Clinical trial NCT02863458: DNA Hypomethylating Agents and Lenalidomide in Elderly Patients With Myeloid Malignancies in the US
Official title: Effectiveness of DNA Hypomethylating Agents and Lenalidomide in Elderly Patients With Myeloid Malignancies in the United States
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Acute Myeloid Leukemia
Myelodysplastic Syndromes
Keywords: MDS
AML
hypomethylating agents (HMA)
Surveillance Epidemiology and End Results (SEER)
Lead sponsor: The Cleveland Clinic
Sponsor class: OTHER
Collaborators: name: Celgene
First posted: 2016-08-11
Enrollment: 0
Sex: ALL
Minimum age (years): 66
Standard ages: OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The overall sample will include cases of MDS and AML newly diagnosed between 2001 and 2011 with claims from 2000 to 2012. Investigators estimate this will include approximately 50,000 patients. To allow adequate time for HMA usage to begin to stabilize, our analysis will be restricted to patients alive at least 3 months after its approval. Decitabine received FDA approval for all subtypes of MDS in June 2006, but it was not covered by Medicare initially, so our analysis of it will be restricted to patients who were alive in January 2007. Patients will be followed from the date of their first MDS claim (index date) through death or study end, whichever occurred first.

Brief summary: The study aims to comprehensively analyze data from a large and unselected older AML population in the US, both treated and untreated. These data will widen understanding of treatment decisions for the older Acute Myeloid Leukemia (AML) population. Through use of the SEER-Medicare Registry, the effectiveness and impact of HMA treatments as well as the effectiveness of lenalidomide will be studied.

Detailed description: This will be the first study to comprehensively analyze data from a large and unselected older AML population in the US, both treated and untreated. These data will widen understanding of treatment decisions for the older AML population and shed light on the impact of HMA in this vulnerable population. The findings from this study will help identify the role of HMA (with its salient features of better tolerability and lesser toxicity) as a new treatment paradigm for older AML patients and enable comparison of outcomes with HMA vs other "conventional" leukemia therapies (intensive chemotherapy, low dose cytarabine or best supportive care). Additionally, with updated SEER-Medicare file carrying Medicare claims through 2013, this study will help analyze several long term outcome measures extending up to eight years for MDS patients treated with lenalidomide. Finally, the effectiveness of HMA in the SEER-Medicare MDS population will be studied by using SEER-Medicare data. This registry includes information on Medicare MDS beneficiaries - a population age cohort that has the highest incidence rates for MDS and a large sample size with absolute MDS cases approaching 40000, making it the largest MDS registry in the world.

The overall sample will include cases of MDS and AML newly diagnosed between 2001 and 2011 with claims from 2000 to 2012. Investigators estimate this will include approximately 50,000 patients.Therefore, the Target Follow Up design looks at patient records over a span of 12 years. Consists of patients with myeloid malignancies diagnosed during 2001-2011 at the age of 66 years or older and alive in September 2004 (i.e. 3 months after FDA approval of azacitidine for MDS), with known month of diagnosis, and not identified from death certificates or autopsy only, who had continuous Medicare Part A and B coverage, and were not enrolled in a health maintenance organization during the period of interest. The overall sample will include cases of MDS and AML newly diagnosed between 2001 and 2011 with claims from 2000 to 2012.

Variables of interest to be collected will include: MDS subtypes, age at diagnosis, sex, race, comorbidities, median household income at the zip code level, treatments (including supportive care) from diagnosis to AML progression, transfusion status, NCI comorbidity index score, response to treatments (using International Working Group criteria), physician characteristics (speciality data),and hospital characteristics (bed size, teaching status, case volume).

Eligibility criteria: Inclusion Criteria:

* patients with myeloid malignancies diagnosed during 2001-2011 at the age of 66 years or older
* alive in September 2004 (i.e. 3 months after FDA approval of azacitidine for MDS)
* known month of diagnosis, and not identified from death certificates or autopsy only
* continuous Medicare Part A and B coverage, and were not enrolled in a health maintenance organization during the period of interest

Exclusion Criteria:

* Anyone under 66 years

Primary outcomes:
measure: Number of Participants on hypomethylating agents (HMA) and the effectiveness of HMA in the SEER-Medicare MDS population
timeFrame: Analaysis restricted to patients alive at least 3 months after HMA approval. Patients will be followed from date of their first MDS claim (index date) through death or study end, whichever occurred first.
measure: Develop predictive models of HMA treatment outcomes using SEER-Medicare data
timeFrame: Analaysis restricted to patients alive at least 3 months after HMA approval. Patients will be followed from date of their first MDS claim (index date) through death or study end, whichever occurred first.
Secondary outcomes:
measure: Effectiveness of lenalidomide in the SEER-Medicare MDS population
timeFrame: Analaysis restricted to patients alive at least 3 months after HMA approval. Patients will be followed from date of their first MDS claim (index date) through death or study end, whichever occurred first.
measure: Identify the role of HMA (with its salient features of better tolerability and lesser toxicity) as a new treatment paradigm for older AML patients and enable comparison of outcomes with HMA vs other "conventional" leukemia therapies.
timeFrame: Analaysis restricted to patients alive at least 3 months after HMA approval. Patients will be followed from date of their first MDS claim (index date) through death or study end, whichever occurred first.
Other outcomes:
Locations:
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
Study officials:
affiliation: The Cleveland Clinic
name: Sudipto Mukherjee, MD, MPh
role: PRINCIPAL_INVESTIGATOR
References: