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A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)
Trial Status: closed to accrual
Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough
new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow
transplant. Regular treatment for patients with aplastic anemia who have a matched
sibling (brother or sister), or family donor is a bone marrow transplant. Patients
without a matched family donor normally are treated with immune suppressive therapy
(IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary
treatment in patients who did not get better with IST, had their disease come back, or a
new worse disease replaced it (like leukemia).
This trial will compare time from randomization to failure of treatment or death from any
cause of IST versus URD BMT when used as initial therapy to treat SAA.
The trial will also assess whether health-related quality of life and early markers of
fertility differ between those randomized to URD BMT or IST, as well as assess the
presence of marrow failure-related genes and presence of gene mutations associated with
MDS or leukemia and the change in gene signatures after treatment in both study arms.
This study treatment does not include any investigational drugs. The medicines and
procedures in this study are standard for treatment of SAA.
Inclusion Criteria
Inclusion Criteria:
To be eligible to participate in the randomized trial, an individual must meet all the
following criteria:
1. Provision of signed and dated informed consent form for the randomized trial by
patient and/or legal guardian.
2. Age ≤25 years old at time of randomized trial consent.
3. Confirmed diagnosis of idiopathic SAA, defined as:
1. Bone marrow cellularity <25%, or <30% hematopoietic cells.
2. Two of three of the following (in peripheral blood): neutrophils <0.5 x 10^9/L,
platelets <20 x 10^9/L, absolute reticulocyte count <60 x 10^9/L or hemoglobin
<8 g/dL.
4. No suitable fully matched related donor available (minimum 6/6 match for HLA-A and B
at intermediate or high resolution and DRB1 at high resolution using DNA based
typing).
5. At least 2 unrelated donors noted on NMDP search who are well matched (9/10 or 10/10
for HLA-A, B, C, DRB1, and DQB1 using high resolution).
6. In the treating physician's opinion, no obvious contraindications precluding them
from BMT or IST.
Exclusion Criteria:
1. Presence of Inherited bone marrow failure syndromes (IBMFS). The diagnosis of
Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing on
peripheral blood or marrow. Telomere length testing should be sent on all patients
to exclude Dyskeratosis Congenita (DC), but if results are delayed or unavailable
and there are no clinical manifestations of DC, patients may enroll. If patients
have clinical characteristics suspicious for Shwachman-Diamond syndrome, this
disorder should be excluded by pancreatic isoamylase testing or gene mutation
analysis (note: pancreatic isoamylase testing is not useful in children <3). Other
testing per center may be performed to exclude IBMFS.
2. Clonal cytogenetic abnormalities or Fluorescence In-Situ Hybridization (FISH)
pattern consistent with pre- myelodysplastic syndrome (pre-MDS) or MDS on marrow
examination.
3. Known severe allergy to ATG.
4. Prior allogeneic or autologous stem cell transplant.
5. Prior solid organ transplant.
6. Infection with human immunodeficiency virus (HIV).
7. Active Hepatitis B or C. This only needs to be excluded in patients where there is
clinical suspicion of hepatitis (e.g., elevated LFTs).
8. Female patients who are pregnant or breast-feeding.
9. Prior malignancies except resected basal cell carcinoma or treated cervical
carcinoma in situ.
10. Disease modifying treatment prior to study enrollment, including but not limited to
use of androgens, eltrombopag, romiplostim, or immune suppression. Note: Supportive
care measures such as G-CSF, blood transfusion support and antibiotics are allowable
Additional locations may be listed on ClinicalTrials.gov for NCT05600426.
Locations matching your search criteria
United States
California
San Diego
Rady Children's Hospital - San Diego
Status: Active
Name Not Available
Florida
Gainesville
University of Florida Health Science Center - Gainesville
