condition Sickle Cell Disease

Overview

Sickle Cell Disease is an inherited disorder that affects the shape of red blood cells. Babies in the United States are tested at birth to see which type of hemoglobin they have in their blood. Hemoglobin is the protein in red blood cells that carries oxygen. People with sickle cell disease make a different type of hemoglobin than people without it. The abnormal hemoglobin causes the red blood cells to form a hooked – or “sickle” – shape which makes it harder for them to easily move through small blood vessels. The sickled red blood cells can get stuck and block the flow of blood. Without blood, the tissue can die since blood brings the oxygen that the tissues – muscles, bones, nerves – need to live and work. Pain crises caused by blocked blood vessels are a common symptom of sickle cell disease and can require a stay in the hospital. Other symptoms can include weakness, feeling tired, swelling in the hands and feet, and vision problems. Cures for sickle cell disease can include gene therapy or a bone marrow transplant, but not every patient can qualify. Treatment mainly focuses on decreasing the number of pain crises.

Drug Therapies

  • Adakveo

    Adakveo (crizanlizumab-tmca)

    Adakveo (crizlanizumab-tmca) is a monoclonal antibody given by infusion to patients with sickle cell disease to decrease the number of vasoocclusive crises.
    Manufacturer:
    Novartis
    Route of Administration: 
    Intravenous
    Site of Care:
    Outpatient
    Website: 
    • https://www.us.adakveo.com
    phone: 
    1-800-282-7630
    Enrollment Forms:
    Synergen Prescription Form
    Patient Support Form

    Approved Indication:

    to reduce the frequency of vasoocclusive crises in adults and pediatric patients aged 16 years and older with sickle cell disease

    Conditions:

    • Sickle Cell Disease1
      less details
    • Desferal

      Desferal (deferoxamine)

      Desferal (deferoxamine) is an iron-chelating agent given by intravenous or subcutaneous infusion to lower iron levels in the blood.
      Manufacturer:
      Novartis
      Route of Administration: 
      Subcutaneous, Intravenous
      Site of Care:
      Outpatient
      Website: 
        Enrollment Forms:
        Synergen Prescription Form

        Approved Indication:

        • as an adjunct to standard measures for the treatment of acute iron intoxication
        • treatment of transfusional iron overload in patients with chronic anemia

        Conditions:

        • Sickle Cell Disease
        • Anemia
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        • Droxia

          Droxia (hydroxyurea)

          Droxia (hydroxyurea) is an antimetabolite that slows down cell growth. In sickle cell diease, it is approved to reduce the number of painful crises and the need for blood transfusions.
          Manufacturer:
          Waylis Therapeutics
          Route of Administration: 
          Oral
          Site of Care:
          Outpatient
          Website: 
          • https://droxiarx.com
          phone: 
          888-218-8897
          Enrollment Forms:
          Synergen Prescription Form
          Patient Support Form

          Approved Indication:

          • reducing the frequency of painful crises and the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises

          Conditions:

          • Sickle Cell Disease
            less details
          • Endari

            Endari (L-glutamine)

            Endari (L-glutamine) is an amino acid supplement approved to reduce the complications of sickle cell disease.
            Manufacturer:
            Emmaus Medical, Inc.
            Route of Administration: 
            Oral
            Site of Care:
            Outpatient
            Website: 
            • https://www.endarirx.com
            phone: 
            1-855-723-5646
            Enrollment Forms:
            Synergen Prescription Form
            Patient Support Form

            Approved Indication:

            reduction of the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older

            Conditions:

            • Sickle Cell Disease
              less details
            • Exjade

              Exjade (deferasirox)

              Exjade (deferoxamine) is an iron-chelating agent given orally to lower iron levels in the blood. The tablets must be dissolved in water, orange juice, or apple juice before administration.
              Manufacturer:
              Novartis
              Route of Administration: 
              Oral
              Site of Care:
              Outpatient
              Website: 
                Enrollment Forms:
                Synergen Prescription Form

                Approved Indication:

                • treatment of chronic iron overload due to blood transfusions in patients 2 years of age and older
                • treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia syndromes and with a liver iron (Fe) concentration of at least 5mg Fe per gram of dry weight and a serum ferritin greater than 300mcg/L

                Conditions:

                • Sickle Celll Disease
                • Anemia
                • Non-Transfusion-Dependent Thalassemia

                  Therapeutic Area:

                  • Oncology
                  less details
                • Hydrea

                  Hydrea (hydroxyurea)

                  Hydrea (hydroxyurea) is an antimetabolite that slows down cell growth. In sickle cell diease, it is approved to reduce the number of painful crises and the need for blood transfusions.
                  Manufacturer:
                  Bristol Myers Squibb
                  Route of Administration: 
                  Oral
                  Site of Care:
                  Outpatient
                  Website: 
                    Enrollment Forms:
                    Synergen Prescription Form

                    Approved Indication:

                    • treatment of locally advanced squamous cell carcinomas of the head and neck, (excluding lip) in combination with concurrent chemoradiation

                    Conditions:

                    • Chronic Myeloid Leukemia
                    • Squamous Cell Carcinoma
                    • Sickle Cell Disease

                      Therapeutic Area:

                      • Oncology
                      less details
                    • Jadenu

                      Jadenu (deferasirox)

                      Jadenu (deferoxamine) is an iron-chelating agent given orally to lower iron levels in the blood. It is available as film-coated tablets or sprinkle granules.
                      Manufacturer:
                      Novartis
                      Route of Administration: 
                      Oral
                      Site of Care:
                      Outpatient
                      Website: 
                        Enrollment Forms:
                        Synergen Prescription Form

                        Approved Indication:

                        • treatment of chronic iron overload due to blood transfusions in patients 2 years of age and older
                        • treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia syndromes and with a liver iron (Fe) concentration of at least 5mg Fe per gram of dry weight and a serum ferritin greater than 300mcg/L

                        Conditions:

                        • Sickle Cell Disease
                        • Anemia
                        • Non-Transfusion-Dependent Thalassemia

                          Therapeutic Area:

                          • Oncology
                          less details
                        • Siklos

                          Siklos (hydroxyurea)

                          Siklos (hydroxyurea) is an antimetabolite that slows down cell growth. In sickle cell diease, it is approved to reduce the number of painful crises and the need for blood transfusions.
                          Manufacturer:
                          Medunik USA
                          Route of Administration: 
                          Oral
                          Site of Care:
                          Outpatient
                          Website: 
                          • https://siklosusa.com
                          phone: 
                          844-716-4663
                          Enrollment Forms:
                          Synergen Prescription Form
                          Patient Support Form

                          Approved Indication:

                          • reducing the frequency of painful crises and the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises

                          Conditions:

                          • Sickle Cell Disease
                            less details

                          Resources

                          Understanding a diagnosis can feel overwhelming. These resources link to well-established organizations, for in-depth education, research, and support.

                          CDC – Sickle Cell Disease
                          Sickle Cell Disease Association of America
                          Sickle Cell Foundation of Georgia

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