Myelodysplastic Syndrome (MDS)
Conditions
Keywords
Myelodysplastic Syndrome (MDS)
Brief summary
This study will examine quality of life, experiences, and unmet needs among individuals diagnosed with Low Risk Myelodysplastic Syndrome who are erythropoietin stimulating agent naïve and non-transfusion dependent and among individuals with suspected myelodysplastic syndromes with unexplained anemia in Japan
Interventions
Questionnaires include: EORTC-QLQ C30 FACT-An PGI-S EQ-5D-5L
OTHERQualitative interviews
For participants that meet eligibility criteria
Sponsors
Bristol-Myers Squibb
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
The inclusion criteria for the quantitative phase will include either 1 or 2 and all of 3-6: 1. Low Risk Myelodysplastic Syndromes (LR MDS) erythropoietin stimulating agent (ESA) naïve non-transfusion dependent (NTD) patrticipants: identified with confirmed via bone marrow aspirate and \< 5% blasts in bone marrow. Lower-risk is defined by International Prognostic Scoring System (IPSS) or Revised International Prognostic Scoring System (IPSS-R), as follows: * Very low, low, or intermediate-risk (score ≤ 3.5) as assessed by IPSS-R * Low or intermediate-1 (score ≤ 1) as assessed by IPSS 2. Unexplained anemia with suspected MDS: identified anemia with general anemia (iron/vitamin deficiency, bleeding, renal, etc.) excluded 3. Participants with hemoglobin in the most recent blood test \< 10.0 g/dl, or the average of hemoglobin \< 10.0g/dl in the most recent 2 blood tests conducted within 30 days prior to enrollment in this study 4. Participants who are ≥ 18 years of age at the time of signing the informed consent form. 5. Participants who are able and willing to provide informed consent. 6. Participants who are able to complete the protocol requirements. For the qualitative phase (approximately 10 participants), the inclusion criteria will also include: 1\. Participants with severity scores of "moderate" or greater on at least one Patient Global Impression-Severity (PGI-S) item.
Exclusion criteria
Patients meeting the following criteria will be excluded: 1. Participants who have difficulty obtaining informed consent or execution of this study because of insufficient Japanese language proficiency. 2. Participants who are considered to have difficulty answering HRQoL questionnaires and/or responding to questions during cognitive interview 3. Participants who received red-blood cell transfusion (RBC-T) within 16 weeks prior to enrollment. Note: RBC-T of 1 to 2 units within the 16 weeks prior to enrollment are allowed, provided those 1-2 RBC-T units are administered for an acute event/illness (i.e., surgical procedure, bleeding, infection) or presence of comorbidity (including cardiovascular, pulmonary, cerebrovascular), and not for the treatment of low hemoglobin (with or without symptoms) alone. 4. Participants who received prior drug treatment related to anemia; drugs include the following: erythropoiesis stimulating agent, erythroid maturation agent, hypomethylating agent, immunomodulatory drugs, immuno-suppressive agent. 5. Participants with myelodysplastic/myeloproliferative neoplasms (MDS/MPN) according to World Health Organization (WHO) 2016 classification (i.e., chronic myelomonocytic leukemia, atypical chronic myeloid leukemia, breakpoint cluster region-Abelson 12, juvenile myelomonocytic leukemia, MDS/MPN unclassifiable). 6. Participants with secondary MDS (i.e., MDS that is known to have arisen as a result of chemical injury or treatment with chemotherapy and/or radiation for other diseases). 7. Participants with a known history of diagnosis of acute myeloid leukemia. 8. Participants with major surgery within 8 weeks prior to enrollment. Patients must have completely recovered from any previous surgery prior to enrollment. 9. Participants with history of cerebrovascular accident (including ischemic, embolic, and hemorrhagic cerebrovascular accident), transient ischemic attack, deep venous thrombosis (including proximal and distal), pulmonary or arterial embolism, arterial thrombosis, or other venus thrombosis within 6 months prior to enrollment. Note: prior superficial thrombophlebitis is not an exclusion criterion. 10. Participants who have any condition or receive concomitant medication that confounds the ability to interpret data from the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Participant quality of life as assessed by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC-QLQ C30) patient reported outcome questionnaire | Day 1 |
| Participant anemia symptoms as assessed by the Functional Assessment of Cancer Therapy - Anemia (FACT-An) patient reported outcome questionnaire | Day 1 |
| Symptom severity as assessed by the Patient Global Impression-Severity (PGI-S) patient reported outcome questionnaire | Day 1 |
| General health status of participants as assessed by the EQ-5D-5L patient reported outcome questionnaire | Day 1 |
| Participant baseline socio-demographics | Baseline |
| Participant baseline clinical characteristics | Baseline |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Qualitative interview results | Up to 1 month | Qualitative interview questions to include questions to understand the participants: * Experiences of diagnosis and treatment * Symptoms of anemia * Quality of life * Communication with physicians * Satisfaction with current treatments |
Countries
Japan
Contacts
STUDY_DIRECTORBristol Myers Squibb
Bristol-Myers Squibb
STUDY_DIRECTORBristol-Myers Squibb
Bristol-Myers Squibb
Outcome results
None listed