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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Retrospective cohort | 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 | Patients with Waldenström's macroglobulinemia treated with Zanubrutinibin clinical practice enrolled from the study start up to 12 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Zanubrutinib | Drug | Patients will be treated with Zanubrutinib as per routine clinical practice. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Cumulative incidence of treatment discontinuation due to toxicity | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse events (AEs) | 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 related to zanubrutinib. |
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Inclusion Criteria:
Exclusion Criteria:
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Anna Maria Frustaci | ASST Grande Ospedale Metropolitano Niguarda, Milano, Italy | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| A.O. SS. Antonio e Biagio e Cesare Arrigo - S.C.D.U. Ematologia | Alessandria | Italy | ||||
| A.O.U. Ospedali Riuniti - Clinica di Ematologia |
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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 relevant adverse events. | 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 serious adverse events (SAEs). | 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 adverse events leading to death. | 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 treatment discontinuation. | 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 dose reduction/interruption. | 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) |
| Time to first onset of relevant adverse event. | 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) |
| AEs ≥ G3 Hematological and non-hematological. | AEs ≥ G3 Hematological and non-hematological. | 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. | 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) |
| Frequency of transformation of WM to an aggressive lymphoma and frequency of secondary malignancies. | 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) |
| Major response rate (MRR) | Major response rate (MRR) (≥PR) (best reported response). | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) |
| Overall response rate (ORR) | Overall response rate (ORR, defined as MRR plus minor response rate) | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) |
| Best response | Best response (best reported response). | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) |
| Progression-free survival (PFS) | Progression-free survival (PFS) including 6, 12, and 24-month PFS rate | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) |
| Overall survival (OS) | Overall survival (OS) including 6, 12, and 24-month OS rate. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) |
| Time to treatment failure | Time to treatment failure (any treatment definitive discontinuation, incl. patient or investigator decision, toxicity, progression or death). | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) |
| Duration of response (DOR) | Duration of response (DOR) | 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. | 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) |
| Impact of patients and disease characteristics, and biomarker test results (MYD88 and CXCR4, if available) on treatment response. | 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) |
| Hospital stays | 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. | Retrospective cohort: from october 2020 until the study start. Prospective cohort: up to 24 months (duration of study) |
| EORTC-QLQ-C30 | Quality of life (QoL) assessed with EORTC-QLQ-C30 questionnaire | Prospective cohort: up to 24 months (duration of study) |
| EQ-5D-5L | Quality of life (QoL) assessed with EQ-5D-5L questionnaire | Prospective cohort: up to 24 months (duration of study) |
| 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 |
| Azienda Ospedaliera Universitaria Careggi - Unità funzionale di Ematologia | Florence | 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 | Milan | Italy |
| IEO Istituto Europeo di Oncologia - Divisione Ematoncologia | Milan | Italy |
| Ospedale Maggiore Policlinico - Fondazione IRCCS Ca Granda - Ematologia | Milan | 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 |
| ID | Term |
|---|---|
| D008258 | Waldenstrom Macroglobulinemia |
| ID | Term |
|---|---|
| D054219 | Neoplasms, Plasma Cell |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D010265 | Paraproteinemias |
| D001796 | Blood Protein Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006474 | Hemorrhagic Disorders |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| C000629551 | zanubrutinib |
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