The National Institute for Health and Care Excellence (NICE) has issued a Final Appraisal Document (FAD) recommending oral azacitidine for maintenance therapy in adult patients with acute myeloid leukaemia (AML) who achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following induction therapy with or without consolidation treatment and who are not candidates for, including those who choose not to proceed to, hematopoietic stem cell transplantation (HSCT).

Oral azacitidine is the first oral maintenance therapy to demonstrate a significant overall survival and show a relapse-free survival benefit compared to placebo in patients with a broad range of AML subtypes.

“AML is an aggressive type of blood cancer that can quickly progress and become harder to treat at advanced stages,” said Professor Timothy Chevassut, Consultant Haematologist, Royal Sussex County Hospital. “The complexity of the disease is a significant challenge for effective maintenance therapy of
AML.

Currently, around half of patients treated with chemotherapy may relapse, so this recommendation has the potential to change the outlook for people living with the disease.” AML is one of the most common acute leukaemias in adults.2 There are around 3,100 new diagnoses of the disease in the UK per year, and since the early 1990s, AML incidence rates have increased by 20%.

Incidence of the disease is strongly related to age with 42% of all new cases in the UK each year being diagnosed in people aged 75 and over.

AML is a heterogeneous group of diseases associated with diverse genetic mutations.

Following patients' initial response to treatment, about 50% of patients relapse, thus representing a significant unmet need for treatment options that prolong overall survival. “People living with AML in the UK, especially those who are not eligible for stem cell transplants, have remained in need of maintenance therapy options. Today’s decision from NICE is an important step forward in providing an alternative treatment to improve outcomes of those living with the disease,” said Hubert Bland, Executive Medical Director, Bristol Myers Squibb.

“At BMS, we are committed to improving long-term outcomes and extending survival for patients with hard-to-treat diseases, such as AML.” The decision from NICE is supported by results from QUAZAR® AML-001, a Phase 3, international, randomised, double-blind study.

Results demonstrated that oral azacitidine was associated with significantly longer overall and relapse-free survival than placebo among older patients with AML who were in remission after chemotherapy and ineligible for a HSCT.

Median overall survival (OS), the primary endpoint, from the time of randomisation was significantly longer in the oral azacitidine arm (n=238) than in the placebo group (n=234) (24.7 months vs 14.8 months; P<0.001). Median relapse-free survival was also significantly longer with oral azacitidine than with placebo (10.2 months and 4.8 months, respectively; p<0.001).

Overall health-related quality of life was maintained across both treatment arms. The most common adverse events that occurred in both treatment arms were gastrointestinal events including nausea (65% of patients treated with oral azacitidine and 24% with placebo), vomiting (60% and 10% respectively) and diarrhoea (50% and 21% respectively). Grade 3 or 4 adverse events, including neutropenia and thrombocytopenia, occurred in 41% and 22% of patients treated with oral azacitidine compared to 24% and 21% of patients with placebo respectively.

Source: Bristol Myers Squibb