AML

Several oncology experts discuss the FDA approvals they found most significant in 2018.

A look back at all the FDA news that happened in the month of November 2018, including several new approvals, priority reviews, a fast track designation, and an accelerated approval, across a variety of cancer types.

An update on the pivotal phase III&nbsp;QuANTUM-R study presented at the 2018 ASH Annual Meeting demonstrated overall survival benefit across patient subgroups with&nbsp;quizartinib in patients with&nbsp;relapsed/refractory <em>FLT3</em>-ITD&ndash;mutated acute myeloid leukemia.&nbsp;

Among older patients with acute myeloid leukemia who were ineligible for intensive chemotherapy, preliminary findings demonstrated that more than 70% achieved complete responses to venetoclax in combination with hypomethylating agents.

It may be possible for hematologists to determine subtypes of acute myeloid leukemia based on genetic analsysis of blood samples in 7 days or less. According to Amy Burd, PhD, this process could play an important role in diagnosing and treating patients.

According to updated data from the phase II ELIANA study,&nbsp;CD19-targeted CAR T-cell therapy tisagenlecleucel as treatment of&nbsp;pediatric and young adult patients with relapsed or refractory acute lymphoblastic leukemia sustained rates of&nbsp;relapse-free survival and overall survival at 24 and 18 months.&nbsp;

Gilteritinib has been approved by the FDA&nbsp;for the treatment of adult patients with&nbsp;<em>FLT3</em>&nbsp;mutation&ndash;positive relapsed or refractory acute myeloid leukemia.

A new drug application for&nbsp;quizartinib has been granted a priority review by the FDA&nbsp;for the treatment of adult patients with relapsed/refractory&nbsp;<em>FLT3</em>-ITD&ndash;positive acute myeloid leukemia. The designation is based on findings from the&nbsp;phase III QuANTUM-R study.&nbsp;

Glasdegib (Daurismo) has been granted FDA approval for combination use&nbsp;with low-dose cytarabine for the treatment of patients with newly-diagnosed acute myeloid leukemia who are aged 75 years or older or who are ineligible for intensive chemotherapy.

Venetoclax (Venclexta) has been granted an accelerated approval by the FDA for combined use with&nbsp;azacitidine or decitabine or low-dose cytarabine as a treatment for&nbsp;adult patients with newly-diagnosed acute myeloid leukemia who are aged 75 years or older,&nbsp;or who have comorbidities that preclude use of intensive induction chemotherapy.

<br /> Mary-Beth Percival, MD, discusses the ongoing development of FLT3 inhibitors and other current research in patients with AML.

Mutations in 8 high-risk genes are associated with an acute myeloid leukemia (AML) diagnosis, according to a deep sequencing analysis. Hetty Carraway, MD, said that these findings lay the ground work for upcoming research on early detection and novel treatment strategies.

Patients with acute myeloid leukemia now have more treatment options available than ever, due to some major changes to the field over the last year. With 2 drugs already approved for patients with <em>IDH</em> mutations and 4 new drugs expected to receive approval in the next year, it is a more hopeful time than ever for this patient population.

Chronic lymphocytic leukemia is characterized by the clonal proliferation and accumulation of small, mature-appearing CD5-positive B lymphocytes in the blood, bone marrow, and secondary lymphoid tissues.&nbsp;A CLL diagnosis is established by the presence of more than 5x109/L peripheral lymphocytes co-expressing CD5, CD19, and CD23, and weakly expressing CD20, CD79b, and surface immunoglobulin.&nbsp;Small lymphocytic lymphoma represents a clinical variant of CLL and is similarly managed.

Lloyd Damon, MD,&nbsp;discussed novel therapies in AML with a focus on FLT3 and IDH1/2 inhibitors.<br /> &nbsp;

Rami S. Komrokji, MD,&nbsp;reflects on the diverse treatment landscape of AML and touches on the prognostic importance of biomarkers.

In an interview with <em>Targeted Oncology</em>, Jay Yang, MD, discussed the evolution of the treatment landscape for AML and the future outlook for these treatments.

In an interview with&nbsp;<em>Targeted Oncology</em>, Jorge E. Cortes, MD, discussed the results from the QuANTUM-R trial, as well as some other studies investigating the use of quizartinib in different patient populations in AML.

Predisposition to forms of myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) in pediatric patients can be caused by either classical inherited bone marrow failure syndromes or recently discovered genetic predisposition syndrome with autosomal dominant inheritance, Marcin Wlodarski, MD, explained.

Combining cladribine and low-dose cytarabine alternating with decitabine (Dacogen) resulted in high response rates and improved survival compared with currently established low-intensity therapies for patients with acute myeloid leukemia who are older than 60, according to results newly published in <em>Lancet Haematology.</em>

A look back at all the&nbsp;FDA news&nbsp;that happened in the month of&nbsp;August 2018, including several new approvals, a priority review, breakthrough therapy designations, and more in a variety of cancer types.

Preclinical and phase I proof for CYAD-01 as well as preliminary findings from the ongoing THINK trial were presented at the 2018 American Society of Gene and Cell Therapy Annual Meeting.