The FDA has approved ivosidenib (Tibsovo®, Servier Pharmaceuticals) in combination with azacitidine (Vidaza®, Celgene) for patients with newly diagnosed IDH1-mutated acute myeloid leukemia (AML) who are at least 75 years old or who have comorbidities preventing them from receiving intensive induction chemotherapy. "Mutant IDH1 catalyzes the production of D-2-hydroxyglutarate, leading to disruption in cellular metabolism and epigenetic regulation and contributing to oncogenesis," wrote Pau Montes...

For older patients with higher-risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), or acute myeloid leukemia (AML) with 20% to 30% marrow blasts, treatment options are limited. In the PANTHER study, the results of which were recently presented at the American Society of Hematology (ASH) 63rd Annual Meeting & Exposition in Atlanta, Georgia, a team of researchers led by Dr. Mikkael A. Sekeres, Professor of Medicine and Chief of the Division of Hematology at the Univer...

The FDA has approved daunorubicin and cytarabine (Vyxeos®, Jazz Pharmaceuticals) for pediatric patients one year and older with newly diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes. "Effective regimens are needed for children with relapsed acute myeloid leukemia (AML)," wrote Todd Cooper, DO, of Seattle Children's Hospital, and colleagues, in their May publication in the Journal of Clinical Oncology of AAML1421 (NCT02642965), one of the studie...

The oral hypomethylating agent CC-486, when administered in an escalated dosing schedule (>7 days per 28-day treatment cycle), has been shown to be effective and well tolerated for patients with acute myeloid leukemia (AML) experiencing their first relapse, according to results of a study presented at American Society of Hematology (ASH) Annual Meeting. For the phase 3 international, randomized, double-blind QUAZAR AML-001 trial (NCT01757535), 472 patients with AML were enrolled. To be eligib...

Measurable residual disease correlates with disease-free survival and overall survival data for patients with acute myeloid leukemia (AML), according to results of a meta-analysis published today in JAMA Oncology. This finding supports the continued use of measurable residual disease as a surrogate end point in clinical trials. The investigators, led by first author Nicholas Short, MD, Assistant Professor in the Department of Leukemia at the University of Texas MD Anderson Cancer Center in Houst...

The FDA has now granted full approval to venetoclax (Venclexta®, AbbVie Inc. and Genentech Inc.) in combination with azacitidine, decitabine, or low-dose cytarabine (LDAC) for patients with previously untreated acute myeloid leukemia (AML) who are at least 75 years of age or who have comorbidities that prevent them from receiving intensive induction chemotherapy. Previously, this venetoclax combination therapy was granted accelerated approval in 2018 for use in this same patient population. The ...

The FDA has now approved azacitidine tablets (Onureg®, Celgene) for the maintenance treatment of acute myeloid leukemia (AML) after intensive induction chemotherapy in patients who are unable to complete intensive curative therapy and who have achieved complete remission (CR) or complete remission with incomplete blood count recovery (CRi). The approval was based on data from the multicenter, double-blind phase 3 QUAZAR trial (NCT01757535), in which 472 patients with AML who achieved CR or CRi w...

Sorafenib maintenance after transplant has shown to be effective in decreasing chance of relapse for patients with acute myeloid leukemia (AML) that have a FLT3 internal tandem duplication (FLT3-ITD) and who are undergoing allogeneic hematopoietic stem-cell transplantation (HSCT), according to recent phase 3 study results published in The Lancet Oncology. A total of 202 patients with FLT3-ITD AML were enrolled. To be eligible, patients had to be aged 18 to 60 years old, currently undergoing allo...

In this interview, Alan S. Gamis, MD, MPH, speaks with i3 Health about the recent FDA approval of gemtuzumab ozogamicin (MylotargTM, Wyeth Pharmaceuticals, LLC) for pediatric patients aged 1 month and older with newly diagnosed CD33-positive acute myeloid leukemia (AML). Dr. Gamis, lead investigator of the phase 3 Children's Oncology Group Trial AAML0531, which provided the basis for the approval, discusses the significance of the approval and shares his advice for health care providers as they ...

The FDA has extended the approval of gemtuzumab ozogamicin (MylotargTM, Wyeth Pharmaceuticals, LLC) to include pediatric patients aged 1 month and older with newly diagnosed CD33-positive acute myeloid leukemia (AML). Gemtuzumab ozogamicin is a humanized immunoconjugate that targets CD33, a cell surface antigen present in over 80% of patients with AML. The approval was based on AAML0531 (NCT00372593), a phase 3 trial that enrolled 1,063 patients aged 0 to 29 years with newly diagnosed AML. Patie...

