Real World Outcomes Using Novel Agents for AML in the UK
Study Details
Study Description
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
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Venetoclax Venetoclax in newly diagnosed AML |
Drug: Venetoclax
Observational study of venetoclax in AML
Other Names:
|
| FLT3 inhibitors FLT3 inhibitors including gilteritinib in relapsed AML |
Drug: Gilteritinib
Observational study of gilteritinib in AML
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Overall survival [1 year]
Overall survival measured from time of treatment initiation
- Early death rate [Day 60 after starting treatment]
Early death rate measured at day 60 after treatment initiation
Secondary Outcome Measures
- Response rate [After 2 cycles of therapy (each cycle is 28 days although may be extended if recovery is delayed)]
Response rate as defined by ELN 2017
- Incidence of relapse in patients achieving remission [1 year]
Relapse incidence measured from the time of achieving remission
- Relapse-free survival [1 year]
RFS as defined by ELN
- Treatment toxicity 1 [During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)]
Number of days in hospital and number of days of intensive care
- Treatment toxicity 2 [During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)]
Duration of neutropenia and thrombocytopenia
- Treatment toxicity 3 [During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed)]
Number of blood and platelet transfusions
- Comparison of survival between patient sub-groups [1 year]
Overall survival compared between disease groups
Eligibility Criteria
Criteria
Venetoclax cohort Inclusion criteria
-
Newly diagnosed acute myeloid leukaemia
-
No prior therapies for AML, apart from hydroxyurea (or similar) for cytoreduction. Previous treatments for MDS or other conditions are allowed
-
Treated with venetoclax in combination with either azacitidine or LDAC No exclusion criteria
Gilteritinib/FLT3 cohort Inclusion criteria
-
Relapsed acute myeloid leukaemia, including molecular relapse
-
Treated with FLT3 inhibitor No exclusion criteria
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Guy's and St Thomas' NHS Foundation Trust | London | United Kingdom | SE1 9RT |
Sponsors and Collaborators
- Guy's and St Thomas' NHS Foundation Trust
- King's College London
Investigators
- Principal Investigator: Richard Dillon, King's College London
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 305432
- 305432