Clinical trial NCT05166135: Latin American Real-world Study in Acute Leukemia
Official title: Real-world Study in Acute Leukemia: Epidemiology, Treatment Patterns and Outcomes for B-cell ALL and AML in Adult Patients From Latin America - LOYAL Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Acute Myeloid Leukemia
Acute Lymphoid Leukemia
Keywords: Acute Myeloid Leukemia
Acute Lymphoid Leukemia
Relapsed Refractory B-cell Acute Lymphoblastic Leukemia
Acute Leukemia in Latin America
LOYAL study
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-12-21
Enrollment: 589
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients ≥18 years old at diagnosis, with de novo AML or R/R B-cell ALL diagnosed between 01 January 2015 and 31 December 2019, as documented in the medical chart and according to the physician's notes, and with at least 1 line of treatment for AML or R/R ALL within the study period.

Brief summary: The objective of the study is to describe the current epidemiology, treatment patterns, outcomes and healthcare resource use of adult patients diagnosed with relapsed/refractory (R/R) B-cell ALL and de novo AML in 4 Latin American countries.

Detailed description: This is a retrospective multicenter non-interventional study using real-world data collected from medical records of newly diagnosed AML or with relapsed/refractory B-cell ALL diagnosed between 01 January 2015 and 31 December 2019 in 4 Latin American countries: Argentina, Brazil, Chile, and Colombia. In addition, as secondary objectives, the study will also describe molecular profile, cytogenetic risk, clinical outcomes, and healthcare resource utilization of treated B-cell ALL R/R and AML patients.

Eligibility criteria: Inclusion Criteria:

* Patients ≥18 years old at diagnosis
* Confirmed diagnosis of relapsed/refractory B-cell ALL or de novo AML diagnosed between 01 January 2015 and 31 December 2019
* At least 1 line of treatment for R/R B-cell ALL or de novo AML within the study period

Exclusion Criteria:

* Patients with no medical chart available
* Patients with unreliable data as per investigator's opinion (e.g. excessive missing data or inconsistence data)
* Patients that have participated in any interventional clinical trial for relapsed/refractory B-cell ALL or AML at any moment
* Patients with secondary AML
* Patients with any concomitant primary malignancy
* Patients with acute promyelocytic leukemia (APL)

