Austrian Myeloid Registry

Study Overview
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Brief Summary
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The Austrian Myeloid Registry (aMYELOIDr) is a non-interventional study. It collects data from patients with the myeloid diseases, primarily myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML) and acute myeloid leukemia (AML).The aMYELOIDr is multi-center database collecting data at various sites in Austria and potentially also at other centers in other countries in future. The registry has an electronic case report form (eCRF), where all data is entered by clinical trial personnel and/or physicians. It is set up to collect real-world experience in the management of patients with these diseases in Austria.

Official Title
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Austrian Myeloid Registry

Conditions
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Myeloid Diseases

Intervention / Treatment
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* Other: Non-interventional


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age >17 years Diagnosis of myeloid disease according to WHO 2016 Signed patient informed consent (IC) Exclusion Criteria: Patient is unable or unwilling to sign IC

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| AML<br>Patients with WHO 2016 diagnosis of AML | Other: Non-interventional<br>* Only routine clinical data, which has already been recorded in the patient's medical chart, will be documented. Any other data assessments (e.g. quality of life analyses such as EQ-5D and QLQ-C30 have been approved by the Ehtikkommission für das Bundesland Salzburg and are optional.<br>|
| MDS<br>Patients with WHO 2016 diagnosis of MDS | Other: Non-interventional<br>* Only routine clinical data, which has already been recorded in the patient's medical chart, will be documented. Any other data assessments (e.g. quality of life analyses such as EQ-5D and QLQ-C30 have been approved by the Ehtikkommission für das Bundesland Salzburg and are optional.<br>|
| CMML<br>Patients with WHO 2016 diagnosis of CMML | Other: Non-interventional<br>* Only routine clinical data, which has already been recorded in the patient's medical chart, will be documented. Any other data assessments (e.g. quality of life analyses such as EQ-5D and QLQ-C30 have been approved by the Ehtikkommission für das Bundesland Salzburg and are optional.<br>|
| PMF<br>Patients with WHO 2016 diagnosis of PMF | Other: Non-interventional<br>* Only routine clinical data, which has already been recorded in the patient's medical chart, will be documented. Any other data assessments (e.g. quality of life analyses such as EQ-5D and QLQ-C30 have been approved by the Ehtikkommission für das Bundesland Salzburg and are optional.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To assess the treatment patterns (therapeutic landscape) of patients with myeloid diseases. | Due to the non-interventional nature of the aMYELOIDr, treatment indication, the decision to offer treatment, treatment choice, dose, schedule and dose reductions/escalations shall be exclusively based on the risk/benefit estimation of the treating physician. We recommend compliance current guidelines. | Through study completion, median expected within 100 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Impact of front-line treatment on overall survival (OS) |  | Through study completion, median expected within 100 months | 
| Impact of number and choice of treatment lines on OS as of initial diagnosis and/or as of treatment start |  | Through study completion, median expected within 100 months | 
| Overall response rate (ORR) | Response will be assessed according to current guidelines for the respective disease. Due to the non-interventional nature of the aMYELOIDr, timepoints and types of response assessment shall be exclusively based on the risk/benefit estimation of the treating physician. We recommend compliance current guidelines. | Through study completion, median expected within 100 months | 
| Event free survival (EFS) | Events include treatment failure, progressive disease, relapse after CR/CRi, death from any cause. Patients lost to-follow-up or still alive ans without event will be censored at last follow-up date | Through study completion, median expected within 100 months | 
| AML transformation | Time to transformation to AML for patients with a non-AML initial diagnosis | Through study completion, median expected within 100 months | 
| Treatment safety | Investigators should report adverse reactions (for which a causal role of a medicine is suspected) to the concerned competent authorities following regulations in the current or future versions of Austrian legislation (Pharmakovigilanz-Verordnung 2013 (PhVO, Regulation on Pharmacovigilance), Österreichisches Arzneimittel Gesetz (AMG, Austrian Medicinal Products Act). Participation in this registry does not exempt the participating center from their legal reporting obligations. Documentation of causality, duration, frequency and severity of adverse events (AEs) according to Common Terminology Criteria for AE (CTCAE v.5). | Through study completion, median expected within 100 months | 
| Concomitant treatments | Concomitant treatments (number of administration of e.g. prophylactic antibiotics, antivirals, antifungals) | Through study completion, median expected within 100 months | 
| Treatment characteristics | Among others, the following treatment characteristics will be assessed for each treatment line: substance | Through study completion, median expected within 100 months | 
| Treatment characteristics | Among others, the following treatment characteristics will be assessed for each treatment line: application date | Through study completion, median expected within 100 months | 
| Treatment characteristics | Among others, the following treatment characteristics will be assessed for each treatment line: route | Through study completion, median expected within 100 months | 
| Treatment characteristics | Among others, the following treatment characteristics will be assessed for each treatment line: dose | Through study completion, median expected within 100 months | 
| Treatment characteristics | Among others, the following treatment characteristics will be assessed for each treatment line: inpatient or outpatient setting | Through study completion, median expected within 100 months | 
| Concomitant treatments best supportive care (BSC) | Concomitant best supportive care (BSC) measures (e.g. number of transfusions, growth factors, iron chelators) | Through study completion, median expected within 100 months | 
| Quality of life assessment EQ-5D-5L (optional) | EuroQol-5 Dimensions with 5 Levels. The descriptive system comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions. | Through study completion, median expected within 100 months | 
| EORTC Quality of life assessment QLQ-C30 (optional) | European Organisation for Research and Treatment of Cancer Quality of life 30-item questionnaire of cancer patients incorporates five functional scales (physical, role, cognitive, emotional, and social), three symptom scales (fatigue, pain, and nausea and vomiting), a global health status / QoL scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnoea, loss of appetite, insomnia, constipation anddiarrhoea) and perceived financial impact of the disease. It has four-point scale which are coded with Not at all, A little, Quite a bit and Very much | Through study completion, median expected within 100 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CMML, AML, PMF, Austrian Myeloid Registry, MDS