Symptom Burden in Chronic Myeloid Leukemia (CML)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01046305
Recruitment Status : Active, not recruiting
First Posted : January 11, 2010
Last Update Posted : October 26, 2017
Novartis Pharmaceuticals
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:


The objective of this study is to measure and delineate the symptom burden experienced by patients with chronic myeloid leukemia (CML).

The Primary Aim is to develop and validate an M. D. Anderson Symptom Inventory (MDASI) module (the MDASI-CML), compliant with FDA standards for patient-reported outcomes (PROs), to measure the severity of multiple symptoms and the impact of these symptoms on daily functioning in patients with CML.

The Secondary Aims are:

  1. to develop a detailed description of the severity and interference with daily activities of symptoms experienced by patients with CML;
  2. to assess the impact of symptom severity in CML on standard functioning and quality of life (QOL) measures including Eastern Cooperative Oncology Group (ECOG) Performance Status and single-item QOL scale;
  3. to evaluate the MDASI-CML as an estimate of functional status and QOL in patients with CML;
  4. to identify common clusters of symptoms and symptom patterns occurring over multiple measurement time points in patients with CML;

5, to define the qualitative symptom burden of patients with CML;

6. to explore the feasibility of the Interactive Voice Response (IVR) system in measuring symptom severity and interference with daily activities over time in patients with CML.

Condition or disease Intervention/treatment
Leukemia Behavioral: Interview & Questionnaires Behavioral: Rating Questionnaire Behavioral: MDASI-CML Questionnaire

  Show Detailed Description

Study Type : Observational
Actual Enrollment : 210 participants
Observational Model: Case-Control
Time Perspective: Prospective
Official Title: Symptom Burden of Patients With Chronic Myeloid Leukemia (CML)
Actual Study Start Date : November 2009
Estimated Primary Completion Date : November 2019
Estimated Study Completion Date : November 2019

Group/Cohort Intervention/treatment
Part 1: Interview & Questionnaires
Patients interviews and questionnaires about CML symptoms once.
Behavioral: Interview & Questionnaires
30 Minute Interview about what it is like to have CML, followed by symptom questionnaire, quality-of-life questionnaire, and demographic questions taking another 10 minutes for all 3 questionnaires.
Other Name: Survey

Part 2: Symptoms Rating
Importance of symptoms to CML patients rated by physicians, nurses, patients, and caregivers.
Behavioral: Rating Questionnaire
Questionnaire developed in Part 1 to rate the relevance of symptom items, given to 3-5 physicians, 3-5 nurses, 2-4 patients and their family caregivers, taking about 10 minutes.
Other Names:
  • Survey

Part 3: MDASI-CML Questionnaires
Patients questionnaires about CML symptoms over 1 year using M. D. Anderson Symptom Inventory (MDASI) module (the MDASI-CML)
Behavioral: MDASI-CML Questionnaire
Patient questionnaires about CML symptoms over 1 year, completed in person or using Interactive Voice Response (IVR) system every 2 weeks.
Other Names:
  • Survey
  • M. D. Anderson Symptom Inventory

Primary Outcome Measures :
  1. Symptom Burden Experienced by CML Patients [ Time Frame: Patients followed over 1 year with MDASI-CML questionnaire ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
Chronic myeloid leukemia (CML) patients and their caregivers, 18 years of age or older, physicians and nurses.

Inclusion Criteria:

  1. 18 years of age or older (Patient and family caregiver only)
  2. Ability to speak and read English (All participants)
  3. Live in the United States (Patient Phase 3 only)
  4. Diagnosis of Philadelphia chromosome positive CML confirmed by pathological and cytogenetic analysis (Patient only)
  5. Being followed at M.D. Anderson Cancer Center (Patient only)
  6. Consent to participate (All participants)
  7. Treatment for CML for at least 1 year (Patient Phase 2 only)
  8. Family caregiver also willing to receive packet for expert panel participation (Patient Phase 2 only)
  9. Physician or nurse with at least 5 years experience caring for patients with CML (Professional expert only)
  10. At least one publication in the last 5 years dealing with CML (Physician professional expert only)
  11. Identification as a family caregiver by a patient with CML (Family caregiver only)
  12. Patient also willing to receive packet for expert panel participation (Family caregiver only)

Exclusion Criteria:

  1. Medical condition or impaired performance status that would preclude participation in the study (Patient only)
  2. Diagnosis of active psychosis or severe cognitive impairment as determined by primary physician (Patient only)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01046305

United States, Texas
University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Novartis Pharmaceuticals
Study Chair: Loretta A. Williams, PHD, MSN M.D. Anderson Cancer Center

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: M.D. Anderson Cancer Center Identifier: NCT01046305     History of Changes
Other Study ID Numbers: 2008-0728
First Posted: January 11, 2010    Key Record Dates
Last Update Posted: October 26, 2017
Last Verified: October 2017

Keywords provided by M.D. Anderson Cancer Center:
Chronic Myeloid Leukemia
Symptom Burden
M. D. Anderson Symptom Inventory

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Neoplasms by Histologic Type
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases