Clinical Trials

Title: Interventional Validation of an MDS-Specific Measure of Quality of Life: Assessing the Responsiveness of the QUALMS-1 to Different Hypomethylating Agent Regimens for Low and Intermediate Risk Disease (Companion protocol to MD Anderson Cancer Center Study 2014-0112)

Principal Investigtaor: Dr. Gail Roboz
Research Nurse: Yulia Dault, RN
Phone: (212) 746-4829
Sponsor: Dana Farber Cancer Center

Although disease-specific quality of life (QoL) measures allow for more relevant, sensitive, responsive and complete assessments of the impact of interventions in both clinical and research settings, increasingly important for patients with the myelodysplastic syndrome (MDS) given the development of new therapies as well as continued controversy surrounding the utility of hematopoietic growth factors, no disease-specific QoL measures have been developed, validated and widely-adopted for patients with this bone marrow failure syndrome. We wish to engage the MDS Clinical Research Consortium (MDS CRC) in validating a new 38-item myelodysplastic syndrome specific QoL measure that we have recently developed through a grant funded by the AAMDS.

With the structured input of 32 myelodysplastic syndrome patients, caregivers, providers and QoL experts, we developed the “Quality of Life in Myelodysplasia Scale” or “QUALMS-1” and then subjected it to rigorous piloting (cognitive debriefing and behavioral coding) with an additional cohort of myelodysplastic syndrome patients (n=20).

We now wish to administer this scale and another standard scale (the Anemia Subset of FACT, FACT-An) at two time points as part of a consortium clinical trial aiming to assess two different regimens of hypomethylating agents for low and intermediate risk disease.

We will ask for a concurrent brief medical record review at each time point as well. We will use the data we collect to perform detailed psychometric assessments of the scale’s properties through analyses of its responsiveness and predictive validity with respect to survival and the patient-reported outcomes on the FACT-An.

Key Eligibility:

  • Men & women 18 years or older
  • De Novo or Secondary IPSS Low or Intermediate-1 risk MDS,
    including CMML-1
  • ECOG < 3 at study entry
  • Serum Creatinine < 2 mg/dL x ULN
  • Total Bilirubin < 2 x ULN
  • ALT/AST < 2 x ULN

Disease Status
: Low and Intermediate Risk MDS
Study Status: Open
IRB#: 1412015748
Expiration: June 10, 2017