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Vicki and Fred Modell established the Jeffrey Modell Foundation in 1987 in memory of their son Jeffrey, who died at the age of fifteen from complications of Primary Immunodeficiency. JMF is a global patient organization devoted to early and precise diagnosis, meaningful treatments, and ultimately, cures - through clinical and basic research, physician education, patient support, advocacy, public awareness, newborn screening and genetic sequencing.Visit Site
Globla Genes is dedicated to eliminating the burdens and challenges of rare diseases for patients and families. A globally connected community equipped to eliminate the challenges of rare disease, united by a determination to support and provide what they need to take action and thriveVisit Site
Whether you've been recently diagnosed, have been living with a PI for years, or just think you might have a PI, the Immune Deficiency Foundation (IDF) is here to help. With our support, achieve an early and accurate diagnosis, appropriate treatment, and improved quality of life. IDF programs are meant to connect, engage, and empower families to live longer, stronger, healthier lives.Visit Site
NORD (National Organization for Rare Disorders), is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD, along with its more then 300 patient organization members, is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.Visit Site
Immune reconstitution sufficient to protect from infection is unlikely to develop prior to 6-12 months after treatment with RETHYMIC. Given the immunocompromised condition of athymic patients, follow infection control measures until the development of thymic function is established as measured through flow cytometry. Monitor patients closely for signs of infection including fever. If a fever develops, assess the patient by blood and other cultures and treat with antimicrobials as clinically indicated. Patients should be maintained on immunoglobulin replacement therapy until specified criteria are met, and two months after stopping, IgG trough level should be checked. Prior to and after treatment with RETHYMIC, patients should be maintained on Pneumocystis jiroveci pneumonia prophylaxis until specified criteria are met.
RETHYMIC may cause or exacerbate pre-existing graft versus host disease (GVHD). Monitor and treat patients at risk for the development of GVHD. Risk factors for GVHD include atypical complete DiGeorge anomaly phenotype, prior hematopoietic cell transplantation (HCT) and maternal engraftment. GVHD may manifest as fever, rash, lymphadenopathy, elevated bilirubin and liver enzymes, enteritis, and/or diarrhea.
Autoimmune-related adverse events occurred in patients treated with RETHYMIC. These events included: thrombocytopenia, neutropenia, proteinuria, hemolytic anemia, alopecia, hypothyroidism, autoimmune hepatitis, autoimmune arthritis, transverse myelitis, albinism, hyperthyroidism, and ovarian failure. Monitor for the development of autoimmune disorders, including complete blood counts with differential, liver enzymes, serum creatinine, urinalysis, and thyroid function.
Pre-existing renal impairment is a risk factor for death.
In the clinical studies of RETHYMIC, 3 out of 4 patients with pre-existing cytomegalovirus infection died. The benefits/risks of treatment should be considered prior to treating patients with pre-existing CMV infection.
Because of the underlying immune deficiency, patients who receive RETHYMIC may be at risk of developing post-treatment lymphoproliferative disorder. Patients should be monitored for the development of lymphoproliferative disorder.
Transmission of infectious disease may occur because RETHYMIC is derived from human tissue and because product manufacturing includes porcine- and bovine-derived reagents.
Immunizations should not be administered in patients who have received RETHYMIC until immune-function criteria have been met.
All patients should be screened for anti-HLA antibodies prior to receiving RETHYMIC. Patients testing positive for anti-HLA antibodies should receive RETHYMIC from a donor who does not express those HLA alleles. HLA matching is required in patients who have received a prior HCT or a solid organ transplant. Patients who have received a prior HCT are at increased risk of developing GVHD after RETHYMIC if the HCT donor did not fully match the recipient.
Of the 105 patients in clinical studies, 29 patients died, including 23 deaths in the first year (< 365 days) after implantation.
The most common (>10%) adverse events related to RETHYMIC included: hypertension, cytokine release syndrome, rash, hypomagnesemia, renal impairment/failure, thrombocytopenia, and graft versus host disease.
To report suspected adverse reactions, please contact the FDA at 1-800-FDA-1088 or www.fda.gov/safety/medwatch
RETHYMIC® (allogeneic processed thymus tissue–agdc) is indicated for immune reconstitution in pediatric patients with congenital athymia. RETHYMIC is not indicated for the treatment of patients with severe combined immunodeficiency (SCID).
1. Collins C, Sharpe E, Silber A, Kulke S, Hsieh EWY. Congenital athymia: genetic etiologies, clinical manifestations, diagnosis, and treatment. J Clin Immunol. 2021;41(5):881-895. doi.org/10.1007/s10875-021-01059-7
2. Data on file, Enzyvant.
3. Markert ML, Gupton SE, McCarthy EA. Experience with cultured thymus tissue in 105 children. J Allergy Clin Immunol. Published online August 3, 2021. doi:10.1016/j.jaci.2021.06.028
4. Hsieh EWY, Kim-Chang JJ, Kulke S, Silber A, O’Hara M, Collins C. Defining the clinical, emotional, social, and financial burden of congenital athymia. Adv Ther. 2021;38(8):4271-4288. doi.org/10.1007/s12325-021-01820-9
5. RETHYMIC [package insert]. Cambridge, MA: Enzyvant Therapeutics, Inc; 2021.
6. Markert ML. Defects in thymic development. In: Sullivan KE, Stiehm ER, eds. Stiehm’s Immune Deficiencies. 2nd ed. New York, NY: Elsevier; 2020:357-379.
7. Gupton SE, McCarthy EA, Markert ML. Care of children with DiGeorge before and after cultured thymus tissue implantation. J Clin Immunol. 2021;41(5):896-905. doi.org/10.1007/s10875-021-01044-0
8. Markert ML, Devlin BH, Alexieff MJ, et al. Review of 54 patients with complete DiGeorge anomaly enrolled in protocols for thymus transplantation: outcome of 44 consecutive transplants. Blood. 2007;109(10):4539-4547.
9. Markert ML, McCarthy EA, Gupton SE, Lim AP. Cultured thymus tissue transplantation. In: Sullivan KE, Stiehm ER, eds. Stiehm's Immune Deficiencies. 2nd ed.: Academic Press; 2020:1229-1239.