Why is this medication prescribed?

Betibeglogene autotemcel injection is used to treat anemia (a lower than normal number of red blood cells) in people who require regular blood transfusions to treat beta thalassemia (Cooley’s anemia; an inherited condition that causes a low number of red blood cells). Betibeglogene autotemcel injection is in a class of medications called autologous gene therapy, a type of medication prepared using cells from the patient’s own blood stem cells. It works by helping your body to produce enough red blood cells so that regular blood transfusions are not necessary.

How should this medicine be used?

Betibeglogene autotemcel is administered as a one-time infusion of genetically modified hematopoietic stem cells. The process involves several steps:

• Hematopoietic Stem Cell Collection: Hematopoietic stem cells are collected from the patient through a process called apheresis. During apheresis, blood is drawn from the patient, and the stem cells are separated from the rest of the blood components using a machine. This step typically takes several hours and may require multiple sessions to collect a sufficient number of stem cells.
• Gene Modification: The collected stem cells are then sent to a laboratory, where they undergo genetic modification. This involves introducing a functional copy of the beta-globin gene into the stem cells using a viral vector. The modified stem cells are cultured and expanded to increase their numbers.
• Conditioning Regimen: Before the modified stem cells are infused back into the patient, they may undergo a conditioning regimen. This regimen typically involves chemotherapy or radiation therapy to make space in the bone marrow for the infused cells and to suppress the patient’s immune system, reducing the risk of rejection.
• Infusion of Modified Stem Cells: Once the conditioning regimen is complete, the modified stem cells are infused back into the patient’s bloodstream through a vein. The infusion process is similar to receiving a blood transfusion and usually takes several hours. After the infusion, the patient is monitored closely for any signs of complications.
• Follow-Up Care: After receiving Betibeglogene autotemcel, patients require regular follow-up care to monitor their progress and manage any potential side effects or complications. This may include blood tests to assess hemoglobin levels, monitoring for signs of graft-versus-host disease or other immune reactions, and providing supportive care as needed.

It’s important for patients to follow their healthcare provider’s instructions carefully throughout the treatment process and to attend all scheduled follow-up appointments. Additionally, patients should discuss any questions or concerns they have about Betibeglogene autotemcel with their healthcare provider.

Other uses for this medicine

Betibeglogene autotemcel is specifically approved for the treatment of transfusion-dependent β-thalassemia and is not indicated for other conditions. It targets the underlying genetic cause of β-thalassemia by increasing the production of fetal hemoglobin, which can reduce or eliminate the need for blood transfusions in patients with TDT.

What special precautions should I follow?

Regarding special precautions for Betibeglogene autotemcel Injection, here are some important points:

• Administration by Healthcare Professionals: Betibeglogene autotemcel Injection should only be administered by healthcare professionals who have experience in stem cell transplantation and gene therapy. The infusion process requires specialized training and expertise to ensure proper handling and administration of the genetically modified stem cells.
• Risk of Adverse Reactions: Like any medical procedure, Betibeglogene autotemcel Injection carries risks of adverse reactions. Patients should be monitored closely during and after the infusion for signs of potential complications, such as allergic reactions, infusion-related reactions, or graft-versus-host disease.
• Immunosuppressive Therapy: Depending on the patient’s condition and treatment protocol, they may receive immunosuppressive therapy before or after receiving Betibeglogene autotemcel Injection. This is typically done to prevent rejection of the infused stem cells and to reduce the risk of graft-versus-host disease. Patients should follow their healthcare provider’s instructions regarding immunosuppressive therapy and any associated precautions.
• Long-Term Monitoring: After receiving Betibeglogene autotemcel Injection, patients require long-term monitoring to assess the effectiveness of the treatment, monitor for any potential side effects or complications, and manage their ongoing care. Regular follow-up appointments with healthcare providers are essential for ensuring optimal outcomes.
• Genetic Counseling: Because Betibeglogene autotemcel Injection involves gene therapy, patients and their families should receive genetic counseling to understand the implications of the treatment, including potential hereditary factors and the risks and benefits associated with genetic modification.
• Pregnancy and Breastfeeding: The safety and efficacy of Betibeglogene autotemcel Injection during pregnancy and breastfeeding have not been established. Pregnant or breastfeeding individuals should discuss the risks and benefits of the treatment with their healthcare provider before proceeding.

Patients should discuss any questions or concerns about Betibeglogene autotemcel Injection with their healthcare provider before undergoing treatment. It’s essential to understand the potential risks and benefits of the therapy and to receive personalized guidance based on individual medical history and circumstances.

What special dietary instructions should I follow?

Regarding special dietary instructions, there are no specific dietary restrictions associated with Betibeglogene autotemcel Injection. However, maintaining a healthy and balanced diet is important for overall health and well-being, especially during the recovery period following the infusion. Follow any dietary recommendations provided by your healthcare provider, which may include guidelines for managing thalassemia-related symptoms or supporting the recovery process.

What should I do if I forget a dose?

If you forget a dose of Betibeglogene autotemcel Injection, it’s essential to consult with your healthcare provider. However, since Betibeglogene autotemcel is administered as a one-time infusion rather than a medication taken on a regular schedule, the concept of “forgetting a dose” doesn’t apply in the same way as with traditional medications. If you have any concerns or questions about your treatment, including the infusion process or post-treatment care, don’t hesitate to reach out to your healthcare provider for guidance and clarification.

What side effects can this medication cause?

Betibeglogene autotemcel (Zynteglo) is a gene therapy used to treat transfusion-dependent beta-thalassemia, and like any medical intervention, it carries potential risks of side effects. Common side effects associated with Betibeglogene autotemcel Injection include:

• Infusion-Related Reactions: Patients may experience infusion-related reactions during or shortly after receiving the injection. Symptoms may include fever, chills, nausea, vomiting, headache, fatigue, and allergic reactions such as rash or itching.
• Graft-Versus-Host Disease (GVHD): In some cases, the infused stem cells may recognize the recipient’s body tissues as foreign and attack them, leading to graft-versus-host disease. GVHD can cause skin rash, liver dysfunction, gastrointestinal symptoms, and other complications.
• Cytokine Release Syndrome (CRS): CRS is a systemic inflammatory response that can occur following the infusion of genetically modified cells. Symptoms may include fever, fatigue, muscle pain, headache, and in severe cases, respiratory distress or organ dysfunction.
• Infections: Patients may be at increased risk of infections following Betibeglogene autotemcel Injection, particularly if they are receiving immunosuppressive therapy as part of their treatment regimen.
• Hematologic Abnormalities: Changes in blood cell counts, including decreases in red blood cells, white blood cells, and platelets, may occur following treatment. These changes may require supportive care or medical intervention.
• Other Adverse Events: Additional side effects reported in clinical trials of Betibeglogene autotemcel Injection include abdominal pain, diarrhea, peripheral edema, respiratory tract infections, and laboratory abnormalities.

It’s important to note that the safety profile of Betibeglogene autotemcel continues to be monitored as more data is collected from ongoing clinical trials and post-marketing surveillance. Patients should discuss any concerns about potential side effects with their healthcare provider before undergoing treatment, and they should report any adverse reactions or symptoms experienced during or after the infusion. Prompt communication with healthcare providers can help ensure that any side effects are addressed promptly and appropriately.

What should I know about storage and disposal of this medication?

Storage and disposal of Betibeglogene autotemcel Injection:

• Storage: Betibeglogene autotemcel Injection should be stored as directed by the healthcare provider or healthcare facility administering the treatment. It typically requires specialized storage conditions to maintain stability and integrity. Follow the storage instructions provided with the medication or as directed by your healthcare provider.
• Disposal: Because Betibeglogene autotemcel Injection is administered as a one-time infusion in a healthcare setting, disposal is typically managed by the healthcare facility or provider. Any unused or expired medication should be disposed of according to local regulations for the disposal of biological materials or medical waste.

In case of emergency/overdose

• In case of an emergency or overdose, immediate medical attention is crucial.
• Contact your healthcare provider, emergency medical services, or go to the nearest hospital.
• Be prepared to provide information about the patient’s medical history, including any known allergies or previous reactions to medications.
• Healthcare professionals will assess the situation and provide appropriate treatment based on the symptoms and severity of the overdose.

What other information should I know?

• Betibeglogene autotemcel is administered through a process called autologous hematopoietic stem cell transplantation (HSCT), where the patient’s own stem cells are genetically modified and then infused back into the patient.
• This treatment is specifically indicated for patients with transfusion-dependent β-thalassemia who do not have a β0/β0 genotype and for whom HSCT is appropriate but a human leukocyte antigen (HLA)-matched related HSCT donor is not available.
• Prior to treatment, patients should undergo a comprehensive evaluation by healthcare professionals to assess their eligibility and suitability for Betibeglogene autotemcel therapy.
• Patients should be informed about the potential risks and benefits of treatment, as well as the need for long-term monitoring and follow-up care.

Always consult with healthcare professionals for specific guidance on the storage, administration, and handling of Betibeglogene autotemcel Injection, as well as in the event of any emergency or overdose situation.