The Myelodysplasia Transplantation-Associated Outcomes (MDS-TAO) Study



Status:Active, not recruiting
Conditions:Blood Cancer, Blood Cancer
Therapuetic Areas:Oncology
Healthy:No
Age Range:60 - 75
Updated:10/6/2018
Start Date:May 2011
End Date:May 2023

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The purpose of this observational study is to compare overall survival in older adults with
myelodysplastic syndromes (MDS) who receive reduced intensity conditioning hematopoietic stem
cell transplant (RIC HSCT) versus those who do not receive HSCT.

MDS is a hematologic malignancy characterized by cytopenias, bone marrow failure, and a risk
of transformation to acute myeloid leukemia (AML). HSCT is the only curative therapy for MDS.
Despite its increasing use among older patients (age greater than 60), more data are needed
to assess outcomes of HSCT in older adults compared to other therapies.

In this observational study, patients with MDS presenting at the study institutions are
screened for disease characteristics that indicate that they are potentially appropriate for
HSCT (both high-risk disease and fit for the procedure). Patients who meet inclusion and
exclusion criteria and agree to participate in the study are entered into a clinical database
and followed for overall survival. Patients also complete quality of life (QoL) assessments
at enrollment and two years afterward, with the goal of investigating potential relationships
between QoL and MDS treatment (HSCT vs. non-HSCT strategies).

Inclusion Criteria:

- Histologically-confirmed diagnosis of:

- Primary or secondary MDS using the World Health Organization (WHO) 2008
classification:

- Refractory cytopenia with unilineage dysplasia

- Refractory Anemia (RA)

- Refractory Neutropenia (RN)

- Refractory Thrombocytopenia (RT)

- Refractory Anemia with Ring Sideroblasts (RARS)

- Refractory Cytopenia with Multilineage Dysplasia (RCMD)

- Refractory Anemia with Excess Blasts-1 (RAEB-1)

- Refractory Anemia with Excess Blasts-2 (RAEB-2)

- MDS with isolated del (5q)

- MDS-Unclassified (MDS-U)

- Another of the following related disorders:

- Chronic Myelomonocytic leukemia (CMML)

- Myelodysplastic/myeloproliferative neoplasm, unclassifiable (MDS/MPD-U)

- Age 60 to 75 years

- Any of the following (high-risk characteristics):

- Intermediate-2 or High-Risk on International Prognostic Scoring System (IPSS)

- Secondary MDS (any karyotype)

- Documented non-IPSS intermediate- or poor-prognosis karyotype including:

- +8

- t(11q23)

- Rea 3q

- +19

- 3 or greater abnormalities

- del(7q)

- -5

- t(5q)

- Documented significant cytopenia for at least four months prior to enrollment,
defined by the following criteria:

- Red Blood Cell (RBC) Transfusion Dependence: four or more units of RBC
transfusions within an eight-week period for symptomatic anemia with
hemoglobin of ≤ 9.0 g/dL; OR

- Severe Anemia: average of two or more hemoglobin values ≤ 8 g/dL within an
eight-week period not influenced by RBC transfusions (i.e., must be seven
days post transfusion); OR

- Severe Thrombocytopenia: average of two or more platelet counts ≤ 50

× 109/L within an eight-week period not influenced by platelet transfusions
(i.e., must be at least three days post- transfusion) or a clinically
significant hemorrhage requiring platelet transfusions within the prior four
months; OR

- Severe Neutropenia: average of two or more absolute neutrophil counts (ANC)
≤ 500 within an eight-week period, or a clinically significant infection
requiring IV antibiotics in the setting of ANC ≤ 1000 within the prior four
months.

- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.

- Adequate organ function to permit RIC HSCT as indicated by the following:

- Serum bilirubin ≤ 2.5 mg/dL (except when Gilbert's syndrome or MDS-related
hemolysis suspected).

- Aspartate aminotransferase (AST) and Alanine Aminotransferase (ALT) ≤ 2.5 times
the upper limit of normal (ULN).

- Serum creatinine ≤ 2.0 mg/dL.

- Seemingly sufficient baseline cardiac function to undergo HSCT (no echocardiogram
required).

- Seemingly sufficient baseline pulmonary function to undergo HSCT (no pulmonary
function tests required).

- Seemingly sufficient neuro-psychiatric function to undergo HSCT (no specific
neuro-psychiatric evaluation required).

- Willingness to undergo human leukocyte antigen (HLA)-typing and consider subsequent
HSCT.

- Willingness and ability to give informed consent.

Exclusion Criteria:

- Known baseline conversion to AML (eg, ≥ 20% peripheral or marrow blasts).

- Knowledge of potential donor status at study entry. Of note, knowledge of HLA status
WITHOUT a related or unrelated search is allowed.

- History of prior malignancy within the past year, except for adequately-treated
carcinoma in situ of uterine cervix, basal cell or squamous cell skin cancer.

- Any severe concurrent disease, infection, or comorbidity that, in the judgment of the
principal investigator, would make the patient inappropriate for HSCT at the time of
study entry.

- Psychiatric disorders including dementia that would preclude obtaining informed
consent or the ability to participate in an ongoing research study.
We found this trial at
3
sites
330 Brookline Ave
Boston, Massachusetts 02215
617-667-7000
Principal Investigator: David Avigan, MD
Phone: 617-667-9920
Beth Israel Deaconess Medical Center Beth Israel Deaconess Medical Center (BIDMC) is one of the...
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185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
Principal Investigator: Yi-Bin Chen, MD
Phone: 617-726-1124
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450 Brookline Ave
Boston, Massachusetts 2215
617-632-3000
Principal Investigator: Gregory A. Abel, MD, MPH
Phone: 617-632-2304
Dana-Farber Cancer Institute Since it’s founding in 1947, Dana-Farber has been committed to providing adults...
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