Patient Reported Outcomes and Patient Voice Among Patients Diagnosed With Low Risk Myelodysplastic Syndrome (LR-MDS) or Unexplained Anemia In Japan

Bristol-Myers Squibb (BMS)

Status: Enrolling

Conditions: Myelodysplastic Syndrome (MDS)

Treatments: Other: Health-related quality of life questionnaires; Other: Qualitative interviews

Study type: Observational

Funder types: Industry

Identifiers

NCT07008820

CA056-1133

Details and patient eligibility

About

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

Enrollment: 50 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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.

Trial design

50 participants in 2 patient groups

Cohort 1

Description:

Low Risk Myelodysplastic Syndrome erythropoietin stimulating agent naïve and non-transfusion dependent

Treatment:

Other: Qualitative interviews

Other: Health-related quality of life questionnaires

Cohort 2

Description:

Suspected Low Risk Myelodysplastic Syndrome with unexplained anemia

Treatment:

Other: Qualitative interviews

Other: Health-related quality of life questionnaires