People aged 75 years and older are underrepresented in blood cancer clinical trials

December 11, 2017

(Atlanta, Monday, Dec. 11, 2017) -- In the first comprehensive analysis of clinical trial enrollment among older adults with blood cancers, researchers from the U.S. Food and Drug Administration (FDA) found significant gaps in participation among those aged 75 and older when considered against the incidence of these malignancies in this age group, according to research being presented today during the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta. By comparison, adults under 65 years tend to be overly represented in these trials, despite the fact that a majority of blood cancers are most frequently diagnosed in those over 65 years of age.

"Until now, there has been very little information about the enrollment of adults with hematologic cancers. Based on our findings, the occurrence of cancer is much higher in adults over 75 years of age compared with the proportion of patients in this age group who enroll in clinical trials," said lead study author Bindu Kanapuru, MD, medical officer in the Division of Hematology Products, Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "With so few patients aged 75 or older enrolled in clinical trials, critical information on the safety and effectiveness of new therapies in this age group is greatly lacking."

The magnitude of the disparity is particularly concerning given that the number of adults aged 75 and older who are diagnosed with blood cancers is only expected to rise as the population ages. Currently, one out of five patients diagnosed with most blood cancers is age 75 and above.

In this study, FDA researchers analyzed data on more than 44,000 patients who were enrolled in clinical trials of drug treatments for blood cancers that were evaluated by FDA as part of the drug approval process between 2005 and 2015.The researchers grouped patients by disease and age (<65, 65-74,and ≥75 years) and then compared their rates of enrollment in clinical trials to the Surveillance, Epidemiology, and End Results (SEER) Program of the National Cancer Institute 2010-2014 data on the rate of occurrence of each cancer by age group. Most patients were enrolled in trials of treatments for lymphoma (including chronic lymphocytic leukemia [CLL]; 45 percent), chronic myeloid leukemia (CML; 24 percent), or multiple myeloma (22 percent).

Patients under 65 years of age were over-represented in trials for lymphomas, CLL, CML, and multiple myeloma compared with the incidence of those cancers in this age group. For example, researchers found that nearly 80 percent of patients enrolled in trials for CML were under 65, but only half of patients diagnosed with CML are in this age group.

By contrast, patients aged 75 and older account for approximately 29 percent of CML diagnoses, yet this age group made up less than 4 percent of those enrolled in clinical trials to evaluate new treatments for the disease. They were also significantly underrepresented in trials of new treatments for lymphoma, CLL, and multiple myeloma compared with the incidence of each of these cancers in that age group.

"We weren't surprised to see that, overall, adults aged 75 years and older were under-represented in clinical trials, as this is common across cancer trials," said Dr. Kanapuru. "But we were surprised by the magnitude of the gap for this age group, particularly for CML trials."

The good news, she adds, is that among patients aged 65 to 74, data show the proportion enrolled in lymphoma (excluding CLL) and CML trials essentially mirrored the reported incidence of blood cancers in this age group. In multiple myeloma and CLL trials, the proportion enrolled in the 65-74-year-old age group was higher than the reported incidence in this group.

The FDA, through a number of guidance documents, actively encourages clinical trial sponsors to ensure that drug studies include the types of patients who will use the drug once it is approved. But Dr. Kanapuru points to multiple barriers to enrolling patients aged 75 and older in clinical trials. For example, patients with certain characteristics are regularly excluded from clinical trials for cancer treatments, including a history of previous cancers; less-than-optimal functioning of the heart, liver, kidneys, or other organs; or other co-existing illnesses. Many of these criteria serve to exclude older adults, who may be more likely than younger people to have co-existing illnesses or changes in organ function associated with aging. Social and financial factors can also play a role.

"In addition, doctors may hesitate to enroll these older patients because they aren't sure how they will tolerate investigational medications," Dr. Kanapuru said. "The 75-and-older population is also extremely heterogeneous. You can have one 75-year-old who is very healthy and another person of the same age who is frail and has a lot of co-existing illnesses."

