Real World Outcomes Using Novel Agents for AML in the UK

Study Overview
=================
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.

Official Title
-----------------
Real World Outcomes Using Novel Agents for Acute Myeloid Leukaemia in the United Kingdom

Conditions
-----------------
Acute Myeloid Leukemia

Intervention / Treatment
-----------------
* Drug: Venetoclax
* Drug: Gilteritinib


Participation Criteria
=================
Eligibility 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

Ages Eligible for Study
-----------------
Minimum Age: 16 Years

Sexes Eligible for Study
-----------------
All

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Venetoclax<br>Venetoclax in newly diagnosed AML | Drug: Venetoclax<br>* Observational study of venetoclax in AML<br>* Other names: Venclyxto;|
| FLT3 inhibitors<br>FLT3 inhibitors including gilteritinib in relapsed AML | Drug: Gilteritinib<br>* Observational study of gilteritinib in AML<br>* Other names: Xospata;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall survival | Overall survival measured from time of treatment initiation | 1 year | 
| Early death rate | Early death rate measured at day 60 after treatment initiation | Day 60 after starting treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Response rate | Response rate as defined by ELN 2017 | After 2 cycles of therapy (each cycle is 28 days although may be extended if recovery is delayed) | 
| Incidence of relapse in patients achieving remission | Relapse incidence measured from the time of achieving remission | 1 year | 
| Relapse-free survival | RFS as defined by ELN | 1 year | 
| Treatment toxicity 1 | Number of days in hospital and number of days of intensive care | During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed) | 
| Treatment toxicity 2 | Duration of neutropenia and thrombocytopenia | During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed) | 
| Treatment toxicity 3 | Number of blood and platelet transfusions | During the first cycle of therapy (each cycle is 28 days although may be extended if recovery is delayed) | 
| Comparison of survival between patient sub-groups | Overall survival compared between disease groups | 1 year |