A recent phase 3 trial found that treosulfan/fludarabine used as conditioning treatment prior to allogeneic hematopoietic stem cell transplantation (HSCT) led to greater event-free survival compared with busulfan/fludarabine in older or comorbid patients with acute myeloid leukemia or myelodysplastic syndrome. While patients age 50 or older with acute myeloid leukemia or myelodysplastic syndrome are often considered eligible for allogeneic HSCT, they are at increased risk from standard myeloabla...

​A phase 2 clinical trial of a chemotherapy/immunotherapy combination involving azacitidine and nivolumab has produced promising results in patients with relapsed or refractory acute myeloid leukemia (AML). The most common type of leukemia, AML is a deadly cancer of the blood and bone marrow with a 5-year survival rate of only 27.4%. Some patients experience relapse after initial treatment success; others have refractory disease, where some leukemic cells remain in the bone marrow even after agg...

The FDA has approved venetoclax (VenclextaÒ, AbbVie Inc. and Genentech Inc.) to be used in combination with azacitidine, decitabine, or low-dose cytarabine for the treatment of acute myeloid leukemia (AML) in elderly patients or those ineligible for intensive chemotherapy. Approval of venetoclax was determined based on two open-label, non-randomized trials involving patients with newly diagnosed AML aged 75 years or older or with comorbidities that prevent them from receiving intensive induction...

Recently, the FDA approved glasdegib (DaurismoÔ, Pfizer Labs) to be used in combination with low-dose cytarabine (LDAC) for newly diagnosed acute myeloid leukemia (AML) in elderly patients or those ineligible to receive intensive induction chemotherapy. Richard Pazdur, MD, Director of the FDA's Oncology Center of Excellence and Acting Director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research, explained, "Intensive chemotherapy is usually used...

Researchers have found that the quantity of genetic mutations of hematopoietic stem cells (HSCs) in patients with acute myeloid leukemia (AML) is no greater than that of healthy individuals. These mutations are largely generated by normal, age-dependent processes that are constantly active during everyone's life. "We thought that people with leukemia would have more mutations than healthy people, but this is not the case," commented the study's principal investigator, Ruben Van Boxtel, PhD, of t...

Researchers have developed a new treatment that has the potential to work powerfully for some patients with relapsed acute myeloid leukemia (AML). One of the deadliest cancers, AML has a five-year survival rate of around 30%, which drops to under 10% for elderly patients. Around 30% of patients with AML have an activating mutation in the tyrosine kinase domain (TKD) of fms-like tyrosine kinase 3 (FLT3) or an FLT internal tandem duplication. These patients generally respond well initially to...

With unknown risk factors and a varied response to treatment, acute myelogenous leukemia (AML) can be a difficult cancer to treat. In order to improve the understanding of this disease, researchers at The University of Texas at San Antonio (UTSA) and the University of Texas MD Anderson Cancer Center created the AML Proteome Atlas, a database derived from MetaGalaxy analyses that contains the proteomic profiling of 205 patients with AML and 111 leukemic cell lines. "Acute myelogenous leukemia pre...

Even though targeted therapies are often efficacious in acute myeloid leukemia (AML), treatment resistance is sometimes inevitable. Mutations in isocitrate dehydrogenase (IDH) enzymes occur in approximately 20% of AML cases and could be a potential factor in treatment resistance. In order to produce a more effective therapy, researchers tracked the evolution of leukemia from mutant IDH2 dependence to independence and found two vulnerabilities of IDH2-mutant leukemia that can serve as potential t...

The FDA has now approved ivosidenib (Tibsovo®, Agios Pharmaceuticals, Inc.) as a first-line treatment for patients 75 years or older with acute myeloid leukemia (AML) and a susceptible IDH1 mutation who have comorbidities that prevent them from safely using intensive induction chemotherapy. Ivosidenib has previously been approved for patients with AML with no comorbidities. However, AML is mainly a disease found in older adults; nearly a third of all AML cases are diagnosed in those older than 7...

The FDA has now approved the addition of data on overall survival (OS) to the labeling of gilteritinib (Xospata®, Astellas Pharma US, Inc.), which is indicated for adults with previously treated acute myeloid leukemia (AML) with an FLT3 mutation. These new data demonstrate that gilteritinib has been successful in increasing OS. One of the types of leukemia occurring most frequently in adults, AML is still fairly rare, representing around 1% of all cancers. According to American Cancer Society es...