Retrospective-prospective Study to Evaluate Treatment Management and Outcomes of Patients With Waldenström's Macroglobulinemia (WM) Treated in Italy According to the Zanubrutinib (Brukinsa®) Compassionate Use Program (CUP) and in Common Practice Following Commercial Approval.

Fondazione Italiana Linfomi - ETS

Status: Active, not recruiting

Conditions: Waldenström's Macroglobulinemia (WM)

Treatments: Drug: Zanubrutinib

Study type: Observational

Funder types: Other

Identifiers

NCT06441214

FIL_BRUCE-ITA

Details and patient eligibility

About

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.

Enrollment: 212 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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)

Trial design

212 participants in 2 patient groups

Retrospective cohort

Description:

All patients with Waldenström's macroglobulinemia enrolled in Pre-Reimbursement Access Program (PRAP) of Zanubrutinib, as per Compassionate Use Program (CUP) of Zanubrutinib, and patients treated with commercial drug (Brukinsa®) from PRAP closure to the study start.

Prospective cohort

Description:

Patients with Waldenström's macroglobulinemia treated with Zanubrutinibin clinical practice enrolled from the study start up to 12 months.

Treatment:

Drug: Zanubrutinib