroke in people who have already had one stroke. […] A blood and bone marrow transplant is a potential curative therapy for some people with sickle cell disease.

• #14

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

  Sickle cell disease usually requires lifelong treatment. […] Your healthcare team will help you learn more about sickle cell disease, and work with you to come up with an individual care plan that takes into account all your needs and health concerns. […] If you continue to have episodes of pain, a medicine called hydroxycarbamide (hydroxyurea) may be recommended. […] If you have a sickle cell crisis, you can usually manage it at home. […] You may need treatment with very strong painkillers, such as morphine, in hospital for a few days. […] Most people need to take a daily dose of antibiotics, usually penicillin, often for the rest of their life. […] 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.

• #15 Sickle Cell Disease (SCD) Medication: Antimetabolites, P-Selectin Inhibitor, Gene Therapies, Hematologics, Tricyclic Antidepressants, Nutritionals, Adrenergic Agonists, Vaccines

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

  Glutamine reduces acute complications (eg acute chest syndrome) associated with SCD. […] It is recommended to maintain an up-to-date immunization schedule for pneumococcal, haemophilus influenzae and meningococcal vaccine. […] FDA approves new treatment for sickle cell disease. […] Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease. […] Gene therapy in sickle cell disease: Possible utility and impact. […] Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease.

• #16 Sickle cell disease – Wikipedia

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

  Treatment 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. […] A simple blood transfusion can be used to treat SCD when hemoglobin levels drop too low, or to prepare for an operation or pregnancy. […] Transcranial Doppler ultrasound (TCD) can detect children with sickle cell that have a high risk for stroke.

• #17

  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.

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

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

  Hydroxyurea (hydroxycarbamide) has an established role as a safe and effective treatment for 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. […] 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.

• #19 Sickle cell disease – Wikipedia

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

  In 2019, crizanlizumab, a monoclonal antibody targeting P-selectin, was approved in the United States to reduce the frequency of vaso-occlusive crisis in those 16 years and older. […] Acute chest syndrome is caused by vaso-occlusion occurring in the lungs. As with a VOC, treatment includes pain control and hydration. Antibiotics are required because there is a severe risk of pulmonary infection, and oxygen supplementation for hypoxia. Blood transfusion may also be required, or exchange transfusion in severe cases. […] 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. Treatment options include resting the joint, physical therapy, pain-relief medicine, joint-replacement surgery, or bone grafting.

• #20 Sickle cell disease – Wikipedia

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

  Treatment 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. […] A simple blood transfusion can be used to treat SCD when hemoglobin levels drop too low, or to prepare for an operation or pregnancy. […] Transcranial Doppler ultrasound (TCD) can detect children with sickle cell that have a high risk for stroke.

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

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

  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). […] One way to treat anemia is with a blood transfusion. You’ll get healthy red blood cells from a donor whose blood has been screened for infections and matched to your type and needs. […] Transfusions can lower the odds of another stroke by about 90%. […] Theyre the only way to cure sickle cell disease. Stem cells are cells in your bone marrow that grow into new red or white blood cells and platelets (cells that help form blood clots). Using this treatment is risky and relatively new for treating SCD.

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

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

  Hydroxyurea (hydroxycarbamide) has an established role as a safe and effective treatment for 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. […] 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.

• #23

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

  A stem cell transplant is an intensive treatment that carries a number of risks. […] Stem cell transplants are generally only considered in children with sickle cell disease who have severe symptoms that have not responded to other treatments, when the long-term benefits of a transplant are thought to outweigh the possible 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.

• #24

  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.

• #25 Sickle Cell Disease Care | Loma Linda University Health

  https://lluh.org/services/sickle-cell-disease-care

  Dehydration can lead to sickle cell crisis we provide fluids to rehydrate your body and relieve symptoms. […] Certain infections are more common with sickle cell disease. We work with your primary care provider to make recommendations for treatment. […] Sickle cell disease requires ongoing care to manage symptoms and complications. Most people can expect follow-up appointments every few months, depending on individual needs.

• #26

  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.

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

  https://medlineplus.gov/sicklecelldisease.html

  Bone marrow or stem cell transplantation, which can cure SCD. Because these transplants are risky and can have serious side effects, they are usually only used in children with severe SCD. For the transplant to work, the bone marrow must be a close match. Usually, the best donor is a brother or sister. […] Gene therapies to treat SCD in people who are 12 years and older and have had repeated sickle cell crises. These new therapies involve taking some of your blood stem cells and either adding new DNA to them or changing their existing DNA. Then these cells are given back to you, and they can make a type of hemoglobin that is healthy. This can reduce the complications of SCD, including the SCD crises. […] 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.

• #28 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 best way to manage your condition is to see your doctor or hematologist as early as possible and to stick with your treatments long term. […] You can also try: A heating pad or hot baths, Massage, Acupuncture, Relaxation techniques like deep breathing or meditation. […] 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.

• #29

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

  Sickle cell disease usually requires lifelong treatment. […] Your healthcare team will help you learn more about sickle cell disease, and work with you to come up with an individual care plan that takes into account all your needs and health concerns. […] If you continue to have episodes of pain, a medicine called hydroxycarbamide (hydroxyurea) may be recommended. […] If you have a sickle cell crisis, you can usually manage it at home. […] You may need treatment with very strong painkillers, such as morphine, in hospital for a few days. […] Most people need to take a daily dose of antibiotics, usually penicillin, often for the rest of their life. […] 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.

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

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

  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. […] 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.

• #31 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.

• #32 Sickle cell disease – Wikipedia

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

  Gene therapy was first trialled in 2014 on a single patient, and followed by clinical trials in which a number of patients were successfully treated. In 2023, both exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) were approved for the treatment of sickle cell disease. […] Hematopoietic stem cell transplantation (HSCT) involves replacing the dysfunctional stem cells from a person with sickle cell disease with healthy cells from a well-matched donor.

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

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

  Currently, most blood and bone marrow transplants are done in children who have had complications, such as strokes, acute chest crises, and repeated pain crises. […] 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.

• #34 Sickle Cell Anemia Treatment and Advancement Toward Cure Through Stem Cell Therapy: A Comprehensive Review | Published in Georgetown Medical Review

  https://gmr.scholasticahq.com/article/125984-sickle-cell-anemia-treatment-and-advancement-toward-cure-through-stem-cell-therapy-a-comprehensive-review

  A 2020 research letter detailed the findings in a prospective phase 2 multicenter trial consisting of 19 pediatric patients with SCD using a HAPLO stem cell transplant from a family member. […] To best address the toxic nature of myeloablative allogeneic HSCT, there have been efforts to explore nonmyeloablative allogeneic HSCT in adults. […] A follow-up study from Hsieh and colleagues included 20 additional patients, follow-ups on the original 10 patients, and an amended protocol. […] HLA-matched donors have a reduced chance of graft rejection and lower mortality rate than recipients with an HLA-unmatched donor. […] A single-center, open-label, pilot study explored the use of BCH-BB694 lentiviral vector-transduced CD34+ cells from 6 patients with severe SCD for autologous HSCT. […] Another potential lentiviral vector-based autologous stem cell gene therapy has been researched.

• #35 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. […] But trying to find a good match for a transplant is a big barrier. 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. […] Many can’t have bone marrow transplantation because only about 15% of patients have a matched sibling, and we can find an unrelated donor for only another 10% to 12%. That means we are only helping 25% of patients, says Dr. Krishnamurti, who was an author on the Lyfgenia study published in The New England Journal of Medicine. This is a big step forward. […] However, the gene therapies are time-intensive taking about a year to complete the process and grueling.

• #36 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

  Doctors at Hassenfeld Childrens Hospital perform stem cell transplantation in children with severe pain, anemia, chronic lung problems, or a high risk of stroke. […] In the weeks before this procedure, doctors may treat your child with chemotherapy or radiation therapy to destroy abnormal blood-producing cells in the bone marrow. […] Immunosuppressant medication is also given to reduce the risk of graft-versus-host disease, a condition in which the donated stem cells attack the hosts cells, usually in the skin and lining of the intestine. […] Although stem cell transplantation offers some children symptom relief and can help to prevent serious complications, it is a procedure that requires careful consideration. […] Our specialists can counsel you about the benefits and risks of stem cell transplantation for your child.

• #37 Sickle Cell Anemia Treatment and Advancement Toward Cure Through Stem Cell Therapy: A Comprehensive Review | Published in Georgetown Medical Review

  https://gmr.scholasticahq.com/article/125984-sickle-cell-anemia-treatment-and-advancement-toward-cure-through-stem-cell-therapy-a-comprehensive-review

  A 2020 research letter detailed the findings in a prospective phase 2 multicenter trial consisting of 19 pediatric patients with SCD using a HAPLO stem cell transplant from a family member. […] To best address the toxic nature of myeloablative allogeneic HSCT, there have been efforts to explore nonmyeloablative allogeneic HSCT in adults. […] A follow-up study from Hsieh and colleagues included 20 additional patients, follow-ups on the original 10 patients, and an amended protocol. […] HLA-matched donors have a reduced chance of graft rejection and lower mortality rate than recipients with an HLA-unmatched donor. […] A single-center, open-label, pilot study explored the use of BCH-BB694 lentiviral vector-transduced CD34+ cells from 6 patients with severe SCD for autologous HSCT. […] Another potential lentiviral vector-based autologous stem cell gene therapy has been researched.

• #38 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/

  The new trial, conducted through the National Institutes of Health (NIH)-supported Blood and Marrow Transplant Clinical Trials Network, is a direct extension of our previous study, DeBaun said. The results are just as good, if not better, than gene therapy or gene editing therapy. […] More SCD patients with complicating medical conditions should be able to qualify for curative therapy because the new regimen has fewer toxic side effects and is about one-fifth the cost of myeloablative gene therapy, said DeBaun, senior co-author of the paper with Johns Hopkins Robert Brodsky, MD, who trained at VUMC. […] 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).

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

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

  Currently, most blood and bone marrow transplants are done in children who have had complications, such as strokes, acute chest crises, and repeated pain crises. […] 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.

• #40 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.

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

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

  The first gene editing therapy for sickle cell disease to be approved by the Food and Drug Administration has deep roots at Boston Childrens Hospital. […] Several types of gene therapy are now available for teens with sickle cell disease. All involve collecting a persons own blood stem cells, treating them with gene therapy in a special facility, and then giving them back through an IV infusion. […] CASGEVY, approved by the Food and Drug Administration in 2023, draws on decades of research at Dana Farber/Boston Children’s. It uses CRISPR gene editing to silence a gene in red blood cells, called BCL11A. This enables the body to produce a fetal form of hemoglobin, which does not cause cells to sickle. […] 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.

• #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 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. […] With Casgevy, the patients blood stem cells are edited in a way that makes them produce high levels of fetal hemoglobin, which is produced during fetal development and binds to oxygen more strongly than adult hemoglobin. […] Essentially, Casgevy works by reducing the number of sickle cells in the body to a negligible amount, which may permanently ease symptoms. […] Lyfgenia, meanwhile, works by taking a piece of a virus (a lentivirus, which belongs to the HIV family) and using it to deliver a functional version of a hemoglobin-producing gene.

• #43 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.

• #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 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

  https://www.nbcnews.com/health/health-news/fda-approves-cure-sickle-cell-disease-first-treatment-use-gene-editing-rcna127979

  The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the majority of whom are Black. […] The therapy, called Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine to be approved in the United States that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in chemistry in 2020. […] The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio. Both treatments work by genetically modifying a patients own stem cells. […] Casgevy, which was approved for people ages 12 and older, removes the need for a donor. Using CRISPR, it edits the DNA found in a patients stem cells to remove the gene that causes the disease.

• #46 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 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. […] 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.

• #47 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

  Both products are made from the patients own blood stem cells, which are modified, and are given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. […] The safety and effectiveness of Casgevy were evaluated in an ongoing single-arm, multi-center trial in adult and adolescent patients with SCD. […] The safety and effectiveness of Lyfgenia is based on the analysis of data from a single-arm, 24-month multicenter study in patients with sickle cell disease and history of VOEs between the ages of 12- and 50- years old.

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

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

  Currently, most blood and bone marrow transplants are done in children who have had complications, such as strokes, acute chest crises, and repeated pain crises. […] 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.

• #49 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

  In the clinical trial the therapy completely eliminated severe pain crises in the months following infusion (follow-up ranged from 4 to 38 months)the longest period in which a gene therapy for sickle cell disease has been studied. […] The effects have been sustained throughout the trial period, which suggests that the results may be durable, says Mapara, who is director of the Bone Marrow Transplantation and Cell Therapy Program at Columbia University Irving Medical Center/NewYork-Presbyterian. […] Because LentiGlobin uses a patients own stem cells, theres no risk of rejection, a common complication of conventional bone marrow transplants, Mapara adds. […] Researchers are currently working on less toxic approaches to conditioning the bone marrow before gene therapy. The eventual goal will be to give this treatment as early as possible, well before patients develop organ damage and other complications of sickle cell disease, says Mapara.

• #50 Sickle Cell Disease | Mount Sinai – New York

  https://www.mountsinai.org/care/cancer/services/benign-hematology/sickle-cell-disease

  The advantages of gene therapy versus bone marrow transplant are that there is no need for a matched donor, and there is no need for immune suppression to prevent rejection. […] Both bone marrow transplant and gene therapy have significant risks and side effects and are considered on a case-by-case basis. […] We are continually investigating new treatment options that are well-tolerated and accessible to many patients. […] Our goal is to cure sickle cell disease through collaboration with other leading researchers throughout the United States. […] Our team is composed of leading clinicians and researchers who offer comprehensive care and the most advanced breakthroughs in treatments that dramatically improve outcomes for patients.

• #51 Sickle cell disease | Novartis

  https://www.novartis.com/diseases/sickle-cell-disease

  The Novartis-Gates Foundation collaboration envisions an in vivo gene therapy administered directly to patients, where the goal would be to mitigate the need for extended hospital stays or specialized infrastructures. […] Contributing to this program for an accessible in vivo gene therapy for SCD and other inherited hemoglobinopathies, Novartis recently entered into a collaborative agreement with Precision BioSciences to design a protein that can cut DNA at a specific site, allowing for the insertion of a functional gene that can act in place of the mutated gene that causes red blood cells to become sickle-shaped.

• #52 Vertex Pharmaceuticals | R&D Pipeline | Sickle Cell Disease

  https://www.vrtx.com/our-science/pipeline/sickle-cell-disease/

  We are working to discover and develop conditioning regimens aimed at improving the hematopoietic stem cell transplant process, which is required for gene therapies. […] In addition, we have internal research programs exploring in vivo gene editing approaches and small molecule treatment options targeting BCL11A for people living with SCD. […] We’re investigating multiple approaches to develop an additional conditioning regimen for people living with SCD. […] We’re researching in vivo gene editing approaches to target the underlying cause of SCD. […] We’re investigating small molecule medicines to target the underlying cause of SCD.

• #53 Dana-Farber/Boston Children’s Pediatric Sickle Cell Disease Program | Boston Children’s Hospital

  https://www.childrenshospital.org/programs/sickle-cell-disease-program

  The Sickle Cell Disease Program at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center provides diagnosis, treatment, and long-term health management for infants, children, adolescents, and young adults with sickle cell disease. […] Research, much of it done right here at Boston Childrens, has created treatment options for sickle cell disease, including stem cell transplantation and gene therapy. […] In the 1980s, our physicians were the first to recognize the potential of hydroxyurea to treat sickle cell disease and to use the medication in children. More recently, they have developed stem cell transplantation and gene therapy as treatments for sickle cell disease. […] This work led to the approval of CASGEVY gene therapy for sickle cell disease in 2023. […] Newer approaches were exploring include base editing, a highly precise form of gene therapy that we are testing in a clinical trial, and a potential pill that targets BCL11A, currently in the early stages of laboratory research.

• #54 Current and Future Therapeutics for Treating Patients with Sickle Cell Disease

  https://www.mdpi.com/2073-4409/13/10/848

  Mitapivat (AG-348) is a first-in-class small-molecule allosteric activator of pyruvate kinase and was approved by the FDA in February 2022 for the treatment of hemolytic anemia. […] Gene therapy is being extensively researched as a potential cure for SCD. […] BCH-BB694 is a lentiviral vector that encodes a miRNA-adapted short hairpin RNA (shRNAmiR) targeting BCL11A. […] Casgevy (CTX001) is an autologous gene therapy based on CD34+ hematopoietic stem and progenitor cells (HSPCs). […] BB305 is a globin lentiviral vector encoded with a healthy β-globin gene that codes for antisickling hemoglobin, HbAT87Q. […] Overall, treatments for patients with SCD focus on managing symptoms, preventing complications, and improving the patient’s overall quality of life. […] While the only cure for SCD is bone marrow transplantation, recently, the FDA approved two gene therapies (Casgevy and Lyfgenia) for treating patients with SCD.

• #55 Advances in Sickle Cell Disease

  https://www.physiology.org/publications/news/the-physiologist-magazine/2024/may/advances-in-sickle-cell-disease

  There are also new drugs in clinical trials. […] We hope that memantine can be used to stabilize the red blood cells and also to somewhat relieve pain, inflammation and organ damage. […] Its too early to know its long-term effects, but the first outcomes for the SCD patients treated with memantine for 12 months are promising. […] Van Doren believes that this small molecule can be combined with hydroxyurea for a more comprehensive treatment approach.

