Myelofibrosis (MF)
Conditions
Keywords
Myelofibrosis, Pacritinib, Sobi, Sobi.PACRIT-RWE-101, PACER
Brief summary
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.
Detailed description
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.
Interventions
OTHERNot applicable- observational study
Not Applicable - Observational Study
Sponsors
Swedish Orphan Biovitrum
IQVIA RDS Inc.
Study design
Observational model
CASE_ONLY
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| ≥50% spleen length reduction | From the Index Date to Week 24 or best response in spleen reduction, assessed up to 49 months. |
| ≥20% spleen length reduction | From the Index Date to Week 24 or best response in spleen reduction, assessed up to 49 months. |
| Change in spleen length | Index to Week 24 and at other timepoints, assessed up to 49 months. |
| Improvement in spleen size category | 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 | 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 | 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 | 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) | 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 | 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 ≥50% reduction in number of RBC units transfused | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 12-Week Period without being fully RBC-TI |
| Physician-reported progression to leukemia (AML) | Index to Follow-up, assessed up to 49 months. |
| Overall survival | Index to Follow-up, assessed up to 49 months. |
| Leukemia-free Survival | Index to Follow-up, assessed up to 49 months. |
| Type, dose, and number of MF-directed therapies administered | At Index (Baseline) |
| Reasons for treatment switch from a prior MF-directed therapy to pacritinib | At Index (Baseline) |
| Method used to switch from prior JAKi to pacritinib | At Index (Baseline) |
| Physician reported (or Physician confirmed) JAKi withdrawal syndrome after switching to pacritinib from a different JAKi | 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 | Prior to (index date) and after initiation of pacritinib (up to 49 months). |
| Duration of treatment with pacritinib | Prior to (index date) and after initiation of pacritinib (up to 49 months). |
| Concomitant MF-related medications | Prior to (index date) and after initiation of pacritinib (up to 49 months). |
| Reason for discontinuation of pacritinib | 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 | 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 | At Index (Baseline) |
| Body mass index (BMI) for all participants enrolled | At Index (Baseline) |
| Receipt of allo-HSCT among patients referred to allo-HSCT | Index to Week 24 |
| Duration of treatment with pacritinib prior to allo-HSCT | Index to Week 24 |
| Dose of pacritinib prior to allo-HSCT | Index to Week 24 |
| Discontinuation of pacritinib prior to allo-HSCT | Index to Week 24 |
| Treatment with pacritinib during conditioning | Index to Week 24 |
| Dose of pacritinib during condition | Index to Week 24 |
| Duration of treatment with pacritinib after allo-HSCT | Index to Week 24 |
| Dose of pacritinib after allo-HSCT | Index to Week 24 |
| Type of MF for all participants enrolled | At Index (Baseline) |
| Comorbidities for all participants enrolled | At Index (Baseline) |
| MF Risk Category for all participants enrolled | At Index (Baseline) |
| Bone Marrow Fibrosis Grade for all participants enrolled | At Index (Baseline) |
| Type of MF Mutations for all participants enrolled | At Index (Baseline) |
| Variant Allele Frequency (VAF) for all participants enrolled | At Index (Baseline) |
| Number of Driver Mutations for all participants enrolled | At Index (Baseline) |
| Number of High-Risk Mutations for all participants enrolled | At Index (Baseline) |
| Karyotype for all participants enrolled | At Index (Baseline) |
Countries
United States
Contacts
CONTACTIQVIA Study Team
CONTACTClinical Operations Lead
PRINCIPAL_INVESTIGATORRaman Garcha, MD
Swedish Orphan Biovitrum
Outcome results
None listed