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

Myelodysplastic Syndromes (MDS) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02390414
• Other study ID #: 11-056
• Status: Completed
• Start date: May 2011
• Est. completion date: September 2020

Study information

• Verified date: March 2023
• Source: Dana-Farber Cancer Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 290
• Est. completion date: September 2020
• Est. primary completion date: September 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 60 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Myelodysplastic Syndromes
• Myelodysplastic Syndromes (MDS)
• Preleukemia

Locations

Country	Name	City	State
United States	Beth Israel Deaconess Medical Center	Boston	Massachusetts
United States	Dana Farber Cancer Institute	Boston	Massachusetts
United States	Massachusetts General Hospital	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Dana-Farber Cancer Institute

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall Survival	To prospectively compare the overall survival of patients in the HSCT group to that of patients in the non-HSCT group.	3 Years

External Links

•   Fit older adults with advanced myelodysplastic syndromes: who is most likely to benefit from transplant?