• #56 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. […] The goals of treatment are symptom control and management of disease complications. Treatment strategies include the following seven goals: Management of vaso-occlusive crisis, Management of chronic pain syndromes, Management of chronic hemolytic anemia, Prevention and treatment of infections, Management of the complications and the various organ damage syndromes associated with the disease, Prevention of stroke, Detection and treatment of pulmonary hypertension.

• #57 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. […] 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. […] Clinical trials provide patients with improved treatment options that can impact patient outcomes.

• #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 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. […] Some children may be cured of sickle cell disease by a transplant of blood-forming stem cells from a healthy donor. […] For certain types of sickle cell, the doctor will recommend a medicine to reduce or prevent some of the complications of the disease. […] Transfusing blood means giving red blood cells from a healthy donor. […] If transplant may be an option for your child, we will talk with you about the risks and benefits.

• #59

  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.

• #60 Sickle cell disease – Wikipedia

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

  Treatment 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. […] A simple blood transfusion can be used to treat SCD when hemoglobin levels drop too low, or to prepare for an operation or pregnancy. […] Transcranial Doppler ultrasound (TCD) can detect children with sickle cell that have a high risk for stroke.

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

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

  Health maintenance for patients with sickle cell disease starts with early diagnosis, preferably in the newborn period and includes penicillin prophylaxis, vaccination against pneumococcus bacteria and folic acid supplementation. […] Treatment of complications often includes antibiotics, pain management, intravenous fluids, blood transfusion and surgery all backed by psychosocial support. Like all patients with chronic disease patients are best managed in a comprehensive multi-disciplinary program of care. […] First approved by the FDA for SCD in 1998, hydroyurea is considered to be standard of care for treatment. […] The National Heart, Lung and Blood Institute recommends starting hydroxyurea in patients as young as nine months old, and it is FDA approved for pediatric patients who are two years or older. It is strongly recommended to treat sickle cell disease.

• #62 Mental Health and Wellness – Sickle Cell Disease Association of America Inc.

  https://www.sicklecelldisease.org/mental-health-and-wellness/

  Sickle Cell Disease (SCD) is a chronic illness that affects both physical and emotional health. […] Seeking therapy can provide warriors, caregivers, and healthcare workers with the emotional support and coping skills needed to manage the mental health challenges associated with SCD. […] Seeking therapy, however, is far from weakness; in fact, its a proactive step towards a path of healing and mental well-being. […] Talk to a therapist: A mental health professional is a licensed and knowledgeable resource dedicated to treating the mental health challenges associated with SCD. […] Counseling and Support Groups: warriors, caregivers, and healthcare workers should be encouraged to seek counseling to help them manage the emotional toll of this chronic illness. […] Mind-Body Therapy: Mind-body therapy techniques such as meditation, mindfulness, and breathing exercises can help manage the pain and anxiety associated with sickle cell disease.

• #63 Sickle Cell Disease Treatment (Topic Center) (for Teens) | Nemours KidsHealth

  https://kidshealth.org/en/teens/center/sickle-cell-treatment.html

  Learn how medicines can help you stay well. […] Use your voice to get the care you need. Feel good and live a healthy life. […] What’s a preventive medicine? Use this chart (PDF) to see how the meds compare. […] Hydroxyurea […] Crizanlizumab […] L-Glutamine […] Ask your doctor how smoking, drugs, and alcohol can interact with preventive medicines and sickle cell disease. […] Life gets busy. It can be hard to remember to take your meds and follow your treatment plan. Tell your doctor if you miss doses or have side effects. Together you can make a plan that fits your life. […] Taking preventive medicines can help. Doctors are still learning about some newer medicines and their effect on fertility. Talk to your doctor about which preventive medicine is right for you.

• #64 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 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. […] 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.

• #65 Will the High Price of Gene Therapy for Sickle Cell Disease Put This Cure out of Reach? | AAFP

  https://www.aafp.org/pubs/afp/afp-community-blog/entry/will-the-high-price-of-gene-therapy-for-sickle-cell-disease-put-this-cure-out-of-reach.html

  On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved Casgevy, the first gene therapy utilizing clustered, regularly interspaced short palindromic repeats (CRISPR) for the treatment of sickle cell disease in patients 12 years and older. […] The modified stem cells are transplanted back to the patient and result in the production of high levels of HbF, preventing the sickling of red blood cells and eliminating or greatly reducing future painful vaso-occlusive (VOC) crises. […] The approval of Casgevy, which has a list price of $2.2 million for the single course of treatment, had been anticipated for months. […] Although the lifetime medical costs associated with sickle cell disease average $1.7 million, insurance companies may be unwilling to pay the exceptionally high up-front cost of this curative therapy.

• #66 Will the High Price of Gene Therapy for Sickle Cell Disease Put This Cure out of Reach? | AAFP

  https://www.aafp.org/pubs/afp/afp-community-blog/entry/will-the-high-price-of-gene-therapy-for-sickle-cell-disease-put-this-cure-out-of-reach.html

  In Africa and India, which are home to most of the worlds population living with sickle cell disease, many patients die in childhood because of lack of access to standard-of-care treatments. […] For example, hydroxyurea, which reduces the frequency of VOCs and prolongs survival, was approved by the FDA in 1998 but remains unavailable to most patients. […] Experts recently proposed expanding the U.S. Presidents Emergency Plan for AIDS Relief (PEPFAR) to provide hydroxyurea therapy for $67 per person to sickle cell patients in sub-Saharan Africa at a total cost of less than $100 million per year.

• #67

  https://www.nbcnews.com/health/health-news/fda-approves-cure-sickle-cell-disease-first-treatment-use-gene-editing-rcna127979

  Although Casgevy has been shown to be effective, experts still dont know about potential long-term effects, since the trial is only set to run for two years. […] Bhatia is following the patients for 15 years as part of a post-approval study for Casgevy and will be monitoring for signs of long-term effects.

• #68 Sickle cell disease gene therapy FAQ

  https://www.genome.gov/research-at-nhgri/Projects/Democratizing-Education/sickle-cell-disease-gene-therapy-FAQ

  Because gene therapy will not change the DNA in your sperm or egg cells, you will still pass on the sickle cell gene to your child. […] Your clinical trial team will create a follow-up plan to monitor your health. Researchers will use your data (and the data of others) to determine if gene therapy can be considered a cure for sickle cell disease. […] You will be under the care of your clinical trials team and physicians for about two to three years. […] Experts recommend that you are followed for 15 years after your gene therapy treatment as part of the clinical trial. […] The gene therapy process may not work for a few reasons: […] After the process, gene therapy may not have successfully lowered the amount of sickled hemoglobin in your blood. If this happens, your clinical trial doctor and general provider will talk to you about next steps for your long-term care.

• #69 Treatment for Sickle Cell Disease | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/sickle-cell-disease/treatment.html

  Sickle cell disease treatment is highly customized for each patient’s needs. […] Our doctors provide the full range of sickle cell disease treatments. Based on your exact case, your treatment may include medicines, like hydroxyurea, crizanlizumab, L-glutamine and voxelotor, and blood transfusions. […] With the array of medications that we have available, no sickle cell patient should be stuck with the same treatment for their whole life. We can always do better. We can always adjust over time to meet their changing needs. […] Because sickle cell disease can range from mild to severe and it can change over time, there is no single way to treat everyone. However, most people get medicines to prevent sickling (when red blood cells become crescent-shaped), to prevent or reduce pain or to manage other effects of the disease.

• #70 Treatment for Sickle Cell Disease | Fred Hutchinson Cancer Center

  https://www.fredhutch.org/en/diseases/sickle-cell-disease/treatment.html

  We update your treatment plan based on the best scientific evidence as well as how your disease responds and what you prefer. […] We have many tools to help you feel better, such as nutrition care and medicines to help with digestive problems, antibiotics and antiviral drugs to prevent or treat infections, and medicines to remove excess iron. […] Along with treating your sickle cell disease, Fred Hutch provides a range of services to support you and your caregiver. […] Throughout treatment, your team provides follow-up care and support on a schedule designed for you.

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

  https://www.childrenshospital.org/conditions/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. In sickle cell disease, the best transplant outcomes are almost always when the donor is a healthy sibling with compatible stem cells. […] 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.

• #72 Comprehensive Guide to Sickle Cell Disease Symptoms and Treatment | Baystate Health

  https://www.baystatehealth.org/articles/sickle-cell-disease

  Recently, several new medications have been approved for SCD treatment: […] Stem Cell or Bone Marrow Transplantation: As promising as transplantation is as potential cure for SCD, its also fraught with risk and challenges. […] Gene therapy involves modifying a patient’s own stem cells to produce healthy hemoglobin. […] Both options require the patient to undergo chemotherapy prior to being infused with the modified genes but both aim to provide a long-lasting or potentially curative treatment by addressing the cause of SCD at the genetic level. […] At this point, research shows gene therapy has an encouraging success rate of 90%. […] He adds, As research continues, there is hope for even more effective treatments and potential cures in the future.

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