Primary outcomes:
measure: Number of Participants According to Health Insurance Type
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Country of Residence
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants With Comorbidities
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Family History of Hematological Malignancies
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants With Prior Exposure to Toxic Agents
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants With Prior Exposure to a High Dose of Radiation
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Reason for Exposure to High Dose of Radiation
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants With Bleeding History
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants With Tobacco Consumption Habits
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Eastern Cooperative Oncology Group (ECOG) Performance Status Scores
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Karnofsky Performance Scores
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Year of Diagnosis
timeFrame: At index (anytime between 01-Jan-2015 and 31-Dec-2019; approximately 5 years); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Classification for Standard Newly AML Therapy
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Drug Regimen Prescribed for Newly AML
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Drug Regimen Prescribed for R/R B-cell ALL
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants Prescribed Gemtuzumab, Midostaurin or Venetoclax Treatment in Newly AML
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Regimen Type in Newly AML
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Treatment Duration
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Time to Next Treatment
timeFrame: From start of front-line therapy until start of subsequent line of therapy, last visit/contact/death (up to maximum of 94.6 months);data collected and observed retrospectively over 11 month
measure: Total Number of Cycles
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants Who Withdrew Regimen
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Reasons for Withdrawing Regimen
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants With Dose Reduction
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants With Central Nervous System (CNS) Involvement at Disease Progression
timeFrame: At disease progression (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Duration of Radiotherapy
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Dose of Radiotherapy
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Location of Application of Radiotherapy
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Intrathecal Chemotherapy
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants With Stem Cell Transplant (SCT)
timeFrame: From start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Absolute Value of Hemoglobin Before Treatment Line
timeFrame: Up to 48 hours before each treatment cycle till the end of treatment, disease progression and/or death from any cause; data collected and observed retrospectively over 11 months
measure: Absolute Value of Hemoglobin After Start of Treatment Line
timeFrame: After start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Absolute Value of White Blood Cell Count Before Treatment Line
timeFrame: Up to 48 hours before each treatment cycle till the end of treatment, disease progression and/or death from any cause; data collected and observed retrospectively over 11 months
measure: Absolute Value of White Blood Cell Count After Start of Treatment Line
timeFrame: After start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Absolute Neutrophil Count (ANC) Before Treatment Line
timeFrame: Up to 1 week before treatment initiation; data collected and observed retrospectively over 11 months
measure: Absolute Neutrophil Count (ANC) After Start of Treatment Line
timeFrame: After start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Absolute Value of Blast Count Before Treatment Line
timeFrame: Up to 1 week before treatment initiation; data collected and observed retrospectively over 11 months
measure: Absolute Value of Blast Count After Start of Treatment Line
timeFrame: After start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Absolute Value of Platelets Count Before Treatment Line
timeFrame: Up to 1 week before treatment initiation; data collected and observed retrospectively over 11 months
measure: Absolute Value of Platelets Count After Start of Treatment Line
timeFrame: After start of treatment until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
Secondary outcomes:
measure: Number of Participants With Molecular Test Performed
timeFrame: At diagnosis (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to AML Translocation Results: AML Arm Only
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months ); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Molecular Profile in Newly AML Arm
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months ); data collected and observed retrospectively over 11 months
measure: Number of Participants According to AML WHO Classification: AML Arm Only
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to ALL WHO Classification: R/R B-cell ALL Arm Only
timeFrame: At diagnosis (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Immunophenotyping Results for AML Arm Only
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Immunophenotyping Results for R/R B-cell ALL
timeFrame: At diagnosis (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Molecular Profile in R/R B-cell ALL
timeFrame: At diagnosis (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to ALL Cytogenetic Risk Classification: R/R B-cell ALL Arm Only
timeFrame: At diagnosis (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to AML Cytogenetic Risk Classification Based on 2017 European Leukemia Net (ELN)
timeFrame: From diagnosis until loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Drug Regimen Prescribed Since Diagnosis of de Novo ALL in R/R B-cell ALL
timeFrame: From the diagnosis for de novo ALL diagnosis date to study enrolment date (anytime between 2001-2014 approximately 13 years); data collected and observed retrospectively over 11 months
measure: Time From Start of First-Line Treatment to Start of the Adverse Event
timeFrame: From start of 1st line treatment until Adverse Event (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Clinical Events
timeFrame: From diagnosis until end of treatment, disease progression, death or loss of follow-up (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Event Free Survival (EFS)
timeFrame: From start of treatment until failure to achieve CR, PD or death or censoring date (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Overall Survival (OS)
timeFrame: From start of treatment until disease progression, loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Secondary: Percentage of Participants Alive at 1,3 and 5 Years Since Treatment Initiation
timeFrame: 1, 3 and 5 years since treatment initiation; data collected and observed retrospectively over 11 months
measure: Relapse Free Survival-Newly Diagnosed AML Participants Only
timeFrame: From date of remission until relapse or death or censoring date (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Treatment Response
timeFrame: From start of treatment until disease progression, loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Hospitalizations
timeFrame: From diagnosis until end of treatment, loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Reason for Hospitalization
timeFrame: From diagnosis until end of treatment, loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Duration in Intensive Care Unit
timeFrame: From diagnosis until end of treatment, loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Procedures
timeFrame: From diagnosis until end of treatment, loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Participants According to Surgery
timeFrame: From diagnosis until end of treatment, loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Blood Transfusions
timeFrame: From diagnosis until end of treatment, loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
measure: Number of Concomitant Medications
timeFrame: From diagnosis until end of treatment, loss of follow-up or death (up to maximum of 94.6 months); data collected and observed retrospectively over 11 months
Other outcomes:
Locations:
city: La Plata
country: Argentina
facility: Hospital Italiano de La Plata
state: Buenos Aires
city: Pilar
country: Argentina
facility: Hospital Universitario Austral
state: Buenos Aires
city: Buenos Aires
country: Argentina
facility: FUNDALEU - Fundacion para combatir la Leucemia
state: Ciudad Autonoma Buenos Aires
city: Cordoba
country: Argentina
facility: Hospital Privado Centro Medico de Cordoba S.A.
city: Cordoba
country: Argentina
facility: Sanatorio Allende
city: Salvador
country: Brazil
facility: Hospital São Rafael
state: Bahia
city: Jaú
country: Brazil
facility: Fundação Doutor Amaral Carvalho
state: SAO Paulo / Brazil
city: Rio de Janeiro
country: Brazil
facility: Instituto COI de Pesquisa
city: São Paulo
country: Brazil
facility: ICESP - Instituto do Câncer do Estado de São Paulo Octavio Frias de Oliveira
city: São Paulo
country: Brazil
facility: Hospital Beneficência Portuguesa de São Paulo
city: São Paulo
country: Brazil
facility: Hospital Israelita Albert Einstein
city: Concepcion
country: Chile
facility: Hospital Guillermo Grant Benavente
city: Bogotá
country: Colombia
facility: Fundacion Santa Fe de Bogota
city: Montería
country: Colombia
facility: Oncomedica SA
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: