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

NCT04937777 | Completed

• 1 other identifier: EMN23
• Study Type: observational
• Target: 4,481
• Locations: 10 countries
• Sites: 13

Brief Summary

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.

Conditions

AL Amyloidosis

Outcome Measures

Primary Outcomes (8)

• 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

Study Arms (2)

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).

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• European Myeloma Network B.V.: lead

Study Sites (13)

Allgemeines Krankenhaus Der Stadt Wien (General Hopsital), Universitätsklinik Für Innere Medizin I Klinische Abteilung Für Onkologie (Clinic of Internal Medicine I), Medizinische Universität Wien

Vienna, Austria

Location

University Hospital Ostrava, Clinic of Hematooncology

Ostrava, Czechia

Location

Centre Hospitalier Universitaire de Limoges, Department of Clinical Hematology and Cell Therapy

Limoges, France

Location

Service de Néphrologie, Hémodialyse Et Transplantation Rénale, Hôpital Jean Bernard, Chu Poitiers

Poitiers, France

Location

Universität Heidelberg, Department of Internal Medicine V: Hematology, Oncology and Rheumatology

Heidelberg, Germany

Location

Alexandra Hospital, University of Athens School of Medicine, Department of Clinical Therapeutics

Athens, Greece

Location

Centro Per Lo Studio E La Cura Delle Amiloidosi Sistemiche Padiglione Forlanini

Pavia, Italy

Location

University Medical Center Groningen

Groningen, Netherlands

Location

University Medical Center Utrecht, Umc Utrecht, Department of Hematology

Utrecht, Netherlands

Location

Fundação Champalimaud, Hematology Research, Imunology - Medical School, Nova University

Lisbon, Portugal

Location

Centro Hospitalar E Universitário São João

Porto, Portugal

Location

Servicio de Hematología, Hospital Clínic de Barcelona

Barcelona, Spain

Location

University College London Medical School, Uk Royal Free Hospital

London, United Kingdom

Location

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Paraproteinemias

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 6, 2021
• First Posted: June 24, 2021
• Study Start: April 23, 2019
• Primary Completion: April 30, 2021
• Study Completion: April 30, 2021
• Last Updated: September 27, 2022

Record last verified: 2022-09

Locations

AU (1); GR (1); NL (2); IT (1); CZ (1); GB (1); PT (2); FR (2); DE (1); ES (1)