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.
Study Type
OBSERVATIONAL
Enrollment
200
Onkologisches Studienzentrum Dr. med. Ingo Zander & Dr. med. Eyck von der Heyde
Hanover, Lower Saxony, Germany
RECRUITINGSymptom 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.
Time frame: From Time of enrollment until month 36.
Effectiveness: Complete hematologic response (CHR) rate
CHR rate is defined as the proportion of patients with * Hematocrit (HCT) \<45% and * Phlebotomy-free (absence of phlebotomy during the previous 3 months) and * Platelet count ≤400 x 10\^9/L and * white blood cell count ≤10 x 10\^9/L
Time frame: From time of treatment start until end of study (max. 54 months after FPI)
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.
Time frame: 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)
Time frame: 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)
Time frame: From time of treatment start until end of study (max. 54 months after FPI)
Effectiveness: Proportion of patients with HCT value <45%
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HCT value (descriptive statistics and frequencies \<45% vs. ≥45%)
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: From start until end of treatment (max. 54 months after FPI)
Treatment discontinuation
Frequency of treatment discontinuation and reasons thereof
Time frame: 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
Time frame: 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.
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: From time of treatment start until end of study (max. 54 months after FPI)