A Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe

A Retrospective Observational Multicenter Study on the Management and Outcome of Patients With Systemic AL Amyloidosis in Europe

This is a retrospective, observational, multicenter study to collect Real-World Evidence (RWE) data on systemic AL-AMY patients in Europe. Data from paper/electronic medical records and/or electronic databases from key reference centers in Europe will be used. Data will either be entered by the site staff in the electronic Case Report Form (eCRF) or, where feasible, transferred directly, always in accordance to local regulations.

Study Overview

• Status: Completed
• Conditions: AL Amyloidosis

Detailed Description

The lack of regulatory approved treatment options for AL amyloidosis (AL-AMY) justifies the need to understand the current treatment practice and outcomes of this disease. There is a need for objective Real-World Evidence (RWE) that reflects how treatments are initiated, combined and sequenced, and how their relative effectiveness and safety profiles emerge outside a clinical trial setting. Therefore, the aim is to generate RWE on systemic AL-AMY patients in Europe, including patient characteristics, resource use, treatments and associated patient outcomes.

• Study Type: Observational
• Enrollment (Actual): 4481

Contacts and Locations

Study Locations

• Austria
  • Wien, Austria
    • Allgemeines Krankenhaus Der Stadt Wien (General Hopsital), Universitätsklinik Für Innere Medizin I Klinische Abteilung Für Onkologie (Clinic of Internal Medicine I), Medizinische Universität Wien
• Czechia
  • Ostrava, Czechia
    • University Hospital Ostrava, Clinic of Hematooncology
• France
  • Limoges, France
    • Centre Hospitalier Universitaire de Limoges, Department of Clinical Hematology and Cell Therapy
  • Poitiers, France
    • Service de Néphrologie, Hémodialyse Et Transplantation Rénale, Hôpital Jean Bernard, Chu Poitiers
• Germany
  • Heidelberg, Germany
    • Universität Heidelberg, Department of Internal Medicine V: Hematology, Oncology and Rheumatology
• Greece
  • Athens, Greece
    • Alexandra Hospital, University of Athens School of Medicine, Department of Clinical Therapeutics
• Italy
  • Pavia, Italy
    • Centro Per Lo Studio E La Cura Delle Amiloidosi Sistemiche Padiglione Forlanini
• Netherlands
  • Groningen, Netherlands
    • University Medical Center Groningen
  • Utrecht, Netherlands
    • University Medical Center Utrecht, Umc Utrecht, Department of Hematology
• Portugal
  • Lisboa, Portugal
    • Fundação Champalimaud, Hematology Research, Imunology - Medical School, Nova University
  • Porto, Portugal
    • Centro Hospitalar e Universitário São João
• Spain
  • Barcelona, Spain
    • Servicio de Hematología, Hospital Clínic de Barcelona
• United Kingdom
  • London, United Kingdom
    • University College London Medical School, Uk Royal Free Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

The study will include adult (male and female) patients diagnosed with systemic AL-AMY and symptomatic organ involvement who have initiated first line treatment in the period 2004-2018.

Patients who have received treatment for AL-AMY as part of an interventional Clinical Trial will be identified but will not be included in the analysis due to the controlled environment of a clinical trial (not RWE data).

Patients with comorbid multiple myeloma will not be excluded.

Description

Inclusion Criteria:

• Age>18 years
• Confirmed diagnosis of AL-AMY and symptomatic organ involvement.
• Initiated first line treatment for AL-AMY in the period 2004-2018.
• Patients who have signed a participation agreement/ICF allowing data collection and source data verification in accordance with local requirements.

The inclusion of deceased subjects in the study is permitted under the condition that consent waiver has been granted by the Scientific Committee and/or Administrative Board of the participating sites and/or any applicable regulatory body, as per local regulations, to either implement a hospital informed consent form (ICF) already in place or provide written approval of this study-specific waiver. In the occasion that waiver of consent is not granted deceased subjects will not be enrolled in the study.

Exclusion Criteria:

• Patients under the age of 18 will not be considered eligible for this study

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Group pre-2010; Patients who initiated first-line treatment between 2004 and 2010 (pre-2010).
Group post-2010; Patients who initiated first-line treatment between 2011 and 2018 (post-2010).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Patient and disease characteristics	To describe the proportion of patients belonging to different age groups, and the proportion of patients per gender, stage, organ involvement, performance status, and hematologic biomarkers at screening or at baseline; by first-line treatment initiation period (pre-2010 or post-2010) and overall.	2004-2018
Treatment patterns	To describe the proportion of patients who had an autologous stem cell transplantation by first-line initiation period (pre-2010 or post-2010) and overall; the proportion of regimen combinations used by line of treatment, and by first-line initiation period; the proportion of patients receiving specific regimen combinations following different first-line regimens, by first-line initiation period and overall.	2004-2018
Response evaluation and efficacy outcomes	To describe the treatment effectiveness in terms of hematologic response rates at 3, 6, 12, and 24 months after first-line initiation, by regimen and first-line initiation period (pre-2010 or post-2010); overall survival, progression-free survival, and time on treatment, overall, by first-line treatment and by first-line initiation period.	2004-2018
Healthcare resource utilization: hospitalizations	To describe the healthcare resource utilization patterns during the treatment of AL amyloidosis by first-line treatment initiation period (pre-2010 or post-2010) and overall, in terms of hospitalization information (number of hospitalizations, duration per hospitalization); use of concomitant treatment (frequency of regimens used, proportion of patients receiving concomitant treatment); proportion of patients undergoing imaging exams; proportion of patients undergoing cardiac exams; proportion of patients receiving dialysis.	2004-2018
Healthcare resource utilization: concomitant therapy	To describe the healthcare resource utilization patterns during the treatment of AL amyloidosis, by first-line treatment initiation period (pre-2010 or post-2010) and overall, in terms of the proportion of patients receiving concomitant therapy.	2004-2018
Healthcare resource utilization: imaging and cardiac assessments	To describe the healthcare resource utilization patterns during the treatment of AL amyloidosis, by first-line treatment initiation period (pre-2010 or post-2010) and overall, in terms of the proportion of patients undergoing imaging and cardiac exams.	2004-2018
Healthcare resource utilization: dialysis	To describe the healthcare resource utilization patterns during the treatment of AL amyloidosis, by first-line treatment initiation period (pre-2010 or post-2010) and overall, in terms of the proportion of patients receiving dialysis.	2004-2018
Safety management	To describe the proportion of patients having serious adverse events or adverse events of special interest, by first-line treatment initiation period (pre-2010 or post-2010) and overall.	2004-2018

Collaborators and Investigators

• Sponsor: European Myeloma Network

Study record dates

Study Major Dates

• Study Start (Actual): April 23, 2019
• Primary Completion (Actual): April 30, 2021
• Study Completion (Actual): April 30, 2021

Study Registration Dates

• First Submitted: April 6, 2021
• First Submitted That Met QC Criteria: June 16, 2021
• First Posted (Actual): June 24, 2021

Study Record Updates

• Last Update Posted (Actual): September 27, 2022
• Last Update Submitted That Met QC Criteria: September 26, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Immunoproliferative Disorders; Paraproteinemias; Proteostasis Deficiencies; Neoplasms, Plasma Cell; Immunoglobulin Light-chain Amyloidosis; Amyloidosis
• Other Study ID Numbers: EMN23

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No