Clinical trial NCT05312112: Real World Outcomes Using Novel Agents for AML in the UK
Official title: Real World Outcomes Using Novel Agents for Acute Myeloid Leukaemia in the United Kingdom
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Acute Myeloid Leukemia
Lead sponsor: Guy's and St Thomas' NHS Foundation Trust
Sponsor class: OTHER
Collaborators: name: King's College London
First posted: 2022-04-05
Enrollment: 1000
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The NHS criteria for access to venetoclax was that a patient was fit for IC and was:

* Aged \>16y with NPM1 mutation without FLT3 internal tandem duplication (ITD)
* Aged \>50y with NPM1, IDH1 or IDH2 mutations (regardless of FLT3 status)
* Patients aged \>60y without favourable-risk cytogenetics

Gilteritinib was made available to all patients aged \>16y with relapse or refractory FLT3 mutated AML. Other FLT3 inhibitors are available to patients through various access schemes

Brief summary: This project will collect data on patients with acute myeloid leukemia in the United Kingdom who were treated with two new targeted therapies during the coronavirus pandemic

Detailed description: Acute myeloid leukaemia (AML) is a blood cancer which in fit young adults is typically treated with intensive chemotherapy. While this is potentially curative, it is associated with significant side effects and the requirement for long hospital admissions. Infection is a major issue during AML treatment, as both the disease and the chemotherapy impair the immune system.

Early data suggested that COVID-19 is associated with a very high rate of death in AML patients undergoing intensive chemotherapy. Because of this, and the need for significant hospital resources to deliver intensive chemotherapy, the NHS made available two new, less intensive, targeted therapies for the treatment of AML during the COVID-19 pandemic - venetoclax and gilteritinib. The aim was to reduce mortality and healthcare resource use.

Many hundreds of patients across the UK have been treated with these two medications on the temporary access scheme. The research aims to collect de-identified data from treating patients to describe the outcomes of patients treated with these approaches, both in terms of the safety and effectiveness.

Eligibility criteria: Venetoclax cohort Inclusion criteria

1. Newly diagnosed acute myeloid leukaemia
2. No prior therapies for AML, apart from hydroxyurea (or similar) for cytoreduction. Previous treatments for MDS or other conditions are allowed
3. Treated with venetoclax in combination with either azacitidine or LDAC No exclusion criteria

Gilteritinib/FLT3 cohort Inclusion criteria

1. Relapsed acute myeloid leukaemia, including molecular relapse
2. Treated with FLT3 inhibitor No exclusion criteria

Primary outcomes:
measure: Overall survival
timeFrame: 1 year
measure: Early death rate
timeFrame: Day 60 after starting treatment
Secondary outcomes:
measure: Response rate
timeFrame: After 2 cycles of therapy (each cycle is 28 days although may be extended if recovery is delayed)
measure: Incidence of relapse in patients achieving remission
timeFrame: 1 year
measure: Relapse-free survival
timeFrame: 1 year
measure: Treatment toxicity 1
timeFrame: During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)
measure: Treatment toxicity 2
timeFrame: During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)
measure: Treatment toxicity 3
timeFrame: During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)
measure: Comparison of survival between patient sub-groups
timeFrame: 1 year
Other outcomes:
Locations:
city: London
country: United Kingdom
facility: Guy's and St Thomas' NHS Foundation Trust
Study officials:
affiliation: King's College London
name: Richard Dillon
role: PRINCIPAL_INVESTIGATOR
References: