Stem Cell Transplant for Primary Immunodeficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
This is a standard of care treatment guideline for allogeneic hematopoetic stem cell transplant (HSCT) in patients with primary immune deficiencies.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is stem cell transplant generally safe for treating primary immunodeficiency?
Stem cell transplants, specifically allogeneic hematopoietic stem cell transplants, have been shown to be generally safe and effective for treating primary immunodeficiency in both children and adults. While there are risks such as graft-versus-host disease (a condition where the donor cells attack the recipient's body) and graft failure, survival rates are high, and safety has improved over the years with advancements in treatment protocols.12345
How is the treatment Allogeneic Hematopoietic Stem Cell Transplant unique for primary immunodeficiency?
Allogeneic Hematopoietic Stem Cell Transplant is unique because it can potentially cure primary immunodeficiencies by replacing the patient's faulty immune system with healthy stem cells from a donor. This treatment is especially important when no matched donor is available, as it allows for the use of partially matched (haploidentical) donors, expanding the pool of potential donors.16789
What data supports the effectiveness of the treatment Allogeneic Hematopoietic Stem Cell Transplant for Primary Immunodeficiency?
Research shows that allogeneic hematopoietic stem cell transplant (a procedure where healthy stem cells are transferred from a donor to a patient) can cure many primary immunodeficiency disorders, with an 80% success rate when using a matched sibling donor. This treatment has significantly improved the prognosis for patients with these conditions.110111213
Who Is on the Research Team?
Christen Ebens, MD
Principal Investigator
Masonic Cancer Center, University of Minnesota
Are You a Good Fit for This Trial?
This trial is for people aged 0-50 with primary immune deficiencies or histiocytic disorders, such as SCID, Omenn's Syndrome, Wiskott-Aldrich syndrome. Participants need a suitable stem cell donor and good organ function. Those with acute hepatitis, severe liver issues, active infections including HIV, or who are pregnant/breastfeeding cannot join.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Preparative Regimen
Participants receive a preparative regimen based on their condition, which may include myeloablative, reduced toxicity, or reduced intensity conditioning, followed by stem cell infusion on day 0.
Engraftment Monitoring
Participants are monitored for neutrophil engraftment, defined as the first day of three consecutive days where the neutrophil count is 500 cells/mm3 or greater.
Follow-up
Participants are monitored for safety and effectiveness, including incidence of acute and chronic Graft-Versus-Host Disease, overall survival, and transplant-related mortality.
Long-term Follow-up
Participants are monitored for long-term outcomes such as disease-free survival and incidence of chronic Graft-Versus-Host Disease.
What Are the Treatments Tested in This Trial?
Interventions
- Allogeneic Hematopoetic Stem Cell Transplant
Allogeneic Hematopoetic Stem Cell Transplant is already approved in European Union, United States, Canada, Japan, China, Switzerland for the following indications:
Find a Clinic Near You
Who Is Running the Clinical Trial?
Masonic Cancer Center, University of Minnesota
Lead Sponsor