Observational Study Towards the Impact of Newly Started Treatment in MDS on QoL (Be-QUALMS)

A Multicenter Observational Belgian Study Assessing the Impact of Newly Started Treatment on the QOL in Patients Suffering From Myelodysplastic Syndromes.

Study type An observational study conducted in different hematological centers in Belgium.

Study objectives

Primary objective:

To assess the impact of newly started treatments on the QOL of patients suffering from myelodysplastic syndromes.

Secondary objectives:

• To assess the impact of newly started therapy on disease perception in MDS patients
• To study the relation between disease perception and quality of life
• To examine which clinical and disease specific factors determine QOL in MDS patients
• Collect information on the transfusion threshold in Belgian hematological centers and evaluate the impact on quality of life.
• To evaluate whether changes in QOL are related to hematological respons.

Study design

• Newly diagnosed MDS patients who are about to start a treatment or previously diagnosed MDS patients who are starting with a new line of therapy.
• QOL assessment with the QUALMS.
• Disease perception measurement using the B-IPQ.
• Measurement at diagnosis/before start of therapy, at 4 weeks, 12 weeks, and at 24 weeks into treatment.

Study endpoints

Primary endpoint:

Change in QUALMS score at visit timepoints 4 - 12 - 24 weeks after the start of a new treatment.

Secondary endpoint:

• Change in B-IPQ score at visit timepoints 4 - 12 - 24 weeks after the start of a new treatment
• Association between B-IPQ and QUALMS score.
• Association between clinical and disease specific factors and QUALMS score
• Association between transfusion threshold and QUALMS score.
• Association between hematological response and QUALMS score

Summary of eligibility criteria

• Adult patients with a new diagnosis of MDS (according to WHO 2016 definitions (3) or known patients with MDS who are about to start a new treatment.
• Signed informed consent.
• Patients enrolled in an unblinded interventional therapeutic trial are eligible.

Exclusion criteria

• Patients with acute leukemia defined as >20% bone marrow blasts.
• Patients suffering from an overlap syndrome myelodysplastic/myeloproliferative disease.
• Patients in post allogeneic transplant setting.
• Patients enrolled in a blinded interventional therapeutic trial.
• Patients starting with multiple treatments under investigation at the same moment apart from intensive chemotherapy.
• Newly diagnosed patients who do not start with treatment.
• Patients who started a previous treatment less then 12 weeks ago apart from packed cell transfusion (up to 4 weeks allowed).
• Diagnosis of any previous or concomitant malignancy except when the patient successfully completed treatment (chemotherapy and/or surgery and/or radiotherapy) with curative intent for this malignancy at least 3 months prior to inclusion.
• Patients refusing to sign informed consent.

Study Overview

• Status: Completed
• Conditions: Myelodysplastic Syndromes

• Study Type: Observational
• Enrollment (Actual): 70

Contacts and Locations

Study Locations

• Belgium
  • Antwerpen, Belgium, 2020
    • Ziekenhuis Netwerk Antwerpen
  • Antwerpen, Belgium, 2610
    • GasthuisZusters Antwerpen
  • Namur, Belgium, 5000
    • CHR Namur
  • Ottignies-Louvain-la-Neuve, Belgium
    • BR Clinic Saint Pierre Ottignies
  • Antwerp
    • Edegem, Antwerp, Belgium, 2650
      • UZA
  • Antwerpen
    • Bonheiden, Antwerpen, Belgium, 2820
      • IMELDA
    • Brasschaat, Antwerpen, Belgium, 2930
      • AZ Klina
    • Geel, Antwerpen, Belgium, 2240
      • Sint-Dimpna Ziekenhuis Geel
    • Lier, Antwerpen, Belgium, 2500
      • Heilig Hart Ziekenhuis
  • Henegouwen
    • La Louvière, Henegouwen, Belgium, 7100
      • CH Jolimont
    • Mons, Henegouwen, Belgium, 7000
      • CHU Ambroise Pare
    • Mons, Henegouwen, Belgium, 7000
      • CHR Mons Hainaut
  • Namur
    • Yvoir, Namur, Belgium, 5530
      • CHU - UCL Namur site Godinne
  • Oost Vlaanderen
    • Gent, Oost Vlaanderen, Belgium, 9000
      • UZ Gent
    • Sint-Niklaas, Oost Vlaanderen, Belgium, 9100
      • AZ Nikolaas
  • Vlaams Brabant
    • Brussel, Vlaams Brabant, Belgium, 1000
      • Institute Jules Bordet
    • Jette, Vlaams Brabant, Belgium, 1090
      • UZ Brussel
    • Leuven, Vlaams Brabant, Belgium, 3000
      • UZ Leuven Gasthuisberg
    • Woluwe-Saint-Lambert, Vlaams Brabant, Belgium, 1200
      • Cliniques Universitaires Saint-Luc
  • West Vlaanderen
    • Brugge, West Vlaanderen, Belgium, 8000
      • Az Sint Jan Brugge
    • Oostende, West Vlaanderen, Belgium, 8400
      • AZ Damiaan Oostende

