How We Treat Acute Myeloid Leukemia

Our Treatment Approach

As a highly specialized center within Dana-Farber Brigham Cancer Center's Center for Hematologic Oncology, the Adult Leukemia Program focuses on the distinct needs of leukemia patients. Our center is one of the most experienced leukemia centers in the nation, seeing a high volume of patients for consultations and treatment, and offering many clinical trials. This expertise supports our ability to treat complex cases for both newly diagnosed and relapsed/refractory patients.

Your care will involve the latest treatments currently available, including the opportunity to participate in clinical trials of new therapies based on research in our laboratories and elsewhere in the field of leukemia treatment.

We take a very personalized approach to your care. Using the most advanced sequencing techniques, we identify the mutations specific to your disease in order to plan the best treatment for you and identify if a clinical trial may be appropriate. With the Rapid Heme Panel, we get these results back in real time so we can quickly develop your treatment plan and start therapy.

A Team of Experts

You receive world-class care from specialists with years of expertise in caring for adults with AML. These experts, including hematologists/oncologists, physician assistants, nurse practitioners, and infusion room nurses, manage your care as a team in order to decide what approach will best treat your condition at each stage. We work closely with our transplant service team when patients require bone marrow/stem cell transplantation to ensure early referral for transplant evaluation. We have extensive experience in standard and emerging therapies, and in monitoring the disease at a molecular level to ensure that your care plan offers the best possible outcomes.

We view every patient as an individual with unique needs and expectations. We take time to involve you and your family in each step of the treatment process. As you go through treatment, you and your family will have access to a wide range of support resources such as nutritionsocial workgeriatricsZakim Center for Integrative Therapies and Healthy LivingAdolescent and Young Adult ProgramOlder Adult Hematologic Malignancies Program, and survivorship.

Experimental Drug Improves Survival in Certain AML Patients

Dana-Farber's Richard Stone, MD, discusses the drug midostaurin, which, added to standard chemotherapy is the first targeted treatment to improve survival of a high-risk, genetically defined subgroup of patients with acute myeloid leukemia.

Types of Treatment

Treatment for AML is a long-term process. Chemotherapy and other treatment for the disease may take 6 to 12 months to complete.

The treatment of adult AML is usually done in phases. In addition to standard approaches outlined here, we offer clinical trial options for patients in all phases of treatment.

Remission Induction Therapy

Traditionally, this was the phase of your therapy when you get intensive chemotherapy. However, with more effective frontline treatment options now available, the ‘remission induction’ phase may now include intensive or less intensive treatments to get patients into remission. The treatment plan is based on a patient’s disease mutations, age, and health status. Some treatment in this stage may require that a patient be admitted to the hospital for support and monitoring.

There are also treatment options for patients not recommended for intensive therapy. For these patients, azacitidine and venetoclax-based treatments may be considered. We also have special treatment plans for older patients through our Older Adult Hematologic Malignancy Program for patients who may benefit from a less intensive, though still beneficial, treatment regimen.

If patients go into remission, we look at molecular and chromosomal tests to see if a stem cell transplant would be beneficial to maintain the remission. Patients may continue with chemotherapy while moving to the transplant stage.

Post-Remission Therapy (Also Called Consolidation Therapy)

The goal of consolidation therapy is to prevent relapse by killing any remaining leukemia cells that may not be active but could begin to regrow. Consolidation therapy usually involves chemotherapy. Some patients may also be recommended for allogeneic stem cell transplant. These patients may receive chemotherapy until their stem cell transplant.

Treatment of AML may also include the following:

Stem Cell Transplant

Some patients, including older adults who would not benefit from intensive initial chemotherapy, may benefit from an allogeneic stem cell transplant as part of initial treatment or as part of treatment for relapsed disease. These re-infused stem cells grow into (and restore) the body's blood cells. Dana-Farber Brigham Cancer Center has one of the largest and most experienced stem cell transplantation programs in the country.

Targeted Therapy

Targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific cancer cells without harming normal cells. Targeted therapy may target a specific mutation in a cancer cell or help stimulate the immune system to attack the cancer.

In 2017, a targeted drug whose clinical testing was led by Richard Stone, MD, (director of translational research in the Adult Leukemia Program), became a new treatment for newly diagnosed AML. The drug, midostaurin (Rydapt®), was approved by the FDA as a combination treatment with chemotherapy for adult patients newly diagnosed with AML with a mutation in the gene FLT3.

Since then, additional therapies have been approved for specific mutations such as FLT3, IDH 1, and IDH 2.

Clinical Trials Questions?


Clinical Trials

The Adult Leukemia Program has an active research program that aims to better understand and treat AML. We offer many clinical trials for adults of all ages and disease subtypes. A few examples include:

For younger patients with a mutation in FLT3, we have a phase 3 clinical trial comparing 7+3 plus a FLT3 inhibitor midostaurin versus crenolanib (17-623). For those without a mutation in FLT3 we have a clinical trial with 7+3 plus the oral BCL2 inhibitor venetoclax (18-351).

For less intensive chemotherapy approaches, we have phase 1/2 clinical trials adding to azacitidine plus venetoclax with SL-401 (tagraxofusp) (17-056) based on laboratory work from Andrew Lane, MD, PhD.

For targeted therapy options, we offer clinical trials with novel single agent therapies including menin inhibition with SNDX-5613 (19-467) for patients with mutations in NPM1 or MLL-gene rearrangement. For AML or MDS patients with splicing or FLT3 mutations, we have a clinical trial with CURIS CA-4948 (IRAK4 inhibitor) (20-168).

We are also exploring cellular therapies for AML. One trial (18-233) explores an allogeneic CAR T product targeted CD123 in patients with relapsed/refractory AML or newly-diagnosed high-risk AML.

Patients also have access to trials at other Harvard-affiliated institutions, those sponsored by private industry, as well as those that are part of a collaborative effort, such as the Alliance for Clinical Trials in Oncology, of which our program is a member.

Treatment for Relapsed or Persistent Disease

If your disease comes back (relapses) after achieving a remission, our clinicians will discuss standard treatment options as well as clinical trials, with the goal of achieving a second remission.

Other Drug Therapy

Arsenic trioxide and all-trans retinoic acid (ATRA) are anticancer drugs that kill leukemia cells, stop the leukemia cells from dividing, or help the leukemia cells mature into white blood cells. These drugs are used in the treatment of a form of AML called acute promyelocytic leukemia (APML).

BPDCN Treatment

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare subtype of AML. Learn more about our specialized BPDCN Center.

Specialized Care for Older Adults

Geriatricians in our Older Adult Hematologic Malignancy Program work closely with our oncologists to create individualized treatment plans for older patients. These plans consider other illnesses or concerns that affect older adults in particular.

Your Care

You will be assigned to one of our leukemia physicians as your primary oncologist overseeing all aspects of your care, both inpatient and outpatient.

All outpatient therapy is provided at the Yawkey Center for Cancer Care at Dana-Farber Cancer Institute, one of the most advanced outpatient cancer centers in the country.

If you need to be hospitalized during your care, or if you undergo stem cell transplantation, you will be admitted to Brigham and Women's Hospital (BWH) or the Dana-Farber Inpatient Hospital located within BWH. Your primary oncologist and nurse will closely monitor your care and will coordinate your care with the inpatient team and additional specialists, who will address any other symptoms you may be experiencing. This model ensures seamless care from the outpatient to the inpatient setting. Learn more about your stay.

Second Opinions

Get Started

Consultations and Second Opinions

  • Because adult acute myeloid leukemia is a quickly progressing blood cancer, there is great value in patients with suspected or diagnosed AML consulting with our team, which has deep experience in treating patients with this disease.
  • We routinely evaluate specimens sent to us from outside centers. These specimens are evaluated by our entire team, including our hematopathologists.

Reasons to consider a consultation or second opinion include:

  • To confirm your diagnosis.
  • If you have received a diagnosis elsewhere and want to be treated at Dana-Farber Brigham Cancer Center.
  • To determine the optimal therapy and timing of treatment.
  • To learn more about your cancer from specialists who are world leaders in this disease, and who have treated hundreds of other patients like you.
  • To learn if you're eligible for a clinical trial based on your disease features and treatment history.

Phone: 617-632-6028 or 617-632-5138
Online: Complete the Appointment Request Form

If you cannot travel to Boston in person, you can take advantage of our Online Second Opinion service; however, access to clinical trial opportunities may require travel to Boston.

For Referring Physicians

Because you, the referring physician, are an integral part of your patient's care team, we are committed to collaborating with you to provide the best care for your patient.

If you are a physician and have a patient with diagnosed or suspected AML, we look forward to working with you. Learn how to refer a patient.