Clinical trial NCT04053933: Observational Study Towards the Impact of Newly Started Treatment in MDS on QoL
Official title: A Multicenter Observational Belgian Study Assessing the Impact of Newly Started Treatment on the QOL in Patients Suffering From Myelodysplastic Syndromes.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Myelodysplastic Syndromes
Keywords: QUALMS
MDS
Be-QUALMS
Quality of life
QoL
Lead sponsor: University Hospital, Antwerp
Sponsor class: OTHER
First posted: 2019-08-13
Enrollment: 70
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients suffering from myelodysplastic syndromes

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Change in QUALMS-score
timeFrame: 6 months
measure: Change in IPQ-score
timeFrame: 6 months
Secondary outcomes:
Other outcomes:
Locations:
city: Bonheiden
country: Belgium
facility: Imelda
state: Antwerpen
city: Brasschaat
country: Belgium
facility: AZ KLINA
state: Antwerpen
city: Geel
country: Belgium
facility: Sint-Dimpna Ziekenhuis Geel
state: Antwerpen
city: Lier
country: Belgium
facility: Heilig Hart Ziekenhuis
state: Antwerpen
city: Edegem
country: Belgium
facility: UZA
state: Antwerp
city: La Louvière
country: Belgium
facility: CH Jolimont
state: Henegouwen
city: Mons
country: Belgium
facility: CHR Mons Hainaut
state: Henegouwen
city: Mons
country: Belgium
facility: CHU Ambroise Paré
state: Henegouwen
city: Yvoir
country: Belgium
facility: CHU - UCL Namur site Godinne
state: Namur
city: Gent
country: Belgium
facility: UZ Gent
state: Oost Vlaanderen
city: Sint-Niklaas
country: Belgium
facility: AZ Nikolaas
state: Oost Vlaanderen
city: Brussel
country: Belgium
facility: Institute Jules Bordet
state: Vlaams Brabant
city: Jette
country: Belgium
facility: UZ Brussel
state: Vlaams Brabant
city: Leuven
country: Belgium
facility: UZ Leuven Gasthuisberg
state: Vlaams Brabant
city: Woluwe-Saint-Lambert
country: Belgium
facility: Cliniques Universitaires Saint-Luc
state: Vlaams Brabant
city: Brugge
country: Belgium
facility: AZ Sint Jan Brugge
state: West Vlaanderen
city: Oostende
country: Belgium
facility: AZ Damiaan Oostende
state: West Vlaanderen
city: Antwerpen
country: Belgium
facility: Ziekenhuis Netwerk Antwerpen
city: Antwerpen
country: Belgium
facility: GasthuisZusters Antwerpen
city: Namur
country: Belgium
facility: CHR Namur
city: Ottignies-Louvain-la-Neuve
country: Belgium
facility: BR Clinic Saint Pierre Ottignies
Study officials:
affiliation: University Hospital, Antwerp
name: Zwi Berneman, MD,PhD
role: PRINCIPAL_INVESTIGATOR
References: