Making inroads to the cure: Barriers to clinical trial enrollment in hematopoietic cell transplantation

Abstract

A significant barrier to advancing the standard of care for patients with hematologic malignancies undergoing stem cell transplantation is access and willingness to participate in clinical trials. The importance of clinical trial enrollment is magnified in an era of targeted therapies, accelerated drug discovery, and investment by the pharmaceutical industry. As disease targets are identified, novel therapies are being evaluated in efforts to reduce treatment-related toxicity and improve progression-free and overall survival. The enrollment of hematopoietic cell transplantation (HCT) patients on clinical studies is essential to promote the development of such therapies. Increasing clinical trial participation requires understanding of potential barriers to enrollment, including patient concerns, institutional and provider hurdles, and disease-specific characteristics.

Maintenance Strategies After Hematopoietic Cell Transplantation

“…56 In most cases, post-allo-HCT relapse is predictable, and deaths occur due to a limited number of effective treatment options and reduced access to novel compounds in clinical trials. [57][58][59] Effective, low-intensity maintenance therapies are necessary to improve disease-related outcomes after allo-HCT. At present, there are no maintenance therapies approved for MDS/AML patients after allo-HCT.…”

Salvage Therapy after Allogeneic Hematopoietic Cell Transplantation: Targeted and Low-Intensity Treatment Options in Myelodysplastic Syndrome and Acute Myeloid Leukemia

“…Finally, patients who relapse with active GVHD have historically poor outcomes [ 6 , 7 ]. These patients generally do not qualify for clinical studies due to inclusion/exclusion criteria [ 23 ]. The standard of care in MDS/AML treatment is challenging, as the safety of combining anti-GVHD and leukemia therapies is not well characterized.…”