Waldenström's Macroglobulinemia (WM)
Conditions
Brief summary
This is a non-interventional, observational, retrospective and prospective multicenter Italian study, to describe treatment management and outcomes of Waldenström's Macroglobulinemia (WM) patients treated according to the Italian Compassionate Use Program (CUP) and receiving zanubrutinib following its commercial approval.
Interventions
DRUGZanubrutinib
Patients will be treated with Zanubrutinib as per routine clinical practice.
Sponsors
Fondazione Italiana Linfomi - ETS
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Signed and dated informed consent form * Waldenström's macroglobulinemia diagnosis in need of treatment according to ESMO (European Society for Medical Oncology) guideline 2018 * Patients who received Zanubrutinib according to the Italian CUP or in common practice following Zanubrutinib commercial approval * Treatment with zanubrutinib according to current SmPC (Summary of Product Characteristics) * Treatment decision before inclusion into this non-interventional study * Age ≥18 years
Exclusion criteria
* Contraindications according to SmPC for patients with WM * Participation in an interventional clinical trial during zanubrutinib treatment * Patients with disease progression during a BTKi treatment (if pre-treated with BTK, only those intolerants are considered eligible)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Cumulative incidence of treatment discontinuation due to toxicity | From the date of first zanubrutinib administration to the date of definitive treatment discontinuation or dose reduction for any cause or death from any cause, from october 2020 up to 24 months since study start | Cumulative incidence of treatment discontinuation due to toxicity |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Hospital stays | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Hospital stays comprises all planned and unplanned hospitalizations as well as emergency unit visits, regardless of whether there is an association with WM or not. All hospitalizations and emergency unit visits that started during therapy with zanubrutinib will be considered for hospital stays. |
| EORTC-QLQ-C30 | Prospective cohort: up to 24 months (duration of study) | Quality of life (QoL) assessed with EORTC-QLQ-C30 questionnaire |
| EQ-5D-5L | Prospective cohort: up to 24 months (duration of study) | Quality of life (QoL) assessed with EQ-5D-5L questionnaire |
| Incidence of adverse events (AEs) | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Incidence of adverse events (AEs) |
| Incidence of adverse events related to zanubrutinib. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Incidence of adverse events related to zanubrutinib. |
| Incidence of relevant adverse events. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Incidence of relevant adverse events. |
| Incidence of serious adverse events (SAEs). | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Incidence of serious adverse events (SAEs). |
| Incidence of adverse events leading to death. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Incidence of adverse events leading to death. |
| Incidence of adverse events leading to treatment discontinuation. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Incidence of adverse events leading to treatment discontinuation. |
| Incidence of adverse events leading to dose reduction/interruption. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Incidence of adverse events leading to dose reduction/interruption. |
| Time to first onset of relevant adverse event. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Time to first onset of relevant adverse event. |
| AEs ≥ G3 Hematological and non-hematological. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | AEs ≥ G3 Hematological and non-hematological. |
| Impact of patients' characteristics and comorbidities on G3 or higher AEs and relevant adverse events development. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Impact of patients' characteristics and comorbidities on G3 or higher AEs and relevant adverse events development. |
| Frequency of transformation of WM to an aggressive lymphoma and frequency of secondary malignancies. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Frequency of transformation of WM to an aggressive lymphoma and frequency of secondary malignancies. |
| Major response rate (MRR) | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Major response rate (MRR) (≥PR) (best reported response). |
| Overall response rate (ORR) | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Overall response rate (ORR, defined as MRR plus minor response rate) |
| Best response | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Best response (best reported response). |
| Progression-free survival (PFS) | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Progression-free survival (PFS) including 6, 12, and 24-month PFS rate |
| Overall survival (OS) | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Overall survival (OS) including 6, 12, and 24-month OS rate. |
| Time to treatment failure | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Time to treatment failure (any treatment definitive discontinuation, incl. patient or investigator decision, toxicity, progression or death). |
| Duration of response (DOR) | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Duration of response (DOR) |
| Impact of patients and disease characteristics, and biomarker test results (MYD88 and CXCR4, if available) on treatment response. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Impact of patients and disease characteristics, and biomarker test results (MYD88 and CXCR4, if available) on treatment response. |
| Change of IgM levels until end of zanubrutinib treatment. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) | Change of IgM levels until end of zanubrutinib treatment. |
Countries
Italy
Contacts
PRINCIPAL_INVESTIGATORAnna Maria Frustaci
ASST Grande Ospedale Metropolitano Niguarda, Milano, Italy
Outcome results
None listed