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.
- Registration Number
- NCT06441214
- Lead Sponsor
- Fondazione Italiana Linfomi - ETS
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 125
- 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
- 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)
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Prospective cohort Zanubrutinib Patients with Waldenström's macroglobulinemia treated with Zanubrutinibin clinical practice enrolled from the study start up to 12 months.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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.
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.
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.
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).
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).
EORTC-QLQ-C30 Prospective cohort: up to 24 months (duration of study) Quality of life (QoL) assessed with EORTC-QLQ-C30 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 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.
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.
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).
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.
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.
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.
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
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.
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.
EQ-5D-5L Prospective cohort: up to 24 months (duration of study) Quality of life (QoL) assessed with EQ-5D-5L questionnaire
Trial Locations
- Locations (28)
Azienda Ospedaliera Universitaria Careggi - Unità funzionale di Ematologia
🇮🇹Firenze, Italy
A.O. SS. Antonio e Biagio e Cesare Arrigo - S.C.D.U. Ematologia
🇮🇹Alessandria, Italy
A.O.U. Ospedali Riuniti - Clinica di Ematologia
🇮🇹Ancona, Italy
Ospedale C. e G. Mazzoni - U.O.C. di Ematologia
🇮🇹Ascoli Piceno, Italy
AOU Policlinico Consorziale - U.O. Ematologia con Trapianto
🇮🇹Bari, Italy
Nuovo Ospedale degli Infermi - SSD Ematologia
🇮🇹Biella, Italy
Policlinico S. Orsola-Malpighi - Istituto di Ematologia "Seragnoli"
🇮🇹Bologna, Italy
Ospedale Centrale di Bolzano - Divisione di Ematologia e T.M.O.
🇮🇹Bolzano, Italy
Azienda Ospedaliera Universitaria Policlinico - S. Marco - UOC di Ematologia
🇮🇹Catania, Italy
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant'Anna - Ematologia e fisiopatologia della coagulazione
🇮🇹Ferrara, Italy
Ospedale Policlinico San Martino S.S.R.L. - IRCCS per l'Oncologia - Ematologia e terapie cellulari
🇮🇹Genova, Italy
ASST Grande Ospedale Metropolitano Niguarda - S.C. Ematologia
🇮🇹Milano, Italy
IEO Istituto Europeo di Oncologia - Divisione Ematoncologia
🇮🇹Milano, Italy
Ospedale Maggiore Policlinico - Fondazione IRCCS Ca Granda - Ematologia
🇮🇹Milano, Italy
AOU Maggiore della Carità di Novara - SCDU Ematologia
🇮🇹Novara, Italy
AOU di Padova - Ematologia
🇮🇹Padova, Italy
A.O. Ospedali Riuniti Villa Sofia-Cervello - Divisione di Ematologia
🇮🇹Palermo, Italy
RCCS Policlinico S. Matteo di Pavia - Div. di Ematologia
🇮🇹Pavia, Italy
P.O. Spirito Santo di Pescara - UOC Ematologia Dipartimento Oncologico Ematologico - ASL Pescara
🇮🇹Pescara, Italy
AOU Pisana - U.O. Ematologia
🇮🇹Pisa, Italy
Ospedale delle Croci - Ematologia
🇮🇹Ravenna, Italy
Azienda Unità Sanitaria Locale-IRCCS - Arcispedale Santa Maria Nuova - Ematologia
🇮🇹Reggio Emilia, Italy
Università Cattolica Sacro Cuore - Ematologia
🇮🇹Roma, Italy
AOU Senese - U.O.C. Ematologia
🇮🇹Siena, Italy
Ospedale "G. Mazzini" - UOS Ematologia
🇮🇹Teramo, Italy
A.O.U. Città della Salute e della Scienza di Torino - Ematologia Universitaria
🇮🇹Torino, Italy
A.O.U. Città della Salute e della Scienza di Torino - S.C. Ematologia
🇮🇹Torino, Italy
Azienda Sanitaria Universitaria Friuli Centrale (ASU FC) - SOC Clinica Ematologica
🇮🇹Udine, Italy