Patient-reported quality of life and experiences in adults with low-risk MDS or unexplained anemia in Japan

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

This project will try to understand quality of life, symptoms, and unmet needs in adults in Japan who have low-risk MDS and are ESA-naïve and non-transfusion dependent, or who have unexplained anemia suspected to be MDS.

Quick facts

• Study type: Observational
• Enrollment: 50 (estimated)
• Ages: 18 Years to 80 Years
• Sex: All
• Sponsor: Bristol-Myers Squibb (Industry-sponsored)
• Drugs / interventions: chemotherapy, radiation
• Locations: 1 site (Minato-ku, Tokyo)
• Trial ID: NCT07008820 on ClinicalTrials.gov

What this trial studies

This observational study combines quantitative health-related quality of life questionnaires with qualitative interviews to capture patient experiences among two groups: adults with confirmed low-risk myelodysplastic syndrome who are erythropoietin-stimulating agent naïve and non-transfusion dependent, and adults with unexplained anemia suspected to be MDS. Eligible participants must be 18 or older and have recent hemoglobin <10.0 g/dL. Data collection occurs at a single site in Tokyo and is sponsored by Bristol-Myers Squibb. The goal is to identify symptom burden, daily-life impacts, and gaps in care to inform future support and care strategies.

Who should consider this trial

Good fit: Adults (≥18 years) in Japan with confirmed low-risk MDS who are ESA-naïve and non-transfusion dependent, or adults with unexplained anemia suspected to be MDS, and recent hemoglobin <10.0 g/dL are ideal candidates.

Not a fit: People who are regularly transfusion dependent, already receiving erythropoietin-stimulating agents, or whose anemia has a known non-MDS cause are unlikely to benefit from the study's focus.

Why it matters

Potential benefit: If successful, the findings could highlight unmet needs and inform better symptom management and patient support for low-risk MDS and unexplained anemia in Japan.

How similar studies have performed: Previous patient-reported outcome work in MDS has shown notable quality-of-life impacts, but focused qualitative and PRO data in ESA-naïve, non-transfusion low-risk patients and in Japanese populations with unexplained anemia are limited.

Eligibility criteria

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.

Where this trial is running

Minato-ku, Tokyo

• Mebix. Inc — Minato-ku, Tokyo, Japan (Recruiting)

Study contacts

• Study coordinator: BMS Study Connect Contact Center www.BMSStudyConnect.com

Conditions Myelodysplastic Syndrome