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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) (TransIT)
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.
Kenneth Desantes, M.D.
All
0 Years to 25 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT05600426
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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
Severe Aplastic Anemia, Other Hematopoietic, Hematologic cancers, other
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