Information for caregivers, including Tips for Effective Conversations with Healthcare Providers and a Step-by-Step Guide for Performing Intramuscular Injections, and Questions to Ask Your Healthcare Provider
Hear about Ashley's Revcovi® treatment journey and her experience living with ADA-SCID
LIVING WITH ADA-SCID
Living with ADA-SCID can be challenging for both the patient and the caregiver. However, with education and planning, you and someone affected by ADA-SCID can begin treating the disease.
The goal of treatment for ADA-SCID—as well as other types of SCID—is to strengthen the immune system. This requires restoring the lymphocyte numbers and function that was lost as a result of ADA deficiency.1 Take comfort in knowing there are multiple treatment options available and choosing the right one is an important part of the process. Discuss treatment options with your doctor.
Currently, there are three different types of specific, acceptable treatment options available for ADA-SCID. All patients with ADA-SCID will require life-long management, monitoring, and regular follow-up.
REVCOVI PATIENT SUPPORT
Important information for reimbursement support and patient support services.
ADDITIONAL PATIENT RESOURCES
Chiesi Global Rare Diseases is dedicated to doing more for patients. Get additional support and resources for those living with ADA-SCID.
ADVOCACY AND SUPPORT ORGANIZATIONS
The following organizations offer support and services for ADA-SCID patients and their families:
The Immune Deficiency Foundation improves the diagnosis, treatment, and quality of life of people affected by primary immunodeficiency through fostering a community empowered by advocacy, education, and research.
SCID ANGELS FOR LIFE FOUNDATION
SCID Angels for Life is a not-for-profit public charity committed to increasing awareness, providing a safe environment for families to connect, granting family scholarships, and providing parent and family education for people affected by Severe Combined Immune Deficiency.
Global Genes is a leading rare disease patient advocacy organization that includes over 750 global organizations. Global Genes builds awareness, educates the community, and provides critical connections and resources to rare disease advocates worldwide.
The Jeffrey Modell Foundation (JMF) is a global organization dedicated to early and precise diagnosis, meaningful treatments, and ultimately, cures for primary immunodeficiencies through clinical and basic research, physician education, patient support, advocacy, public awareness, newborn screening and genetic sequencing.
NATIONAL ORGANIZATION FOR RARE DISORDERS
The National Organization for Rare Disorders (NORD) is the leading patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
World PI Week provides the opportunity to inform and educate health policy-makers, schools and families, and the general public about primary immunodeficiencies (PI) to drive the earliest possible diagnosis and optimal treatment.
Important Safety Information
Warnings and Precautions:
- If you have lower than normal platelet counts, you may be at increased risk of bleeding at the injection site. Your doctor should not prescribe Revcovi if you have severely low blood platelet counts.
- There may be a delay in improvement of your immune system’s function after starting Revcovi treatment. During this delay, it is important to be protected from exposure to infections until your immune system improves.
What are the possible side effects of Revcovi?
- Cough and vomiting were the most common side effects reported by patients receiving Revcovi.
- Based on reports of side effects of other enzyme replacement therapies used to treat ADA-SCID, which are similar to Revcovi, patients taking Revcovi may also experience:
- Blood and blood cell–related problems: red blood cells being destroyed faster than they can be made, resulting in anemia; and higher or lower than normal number of platelets in the blood
- Skin-related problems: redness and itching at the injection site
- Cancers of the immune system called lymphomas
What types of monitoring will be needed while I am taking Revcovi?
- Continuous therapy and adherence to the recommended drug schedule is important for the success of the treatment.
- While you are being treated with Revcovi, your doctor will need to do blood tests to monitor the levels of ADA activity and metabolites and overall immune function.
- This monitoring will ensure you are maintaining appropriate levels for therapeutic benefit. If an ongoing decline in your blood’s measured adenosine deaminase (ADA) activity occurs, your immune function and clinical status will be monitored closely and precautions will be taken to help reduce your risk of infection.
What is known about taking Revcovi during pregnancy?
- If you are pregnant or planning to become pregnant, or breastfeeding or planning to breastfeed, please talk to your doctor.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
Indication
Revcovi® (elapegademase-lvlr) is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
Please see the Full Prescribing Information.
Important Safety Information
Warnings and Precautions:
- If you have lower than normal platelet counts, you may be at increased risk of bleeding at the injection site. Your doctor should not prescribe Revcovi if you have severely low blood platelet counts.
- There may be a delay in improvement of your immune system’s function after starting Revcovi treatment. During this delay, it is important to be protected from exposure to infections until your immune system improves.
What are the possible side effects of Revcovi?
- Cough and vomiting were the most common side effects reported by patients receiving Revcovi.
- Based on reports of side effects of other enzyme replacement therapies used to treat ADA-SCID, which are similar to Revcovi, patients taking Revcovi may also experience:
- Blood and blood cell–related problems: red blood cells being destroyed faster than they can be made, resulting in anemia; and higher or lower than normal number of platelets in the blood
- Skin-related problems: redness and itching at the injection site
- Cancers of the immune system called lymphomas
What types of monitoring will be needed while I am taking Revcovi?
- Continuous therapy and adherence to the recommended drug schedule is important for the success of the treatment.
- While you are being treated with Revcovi, your doctor will need to do blood tests to monitor the levels of ADA activity and metabolites and overall immune function.
- This monitoring will ensure you are maintaining appropriate levels for therapeutic benefit. If an ongoing decline in your blood’s measured adenosine deaminase (ADA) activity occurs, your immune function and clinical status will be monitored closely and precautions will be taken to help reduce your risk of infection.
What is known about taking Revcovi during pregnancy?
- If you are pregnant or planning to become pregnant, or breastfeeding or planning to breastfeed, please talk to your doctor.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
Indication
Revcovi® (elapegademase-lvlr) is indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients.
Please see the Full Prescribing Information.
References available upon request.
ADA-SCID Patient & Family Resources