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Rachel Darwin, Senior Manager, Public Relations, 2676226624

New Study in JNCCN Finds Disturbing Lack of Key Leukemia Medication

Researchers call on hematologists and pharmacists to make sure life-saving treatment is kept on-hand for patients with rare leukemia.

JNCCN Cover, November 2021PLYMOUTH MEETING, PA [November 16, 2021] — New research in the November 2021 issue of JNCCN—Journal of the National Comprehensive Cancer Network finds fewer than one-third of hospitals had immediate availability of a crucial blood cancer medication called all-trans retinoic acid (ATRA). ATRA is initiated early in the treatment of acute promyelocytic leukemia (APL)—a form of acute myeloid leukemia (AML), to prevent major bleeding, clotting, and potential death. APL is very treatable and tends to have a better prognosis than other subtypes of AML when treated appropriately.

The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for AML, along with other international evidence-based guidelines, recommend the immediate initiation of ATRA for patients even suspected of having APL while waiting for genetic confirmation of the diagnosis—due to the likelihood of early disseminated intravascular coagulopathy (DIC), a life-threatening bleeding disorder. However, when researchers conducted a telephone survey of 120 randomly selected hospitals from different regions around the United States, they found that only approximately 31% of hospitals—and just 14% of hospitals that refer patients with leukemia elsewhere—had ATRA immediately available. Furthermore, 42% of the hospitals that confirmed they do treat patients with acute leukemia did not have ATRA in stock.

Dale Bixby, MD, PhD Michigan Medicine and University of Michigan Medical School“The loss of a patient with a highly curable leukemia due to the lack of access to a pill for formulary reasons would be devastating,” said Dale Bixby, MD, PhD, Michigan Medicine and University of Michigan Medical School. “We hope our study will lead to more widespread knowledge of the lack of availability of ATRA in hospital systems within the U.S. and spur a call to action from pharmacists and hematologists to ensure it is readily available at their institution.”

According to the survey results, the main reasons for lack of availability of ATRA were related to the rarity of APL and included:

  • the drug had not recently been requested;
  • the inpatient pharmacist was not familiar with the drug;
  • or the facility relied on associated hospitals or cancer centers to provide the drug.

"The lack of immediate availability of ATRA at 42% of centers who treat acute leukemia was shocking and sobering,” commented James K. Mangan, MD, PhD, Associate Clinical Professor of Medicine, Division of Blood and Marrow Transplantation, UC San Diego, Moores Cancer Center.

Dr. Mangan, a Member of the NCCN Guidelines® Panel for AML continued: “Furthermore, given that transfers from referring centers often take days due to bed availability issues, the lack of availability of ATRA at 86% of referring centers is a truly dangerous situation. This illustrates another reason why SEER database analysis shows that real-world outcomes for Acute Promyelocytic Leukemia lag far behind the excellent outcomes reported in clinical trials. It is a clarion call to action to lobby for inclusion of ATRA on the formularies of all hospitals who encounter acute leukemia patients, no matter whether they are a referring or treating center." 

Previous research cited in the study found that 11% of patients with APL died within 30 days of presentation for medical care.[1] When ATRA was administered within one day of a suspected diagnosis of APL 33% of the early deaths were from bleeding, while for those who received ATRA after more than one day from suspected diagnosis, 70% of the early deaths were from bleeding.

To read the entire study, visit Complimentary access to “Clinical Availability of ATRA for Patients With Suspected Acute Promyelocytic Leukemia: Why Guidelines May Not Be Followed” is available until February 10, 2022.

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About JNCCN—Journal of the National Comprehensive Cancer Network

More than 25,000 oncologists and other cancer care professionals across the United States read JNCCN—Journal of the National Comprehensive Cancer Network. This peer-reviewed, indexed medical journal provides the latest information about innovation in translational medicine, and scientific studies related to oncology health services research, including quality care and value, bioethics, comparative and cost effectiveness, public policy, and interventional research on supportive care and survivorship. JNCCN features updates on the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®), review articles elaborating on guidelines recommendations, health services research, and case reports highlighting molecular insights in patient care. JNCCN is published by Harborside. Visit To inquire if you are eligible for a FREE subscription to JNCCN, visit Follow JNCCN on Twitter @JNCCN.

About the National Comprehensive Cancer Network

The National Comprehensive Cancer Network® (NCCN®) is a not-for-profit alliance of leading cancer centers devoted to patient care, research, and education. NCCN is dedicated to improving and facilitating quality, effective, equitable, and accessible cancer care so all patients can live better lives. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) provide transparent, evidence-based, expert consensus recommendations for cancer treatment, prevention, and supportive services; they are the recognized standard for clinical direction and policy in cancer management and the most thorough and frequently-updated clinical practice guidelines available in any area of medicine. The NCCN Guidelines for Patients® provide expert cancer treatment information to inform and empower patients and caregivers, through support from the NCCN Foundation®. NCCN also advances continuing education, global initiatives, policy, and research collaboration and publication in oncology. Visit for more information and follow NCCN on Facebook @NCCNorg, Instagram @NCCNorg, and Twitter @NCCN.

[1] Altman JK, Rademaker A, Cull E, et al. Administration of ATRA to newly diagnosed patients with acute promyelocytic leukemia is delayed contributing to early hemorrhagic death. Leuk Res 2013;37:1004–1009.