What is the management of sickle cell anemia?

From the Guidelines

Management of sickle cell anemia requires a comprehensive approach including preventive care, pain management, and disease-modifying treatments, with hematopoietic stem cell transplantation being the only curative option for eligible patients, as recommended by the American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation 1.

Overview of Sickle Cell Anemia Management

The management of sickle cell anemia involves several key components, including:

• Preventive care to reduce the risk of complications
• Pain management to control acute and chronic pain
• Disease-modifying treatments to reduce the frequency and severity of sickle cell crises
• Regular screenings for complications such as retinopathy, nephropathy, and pulmonary hypertension

Disease-Modifying Treatments

Disease-modifying treatments for sickle cell anemia include:

• Hydroxyurea, typically started at 15-20 mg/kg/day and titrated based on response, which increases fetal hemoglobin production and reduces painful crises
• L-glutamine, 10-30 g daily
• Crizanlizumab, 5 mg/kg IV monthly
• Voxelotor, 1500 mg daily

Acute Pain Crises Management

For acute pain crises, treatment involves:

• Aggressive hydration with IV fluids
• Oxygen therapy if oxygen saturation is below 95%
• Pain control with NSAIDs and opioids as needed

Blood Transfusions

Blood transfusions are used for:

• Severe anemia
• Acute chest syndrome
• Stroke
• Before surgeries Exchange transfusions are preferred for acute complications to prevent iron overload.

Infection Prevention

Infection prevention is crucial, including:

• Pneumococcal, meningococcal, and annual influenza vaccines
• Prophylactic penicillin, 125-250 mg twice daily, for children under 5

Hematopoietic Stem Cell Transplantation

Hematopoietic stem cell transplantation remains the only curative option for eligible patients, as recommended by the American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation 1, and gene therapy shows promising results in clinical trials.

Peri-Operative Management

For patients with sickle cell disease undergoing surgery, careful planning, assessment of comorbidities, and multidisciplinary team working are essential to mitigate peri-operative complications, as outlined in the guideline on the peri-operative management of patients with sickle cell disease: guideline from the association of anaesthetists 1.

From the FDA Drug Label

L-glutamine is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older. The management of sickle cell anemia includes the use of L-glutamine to reduce the acute complications of the disease.

• The recommended dosage is based on body weight, with patients receiving 5-15 grams of L-glutamine orally, twice per day.
• Treatment with L-glutamine has been shown to reduce the number of sickle cell crises, hospitalizations due to sickle cell pain, and cumulative days in hospital.
• L-glutamine has also been found to lower the incidence of acute chest syndrome. The safety and effectiveness of L-glutamine have been established in pediatric patients 5 years and older 2, 2.

From the Research

Sickle Cell Anemia Pathophysiology and Management

• Sickle cell disease (SCD) is a hereditary blood disorder characterized by the production of abnormal hemoglobin molecules that cause red blood cells to take on a crescent or sickle shape 3.
• The primary cause of SCD is a mutation in the gene responsible for producing hemoglobin, leading to abnormal hemoglobin called hemoglobin S, which causes red blood cells to become stiff and sticky, resulting in various health complications 3.
• Patients with SCD may experience recurrent pain, fatigue, anemia, and increased infection susceptibility 3.

Management of Sickle Cell Anemia

• Treatment options for SCD focus on managing symptoms and preventing complications, including pain management with analgesics, hydration, and blood transfusions to improve oxygen delivery 3.
• Hydroxyurea, a medication that increases the production of fetal hemoglobin, is commonly used to reduce the frequency and severity of pain crises 3, 4, 5, 6.
• Bone marrow or stem cell transplants can cure select individuals with severe SCD 3, 4.
• Other treatment options include l-glutamine, which has been shown to reduce the incidence of pain crises among patients with sickle cell anemia or sickle β0-thalassemia 7.
• Current and future treatments for SCD also include hematopoietic stem cell transplantation and in vivo gene therapy, which aim to provide a more accessible and cost-effective solution for curing SCD worldwide 4.

Treatment Benefits and Safety

• Hydroxyurea has been shown to be a safe and effective therapy for SCA, with benefits including reduced mortality, fewer pain crises, and improved quality of life 5, 6.
• L-glutamine has been shown to be effective in reducing the incidence of pain crises and hospitalizations among patients with sickle cell anemia or sickle β0-thalassemia 7.
• The safety profile of hydroxyurea is acceptable, with minimal genotoxicity or carcinogenicity with long-term exposure 5.