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients suffering from myelodysplastic syndromes

Description

Inclusion Criteria:

• Newly diagnosed patients with myelodysplastic syndromes defined by WHO 2016 criteria that are about to start treatment.
• Patients with a known diagnosis of MDS, irrespective of IPSS and irrespective of time of diagnosis that are about to start a new therapy.
• Signed informed consent

Exclusion criteria

• Patients with acute leukemia defined as >20% bone marrow blasts.
• Patients suffering from a myelodysplastic/myeloproliferative overlap syndrome. In this case the disease has both dysplastic and proliferative features but cannot be properly categorized to either group. This category includes chronic myelomonocytic leukemia (CMML), juvenile myelomonocytic leukemia (JMML), atypial chronic myeloid leukemia (aCML) and myelodysplastic/myeloproliferative disease unclassifiable.
• Patients in post allogeneic transplant setting.
• Patients enrolled in a blinded interventional therapeutic trial.
• Patients starting with multiple MDS treatments at the same moment apart from intensive chemotherapy.
• Newly diagnosed patients who do not start with treatment.
• Patients who started a previous MDS related treatment less then 4 weeks ago.
• Patients who started a previous MDS related treatment less then 12 weeks ago apart from packed cell transfusions.
• Diagnosis of any previous or concomitant malignancy except when the patient successfully completed treatment (chemotherapy and/or surgery and/or radiotherapy) with curative intent for this malignancy at least 3 months prior to inclusion.
• Patients refusing to sign informed consent

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

7

Cohorts and Interventions

Group / Cohort
Patients treated with ESA
Patients treated with 5'azacitidin
Patients treated with deferoxamine
Patients treated with deferasirox
Patients treated with transfusion only
Patients treated with lenalidomide
Patients treated with intensive chemotherapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in QUALMS-score	Change in quality of life-score after the start of a new MDS related treatment	6 months
Change in IPQ-score	Change of ilness perception score after the start of a new MDS related treatment	6 months

Collaborators and Investigators

• Sponsor: University Hospital, Antwerp
• Investigators: Principal Investigator: Zwi Berneman, MD,PhD, University Hospital, Antwerp

Study record dates

Study Major Dates

• Study Start (Actual): August 1, 2019
• Primary Completion (Actual): July 31, 2021
• Study Completion (Actual): July 31, 2021

Study Registration Dates

• First Submitted: August 9, 2019
• First Submitted That Met QC Criteria: August 9, 2019
• First Posted (Actual): August 13, 2019

Study Record Updates

• Last Update Posted (Actual): March 30, 2025
• Last Update Submitted That Met QC Criteria: March 25, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: QoL; Quality of life; MDS; QUALMS; Be-QUALMS
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease; Hematologic Diseases; Precancerous Conditions; Bone Marrow Diseases; Syndrome; Preleukemia; Myelodysplastic Syndromes
• Other Study ID Numbers: B300201938708

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No