Overview
A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases.
Description
This is a prospective, multicenter, study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases. The enrollment goal is 375 participants and up to all consenting 100 related donors (approximately 100 are anticipated), accrued over 4 years. Participants will be followed until the end of study, but continued collection of specimens and clinical data may continue to allow up to 15 years of follow-up for all enrolled participants if funds are available. Participants with a diagnosis of Aplastic Anemia or Hemoglobinopathies or bone marrow failure undergoing HCT or GT for management of their underlying disease. There is no treatment intervention in this study. Treatment will be at the provider's discretion. Participants will provide blood and other biospecimens pre and post-intervention at specified intervals. 375 participants and approximately 100 related donors will be enrolled from 35 sites.
Eligibility
HCT/GT Inclusion:
- Patients with a diagnosis of Aplastic Anemia (AA), hemoglobinopathies or bone marrow failure from other causes except for malignant diseases will be eligible for enrollment on this protocol:
- AA will be defined as having peripheral blood cytopenias with a hypocellular bone marrow for age and a clinical diagnosis of aplastic anemia as determined by their treating physicians.
- Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study.
- Individuals with bone marrow failure due to clinical or molecularly diagnosed inherited bone marrow failure, inborn errors of immunity or other cause will be included.
- Patients must receive an HCT or GT for management of their underlying disease. Allogeneic transplants including all conditioning regimens, donors, and GVHD prophylaxis regimens are eligible. This study does not define how the transplant or transplant-supportive care will be performed.
- Patients or their legal guardian must consent to participate in the CIBMTR "Protocol for a Research Database for Hematopoietic Cell Transplantation and Marrow Toxic Injuries" (NCT 1166009) to allow linkage with the longitudinal clinical data collected by CIBMTR.
- All ages minorities, sexes and genders are eligible for the study, but participants must weigh at least 10 kilograms (kg) at the time of study enrollment given the volume and number of blood draws required.
- All participants or parent/legal guardian must sign an informed consent for this study. If there are questions regarding a patient's eligibility for the study, contact the Protocol Team for review and discussion by emailing bmtctn2402@emmes.com.
HCT/GT Exclusion
- Patients with aplastic anemia or hemoglobinopathies who are not pursuing allogeneic HCT or GT.
- Active malignancy.
- Hematologic malignancy or therapy for a prior hematologic malignancy in the previous five 5 years.
- Weight ≤ 10.0 kg at time of study enrollment.
- Prior autologous or allogeneic transplant.
Related Donor Inclusion:
1\. All related donors for eligible recipients undergoing allogeneic HCT for AA, hemoglobinopathies, or bone marrow failure as defined in the recipient eligibility criteria above are eligible. Note: HCT recipient participants will remain eligible if the related donor declines to participate in the study.
Related Donor Exclusion:
1\. Donor weight ≤ 10.0 kg at time of study enrollment
