Hematology Advisor Opinion: Commentary and Perspective on Medicine and Practice

This article was written by Jeffery J. Auletta, MD, and Stella M. Davies, MBBS, PhD, MRCP in their personal capacities. The opinions expressed in this article are their own and do not necessarily reflect the views of Hematology Advisor or Haymarket Media.

Inequality in access and outcomes based on race, ethnicity, and socioeconomic status has been well documented in hematopoietic cell transplantation (HCT) and cellular therapy.1,2 It is time that the HCT and cellular therapy community moves beyond publishing papers, taking steps to address these problems.

That is what the ACCESS Initiative seeks to do. In 2022, the National Marrow Donor Program (NMDP)/Be The Match and the American Society for Transplantation and Cellular Therapy (ASTCT) formed the ACCESS Initiative to reduce barriers to and improve outcomes for HCT and cellular therapy through the implementation of practice and policy changes.

Meaningful, widespread change can only occur with active, sustained engagement from stakeholders throughout the cellular therapy ecosystem, including HCT/cellular therapy clinicians and institutions, program administrators, health policy and health equity experts, health services researchers, payer organizations, and federal and state stakeholders.

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Despite Advances, Disparities in HCT and Cellular Therapy Persist

The HCT and cellular therapy community has more potentially life-saving treatment options for patients than ever before, including the ability to provide a donor for nearly all patients in need by transcending the human leukocyte antigen (HLA) barrier.3

Patients who do not have a fully matched related or unrelated donor — which disproportionately impacts patients who are ethnically diverse — now have options to proceed to HCT with good outcomes.4,5 Research in this area continues with the ACCESS clinical trial (ClinicalTrials.gov Identifier: NCT04904588), which is currently enrolling patients.6

Since 2017, the US Food and Drug Administration (FDA) has approved 6  chimeric antigen receptor (CAR) T-cell therapies for hematologic malignancies.7 Additional allogeneic and autologous cell and gene therapies for hematologic malignancies and disorders are currently in the pipeline.8

Despite these advances, access and outcomes for HCT and cellular therapy remain unequal. For example, researchers performed a retrospective analysis of US unrelated donor (URD) searches of the Be The Match Registry to identify the likelihood a patient would proceed to HCT within 6 months of active search initiation.9 Of those who received URD HCT within 6 months, patients who were non-Hispanic White were more likely to receive HCT than patients who were Black or African American (45% vs 27%; P <0.001).

Outcome disparities also reflect patient socioeconomic status. The Center for International Blood and Marrow Transplant Research (CIBMTR) performed an analysis to study the impact of neighborhood poverty exposure on pediatric HCT outcomes.10 The investigators found neighborhood poverty was associated with higher rates of transplant-related mortality (TRM) in the malignant disease cohort. In addition, when compared to children with private insurance, those receiving Medicaid had lower overall survival and higher TRM.

The landscape of access and outcome disparities in CAR-T therapy is similar, with studies revealing differences in access and outcomes for patients who are Black and African American.11,12

The ACCESS Initiative aims to overcome problems that are deeply rooted in the US health care system. The initiative is a starting point for the HCT/cellular therapy and broader hematology/oncology communities to take collective steps forward.

Initially, the ACCESS Initiative will focus on 3 areas: awareness, poverty, and racial and ethnic inequity.1

Lack of Information is a Barrier to HCT and Cellular Therapy Access

New standards of care in HCT and cellular therapy are often not disseminated to hematologists/oncologists at community practices or academic institutions that do not provide these treatments.1,13,14 Some patients do not understand the options available to them, so they cannot advocate for themselves or make informed decisions about their care.

The ACCESS Initiative Awareness Subcommittee aims to increase patient access to HCT and cellular therapy by increasing awareness among hematologists/oncologists and patients about treatment options and new research.

Among clinicians, the committee will develop partnerships and connections between transplant/cellular therapy centers and hematology/oncology practices, provide education to hematology/oncology practices, and improve access to information about current research.

Activities are already underway. For example, the committee launched a physician exchange program that fosters discussion on how to support patients across the care continuum, from diagnosis through return to the hematology/oncology practice post-treatment.

Within the patient community, the committee will build and strengthen partnerships with national disease-specific groups and health-focused community organizations. The committee aims to develop joint campaigns and local outreach efforts to raise awareness about treatment options and share support information.1,13