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| Name | Class |
|---|---|
| AOP Orphan Pharmaceuticals Germany GmbH | UNKNOWN |
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The primary objective of this non interventional study is to evaluate symptom burden in adult patients with PV without symptomatic splenomegaly during treatment with ropeginterferon alfa-2b in a real-world setting. Further patient-relevant endpoints include effectiveness including complete hematologic response (CHR), event-free survival (EFS), safety and tolerability, treatment reality including dosing details as well as factors affecting treatment decision making.
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| Measure | Description | Time Frame |
|---|---|---|
| Symptom Burden | Absolute values of the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) total symptom score (TSS) at time of study enrollment, during course of study until month 36. | From Time of enrollment until month 36. |
| Measure | Description | Time Frame |
|---|---|---|
| Effectiveness: Complete hematologic response (CHR) rate | CHR rate is defined as the proportion of patients with
| From time of treatment start until end of study (max. 54 months after FPI) |
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Inclusion Criteria:
Exclusion Criteria:
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Adult patients with polycythemia vera (PV) without symptomatic splenomegaly with the decision for treatment with ropeginterferon alfa 2b according to Summary of Product Characteristics (SmPC).
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Laura Serrer | Contact | +49 761 15242-0 | rope@iomedico.com |
| Name | Affiliation | Role |
|---|---|---|
| Eyck von der Heyde, Dr. | Onkologische Schwerpunktpraxis | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Onkologisches Studienzentrum Dr. med. Ingo Zander & Dr. med. Eyck von der Heyde | Recruiting | Hanover | Lower Saxony | 30161 | Germany |
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| ID | Term |
|---|---|
| D011087 | Polycythemia Vera |
| D009196 | Myeloproliferative Disorders |
| ID | Term |
|---|---|
| D019046 | Bone Marrow Neoplasms |
| D019337 | Hematologic Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
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| Effectiveness: Event-free survival (EFS) | EFS is defined as the time from the first administration of ropeginterferon alfa-2b until the first occurrence of a thromboembolic event, disease progression to post-PV myelofibrosis, acute myeloid leukemia (AML) transformation, or death, whichever comes first. Patients without any event at the time point of analysis will be censored with their respective date of last contact. | From time of treatment start until end of study (max. 54 months after FPI) |
| Effectiveness: Proportion of patients with platelet count ≤400 ×109/L | Platelet count (descriptive statistics and frequencies ≤400 vs. > 400 ×109/L) | From time of treatment start until end of study (max. 54 months after FPI) |
| Effectiveness: Proportion of patients with WBC count <10 ×109/L | WBC count (descriptive statistics and frequencies <10 vs. ≥10 ×109/L) | From time of treatment start until end of study (max. 54 months after FPI) |
| Effectiveness: Proportion of patients with HCT value <45% | HCT value (descriptive statistics and frequencies <45% vs. ≥45%) | From time of treatment start until end of study (max. 54 months after FPI) |
| Effectiveness: Proportion of patients without phlebotomy during course of study | Proportion of patients without phlebotomy from treatment start until respective time point | From time of treatment start until end of study (max. 54 months after FPI) |
| Drug safety | Incidence of serious adverse events (SAEs), adverse drug reactions (ADRs) and serious adverse drug reactions (SADRs) related to ropeginterferon alfa-2b as characterized by severity, and seriousness | From time of treatment start until end of study (max. 54 months after FPI) |
| Dosing | Dose intensity (average dose in µg/4 weeks) and dose over time per patient and overall | From start until end of treatment (max. 54 months after FPI) |
| Treatment discontinuation | Frequency of treatment discontinuation and reasons thereof | From start until end of treatment (max. 54 months after FPI) |
| (S)ADRs leading to permanent treatment discontinuation | Frequency of patients with (S)ADRs leading to permanent treatment discontinuation | From start until end of treatment (max. 54 months after FPI) |
| Symptom burden | Absolute values of single items regarding symptom burden during course of study until month 36. | From time of enrollment until month 36 after treatment start (max. 54 months after FPI) |
| Treatment reality: previous cytoreductive therapies | Frequency of distinct previous cytoreductive therapies (if applicable) | From time of treatment start until end of study (max. 54 months after FPI) |
| Treatment reality: Switch to ropeginterferon alfa-2b | Reason for switch to ropeginterferon alfa-2b (if applicable) (i.e., frequencies of answers per reasons in the physician´s questionnaire) | From time of treatment start until end of study (max. 54 months after FPI) |
| Treatment reality: parallel cytoreductive therapies | Frequency of parallel cytoreductive therapies (combinations) | From time of treatment start until end of study (max. 54 months after FPI) |
| Treatment reality: subsequent cytoreductive therapies | Frequency of subsequent cytoreductive therapies (switch to another therapeutic agent) | From time of treatment start until end of study (max. 54 months after FPI) |
| D001855 |
| Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |