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.
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
4,481
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
University Hospital Ostrava, Clinic of Hematooncology
Ostrava, Czechia
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
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Centre Hospitalier Universitaire de Limoges, Department of Clinical Hematology and Cell Therapy
Limoges, France
Service de Néphrologie, Hémodialyse Et Transplantation Rénale, Hôpital Jean Bernard, Chu Poitiers
Poitiers, France
Universität Heidelberg, Department of Internal Medicine V: Hematology, Oncology and Rheumatology
Heidelberg, Germany
Alexandra Hospital, University of Athens School of Medicine, Department of Clinical Therapeutics
Athens, Greece
Centro Per Lo Studio E La Cura Delle Amiloidosi Sistemiche Padiglione Forlanini
Pavia, Italy
University Medical Center Groningen
Groningen, Netherlands
University Medical Center Utrecht, Umc Utrecht, Department of Hematology
Utrecht, Netherlands
Fundação Champalimaud, Hematology Research, Imunology - Medical School, Nova University
Lisbon, Portugal
...and 3 more locations
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 2004-2018