Dr. Kanapuru stresses that it is important for clinicians to evaluate patients' suitability for enrollment in a clinical trial based on all of their characteristics and not to exclude them on the basis of age alone. Assessment tools that can help identify how older patients will fare and efforts to expand inclusion criteria are also needed, she says. Dr. Kanapuru also points out that the FDA has been vocal about the need to expand eligibility criteria and has engaged directly with sponsors and other stakeholders to examine ways to include specific populations of patients who are commonly excluded in cancer trials. She and her team believe these findings are a first step in raising awareness about the clear disparities in participation in blood cancer-related research studies, which are key to gathering evidence that ultimately informs treatment protocols.
-end-
Bindu Kanapuru, MD, of the U.S. Food and Drug Administration, will present the study "Enrollment of Older Adults in Clinical Trials Evaluating Patients with Hematologic Malignancies - the Food and Drug Administration (FDA) Experience [861] on Monday, December 11, at 4:30 p.m. EST in Room B211 of the Georgia World Congress Center.

For the complete annual meeting program and abstracts, visit http://www.hematology.org/annual-meeting. Follow @ASH_hematology and #ASH17 on Twitter and like ASH on Facebook for the most up-to-date information about the 2017 ASH Annual Meeting.

The American Society of Hematology (ASH) is the world's largest professional society of hematologists dedicated to furthering the understanding, diagnosis, treatment, and prevention of disorders affecting the blood. For more than 50 years, the Society has led the development of hematology as a discipline by promoting research, patient care, education, training, and advocacy in hematology. The Society publishes Blood®, the most cited peer-reviewed publication in the field, as well as the online, open-access journal, Blood Advances®.

American Society of Hematology

Related Cancer Articles from Brightsurf:

New blood cancer treatment works by selectively interfering with cancer cell signalling
University of Alberta scientists have identified the mechanism of action behind a new type of precision cancer drug for blood cancers that is set for human trials, according to research published in Nature Communications.

UCI researchers uncover cancer cell vulnerabilities; may lead to better cancer therapies
A new University of California, Irvine-led study reveals a protein responsible for genetic changes resulting in a variety of cancers, may also be the key to more effective, targeted cancer therapy.

Breast cancer treatment costs highest among young women with metastic cancer
In a fight for their lives, young women, age 18-44, spend double the amount of older women to survive metastatic breast cancer, according to a large statewide study by the University of North Carolina at Chapel Hill.

Cancer mortality continues steady decline, driven by progress against lung cancer
The cancer death rate declined by 29% from 1991 to 2017, including a 2.2% drop from 2016 to 2017, the largest single-year drop in cancer mortality ever reported.

Stress in cervical cancer patients associated with higher risk of cancer-specific mortality
Psychological stress was associated with a higher risk of cancer-specific mortality in women diagnosed with cervical cancer.

Cancer-sniffing dogs 97% accurate in identifying lung cancer, according to study in JAOA
The next step will be to further fractionate the samples based on chemical and physical properties, presenting them back to the dogs until the specific biomarkers for each cancer are identified.

Moffitt Cancer Center researchers identify one way T cell function may fail in cancer
Moffitt Cancer Center researchers have discovered a mechanism by which one type of immune cell, CD8+ T cells, can become dysfunctional, impeding its ability to seek and kill cancer cells.

More cancer survivors, fewer cancer specialists point to challenge in meeting care needs
An aging population, a growing number of cancer survivors, and a projected shortage of cancer care providers will result in a challenge in delivering the care for cancer survivors in the United States if systemic changes are not made.

New cancer vaccine platform a potential tool for efficacious targeted cancer therapy
Researchers at the University of Helsinki have discovered a solution in the form of a cancer vaccine platform for improving the efficacy of oncolytic viruses used in cancer treatment.

American Cancer Society outlines blueprint for cancer control in the 21st century
The American Cancer Society is outlining its vision for cancer control in the decades ahead in a series of articles that forms the basis of a national cancer control plan.

Read More: Cancer News and Cancer Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.