Anemia, Myelofibrosis, Myelofibrosis; Anemia, Post-essential Thrombocythemia Myelofibrosis, Post-polycythemia Vera Myelofibrosis, Primary Myelofibrosis, Secondary Myelofibrosis
Conditions
Brief summary
The purpose of the project is to set up a national, prospective, longitudinal, multicenter cohort study, a tumor research platform, to document uniform data on characteristics, molecular diagnostics, treatment and course of disease and to collect patient-reported outcomes for patients with primary and secondary myelofibrosis and anemia in Germany.
Detailed description
RHODOLITE is a national, prospective, open-label, longitudinal, non-interventional multicenter cohort study (research platform) to describe treatment in routine clinical practice of myelofibrosis patients in routine care in Germany. RHODOLITE will follow patients for up to three years with the aim to identify common therapeutic sequences and changes in the treatment of the disease. At inclusion, data in patient characteristics, comorbidities, tumor characteristics and previous treatments are collected. During the course of observation data on all systemic treatments and outcome are documented. Health-related quality of life (HRQoL) will be evaluated for up to three years. The RHODOLITE project is a joint project with the German Study Group for Myeloproliferative Neoplasms (GSG-MPN) and its GSG-MPN Bioregistry (NCT03125707). Details on treatment and outcome will be collected in RHODOLITE for up to three years, while long-term follow-up for a minimum of additional five years will be performed in the GSG-MPN Bioregistry.
Interventions
None listed
Sponsors
iOMEDICO AG
German Study Group for Myeloproliferative Neoplasms (GSG-MPN)
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Confirmed diagnosis of primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis (MF) (Note: diagnosis according to WHO-2017, ICC-2022 or WHO-2022 or IWG-MRT criteria, respectively). * Diagnosis of anemia at the time of enrollment as per individual, clinical assessment by the local physician. * Start of first or subsequent systemic treatment for MF. * Informed consent and registration for the GSG-MPN Bioregistry. * Willingness and capability to participate in PRO assessment. * Signed and dated informed consent form for RHODOLITE at the latest six weeks after start of the respective systemic MF treatment.
Exclusion criteria
* No systemic therapy for diagnosed primary or secondary MF. * Planned allogenic stem cell transplantation (allo-SCT) or active participation in an interventional clinical trial.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Documentation of systemic treatment for MF and therapy sequences | 3 years per patient | Types and frequencies of systemic treatment for MF during observation time. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Hematologic response | 3 years per patient | Frequencies of hematologic response (improved, stable, worsened) per line of treatment |
| Splenic response | 3 years per patient | Frequencies of splenic response (improved, stable, worsened) per line of treatment |
| Overall response rate (ORR) | 3 years per patient | Documentation of ORR per line of treatment |
| Progression free survival (PFS) | 3 years per patient | Documentation of PFS per line of treatment |
| Overall survival | 3 years per patient | Documentation of OS per line of treatment |
| Health-related quality of life (Patient-reported outcome, PRO) | 3 years per patient | European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life Questionnaire (EORTC QLQ-C30, the lower the score the better the QoL) |
| Assessment of Myelofibrosis-related symptoms | 3 years per patient | Myelofibrosis Symptom Assessment form (MFSAF) questionnaire 7-day Recall |
Countries
Germany
Contacts
CONTACTiOMEDICO AG
STUDY_CHAIRKonstanze Döhner, Prof. Dr. med.
University Hospital Ulm, Germany
STUDY_CHAIRSteffen Koschmieder, Prof. Dr. med.
University Hospital Aachen, Germany
Outcome results
None listed