Pacritinib Effectiveness in Real-world Settings

Swedish Orphan Biovitrum60 enrolled

Overview

This study aims to evaluate real-world treatment patterns and effectiveness of pacritinib, including hematologic and clinical outcomes, and survival through a site-based retrospective chart review of medical records of patients with MF.

This is a multicenter, observational, retrospective chart review study of patients with Myelofibrosis (MF) who received treatment with pacritinib in routine clinical settings with platelet count ≥50 x 109/L at the time of treatment initiation with pacritinib. This study will be conducted entirely through medical chart abstraction; all data will be taken from the patient's medical record, with no additional assessments.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Myelofibrosis (MF)

Interventions

Not applicable- observational studyOTHER

Not Applicable - Observational Study

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Adult patients must be ≥18 years of age at the index date * Patients diagnosed with MF with platelet counts ≥50 x 109/L at the time of treatment initiation with pacritinib. If multiple values are available within 30 days prior to initiating treatment with pacritinib will use the value closest to index date * Patients will be required to have ≥1 month of treatment with pacritinib and ≥6 months of observation from the start of pacritinib unless the patient died within 6 months of starting pacritinib * If peripheral blasts were evaluated prior to index, they must be \<10%. If not evaluated, patients will be included unless a healthcare provider has indicated in the medical record that there is a concern that the patient has transitioned to accelerated/blast phase disease at or prior to index * According to local regulations, waivers of consent will be sought for study patients from the appropriate regulatory authorities and/or the independent ethics committee (IEC)/institutional review board (IRB). For patients not covered by waivers of consent, signed and dated informed consent provided by the patient, or the patient's legally authorized representative(s) for patients under the legal age (with patient assent, as applicable), should be obtained before any study-related activities are undertaken. Exclusion Criteria: * Diagnosis of acute myeloid leukemia prior to index * Physician-concern that the patient has transitioned to accelerated/blast phase disease if peripheral blasts were not evaluated * Treated with 2 or more JAK inhibitors prior to initiating treatment with pacritinib * Treated with pacritinib in a clinical trial setting

Locations (3)

Yale School of Medicine

New Haven, Connecticut, United States

RECRUITING

The Ohio State University

Columbus, Ohio, United States

RECRUITING

Medical University of South Carolina

Charleston, South Carolina, United States

RECRUITING

Outcomes

Primary Outcomes

≥50% spleen length reduction

Time frame: From the Index Date to Week 24 or best response in spleen reduction, assessed up to 49 months.

≥20% spleen length reduction

Time frame: From the Index Date to Week 24 or best response in spleen reduction, assessed up to 49 months.

Change in spleen length

Time frame: Index to Week 24 and at other timepoints, assessed up to 49 months.

Improvement in spleen size category

Time frame: Index to Week 24 and at other timepoints, assessed up to 49 months.

Among those with ≥1 MF-related symptom at index Symptom-specific resolution

Time frame: Index to Week 24 and at other timepoints, assessed up to 49 months.

Among those with ≥1 MF-related symptom at index Decrease in total number of MF-symptoms

Time frame: Index to Week 24 and at other timepoints, assessed up to 49 months.

Among those with ≥1 MF-related symptom at index Change in total number of MF-symptoms

Time frame: Index to Week 24 and at other timepoints, assessed up to 49 months.

Change in blood counts (i.e., white blood cells [WBC], platelets, absolute neutrophil counts, peripheral blast percentage)

Time frame: Index to Week 24 and at other timepoints, assessed up to 49 months.

Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index RBC-TI - absence of RBC transfusions over any 12-week period

Time frame: Index to Week 24 and at other timepoints, assessed up to 49 months.

Central Contacts

IQVIA Study Team

CONTACT

617-621-1600 PACER-study@mail.iqvia.com

Clinical Operations Lead

CONTACT

sara.norris@iqvia.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index ≥50% reduction in number of RBC units transfused

Time frame: Index to Week 12 & Index to Week 24

Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index ≥50% reduction in monthly rate of RBC units transfused

Time frame: Index to Week 12 & Index to Week 24

Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index change in the number of RBC units transfused

Time frame: Index to Week 24

Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index change in the monthly rate of RBC units transfused

Time frame: Index to Week 24

Among those with a platelet count <100 x 109/L at index date Absolute increase in platelet counts ≥30 x 109/L without a platelet transfusion in the prior 2 days

Time frame: Index to best response, assessed up to 49 months.

Among patients with Hb <10 g/dL at index and who are RBC-transfusion dependent (TD) (≥3 RBC units transfused in prior 12 weeks) at index: achieved RBC-TI

Time frame: 12-Week Period with no RBC transfusions administered during the period

Among patients with Hb <10 g/dL at index and who are not RBC-transfusion dependent (TD) (≥3 RBC units transfused in prior 12 weeks) at index: ≥1.5 g/dL increase in average Hb

Time frame: 12-Week Period with no RBC transfusions administered during the period

Among patients who have IWG Hb Minor Response at index and are RBC-TD at index ≥50% reduction in RBC transfusion

Time frame: 12-Week Period without being fully RBC-TI

Among patients who have IWG Hb Minor Response at index and are not RBC-TD at index ≥1 g/dL increase in average Hb

Time frame: 12-Week Period without being fully RBC-TI

Physician-reported progression to leukemia (AML)

Time frame: Index to Follow-up, assessed up to 49 months.

Overall survival

Time frame: Index to Follow-up, assessed up to 49 months.

Leukemia-free Survival

Time frame: Index to Follow-up, assessed up to 49 months.

Type, dose, and number of MF-directed therapies administered

Time frame: At Index (Baseline)

Reasons for treatment switch from a prior MF-directed therapy to pacritinib

Time frame: At Index (Baseline)

Method used to switch from prior JAKi to pacritinib

Time frame: At Index (Baseline)

Physician reported (or Physician confirmed) JAKi withdrawal syndrome after switching to pacritinib from a different JAKi

Time frame: Prior to (index date) and after initiation of pacritinib (up to 49 months).

Pacritinib line of therapy, treatment dose and frequency, changes in dose/and or frequency, frequency of dose changes and reasons for change

Time frame: Prior to (index date) and after initiation of pacritinib (up to 49 months).

Duration of treatment with pacritinib

Time frame: Prior to (index date) and after initiation of pacritinib (up to 49 months).

Concomitant MF-related medications

Time frame: Prior to (index date) and after initiation of pacritinib (up to 49 months).

Reason for discontinuation of pacritinib

Time frame: Prior to (index date) and after initiation of pacritinib (up to 49 months).

Subsequent MF-therapy following discontinuation of pacritinib and time to next Treatment

Time frame: Prior to (index date) and after initiation of pacritinib (up to 49 months).

Demographic characteristics include age, sex, race/ethnicity, geographic region, and insurance type

Time frame: At Index (Baseline)

Body mass index (BMI) for all participants enrolled

Time frame: At Index (Baseline)

Receipt of allo-HSCT among patients referred to allo-HSCT

Time frame: Index to Week 24

Duration of treatment with pacritinib prior to allo-HSCT

Time frame: Index to Week 24

Dose of pacritinib prior to allo-HSCT

Time frame: Index to Week 24

Discontinuation of pacritinib prior to allo-HSCT

Time frame: Index to Week 24

Treatment with pacritinib during conditioning

Time frame: Index to Week 24

Dose of pacritinib during condition

Time frame: Index to Week 24

Duration of treatment with pacritinib after allo-HSCT

Time frame: Index to Week 24

Dose of pacritinib after allo-HSCT

Time frame: Index to Week 24

Type of MF for all participants enrolled

Time frame: At Index (Baseline)

Comorbidities for all participants enrolled

Time frame: At Index (Baseline)

MF Risk Category for all participants enrolled

Time frame: At Index (Baseline)

Bone Marrow Fibrosis Grade for all participants enrolled

Time frame: At Index (Baseline)

Type of MF Mutations for all participants enrolled

Time frame: At Index (Baseline)

Variant Allele Frequency (VAF) for all participants enrolled

Time frame: At Index (Baseline)

Number of Driver Mutations for all participants enrolled

Time frame: At Index (Baseline)

Number of High-Risk Mutations for all participants enrolled

Time frame: At Index (Baseline)

Karyotype for all participants enrolled

Time frame: At Index (Baseline)